[Recorded by Electronic Apparatus]
Thursday, December 14, 1995
.0916 
[English]
The Chairman: Good morning.
[Translation]
Good morning, everyone and welcome to this fourth session of our roundtable on compassionate access to experimental drugs. This morning, we are going to deal with the important issue of responsibility.
[English]
The crucial issue today certainly is, whose responsibility is it to take a leadership role concerning an expanded access to investigational drugs? Another important question is, who should be responsible for paying the costs of compassionate access? We will also discuss the respective roles of the federal and provincial governments, the pharmaceutical industry and the insurance companies.
[Translation]
We will proceed in our usual manner. First, each participant will have a maximum of ten minutes to make a presentation, after which the members of the sub-committee will have an opportunity to put questions to the witnesses of their choice. The roundtable will close with a discussion between all members of the sub-committee and the participants.
This morning, to start with, we will hear Dr. Sophia Fourie, Dr. Michael Levy and Mr. William Milligan who represent the Pharmaceutical Manufacturers' Association of Canada.
Dr. Fourie, please.
[English]
Dr. Sophia Fourie (Pharmaceutical Manufacturers' Association of Canada):Mr. Chairman, thank you again for this opportunity to speak.
The responsibilities of manufacturers can be divided into three areas: moral responsibilities - the responsibilities to society; legal responsibilities - issues relating to product liability; and fiduciary responsibilities.
In the first two areas - societal and legal responsibilities - the conduct of manufacturers, investigators and treating physicians is governed by guidelines and codes of conduct such as the Helsinki code and MRC, FDA, and HPB guidelines. With regard to fiduciary responsibility, manufacturers are diligent in considering issues of proper use of their products and they pay specific attention to the status of knowledge of a new drug when deciding on the provision of early access.
Furthermore, the burden of administration and provision of experimental access can be significant. The financial cost can range between $30 million and $40 million. The creation of an infrastructure to administer the program, the provision of case records, the collecting of data - all these issues add up to quite a significant financial burden for this process.
The core issues that will have to be addressed are, first, the issues of product liability, as we discussed in great detail yesterday. Even due process and optimal conditions for ensuring that patients do make informed decisions on their exposure to experimental drugs do not safeguard against legal action.
The issue of financial burden and how this should be borne should be considered.
Finally, we all have the responsibility to consider the broader issue around access to advances in treatment. Having a mechanism for early access to a single experimental drug may not be as advantageous as having the drug approved in the shortest time period.
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In AIDS and cancer, combination therapy early in the disease seems to be the most promising approach.
We have heard the success story of 3TC, which was approved in Canada in five months, and in looking at the FDA record of approval times, periods range from three months recently with saquinavir, to six months for other AIDS drugs approved to date.
The drugs directorate should be supported through the commitment of resources to continue to speed up the approval process. The FDA has additional tools such as partial submissions and conditional approvals to help speed up the review process. The question is, are these initiatives ones we should be pursuing in Canada?
Manufacturers have shown, through the consistent implementation of early access programs in Canada, that the provision of expanded access is a priority and a recognized responsibility, and that companies have and will continue to shoulder this responsibility.
Thank you, Mr. Chair.
The Chairman: Thank you, Dr. Fourie.
Dr. Levy.
Dr. Michael Levy (Pharmaceutical Manufacturers' Association of Canada): Thank you. I support the comments of Dr. Fourie and I have nothing further to add.
Mr. William D. Milligan (Pharmaceutical Manufacturers' Association of Canada): The same - nothing further to add at this time, Mr. Chairman.
The Chairman: Thank you.
Mr. Arn Schilder from the B.C. People with AIDS. Mr. Schilder, please.
Mr. Arn Schilder (Vice-Chair, B.C. People with AIDS): Thank you, Mr. Chair. I have prepared responses to the five questions on responsibilities outlined in your communication with us last week, and I'll present those here.
On the issue of responsibility, the first question was, where does the responsibility for leadership lie in the effort to expand compassionate access? In Canada the responsibility lies with the federal Parliament because of its redirective capacity to determine policy. It is up to Parliament to set the ends and the health protection branch to establish and carry out the means. No clinical trials should be allowed to take place in Canada unless compassionate access is a provision.
The second question was, should the government take a legislate-and-command stand?
The Canadian government has a responsibility and an obligation to protect and provide for its citizens. HIV disease is a critical national issue. A large part of the strategy around AIDS must be devoted to creating incentives for the pharmaceutical industry to expand and improve their research agendas, for example, in studying basic science. We want AIDS to be a big business for this industry. In many ways our lives depend upon it. If economic incentives are what it takes to find effective and non-toxic treatments, then economic incentives are what they must be given.
The third question is, do federal and provincial governments have a responsibility to fund clinical trials on agents that are not of interest to the pharmaceutical companies?
Yes, absolutely. The pharmaceutical industry's interests lie in making HIV disease a lifelong chemically controlled illness. They want expensive and patentable compounds. If the government, particularly at the federal level, does not take some responsibility to allow for independent trials of potentially cheap and unpatentable products, then who will?
Furthermore, the government should be sponsoring through universities basic science investigations into HIV pathogenesis - immunological and virologic and genetic processes - ensuring a high degree of gender equity.
There are important ramifications as to whether a piece of research is curiosity driven or specifically directed. In its funding decisions, the government must be able to make this distinction and allocate funds appropriately.
The pharmaceutical industry has vested an interest in having direct basic science research happen. This is another opportunity for collaboration between industry and the public sector and an opportunity that could benefit everyone concerned.
The fourth question is, what responsibilities does the Canadian HIV Trials Network have to strongly encourage pharmaceutical companies to extend compassionate access?
The Canadian HIV Trials Network is a body that enacts the policies and standards set by the government. It advises on ends and executes means simultaneously. The Canadian HIV Trials Network currently recognizes and exercises its own responsibility in ensuring compassionate access by not accepting to undertake or sponsor any trial that does not provide compassionate access. Any kind of collaborative work between the Canadian HIV Trials Network and the industry must involve discussions about compassionate access and should be a focus for lobbying efforts and setting priorities around these issues.
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The fifth question is, do pharmaceutical companies have a responsibility beyond running a successful enterprise? Not according to them. They are a major international industry supplying essential products to a huge global market. Their primary concern is profit.
Through the Patented Medicine Prices Review Board of Canada, the PMPRB, the pharmaceutical companies are however legislated to spend 5% to 10% of their marketing budget on donations to community organizations, physician mentorship programs, etc. Our understanding is that the pharmaceutical industry reaps the profits from human disease and suffering. It must therefore bear the social responsibility that is associated with this power and privilege. Its responsibility is a moral one. Thank you.
The Chairman: Thank you very much, Mr. Schilder.
Now, from Access to Effective Cancer Care in Ontario, we have Ms Carol Harkness.
Ms Carol Harkness (Directing Officer, Access to Effective Cancer Care in Ontario): Good morning. My name is Carol Harkness, and I'm a directing officer of Access to Effective Cancer Care in Ontario, also known as AECCO. As well, I am a breast cancer survivor.
Attempting to join me this morning was one of our directing officers, a clinical trials nurse, Jan Stewart, who is weather-bound in Toronto. Carole Spiro, the president of Breast Cancer Action, is with me this morning.
We welcome the opportunity to participate in this national roundtable discussion, as access to drugs has been and continues to be a principal concern of AECCO.
While we recognize that the focus of this discussion is experimental drugs, the subcommittee should know that cancer patients continue to face significant problems in gaining access to approved breakthrough drugs. We hope this government will assert its role as enforcer of the Canada Health Act to ensure that cancer patients have equal access to the important drugs, experimental and approved, we need to beat this disease, whether we are rich or poor or whether we hail from Ontario or Saskatchewan.
I'd like to tell you a little bit about AECCO, how we started, what we've achieved, and what we're working on. Then I'll offer a few brief, preliminary thoughts on access to experimental drugs from the perspective of all that underpins AECCO's activities, that of the cancer patient.
AECCO was formed in September 1973 when a group of cancer survivors, oncologists, oncology nurses, and other cancer care workers who shared a concern about access to cancer treatment in Ontario came together. That night there was much discussion on several issues, including treatment waiting lists, shortage of radiation machines and therapists, and access to mammography, but the consensus emerged that the group's first priority ought to be to address an issue of growing concern to cancer patients and oncologists alike, that is, access to breakthrough cancer drugs.
The oncologists involved with AECCO are regularly faced with the dilemma of whether to tell patients who are not covered by a drug plan that they may benefit from a particular new drug while knowing it is financially out of reach or simply to pose other less effective treatment alternatives without even mentioning the breakthrough drug.
It was clear that night at our founding meeting, listening to oncologists and patients tell their stories, that we do in fact have a two-tiered cancer care system in Ontario. Wealthy cancer patients and those covered by a drug plan have much better access to these new breakthrough drugs, while the majority of patients receive suboptimal care.
AECCO decided that night over two years ago to broaden public awareness of this issue and to focus the Ontario government's attention on the need to improve cancer patients' access to breakthrough drugs. To put a human face on the issue, AECCO used access to granulated colony stimulating factor, or GCSF, as an important drug used in conjunction with chemotherapy to illustrate our concern with the limited availability of certain new medications. GCSF, considered a breakthrough by the federal government when it was fast-tracked and approved a few years ago, is a drug used to stimulate the rapid regrowth of white blood cells, which are the body's natural defence against infection.
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Chemotherapy patients susceptible to infection are often forced to have their chemotherapy programs delayed or reduced in intensity, which can compromise their treatment.
In every province in Canada except Ontario provincial health ministries have put in place policies and procedures whereby patients who really needed GCSF can have it covered by provincial drug plans in a timely way. You will understand that the patient's condition almost always requires a timely response.
In Alberta, oncologists' requests for GCSF are answered within a week; in Saskatchewan, within three to four days; and in Quebec, New Brunswick, Newfoundland and Nova Scotia, within one to two days. The story is quite different in Ontario.
Data gathered by AECCO under the Freedom of Information Act showed that on average, Ontario oncologists - and more importantly their patients - waited 61 days before receiving a response to their request for GCSF. Compared to a one- to two-day turnaround in most other provinces, an average response time of 61 days in Ontario is unacceptable and AECCO said so in a news conference in June 1994. For cancer patients, 61 days is a long, long time.
Partially as a result of AECCO's efforts the government's response time has been shortened significantly, but the access problem did not end there. Last February the Ontario government introduced treatment guidelines for GCSF to ensure its appropriate use. These guidelines explicitly excluded breast cancer patients despite a growing body of research that highlights the importance of maintaining patients' fully prescribed chemotherapy programs, something that GCSF can make possible for women with breast cancer.
AECCO is continuing its effort to enhance access to GCSF and other important breakthrough cancer treatment drugs.
We realize that we have spoken so far about approved cancer drugs and we have done so with good reason. We respectfully ask the subcommittee not to overlook the accessibility barriers to approved drugs when it considers access to experimental drugs. It is an issue that plagues many cancer patients every day.
The issue before the subcommittee, which is access to experimental drugs, raises difficult questions. Who should have access? Who should pay? Which drugs should be available? Which should not? That lines need to be drawn seems clear. Where they should be drawn is less obvious.
These questions are made even more challenging when viewed in the context of the government's current fiscal situation. In an ideal world the cost of treatment would have no bearing on patient access. However, we are reminded by every federal and provincial budget that we do not live in a perfect world.
We do not profess to have the answers to these difficult questions. Rather we come to this roundtable as an organization of patients with views shaped by the daily emotional and physical struggles of cancer patients desperate for a treatment breakthrough that will save our lives.
We believe that access to experimental drugs should be granted according to a set of principles or criteria to be applied on a case-by-case basis.
In the development of the access criteria, we believe the following should be considered: increased probability of survival should not be the guiding criterion; quality-of-life improvements, irrespective of survival, should be an important factor; priority access should not be given to certain disease groups at the expense of others; and finally, the financial wherewithal of the patient should not be the only factor.
Canada is recognized around the world for its public health care system, where all Canadians are treated equally. Unfortunately, when it comes to drugs, the rhetoric does not square with reality.
For cancer patients without a drug plan, Canada does have a two-tiered health care system. It seems unfair and un-Canadian that cancer patients with no drug plan who are treated on an outpatient basis with powerful drugs face enormous financial costs, while cancer patients treated with surgery in hospital have their costs underwritten by the government.
Drugs are just as much a part of our health care system as hospital beds and operating rooms. With this in mind, we believe that for Canada's health care system to be truly universal, drug care costs should be covered as well. We acknowledge that this notion flies in the face of the dramatic health care cutbacks being planned and made in every province in Canada, but imagine for a moment having a life-threatening disease and finding a drug that could improve your chances of survival or the quality of your life. Now imagine holding that drug in your hand and never being able to take it because you can't afford it or you have no drug plan.
.0935
This is not the United States; this is Canada. This happens to cancer patients with alarming frequency. Jan could tell you of oncologists who decide not even to inform some patients of GCSF or other breakthrough cancer drugs if they know there is no drug plan to cover the cost. These are drugs that can help their treatment, improve their quality of life, and, in some cases, even save their lives.
The bottom line for us at AECCO is that if there is a reasonable chance that an experimental drug can help a patient live longer or have a more comfortable life, it should be available. The costs should be borne by insurance companies, the government, or through some shared arrangement that might include the patient as well. The sanctity of Canada's health care system depends on it.
As well, it should not be forgotten that, in many cases, these powerful drugs help to keep patients out of hospitals and decrease provincial governments' health care costs. Unfortunately, being discharged from hospital usually means more than returning to the comfort and support of a patient's family; it also means they begin to pay for all of their drugs.
Finally, we are not experts in this field. It seems odd that so many of the new and powerful drugs that hold such promise for cancer patients are approved in the United States for general use, while in Canada they are still considered experimental. We would hope that all is being done by pharmaceutical companies and the federal government to move new drugs through the approval process as quickly as possible. As a cancer survivor, I can tell you there is nothing worse than reading about a new breakthrough drug available in the United States that is on the experimental list in Canada.
In summary, AECCO supports the availability of experimental drugs if there is a reasonable prospect of an improvement in the patient's quality of life. As well, we encourage the subcommittee to broaden its focus to encompass the problems cancer patients face in gaining access to approved drugs.
Finally, despite the fiscal crisis in which the federal and provincial governments are mired, Canada will never have a truly public health care system until drugs are publicly funded, like virtually every other aspect of health care delivery from surgery to doctors on house calls.
We are not public policy analysts or economists. We simply do not have the background and expertise to propose elaborate financial models to support our position. We are cancer survivors who are here not only to represent current patients, but to honour those who lost their fight with this terrible disease.
At an AECCO general meeting in the spring of 1994, a young woman recounted what she called the struggle of life. It was not with her cancer, but with her battle against the health care system that denied her access to an important breakthrough drug unless she paid for it herself. She won neither her battle with the system nor her fight against cancer. This morning we are here for her.
We appreciate the time the subcommittee has granted AECCO this morning, and we wish you well in your important deliberations. We would be pleased to answer any questions. Thank you.
The Chairman: Thank you, Ms Harkness.
Our next witnesses are from the Canadian Life and Health Insurance Association, Mr. Charles Black, senior adviser, and from Liberty Health, Mr. Gerry Byrne.
Gentlemen, please.
Mr. Charles Black (Senior Adviser, Insurance Operations, Canadian Life and Health Insurance Association): Thank you, Mr. Chairman.
My name is Charlie Black, and I appreciate the opportunity to participate in this morning's roundtable discussion on behalf of the Canadian Life and Health Insurance Association, which is the industry or trade association representing life and health insurance companies operating in Canada.
CLHIA's 90 member companies provide 92% of the life insurance, disability income insurance, supplemental health care insurance, and dental insurance purchased by Canadians.
In this morning's discussion, I want to focus on one segment of the industry's activities, namely the provision of supplemental health care insurance or extended health care benefit plans. In particular, I want to focus on the funding aspect of the question being deliberated by the subcommittee, and, in one subsection of that, the potential role of the private sector, particularly the private insurance sector, in any funding arrangements of that nature.
.0940
Supplementary health care insurance, or extended health care, which I'll refer to as EHC, is intended to supplement, more properly to complement, the important health insurance provided by public sector plans, both those established in accordance with the Canada Health Act and those established by various provinces to cover other important health care costs.
There is considerable variation in that latter category of provincial insurance plans, which are those that go beyond the Canada Health Act. For example, there's British Columbia's Pharmacare plan and Ontario's assistive devices plan. Because of this, there is also considerable variation under the complementary private coverages.
Typically, however, the extended health care plans cover much of the cost of prescription drugs for some 20 million Canadians, as well as other essential health care services, such as ambulance services, assistive devices, paramedical services and out-of-Canada health care costs, as well as some less essential but nonetheless desirable services, such as semi-private hospital surcharges.
In today's environment, this supplementary coverage is provided almost entirely for more than 90% through group insurance plans that are established by an employer, a union or other plan sponsor for the benefit of the employees or the plan members.
There are three aspects of the EHC segment of the insurance industry that I would like to highlight briefly, which is its voluntary nature, competitive nature, and diversity.
First, such benefit plans are entirely voluntary. There is no law mandating an employer or other plan sponsor to establish an EHC plan, or to include particular services under it. I should note, however, that collective bargaining agreements frequently cover this area. Thus, the sponsor must be convinced that it is beneficial to establish such a plan, or to include certain items under it, for the collective benefit of the plan members.
Second, this is a very competitive market. Insurers compete very actively with each other to obtain and retain business in this area. In addition, other major participants, such as prepayment organizations like the commonly known Blue Cross and Green Shield, and administrative services organizations that deal with uninsured benefit plans, compete actively with insurers to be the intermediary in providing comparable coverage.
Consistent with the voluntary and competitive natures of this market, there is considerable diversity. The plan sponsor calls the shots, usually with the assistance of a benefits consultant, and instructs the insurer or other intermediary on essential features of the plan design.
Thus, while there are common features in such plans, there are also major variations. For example, in the design of the formulary, which Mr. Byrne will comment on in more detail in a moment, there are major variations from one plan to another, and even among plans being administered by a single insurer.
To be successful in a voluntary, competitive market, an insurer must listen to its clientele. Today, a very loud message from employers and other plan sponsors is the need to constrain, or indeed to cut, costs, or conversely, to add to the services covered under such plans only after very careful consideration.
While compensation costs have been stabilized in recent years, the cost of EHC coverage has been increasing by 12% to 15% per year, on average. Most of that increase arises in the prescription drug area from increased utilization, technological advances and the introduction of new effective drugs, and from general increases in cost. Costs shifting from the public sector to the private sector have also become a significant contributor to increasing costs through constraints in provincial insurance plans.
In addition, some jurisdictions have decided to burden such plans with higher taxes. As a result, very substantial resistance to increasing costs has developed among plan sponsors. Thus, it should certainly not be assumed that additional costs from this or any other area can be imposed on private insurance plans automatically.
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Comments were made early this morning about costs being borne by insurance companies. Insurance companies are intermediaries. They do not bear the costs. They may allocate the costs among different clients, but the ultimate decision is made by the plan sponsor.
Insurance companies are certainly willing to participate in this and any further discussions regarding the potential role as intermediaries to the extent that it is decided that extra costs in this or any other areas of our health care system should be imposed on the recipients. But it must be remembered that the costs are paid by individuals or by plan sponsors, not by the insurance industry.
Thank you very much, Mr. Chairman. I'll now turn it over to Mr. Byrne.
Mr. Gerry Byrne (Liberty Health, Member Company, Canadian Life and Health Insurance Association): Thank you. Good morning.
I would like to begin by expanding or elaborating on Mr. Black's definition of the term ``formulary''. Formularies are lists of eligible drugs that become the basis for drug reimbursement in the private sector. Eligible drug lists vary from insurer to insurer and employer to employer. Liberty Health today reimburses on twenty various formularies.
Drugs listed on the formularies must be legal for sale in Canada, and therefore they do not include experimental medications. Should the subcommittee decide to recommend expanded access to experimental medications on a compassionate basis, Liberty Health would recommend that, first of all, the program be singular and mandated nationally by the federal government. In this sense, the leadership role would be federal.
Secondly, medications released for access would be identified by the federal government. We would suggest as a third point that pharmaceutical manufacturers be approached, as they may wish to play a compassionate low-cost or no-cost role. Our fourth suggestion or recommendation would be that administrative functions for eligibility and payment, if applicable, be conducted by an objective third party supplying both expertise and patient confidentiality.
Concerning the administrative function, Liberty Health is willing to assist the government with administrative expertise for compassionate access. For such support we would expect recovery of specific administrative costs associated with supplying the administration for the program. Other members of CLHIA may also be willing to be of assistance in this role or in this capacity.
Our suggestions are consistent with the process used already by Health Canada in distributing payments to Canadians infected with HIV/AIDS through blood transfusions.
We look forward to open dialogue on this issue in the roundtable. Thank you.
The Chairman: Thank you, Mr. Byrne.
Welcome, Dr. O'Shaughnessy.
Dr. Michael O'Shaughnessy (Director, B.C. Centre for Excellence in HIV/AIDS): Thank you. Good morning.
During our discussions today I will present information from two perspectives, first as a director of the Canadian HIV Trials Network and secondly as a director of the B.C. Centre for Excellence in HIV/AIDS.
The centre distributes free of charge all anti-retroviral drugs used to treat HIV disease in the province of British Columbia to the more than 2,000 individuals enrolled in the drug treatment program. These drugs are used in accordance with the centre's guidelines, but the compassionate release of drugs certainly has an impact on our program, and I'll discuss that later.
I have prepared a text but in the interests of time I will skip through it.
With respect to compassionate release, the Canadian HIV Trials Network has been working with Roche on a system for the allocation of individuals seeking access to a limited supply of the drug saquinavir under a compassionate release program. Unfortunately, saquinavir is in short supply and the drug available for compassionate release is quite limited. Hence, a lottery system for patient access to the drug has been developed.
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The CTN has been asked by Roche and their community advisory committee to ensure that the randomization process is equitable. This is not an ideal situation, but it represents an approach when a drug is in very limited supply. We are now in the process of surveying the participants of this program to gain insight into how they feel about a program, i.e. a lottery.
I have also included a breakdown of the distribution of saquinavir by province, and it's attached to the original documents.
That is not to say that distribution through a lottery system meets the expectations of the patients, because, remember, you can give out a drug to 200 individuals under this system, but there may be 1,000 applicants for the drug so that there is always a discrepancy between supply and demand.
The CTN has encouraged pharmaceutical companies to offer a parallel compassionate release program for the programs they sponsor. The CTN has corresponded and had ongoing discussions with several drug companies concerning the compassionate release of new drugs, including nevirapine and saquinavir. In addition, we have revised our protocol form to ensure that investigators approach companies about providing a compassionate release program.
The documents that I and the CTN received prior to this session posed a question to the CTN on whether federal and provincial governments have a responsibility to fund clinical trials on agents that are not of interest to pharmaceutical companies.
One of the most successful clinical trials the CTN has sponsored has been the so-called MAC study in which two treatment regimens for the treatment of mycobacterial disease were compared. The CTN and six pharmaceutical companies supported this highly original and productive study. One of the treatment arms demonstrated not only efficacy but also significant improvement in the quality of life of the participants. Without the pivotal support, both cash and personnel, of the Canadian HIV Trials Network, this study would not have happened.
When phase 2 of the national strategy was announced, we at the CTN were shocked to learn that although funding for phase 2 was higher than that allocated for phase 1, the amount budgeted to the Canadian HIV Trials Network actually decreased. Prior to the actual release of phase 2 of the national AIDS strategy, the national partner organizations unanimously endorsed the increased funding for the CTN since they believed it was important for the network to have the flexibility to initiate trials similar to the MAC study.
In addition to the shortage of money, the network encountered another distinct, major bureaucratic obstacle. Government officers indicated they were uneasy about the CTN co-sponsoring clinical trials. The argument presented to the Canadian HIV Trials Network was that this activity could put the Minister of Health in conflict since some of the drugs used in the clinical trials came under the regulatory authority of the minister.
Although the CTN receives core funding from Health Canada, it is not an arm of the government nor does it have a role in any regulatory activities of any government agency. On the other hand, in the United States the AIDS clinical trial units and groups are directly supported by the American government, and they actually pay all the costs associated with running clinical trials, from purchasing the drugs to providing laboratory tests.
A consequence of the two impediments was the absolute requirement that the Canadian HIV Trials Network restrict itself to industry-sponsored trials. This constraint limits the scope of the drugs tested and the combination of drugs that can be evaluated, because if you do a combination study, you have to get two companies to agree to work together, which is sometimes not that easy, sometimes impossible, and the sites that will be allowed to enrol patients.
It is far more economical for a drug company to enrol participants in clinical trials at two or three major sites - and they will be, believe me, Toronto, Montreal and Vancouver because that's where the patients are - than to enrol patients from small venues across the country. So you have trials, but if they're totally industry-sponsored, they will probably not occur at small sites.
The MAC trial we spoke about had 24 active sites across the country. The broad-based support for a clinical trial would never have occurred if not for the financial and personnel support the CTN committed to the conduct of the trial.
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On the other hand, drug treatment programs in Canada are greatly affected by the compassionate release of drugs for the treatment of HIV disease. For example, in British Columbia drugs are supplied free to HIV-positive individuals, but the drugs must be used in accordance with the guidelines published by the centre. The guidelines committee consists of experienced physicians, representatives of the centre, physicians who carry out clinical trials, representatives from the large community organizations in British Columbia, pharmacists and health economists. The guidelines are considered to be progressive; in fact, B.C. was the first jurisdiction to pay for combination therapy beginning in 1992.
However, the compassionate release program may not conform to the provincial standards for the use of drugs. Hence, when the compassionate provision for the drug terminates, the provincial programs are usually expected to assume the cost.
In British Columbia there were approximately 300 individuals receiving 3TC under the compassionate release program. These individuals were already receiving AZT under the Centre for Excellence guidelines, but the cost of providing the new drug, 3TC, to them is about $1 million a year. Consequently, terminating the provision of the free drug will force the guidelines committee to accept the criteria for compassionate access for inclusion into the guidelines.
In this case, the use of 3TC in combination with AZT is appropriate under the B.C. guidelines, but there are many jurisdictions in which it will not be appropriate. So in other words, 3TC will come on the market, and the people who have been using the drug and the provinces may or may not decide to pay for that. We see an inherent conflict in that.
My only job in B.C., because of the conformity to our guidelines, is now to find the money. That is probably the most difficult job - finding $1.2 million.
I have also attached to the presentation a figure that shows the increase in the number of individuals in the centre's program who have been receiving AZT alone; 48% of those individuals receive 3TC. It took a great deal of persuasion from me to get this information from industry. In other words, this figure was not forthcoming.
There are approximately 2,000 individuals across Canada who receive free 3TC. They have been provided with assurances from the company that they will receive a free drug for 30 days after the company receives full permission to sell the drug in Canada.
It's a good drug, 3TC. It was developed in Canada with, we think, a very good potential. However, the compassionate release program has created an immediate market niche for the drug, and its introduction could cost the provinces at least $6.6 million. It is obvious that the compassionate release of new drugs and the open arm in clinical trials represent advances from the paradigm that existed 15 years ago. However, the high cost of drugs, the limited availability of new drugs, and the potential impact of the introduction of new drugs on provincial drug programs necessitate that we all must find better ways to work together and communicate with each other, since so many of our initiatives are interdependent.
Thank you.
The Chairman: Thank you very much, Dr. O'Shaughnessy.
Dr. Robert Voigt (Individual Presentation): I'm a general practitioner. I see primarily people with HIV and AIDS. This issue of compassionate access to drugs is of great interest to me and of concern to my fellow physicians involved in primary HIV caregiving.
From my perspective, the situation with respect to compassionate access to drugs is worsening at an alarming rate. For patients, physicians, researchers, government, the pharmaceutical industry and insurance companies, this is both a boon and a burden. I'm alarmed at the increasing amount of data collection being done on behalf of pharmaceutical companies in order to comply with the regulations for the emergency drug release program that is being carried out by primary care physicians. Along with the work of treating patients' medical problems, there is an ever-increasing amount of paperwork involved with the compassionate access protocols.
I currently care for 45 active AIDS patients with CDC-defined AIDS and 177 patients who are HIV positive. In the last five years 161 of my patients have died of AIDS. The vast majority of these patients have been or are involved in one or more research protocols, or are on compassionate access drugs. At the moment, 96 of these are on compassionate release 3TC. It's not unusual for patients to be on two, three or more different protocols.
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In the past, physicians would access drugs through compassionate release programs only once or twice a year at most. For physicians caring for people with HIV, it's a daily practice. This is becoming a significant burden and has reached the point where the time spent filling out forms for compassionate access is equal to the time spent caring for individuals' medical needs.
Primary care physicians expend considerable effort advising patients of new trials, associated risks and potential benefits in discussing the informed consent. Add to this the repeated follow-up, administration of forms, ordering, receiving, tracking, dispensing and logging of medications dispensed and dealing with side effects and quickly the amount of time added to regular medical follow-up becomes substantial. This is all work that occupies significant time, work that is entirely donated by primary care physicians. There's no mechanism for reimbursement for these services. This is a shift away from the previous standard of having research assistants at a central base conducting patient follow-up with respect to trial drugs, and medical follow-up by physicians. This shift has been slow but progressive.
This is relevant to this discussion regarding responsibility from several perspectives. The emotional damage is significant to patients if they hear about new drugs and are unable to get them, and there are economic consequences as well. The government loses financially by paying for unnecessary visits to physicians. Primary care physicians lose inordinate amounts of time dealing with situations where they must explain why patients can't get drugs they hear about. It is not the primary care physician's responsibility to subsidize research or compassionate release.
Increasingly, there are limited numbers of places available in drug trials. Most of these trials have compassionate access arms for people who still wish to be on drugs but do not qualify for control trials. Patients and physicians often think of compassionate access as access to treatments. It is not; it is access to an uncontrolled experiment that will not provide any useful information other than adverse events.
With the information from compassionate access, there is no means of determining efficacy without a control arm. Everyone loses out with this approach. The patient loses because they start medications that have no evidence that they are superior to standard therapy, apart from the publication of a ``promising therapy'' stamp given by the media and the pharmaceutical companies. There have been and will continue to be drugs that go to trial that are inferior to standard therapy. Patients largely make decisions on which drugs to take on emotional grounds, and the new drug around the corner, which will always be the new cure, is always more appealing than the randomized trial they might get onto.
They also lose out in other areas. As was demonstrated with the AZT-ddI versus AZT-ddC comparative trial, by having a far greater number of patients taking a drug on the compassionate arm than on the trial arm, the time it takes to enrol sufficient patients for a trial to reach a significant conclusion is substantially increased.
With this particular trial, for example, it took three years to recruit sufficient numbers, but if all patients had gone on trial rather than compassionate release, there would have been an answer to the question in 18 months. This is a decidedly poor situation for patients. Some individuals were on drugs for three years without any evidence of superiority of one arm, and the outcome of this would have been demonstrated in half the time if there were no compassionate arm. This isn't acceptable.
One can argue that patients should have the right to access any treatment they wish, and I would agree with that, but I reiterate that compassionate access is to experiments, not to treatments. Can a patient have a right to be on an experiment? I believe so, but an experiment in which you will never know the outcome is a poor experiment. I would advocate for greater availability to trials, but with the trend to increased compassionate access, interest in being on trials is waning. People want the fast track to the cure, but they are deluding themselves.
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I sit on the safety and efficacy review committee for the Canadian HIV Trials Network, and we are frequently placed in a position of having to stop trials because the rate of accrual of patients is too low to ever get an answer to the question being posed. We are therefore losing out on knowing if some of the therapies we would like to use are of benefit. This is not acceptable.
The physicians also lose out. When a patient is on a trial, the pharmaceutical industry is obliged to provide staffing and funding for the data collection, monitoring and dispensing of drugs. If a patient is on compassionate access, the data collection, monitoring, stocking, tracking, dispensing of drugs is done by the physician. This is a time-consuming activity and is a service provided for free.
To give you an idea of how time-consuming this is, I now see patients only four days a week, as I need one full day each week just to catch up on required paperwork. I lose out significantly financially. My patients lose out as I'm not available to see them for their health issues, and we all lose out as the other physicians who might start seeing HIV patients get a glimpse of the bureaucracy involved and run the other way. This is not acceptable.
I propose that several steps be taken to reverse this trend. First, we have to educate the medical profession, the government, the patients and the pharmaceutical industry regarding the value of organized trials versus the real value and drawbacks of compassionate access. The responsibility for this lies with the pharmaceutical industry, physicians and the medical associations, the Canadian HIV Trials Network and the government through the emergency drug release program, and the community groups that need to provide information based on both patient needs and scientific information. The process of lotteries does not guarantee equitable access to trials and is a time-consuming process for primary care physicians.
Second, I suggest that the pharmaceutical companies absorb the cost of representatives in each major centre and have those people collect the data and do the forms for compassionate access. I believe this responsibility lies with the pharmaceutical industry, as they are the ones who ultimately will benefit financially from the drug. Government may take a regulatory role in this matter, and with respect to changes in the legislation, regarding and defining the scope of compassionate access.
Third, I recommend that a standard, single-page format be developed that will provide the necessary information for follow-up and change from the multi-page forms currently in use. This would make collection of the appropriate data less onerous and expensive. With the computerization of most medical offices, there may even be a simple means of entering this electronically and transmitting. The responsibility lies with the government, the pharmaceutical companies and the CTN, and needs input from the primary care physicians to meet their needs in optimizing the use of their time and to prevent unnecessary information from being gathered and placing an unnecessary burden on patients.
Fourth, I recommend that along with the government agencies involved, a single application for status as research centre be adopted so that the laborious process of applying for approval with the drug company, the FDA in the U.S. for U.S.-centre drugs and/or HPB...the local IRB be done and then be good for a specified period. This process of approval as a physician to dispense compassionate release drugs can take months and makes one question how a process that can take months to organize can be considered ``emergency''. The responsibility for this lies with the government, the pharmaceutical industry and the physicians.
Fifth, I recommend that the process of paying for some drugs be discontinued, as it is onerous for the patient to receive a bill for medications ordered through EDRP, without being first notified that there is a cost, and for the physician to be left with the cost if the patient either has no funds or expires. Currently, I am in this situation.
Several questions were posed on the brief I received requesting me to be here. One question asked where the responsibility for leadership lies in the effort to expand compassionate access. I'm unconvinced that this is the appropriate question. I think it should be rephrased to ask ``should compassionate access be expanded?''
As posed in the brief, I do not feel the government should take a ``legislative and command stand''; however, the cooperation of the government in adjusting legislation and to facilitate change is essential.
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With respect to the federal and/or provincial governments funding research clinical trials on agents that are not of interest to the pharmaceutical companies, I feel the governments should certainly allocate increased funds to research, but leave this to the already established research organizations. The per capita expenditure in Canada on research is dismally low compared to our neighbours to the south.
I hope it's possible to consider these suggestions, as I honestly believe that the source of subjects for trials will dry up as the physicians caring for these patients are unable and/or unwilling to be saddled with the expense of running large compassionate release programs that hinder useful information-gathering that will result in patients benefiting from research more quickly than if extended access increased.
The Chairman: Thank you, Dr. Voigt. Our next witness is Dr. Sharon Walmsley.
Dr. Sharon Walmsley (Individual Presentation): Thank you, Mr. Chairman. I appreciate the opportunity to speak with your panel again today.
I will be addressing the issue of responsibility from my perspective as a specialist in HIV. My practice is largely in patients with advanced stages of illness, and I would say that more than half of my patients are currently involved in compassionate programs.
The issue of who should pay for the compassionate use of drugs is certainly very complex. I would like to re-emphasize some of the points that were raised by Dr. Voigt, that when we're discussing this cost, we must remember that the cost is more than that of the agent. These compassionate-use programs are very time-consuming. Physicians spend a lot of time with these patients discussing these compounds, what the risks and benefits may be, and ensuring that they are making true informed consent about their involvement in the programs.
The administrative time and costs are enormous. My nurses only too happy for a drug to be approved; then the paperwork falls out of their hands. Following these patients is a large effort in terms of surveillance. If adverse events do take place, we have more duties to perform.
Last, we have to evaluate the outcomes of these programs.
All these factors serve to expand the scope of care for patients involved in these programs.
Another point I would like to raise is that all agents are not equal. Over the last few days we've been talking largely about pills. What are we going to do about intravenous therapies that patients may require as compassionate access? What are we going to do when gene therapy becomes a more scientifically acceptable process? What are we going to do then?
When we talk about compassionate release of programs, we may not be able to consider them all in the same framework, and our policies may have to be different for different types of compounds.
Also, when we're looking at non-validated therapies, we would also have to address conventional therapies. What about the newest of the anti-retroviral drugs as opposed to non-conventional therapies? We're often being faced with requests for ozone therapy, hyperbaric oxygen, and other forms of non-conventional therapies.
We do recognize that our resources are limited. We need to avoid those situations where expenditures of resources for these unproven therapies would deprive other patients of truly beneficial treatments.
The next question we might raise is, who's going to contribute to this process? In the context of clinical trials, the pharmaceutical companies or the research agency overseeing the trial usually cover the cost of agents being studied. If we view compassionate access release as an extension of this clinical trial in which we're collecting data on the safety and toxicity of these compounds in catastrophically ill patients, I think the same criteria should apply.
Many patients with catastrophic illness, whose death or disability may ensue before the results of the clinical trials are available, should not be expected to cover the costs of these unproven therapies themselves. On the other hand, if these programs were expanded to the degree that patients at earlier stages might receive these compounds, and a patient requests the therapy in this earlier stage of their disease and is unwilling to participate in the clinical trial, then perhaps that patient might be expected to contribute to the costs.
As Mr. Byrne has previously pointed out, third-party payers have traditionally used regulatory approval as a means to determine whether or not they will reimburse for these programs.
I strongly believe that patients should not be denied access to compassionate programs if they are unable to pay. In my experience, most patients in the later stages of HIV disease have depleted all of their personal resources. In Canada, the shifts are apparent and the average age of newly infected HIV patients is now 23 years. These individuals, for the most part, have not developed careers; they don't have jobs and they don't have drug plans. Many of them have been declined disability plans because of their HIV status.
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Also, in Canada, the percentage of women living with HIV is also increasing. Women often have lower incomes than their male counterparts and are often part of a family in which more than one individual might be infected with HIV and require access to these compounds.
Many patients, because of the stigma of their disease, have lost any potential financial support from their families.
I think the pharmaceutical companies should be expected to provide access to drugs through these compassionate release programs. But I think we have to be very aware of the limitations of this contribution. We know that the cost of research and development are high. We know that their long-term profits can be threatened by patent laws. If we don't have limits on these compassionate use programs the drug companies may be then unwilling to undertake clinical trials in Canada if this is an expectation, and they may switch to other parts of the world or to other interests. In either case, this is going to serve to limit access of these compounds to our patients.
Lastly, I think the government will be required to make some contribution to these programs. Perhaps some of the roles they may have would involve contributing to the research and development of the pharmaceutical companies, with the expectation that there will be compassionate release as drugs are being developed. Some of the administrative costs that we've outlined previously also require support. I think another role for government is to support research of independent investigators who are looking at products in populations or perhaps in combinations that are of lesser interest to the pharmaceutical companies.
In summary, the expectation of all the groups contributing to this process is that the use of compassionate therapies can contribute to the health of the persons living with HIV, and we hope that this could potentially decrease the overall health care costs, but it may not.
Although we need to be fair and ethical to the individual living with HIV and AIDS, I think there is also a responsibility to be fair to the population as a whole and to ensure that we use our resources wisely.
The Chairman: Thank you very much, Dr. Walmsley.
Now, from the Ministry of Health, Government of Ontario, Mr. Lance Payne.
Mr. Lance Payne (Coordinator of Special Projects, Drug Programs Branch, Ministry of Health, Government of Ontario): Thank you, Mr. Chairman. I appreciate the opportunity to participate in this morning's discussion.
In Ontario, outpatient HPB-approved medication used to treat HIV and HIV-related illnesses are covered for individuals through various programs without cost to these individuals. Through the special drugs program, which is a program that covers drugs for specific disease conditions, including those for HIV, four specific medications are covered. Individuals must meet clinically approved criteria and are enrolled through the HIV project centre.
Through the Ontario drug benefit, specific medications are provided, again free of charge to eligible recipients. The eligible recipients include those on social assistance, individuals 65 years or older, and those on long-term care and home care.
The drugs that are provided through this mechanism are those listed in the Ontario drug benefit formulary or on the non-formulary benefit list. As well, ten drugs used to treat specific HIV-related illnesses can be prescribed by physicians who treat patients with HIV or AIDS without the usual the paperwork. There is also a process for considering coverage of approved drugs that are not covered through any of these mechanisms through an individual request program.
In April 1995 the Ministry of Health implemented the Trillium Drug Fund. This program was designed to provide assistance to any Ontario resident who has high drug costs in relation to their income. The program is based on a deductible based on net taxable income, and individuals are required to pay for their prescriptions up to the required deductible.
The Trillium Drug Fund is unique and provides potential assistance for drug coverage for any individual with excessive drug costs, and it's not disease specific. I'm unaware of other provinces that have similar programs, and the ministry feels it has undertaken appropriate steps to assist all Ontarians with coverage for prescription medications.
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For all of the above programs, investigational and experimental drugs are not considered for coverage. Once drugs are approved by the federal government, manufacturers may request coverage for drugs through the Ontario drug benefit program or the Trillium Drug Fund. Additional drugs will not be added to the special drugs program.
Upon request, there is a formal review process for evaluating coverage. The ministry relies on the advice and recommendations from its expert advisory committee, the drug quality and therapeutics committee, in reviewing and determining the most suitable mechanism for coverage.
As well, there is a fast-tracking process for new chemical entities that represent breakthrough therapies.
In summary, the province relies on the HPB to approve the drug for sale in Canada and thereafter will consider coverage in relation to the various available programs. We certainly do continue to work with stakeholders, including the HIV project centre, to evaluate how to provide better coverage for HIV-related drugs to Ontario residents.
The Chairman: Thank you, Mr. Payne.
From Health Canada we have Mrs. Pieterson and Dr. Jacques Bouchard. They don't have a brief to present to the subcommittee this morning. They're here to listen to the witnesses and to participate in the roundtable...if you have any questions to be submitted to them.
Now we'll have some questions from the members of the subcommittee for the panellists, and after that we'll take a five- to ten-minute break for coffee. We're going to end with a roundtable with the panellists and the members of the subcommittee.
First question, please, Monsieur Ménard.
[Translation]
Mr. Ménard (Hochelaga - Maisonneuve): I am interested in getting more information from the Director of the Canadian HIV Trials Network. First of all, I would like to know what you think, in general terms, about the support provided by Health Canada, not only in the area of access to emergency drugs, but rather in terms of the overall approval process.
As parliamentarians, we have undertaken the task of trying to find the ideal way for Canada to approve drugs. In your brief, you give the example of saquinavir. It's a happy coincidence that you mentioned this drug since, being from Montreal, I recently also had the opportunity to take an interest in this matter. What could Canada do to "speed up" the process?
Let me rephrase my question. If you became Minister of Health tomorrow, what suggestions would you make to this committee so that the whole drug approval process could be speeded up and become more efficient? I say this with all due caution, because I know that Health Canada officials do everything in their power to meet the needs of all concerned stakeholders.
Now, for my second question, as far as I understand, you do wish that pharmaceutical companies make available, at whichever site clinical trials are conducted, a representative who would be responsible to collect the data. It seems that administration is a real problem - and others have said the same thing - because of the need to fill out forms and update requests when research protocols are set up, and also when a physician wants to have access to an emergency drug program established by a manufacturer.
Could you give us more information about that.
[English]
Dr. O'Shaughnessy: I think you asked specifically what we could do to improve the approval process in Canada. I think one of the first things we would like to see reinstituted...I say reinstituted because at one time in fact there was a big push in the health protection branch to harmonize the release program and to work with the Food and Drug Administration in the United States on the joint approval of drugs so that we wouldn't be in a situation in Canada where agents would be released in the United States and we would not have access to them in this country because our regulatory process took longer.
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A few years ago there was a great push on harmonization, and the panels that reviewed data would in fact consist of a combination of American and Canadian investigators. I thought that was a good idea. There seems to be much less emphasis on that today, and I'm not sure why that is, why they have disjointed the process and gone back to the way things were 15 years ago.
The other thing I would suggest on the approval side is that although there have been some successes of late, the process should be sped up. We're not pointing fingers at the bureaucracy. I worked for ten years in the federal system, and individuals in the system work as hard as they can. Sometimes through downsizing institutions are shrunk so that they in fact become no longer effective and able to respond to, say, the increasing number of new submissions on HIV.
So I think people need to look at the process and the manpower requirements and at all this being done in the context of shrinking dollars.
I think there really has to be a better way of exchanging information among the stakeholders, that is, the pharmaceutical industry, the government and the provinces that in fact pay for drugs.
Recently, from the B.C. perspective, we've had some problems getting accurate estimates of what the cost implications of specific drugs would be, and I would say that is an unsettling event for individuals in charge of provincial programs.
There's one other issue that perhaps is not an easy one to deal with, but it certainly relates to the approval process, and that is what is a fair cost for a drug. We have been asked to look at a drug for which the cost would be $18,000 a year per patient in Canada. We asked if we could see the rationale for these costs, because we have health care economists in the centre. When we looked at the information presented to the government, we were more than mildly surprised that regulators would approve a drug at that price based on that information. It surprised us greatly. I don't think I want to get into it much more than that except to say that when they explained the data to us, our economists and myself felt that the estimates as provided to the drug pricing board were clearly out of touch with reality.
So that's something else that could be done. I think the whole process is a little bit loose.
[Translation]
Mr. Ménard: There is one thing I would like to understand before leaving the floor to somebody else. Since this committee started working on this, the Canadian HIV Trials Network has remained, for me, a kind of fantasy entity. It's something I hear about, but that I have never really been able to put my finger on.
So, I would like to understand one thing once and for all. When a manufacturer wants to work in cooperation with the Canadian HIV Trials Network to conduct a research protocol, what are the various steps of the process? What does it imply? Tell us as if we were hearing about it for the first time. The information given to us by manufacturers does not seem to be the same as the one provided by the Canadian HIV Trials Network.
You represent the CTN. When you appeared before us, you told us that Canada is the only country in the world, I mean among industrialized countries, which finances only the infrastructure and that our contribution in this area should be 5.5 million dollars rather than 2.9 million dollars. You protested - quite rightly, I believe - the fact that the research program is driven by the pharmaceutical industry.
I would like to understand how that translates when Roche, Glaxo, Biochem or any other pharmaceutical company comes to you with a research protocol to launch a trial. What are the main steps of the process? What kind of role does the ethics committee play? How are patients involved? How do you proceed, practically speaking?
Please forgive me for using the word "fantasy" earlier; it was a slip of the tongue.
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[English]
Dr. O'Shaughnessy: All right. Well, first of all the company has to come to us, or more specifically an investigator will have an idea. A single investigator will go to a company and say, here's my idea for the treatment of whatever with, say, 3TC in combination with another drug. That proposal will be sketched out, and if the investigator gets permission from the company they will bring it to the CTN. The CTN will look at it. An application form is filled out that includes questions on scientific validity. Is the trial design correct? What ethical approvals are required for this study? What are the ethical implications? That will go.
So we have several processes that will go on almost independently. One is that the committee is reviewed by scientific merit, by a scientific review board. The second is that a proposal will be reviewed by the ethics review committee. The third is that the community advisory committee will look at a proposal and ask what the implications of this trial are to the HIV-positive person.
When all of those committee recommendations come together, if there's a pass - if there are no ethical dilemmas or no problems with the scientific merit - then it will go to a steering committee. The steering committee will discuss issues further with the community advisory on, say, compassionate release or the economic implications.
Remember, the network only has the infrastructure. We don't pay for the nurses to go to the patients and collect the data. We don't pay for the laboratory tests. The company has to pay for that. So we have to put all that information in the pot, and if an approval comes out the trial can go forward.
The most important criterion is to understand that this all begins if industry is interested in doing a trial and putting up its money to do a trial. This is not a small event; some trials probably cost the pharmaceutical industry $2 million to $5 million, especially on the newer drugs where they require 600 or 800 individuals to be enrolled. You're looking at a million-dollar drug.
So we get through the approval, we get approval and then we negotiate sites. Where is the study going to be done? As I said, there's a great tendency to want to do it in the big sites because it's cheaper if you have to hire only one nurse. Then there is the discussion about sites because it's an ethical question. It's raised all the time by the ethics committee and the community advisory committee. We address that and usually reach a compromise. The trial is most often held in more places than the industry wanted, but fewer than we wanted. Then we go forward and the study will begin.
You heard earlier that sometimes studies never finish and in fact have to be cancelled because enrolment is too slow. But let's assume it's a good study and we go forward. The study enrols the patients and the trial is completed.
One of the big advantages the network provides to manufacturers is that the network has been responsible for analysis of the data. Although we can't pay for the drug like the Americans do, and we can't pay for the lab tests because we don't have the money, we can do the analysis. We can be sure that the analysis is done in a timely fashion and is done correctly. That's a very strong advantage of the network.
That's essentially how a trial runs. I've condensed three years' worth of work in three minutes.
[Translation]
Mr. Ménard: Mr. Chairman, I don't want to take too much time, although I do, I am afraid, but after all, it's Christmas and we're very much interested in the subject. It is possible to get a list? When you came to see us, I recall you said that 40 clinical trials had been done by the CTN. Is it possible to get a list of those clinical trials and to know how many there were and how much they cost.
If I understood what Dr. Levy said, it seems that the cost of a clinical trial is between 25 and30 million dollars. It is estimated that, to complete the marketing process for an investigational drug, you could easily have to invest 250 million dollars.
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You tell us that it could cost between 1 and 5 million dollars. Of course, there have to be qualifiers. A clinical trial involving 300 people is not the same as one which involves 1,200 people, it's quite different. I would therefore like to have the average cost of the 40 clinical trials done by the CTN which, as we know, is the Cinderella of the Canadian strategy.
I have to tell you that I am in a rather privileged position since, in a few days, in January, I am going to spend a little time with Dr. Bouchard and have a look at the documents which make it possible for... I assume the invitation still stands. I would also like you to send to the clerk the documents which are used to have the clinical trials done by the CTN approved. I don't know if the costs can be made public.
The Chairman: We could ask Dr. O'Shaughnessy to give us a list of those clinical trials.
Mr. Ménard: And the costs. Without the costs, the list doesn't mean anything.
The Chairman: We'll ask him to provide this information.
[English]
Dr. O'Shaughnessy: One of the difficulties is that much of the cost is kept from us. We don't know. Unless we are some of the principal investigators, we don't negotiate the cost. We'll give you what we have, but many times we don't know exactly from industry what their total costs are.
The Chairman: Thank you, Dr. O'Shaughnessy.
Dr. Voigt.
Dr. Voigt: Your question was regarding whether I wanted to see specific things done as far as support for compassionate release is concerned. Is this correct?
[Translation]
Mr. Ménard: As I understand, you said that administration is a problem, in terms of data gathering, and that you wished pharmaceutical companies would get more involved in that area. I would like to know if my interpretation of your statement is correct and how you think all that could work.
Where is the problem for the front line physician? When you have to contact the manufacturer to have access to an investigational drug or when research protocols have to be presented in the context of the clinical trials done by the CTN?
[English]
Dr. Voigt: The compassionate access is quite different from a clinical trial. If I'm having somebody go on to a clinical trial, then I contact whoever the research person is. The patient sees them and they go through that.
My greater concern is with the compassionate access. As a general rule there is no difficulty obtaining compassionate access approval for someone to use a drug, for example 3TC or saquinavir. The difficulty once approval is granted is in having to comply with the regulations for data gathering and safety monitoring. That's the time-consuming difficulty.
We are experiencing some change in this respect, as it has always been done strictly by the physician in the past. When I and a group of five others in Vancouver became too overwhelmed with the paperwork involved for 3TC, we essentially phoned the company and said, forget it, you're going to have to send somebody here. They eventually did come up with funding to hire a nurse whose specific function was to fill out all those forms. We now have somebody working slightly more than full-time doing that.
If a drug trial has an open arm or if there is compassionate access, I think it should be required that the drug company be the one to support this, that they have staff available to go into physicians' offices, get the information and fill out the forms. It isn't strictly filling out forms; it's having to allocate drug, to track it. This is all quite a time-consuming process. This is something that can happen quite easily, strictly by their funding somebody to be there.
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Part of the reason I feel it is the drug companies' responsibility to do this is that at the moment, compassionate access has evolved so that it is no longer compassionate access; it's pre-marketing. As Dr. O'Shaughnessy says, 3TC now has a very large base of people in Vancouver who are going to be extremely angry if it's not approved and used. Likewise with saquinavir - people are really being revved up to get on saquinavir. They want to get into this lottery. It has the same emotional impact of getting your 649 ticket: did I get on saquinavir? Again, they're establishing this base of people for the drug.
We're hearing from some of the more recent conferences in San Francisco that in fact it may be bad to be on saquinavir. So here we are being pushed into getting people on a drug and being led to believe that it's something very helpful for the patients, but we may actually be doing them a disservice.
The Chairman: Thank you, Dr. Voigt.
Mr. Jackson, please.
Mr. Jackson (Bruce - Grey): Mr. Chairman, I want to thank the witnesses for being here today. There's a lot of information and a lot of good stuff flowing.
Our role here is to see what the government's role is and how we can look after the health of Canadians. We've heard from a lot of witnesses that sometimes a lot of scientific information regarding new drugs is overblown, and we've heard a lot about harmonization.
I guess my questions would both be to the department, Mr. Bouchard and his assistant, or his boss. Are we working on harmonizing all these drugs that come down the pipe with the United States and Europe and globally? Is there a mechanism to look at these things realistically and see how quickly we can harmonize rather than duplicate the work being done in the United States, France, Holland, or what have you?
Ms Beth Pieterson (Chief, Submission and Information Policy Division, Department of Health): There's regulatory harmonization going on. From our point of view, this means agreeing internationally with all the regulatory agencies what data requirements are necessary to approve a drug. That's very active. There's an international committee that Canada has...I think we have observer status on a few of the committees and we participate. They're all aimed at speeding up the regulatory process. Obviously that's our goal as much as anyone's goal. That is what harmonization means from our perspective.
Mr. Jackson: How active is it? I know we are doing that with regard to pesticides, for instance, in the farming communities, where we have a border state where the guy sprays the stuff and it goes over next door, and then we have a problem getting it in. I know this was harmonized. Are we doing a similar thing with some of the new drugs coming out?
Ms Pieterson: I don't think it's to that extent.
Mr. Jackson: Second, it seems to me that this is a very knowledge-based thing. The knowledge has to be coordinated because everybody dreams up new ideas. Sometimes people are working concurrently on these new ideas in the university community, in the pharmaceutical community, and of course those people on the ground, like Dr. Voigt. By the way, your five points were quite well taken. You tried from your perspective to indicate how best the thing could be worked out with the paperwork and whose responsibility it is. You've said that we have some pre-marketing by the pharmaceuticals, and they should take that responsibility.
How are we working out with the community? Even if we put more resources in your department, I think the key here is to stay on top of this. Do you have a regular meeting point or place where all this information comes so that you can stay on top of these new ideas and questions with regard to new drugs that might help sick people?
Dr. Jacques Bouchard (Acting Chief, AIDS and Viral Diseases Division, Department of Health): We certainly don't have formal meetings with the community on those issues.
Mr. Jackson: Should we have something like that? Should it come from the physicians? We have all these central organizations. We certainly have a lot of information. I think that both at our level and at your level, it could become very bureaucratic and very mired in all the ideas. Everybody is pushing in different directions, with people sometimes vying for the limited dollars that we have. Personally I think those are two areas that need to be looked at in order to help the processes of government.
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Dr. Bouchard: There's always a problem with potential conflict of interest. For example, if we discuss with the community and we promote in any way the use of one drug over the other, it puts us in a really difficult position when we discuss things with the pharmaceutical company.
Mr. Jackson: Okay.
Thank you, Mr. Chairman.
The Chairman: Thank you, Dr. Bouchard.
Mrs. Ur.
Mrs. Ur (Lambton - Middlesex): Thank you, Mr. Chairman.
My first question is to Dr. O'Shaughnessy, regarding the first graph in your presentation. I just want clarification. The centre graph states the percentage of total numbers of patients registered - and I should stipulate that this is in Ontario. The second portion of that graph is the percentage of total number of patients allocated. The allocation amount appears to be larger than the registered amount.
Dr. O'Shaughnessy: It's a percentage of the total of the AIDS cases, right?
Mrs. Ur: Yes.
Dr. O'Shaughnessy: In Ontario it's 40%. Next is the percentage of the total number of patients registered in tiers one and two, right?
Mrs. Ur: Yes.
Dr. O'Shaughnessy: That means they have more people with early disease.
The third one is the percentage of total number of patients allocated saquinavir. This says about 50% of the total who applied got drugs.
Mrs. Ur: Okay, thanks.
Mr. Byrne, in your presentation, you stated that formularies are a list of eligible drugs that become the basis for drug reimbursement, but that list does not include experimental medication. Would you have any data that would perhaps enlighten us as to what the cost figures would be should that be incorporated?
Mr. Byrne: Cost figures relative to which?
Mrs. Ur: If experimental medication were included, would there be an increase in the cost for the program?
Mr. Byrne: Yes, there would be. That would be very much dependent on the cost, and the cost is of course difficult to identify on experimental drugs.
Mrs. Ur: But it would be substantial.
Mr. Byrne: It could be substantial, and as Mr. Black pointed out, in terms of group insurance it is very group-specific, meaning the exposure in cost is relative to that specific group. You could have a group with, say, 200 employees and if you had three employees on experimental drugs, regardless of the cost it would then be absorbed by those other 200 people. On the other hand, if you had a group of 5,000 and had one individual on an experimental drug at a reasonable cost, the impact would be relatively insignificant. So it's a very variable issue.
Mrs. Ur: Right.
This is not directed to anybody specific, but it was mentioned this morning that there has been a denial of access to compassionate release of certain drugs. Is that a large percentage? I thinkDr. Walmsley related to that in her presentation.
Dr. Walmsley: The compassionate access programs that we've used over the past few years have criteria under which people can get them. So in terms of taking 3TC, for example, the criteria did move but initially it was a CD4 count of less than.... I forget the first time, but it was 100 and then it went to 300. So if a person had a value within that level, there was no problem getting the compassionate access.
In the saquinavir trial, on the other hand, compassionate access has been done by means of a lottery and people have been delegated to tiers based upon severity of illness. The lottery took place within the group of the most severely ill patients, and if a further drug was available it went up the scale. So in terms of patients being able to get that drug, it was dependent upon their severity of illness.
Dr. Voigt: To add to that, though, it also depends on the patients being aware of the drug, on the physician being aware of the drug, and on how willing the physician is to enrol in that process. Where I come from, some of the physicians strictly took a list of all their patients with HIV and filled out forms and sent off a lottery chit on every one of them, and some only sent it off on people they'd actually talked to about the drug.
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So then it becomes a bit of an ethical issue. Do you enter somebody's name in a prize draw without discussing it with them? That becomes quite a difficult issue with regard to these lotteries. When you have as large a number of patients as Sharon and I have, you cannot talk to every patient every time these lotteries come up.
Mrs. Ur: Thank you.
Carol Harkness, I thank you for your presentation and your brief. In it you say you believe access to experimental drugs should be granted according to a set of principles or criteria to be applied on a case-by-case basis. Who should be in charge of that?
You also say priority access should not be given to certain disease groups at the expense of others. Is that happening, to your knowledge?
Ms Harkness: Jan was to have come today, being that she represents the care practitioners and I represent the patient point of view.
We certainly have experienced, as presented in my brief, considerable difficulties with the cancer guidelines. This is strictly Ontario that we're representing. I can't really comment at the expense of other disease entities in terms of catastrophic diseases.
I could certainly discuss that with Jan and get an answer for you on that question.
Mrs. Ur: Thank you.
The Chairman: Thank you, Ms Harkness.
Mrs. Hayes, please.
Mrs. Hayes (Port Moody - Coquitlam): Thank you, Mr. Chairman.
I must admit I haven't heard all the presentations nor all the questions this morning, so you'll have to bear with me if I repeat something, but maybe it will help us all know a few things better.
In the discussion this morning - and I found it very interesting - we talked about the access to drugs both inside and outside the compassionate access programs. In both of those areas, I've heard affordability can be a very big factor to having it available to the person who needs it in some catastrophic illness situation.
Specifically to the health and life insurance representatives, you mentioned 20 million Canadians were involved in supplementary drug coverage through insurance. This is mainly through provincial plans and group work plans. I was wondering if these cover only approved drugs in all cases. I think that's what you said.
Maybe you can elaborate on that and give us some idea of the differences in the plans you know. If this does relate, say, to provincial plans - for instance, I'm from British Columbia - what is covered in B.C. in terms of HIV drugs?
Maybe I'll stop there. Could you clarify some of those questions?
Mr. Black: Perhaps I could begin.
Basically the figure of 20 million is our best estimate of the number of people covered either as plan members or as dependants, mainly the spouses and children of plan members under the group insurance plans.
The coverage of HIV-related drugs does vary from province to province but generally is covered under special provincial plans. Mr. Payne, for example, mentioned the special drug program in Ontario that covers drugs such as AZT and the Trillium plan, which was introduced a few months ago to cover the high-cost drugs, with an income-related deductible.
The Pharmacare program in British Columbia certainly is similar in that it has a significant deductible for people who are covered and it does cover all residents of the province. But once that deductible is satisfied, the Pharmacare plan meets what are often called the catastrophic drug costs. So as I said, the coverage varies greatly from province to province, and the private plans try to take that into account.
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I think it's fair to say the private or supplemental health care plans typically do cover only drugs that have been approved or a selection of drugs that have been approved. There may be exceptional cases, because, as I mentioned, each plan can be tailored quite dramatically. There may be a few exceptions, but it would be a very few. Generally it would be only approved drugs.
Gerry Byrne would have more detail on that.
Mr. Byrne: Yes, ma'am, typically only approved drugs are covered under group programs.
Some employers have a third party, an insurance company, acting as an administrator. They are called ``administrative services only'' employer groups. The employer is actually at risk.
In very rare cases, as Mr. Black pointed out, an employer will approve a drug that is not at that point legal for sale. It would be on a consent basis or a discretionary basis, but employers are very concerned about doing that for some of the reasons you heard this morning. Once they set that precedent with an individual employee, how do they refuse then a similar need, whether it be HIV-associated or similar to what Ms Harkness said earlier?
There may be issues even beyond HIV. Once you get into the discretionary area and the drug does not have a drug identification number, you're in a very dangerous precedent-setting area. In that sense, most employers are very reluctant to do so.
Mrs. Hayes: Some of the discussion was on the availability of drugs. I think it's GCSF for cancer patients, for instance, as opposed to....
Well, the stories are certainly numerous. Even in my own experience, I know patients who put second mortgages on their home in order to get the drug they feel will help. It seems a very great problem with this type of need in Canada.
Specifically to the B.C. Centre for Excellence, from what you have said, HIV drugs are distributed free of charge through your facility. Maybe I could get some clarification on this. Are these both approved and experimental drugs as needed? Could you give us some idea of the cost of the drug distribution to your facility and what the total funding of your facility is from government?
Dr. O'Shaughnessy: First of all, there is no deductible. If a person is eligible to receive any of the twenty drugs on the program, they get that drug free of charge without a deductible.
The centre has an epidemiological arm that also describes the epidemic. We know the epidemic either begins in poverty or forces people into poverty because of the nature of the disease. So poor people get it or you become poor when you get the disease because you're not able to work and have to go on welfare. There's that aspect of it.
Our budget for drugs this year I believe is $4.4 million. I'm arguing - not arguing. I shouldn't say that in Parliament. I'm discussing with the provincial authorities the impact of 3TC, and that will probably push our budget from $4.4 million to almost $6 million. You can see as a percentage the tremendous impact this one drug will have, because we've had a very liberal process in British Columbia. That's the largest component of the budget of the centre. In fact it's three times larger than any other component.
Obviously we cannot pay for drugs that are not approved for release in the country. We can only approve or pay for drugs that are released. For drugs that don't have the status of being for sale, we sometimes can facilitate their use through the EDRP by trying to help on the collection of data, but we haven't been very successful doing that. That's because, as Dr. Voigt said earlier, the requirements differ so tremendously between one company and another. So we haven't been able to help them in that respect much. We've been restricted to buying drugs that are legally for sale in Canada.
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Mrs. Hayes: Is there any movement to have this kind of facility available for other catastrophic diseases?
Dr. O'Shaughnessy: It would make sense. The centre is unique in that we use drugs according to guidelines. So there's a rationality for the use of these very powerful drugs. It's not only an economic rationality, but a rationality behind when you use it.
These are very powerful drugs. This is not the same as taking aspirin with some of these drugs. They have significant side effects. Many individuals, such as the head of MRC and those in many American governments, come to us to look at the model we have. We involve the treaters, patients, and economists in the formulation of guidelines so that there's a linkage between use, supply and demand, and cost. It allows us then to predict costs and usage to look at profiles of an epidemic.
It seems to us that this type of model of integration of all the factors involved in providing health care is an appropriate model for the future, and it could be broadened to include other entities.
Mrs. Hayes: Thank you.
The Chairman: Thank you, Mrs. Hayes.
Before we adjourn for a little coffee break, I have one question myself. It's either forMr. Schilder, Dr. O'Shaughnessy, or any of our panellists.
Given the fact that there have been reports of high drop-out rates with HIV/AIDS in clinical trials, I would like some or one of you to address the question of patient responsibility.
Mr. Schilder: There are two things there. One, in my mind, is that you're dealing in an area of drug therapies that is constantly shifting as we begin to understand more about HIV pathogenesis and as new agents come on the market.
It's really the drug of the month in terms of HIV therapies. So people are constantly saying that this month it will be 3TC, but what are we looking at next month? There's a constant wanting to shift to the best and available drug based on where a person is in their disease cycle.
The other thing is that drug trials, to some degree, encourage some of that behaviour. In the past, before compassionate access was available, you would have people sharing their drugs when there was a placebo in place. You have a very informed community that knows how to manage access within a drug trial and how to get around barriers, to some degree. I think Michael can certainly respond to that.
The Chairman: Are there any other comments?
Dr. O'Shaughnessy: I can't add much more than what Arn said with respect to patients accessing drugs through trials. But the other thing is that there is a high drop-out in HIV clinical trials because of the toxicity of these agents.
Remember that most of the trials that go on restrict the entry of patients who have significant illness. That is, they have a low CD4 count, and they're not doing well, but you're giving them agents that are toxic.
For instance, we did one study with a very high drop out rate because of the toxicity of the agent. Really, that has to be expected in individuals that have CD4 counts below 200, 100 or 50.
So there is some of the shopping that Arn mentioned, because any patient, I think, wants to protect themselves and make sure they're getting the right drug and the best drug. But also, we have to recognize that, in advanced HIV disease, the patients may not tolerate all the drugs that well, so there's a tolerability question as well.
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Dr. Walmsley: There's also the issue of the ethics of participation in trials. All consent forms clearly indicate that the patient is a volunteer, and if for any reason throughout the course of the protocol they wish to remove themselves from the trial, they have the right to do so.
In my experience, patients will sometimes leave trials because they don't perceive a benefit to themselves. They may have their own ideas as to what this drug might do for them. They may feel it should improve their quality of life; they may feel it should improve their immunological or virological markers. If they don't see that happening, they will often choose to leave the trial for that reason.
The other issue is that the field is changing very rapidly. When data come down that perhaps combination therapy is superior to monotherapy and they are participating in a trial where there is a monotherapy arm, again they may choose to leave the trial on the basis of that information.
The Chairman: Thank you very much. We're going to adjourn for five minutes.
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[Translation]
The Chairman: Please, could we start again? As I said at the beginning, we are going to have a roundtable discussion and questions can be directed by one panellist to another or by a member of the sub-committee to one of our experts. It's a very informal, very open process to get your opinions the matter at hand.
Is there a panellist who would have a question to ask to one of the other participants?
[English]
Would anyone like to ask a question to someone else?
Dr. Levy?
Dr. Levy: If it's possible at this time, I'd like to comment on Dr. Voigt's remarks before the break regarding compassionate access.
Dr. Voigt mentioned that compassionate access is a pre-marketing exercise, and I don't believe that is in fact the case. But I think it's an enlightening comment to look at who actually does drive the process and what lessons we can learn in general from this.
I think in the case of 3TC the manufacturer was under huge pressures from the physician and patient communities to make the drug available. This had huge resource implications for the manufacturer, and at certain points in time we almost didn't have enough drug to conduct the clinical research program and to provide compassionate access simultaneously. In fact, at one point we limited the number of patients enrolling in the compassionate use program to 30 new patients a week, which was all we could cope with. There was a huge outcry from physicians and patients alike that this was unfair and that we needed to find different ways to make the drug available.
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I think patients and physicians drive compassionate access, not pharmaceutical manufacturers. This points to a key issue. What does it mean for us when a program gets to the point where we have 2,900 patients receiving a drug such as 3TC through compassionate access? At that point, clearly, many people believe that it's a real therapy and we've gone beyond a situation of access to experimental research and are providing bona fide therapy.
This takes us to the heart of the matter in which we need to find speedy and efficient approval of drugs by the HPB and not regulate compassionate access, which is just a band-aid on the system.
It's interesting that we're focusing today on access for HIV/AIDS therapy, because in some ways this is the most effectively managed therapeutic area. It should be a paradigm for other therapeutic areas. We've already talked about the fact that we do have large compassionate access programs in place for HIV/AIDS and that the HPB should be congratulated on its review process, which is relatively speedy and efficient.
If we consider the average review time for the 24 new drugs that were approved by the HPB in 1994, we find at that time it was 1,142 days, which is actually an increase from the time required in 1993, which was 1,044 days. This takes into account only the time when the HPB is actively reviewing new drugs. It doesn't include the time when it considers the clock to be stopped because it has submitted questions to the manufacturer and the manufacturer is then spending time to answer those questions.
But if we consider the case of HIV/AIDS, in particular the case of 3TC, we find it was approved in only 5 months. I think the HPB and Dr. Bouchard, who is here today, should be congratulated on this. In fact I think this should be the model for other therapeutic areas.
The best access to new drugs is speedy and efficient approval. We can achieve that through harmonization of regulations so that the HPB is not requiring unique data or unique information in Canada. Other solutions should be considered: further resources, perhaps, for the HPB, or perhaps joint reviews with the FDA, as has already been mentioned earlier today.
There are valuable lessons to be learned from the HIV/AIDS therapeutic area that can be applied to all other therapeutic areas in terms of providing access to new therapies for all Canadians in a speedy manner.
Thank you.
The Chairman: Thank you, Dr. Levy.
Do you want to respond, Dr. Voigt?
Dr. Voigt: Yes. I think with the stage that we're at right now with 3TC and saquinavir, the patients are certainly driving the push towards getting compassionate access.
But you have to understand that the impetus for that comes from the releases that happen to the media regarding drugs at a very early stage. We've seen several cases where a drug has yet to be used on a human being, and a press release is issued saying ``this is the new greatest thing for HIV/AIDS,'' and that gets picked up and passed around and is reinforced within the community. I think there is some degree of misinformation that gets issued, resulting in that.
We do have to understand that appropriate treatment for HIV is in fact experimental treatment, rather than approved drug therapy. In light of that, the whole structure under which drugs are tried and in which they are dispensed to patients has to be reviewed to allow for early access to experiments - I really do emphasize that word. Although in the later stages it is the patients that drive the process, I agree that the impetus does come from physicians, but I do think the pharmaceutical industry....
The Chairman: Thank you, Dr. Voigt.
Any other comments?
Mr. Schilder.
Mr. Schilder: This issue has been recurring constantly. In my organization, many times we have people coming in to our offices in response to a piece of information when a drug company starts releasing ``promising results'' - I say that with quotation marks because the term is used so often - and people become very frantic and desperate.
To some degree, in our mind, both the researcher and the pharmaceutical company are essentially pre-marketing with this kind of release to the media. The media is very, very naive about therapeutic agents and research issues, and not educated to any extent. Really, what they are doing is raising capital in the investment community to some degree, when they excite interest through the media. I think there should be federal government guidelines restricting the kind of information that may be released before a notice of compliance is issued, and that certain standards of practice are brought into effect in this question.
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The Chairman: Thank you, Mr. Schilder.
[Translation]
Mr. Ménard: With your permission, I have four questions. The first one is directed toDr. Voigt. Forgive me for asking and please, feel free not to answer if you wish, but I believe that the question is useful for us to understand the issue as parliamentarians.
Strictly in terms of curative value, what is the potential of 3TC as opposed to saquinavir? Which one is more promising?
Some people, particularly within the Montreal scientific community, are really thrilled to hear that saquinavir might be approved soon. Some other members of the scientific community are not as enthusiastic. If you could tell us what you think about that, I believe it might be useful for us.
I am going to ask my four questions quickly, Mr. Chairman, to be done with it.
The Chairman: Are they directed to the same witness?
Mr. Ménard: No.
The Chairman: Then, Dr. Voigt, please.
[English]
Dr. Voigt: On the difference between 3TC and saquinavir, when you use 3TC along with AZT, you are presenting the virus with a combination of drugs, so if it learns how to be resistant to one, it loses resistance to the other. You will always have one drug in the person's system that the virus is sensitive to, so you will get a positive interaction there.
If the virus becomes resistant to saquinavir, which it has the ability to do within a matter of days, it then is resistant to all other protease inhibitors, whereas if you are on some of the other protease inhibitors and develop resistance, you don't have resistance to saquinavir. So individuals may take saquinavir and within a short time develop resistance, and then have nothing they can use in the protease inhibitor group.
[Translation]
Mr. Ménard: Okay. Thank you.
[English]
Dr. Walmsley: I would like to comment as well. In terms of the data that we have available for both of these compounds, when used in combination with AZT, we can raise CD4 counts for a limited period of time and decrease viral loads for a limited period of time, and that period of time is similar for both compounds. Whether that translates into patients living longer, feeling better or whatever, we don't know, and in terms of choosing one over the other, we just don't have the data right now to do that.
[Translation]
Mr. Ménard: It is interesting because some members of the medical community are urging us to press for the approval of saquinavir. Some would even like that to be done before Christmas. There was some action taken, but that's another issue.
My second question is directed to the panellist who is, by now, rather an idol of mine, the Director of the Canadian HIV Trials Network. Would you be in favour of the legislator presenting a bill in the coming months, to make it compulsory for pharmaceutical companies to give access to experimental drugs to catastrophically-ill patients?
It's an issue we cannot avoid to raise in our recommendations to the government. We will have to determine whether we wish this to be discretionary, as it is right now, or to put more constraints on pharmaceutical companies. In that case, of course, we'll have to legislate by amending the Food and Drugs Act. What do you think about that?
[English]
Dr. O'Shaughnessy: Was your question whether I think there must be a compassionate release program? Is that what I get out of that question?
[Translation]
Mr. Ménard: Perhaps I wasn't clear enough. I'm going to try again and make a complete sentence, with a subject, a verb and a complement.
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Do you think that we should make it mandatory, through legislation, for manufacturers to give access to experimental drugs? Should it be discretionary in the case of clinical trials or, on the contrary, should there be an obligation to do so, should it be mandatory, in which case, the legislative arm of the government would have to table a bill to that effect?
[English]
Dr. O'Shaughnessy: That's a very good question, and I'll give you an answer without having time to think about it.
HIV/AIDS has taught us a lot about access to drugs and involving patients in the decision-making process. If a manufacturer was going to bring in a drug for trials, if there was enough drug available so that the trial could be done and you could judge efficacy.... If there was enough drug for that and there was some other drug left over, yes, I would support a mandatory compassionate release program.
The mechanics would have to be worked out so that you could ensure the trial happened, but I think the issue would be how one could verify whether there was enough drug available for compassionate release. I think that would get right at the crux of the problem.
[Translation]
Mr. Ménard: My other question is for Dr. Levy. I believe this is the type of information which is extremely important to have.
Last week, Dr. Fry, a colleague from the government side, asked the people representing PMAC if they could give us an idea of the number of pharmaceutical companies within the association which, up till now, have participated in the compassionate access program.
As I see it, what we have to know is whether, right now, in the absence of legislation to that effect, it's a common practice, if there is no impact other than on the production of the experimental drug and if manufacturers participate as willingly as possible in the compassionate access program. The picture would be different if we knew that it's a rather marginal practice. Could you get figures on that?
[English]
Dr. Levy: I don't have any figures, but I can tell you what I found out. I talked with PMAC president Judy Erola, and she looked into this matter for me last week. Dr. Fry's question was what percentage of pharmaceutical manufacturers have an emergency drug release program in place?
We couldn't answer that specific question without surveying our members, which we couldn't do within the one-week time period. So we looked into it more informally, and it seems that all of the manufacturers endeavour to have compassionate use programs in place. That is the norm rather than the exception. We weren't aware of any big examples of a demand for compassionate release that wasn't being met by a pharmaceutical manufacturer.
Mr. Milligan: Dr. Voigt earlier stated that compassionate access programs are beneficial from a pharmaceutical manufacturers point of view. It gets physicians to get experience with the drug prior to it going to market, and it allows patients to get experience with the drug. Obviously, it's an advantage if you can get a product out in compassionate access in phase 3.
Before you have the information to provide informed consent to patients in phases 1 and 2, you don't know what the safety and efficacy are. That's an area where there's a lot of legal risk, but in phase 3, at least in the HIV/AIDS area, all companies that manufacture drugs have provided compassionate access to the extent that production allows.
[Translation]
Mr. Ménard: Last week, Dr. Levy, your colleague said something which raised quite a few questions in my mind and I would like to try and understand the situation better, particularly since we have Dr. Bouchard with us.
Among other things, your colleague talked about the possibility of improving the approval process through conditional licensing and joint HPB-FDA approvals. You will recall that I asked whether this type of practices were already used.
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Having checked, I don't believe that there is anything in the legislation about conditional licensing or that it's among common practices at Health Canada. I don't believe either that there are joint HPB-FDA approvals. Wouldn't it be two ways to vastly improve the approval process?
I would therefore like Dr. Bouchard to tell me if I am correct or not when I say that it's not possible, at the present time, to get a conditional license and that there are no joint HPB-FDA approvals. Do panellists feel that joint approval by the two concerned agencies would be desirable?
Dr. Bouchard: At the present time, there is no conditional approval mechanism for drugs in Canada. There is nothing to that effect in the Food and Drugs Act.
Mr. Ménard: What about the United States?
Dr. Bouchard: Right now, in the United States, most drugs used for HIV/AIDS are approved through a conditional approval process.
Mr. Ménard: Is it a rather common process in G-7 countries, for instance, in Great Britain?
Dr. Bouchard: Right now, in this area, the United States are the only country to operate this way. As far as I know, it's not done in any other country. May be I am wrong, but...
[English]
Ms Pieterson: I would like to add that the drugs directorate is coming out with a proposal for a new product licensing framework, and included in the proposal is a conditional licence. We are considering this. The proposal will come out at the end of the month, and you will see in it that we recognized the need for it.
[Translation]
Mr. Ménard: As I understand, getting joint approval would mean that once... In the United States, the FDA has many more human resources than we do. If they did the clinical inventory and the data analysis, couldn't Canada take advantage of the analysis they would have done already? By joint approval, do we mean, practically, that the work would be equally shared?
Dr. Bouchard: There is an impression I would like to correct because even if it is not inaccurate, it does not reflect the facts. Up until now, there have been very few joint reviews with the United States. The process has been put in place only 15 years ago; the first such review was done in 1991. As a matter of fact, I believe that it is the only time a drug has been simultaneously, as opposed to jointly, approved, by the two committees. We are talking about two totally different processes. At the moment, individual efforts are made, both in Canada and in the United States, to establish a joint review process. There is no official mechanism. Clearly, this might involve all kinds of international agreements.
Mr. Ménard: Over the past few days, you and I have both been lobbied, but this is a fact of life and there is no point in ranting and raving about it. Some people believe that if the drug is approved in the United States, it means that experts already know about the clinical data and that it has been analyzed. Couldn't that be transferred, even if on the legislation side, there is a problem?
I know that the legislation does not allow this and that the laws are different, but don't you think that, in the context of free trade and an ever greater integration, this could be a possible solution? Does that look like a direction we could take?
Dr. Bouchard: You, as legislators, will have to decide wether Canada is to remain a distinct entity in the area of drug regulation. Some countries follow the decisions made by Canada, the United States or another country to approve their drugs. There is no formal review. Do we want such a process in Canada? I am asking you.
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[English]
The Chairman: Dr. O'Shaughnessy, do you have a few comments?
Dr. O'Shaughnessy: Yes, I'd like to join this discussion.
I have attended tripartite meetings involving the United States, Canada and Great Britain where this joint review was freely discussed and was a priority of earlier government officers who sit in HPB. Dr. Bouchard correctly mentioned the ddI that was done when Sabih Khan sat as a member of the FDA panel, made the contributions, and the approvals were given simultaneously.
My question, if I can give one to you, is why has the health protection branch chosen not to continue that route when at the tripartite meetings they endorsed the policy but the practice is not a completion of the endorsement? I think that's the question. If you say to the United States government and to Great Britain, ``This is a good thing to do; we have a model that worked'', then what happened that it never happened again?
The Chairman: Dr. Levy.
[Translation]
Mr. Ménard: Personally, I would have directed this question to Dr. Bouchard. I am becoming quite a coward with age.
[English]
Dr. Levy: I think it's good that we're discussing the possibility of joint reviews. That is one way to speed up the system. I understand Dr. Bouchard's concerns that perhaps with this system Canada could lose autonomy in a key area, but I'm not sure that's the case. There are other models to consider.
For example, there's a very recent model in the European Community where each of those countries, which are more similar in size to Canada than the United States, had their own independent review agencies. They were all reviewing the same dossiers, the same huge packages of information, eventually coming up with very similar answers. They agreed that this was inefficient. They set up a joint committee that governs all of the EC states.
There are a number of ways in which this can work, with a number of mechanisms, but one of the ways is that they give different dossiers to different countries. When one country approves, reviews it, and shares that information, then the other countries go ahead and do it without repeating all of the work that would otherwise be entailed. So there are models you can use that don't give up the autonomy of the country involved.
The Chairman: Thank you, Dr. Levy.
Mr. Milligan.
Mr. Milligan: The name of the program Dr. Levy's reporting on is the rapporteur system in EC. It has improved times of review and times of approvals in Europe.
The Chairman: Thank you.
Are there any other comments?
Dr. Bouchard.
Dr. Bouchard: Have they approved 3TC yet, those countries you're referring to?
Dr. Levy: No, they have not.
Dr. Bouchard: Thank you.
The Chairman: Ms Harkness.
Ms Harkness: I have just a general comment as a consumer of the health care system. To put into perspective drugs in terms of care, I made the point earlier that hospital stays, doctors' visits and so on, many things, are covered under our provincial coverage, but drugs are not. As we move to community-based practice and community-based health care, we are closing hospitals across the country. Many illnesses - cancer, heart disease, HIV/AIDS, and many other very serious and life-threatening illnesses - are being treated more and more in the community on an out-patient basis. Drugs are a mainstay of this treatment program. They are not covered. This means individuals who have those illnesses have a clear responsibility in terms of how they can access those.
Many are ill. Many do not have the resources to pursue and find out about access to other types of information programs. They rely strictly on the health care professionals. There are support groups in the community.
So I put that forward in terms of considering a total health care package. I implore you to consider, as we move toward community health and as drug treatment programs become more and more important, that there is little additional help - in some cases, none - for many citizens in the country to avail themselves of optimal levels of care in treating their diseases.
The Chairman: Thank you, Ms Harkness.
Are there any other comments?
Dr. O'Shaughnessy: In support of that, I saw some data from the centre last week that looked at just what is the effect of all of these expensive drugs on the life and well-being of an HIV-positive person. What we found from analysing our data is that, in fact, what we've done by use of these drugs early is to prolong the productive life - significantly. So we've pushed back the diagnosis of AIDS to the right-hand side of the life curve. Following diagnosis of AIDS, the time a patient survives after diagnosis may be shorter than it used to be.
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But the point is, the diagnosis of AIDS - in other words, the critical event of total destruction of the immune system - now occurs so much later that the person has stayed out of hospital for two years longer and has used way less institutional resources. So the use of these agents may actually reduce the cost of the health system.
I would just say that's in support of the comments from the breast cancer agency. One has to look at the full picture. So it's just an isolated incident to say ``How much does the drug cost?''. What you want to know is, ``The drug cost this, but what does it cause?'' For instance, does it cause a reduction in the requirement for institutionalized services, which for AIDS is $1,500 a day in hospital?
The Chairman: Thank you, Dr. O'Shaughnessy.
Any other comments?
Mr. Black: Mr. Chairman, I'd like to speak to this point as well. I think Dr. O'Shaughnessy mentioned earlier a comment that I think is worthy of reinforcement. That is, AIDS has taught us a lot. Certainly from my perspective, in so many areas the dramatic and serious nature of AIDS has focused attention on areas that have major implications in other parts of health care, other parts of our daily procedures.
Certainly in our industry it focused attention on the needs of terminally ill individuals who may have substantial amounts of life insurance coverage that will be payable at their death but doesn't help them during the final stages of their illness. So our industry, one of our member companies in Canada, set a worldwide precedent and initiated a program of living benefits. That program is available for all illnesses. It's not restricted to AIDS, but it was AIDS that taught us that this is an important area.
In the area of health care, in terms of the comments Ms Harkness and Dr. O'Shaughnessy have made, AIDS is teaching us that drug therapy is a very important part of health care. Yet in our overall health care system we treat physician care and hospital care under the Canada Health Act while drug care and other important parts of health care are treated separately.
Maybe the message from AIDS is that we need to look at the whole health care area as one entity, as the previous speakers have said. I would very strongly support that.
The Chairman: Thank you, Mr. Black.
Any other comments?
Dr. Voigt: With regard to the issue of third-party insurance, on what I think Dr. Walmsley had said earlier, the majority of our patients don't have that. They're at the other end of the spectrum. I would guess that about 10% of my patients have insurance beyond just their basic medical coverage.
It's not that our drug distribution program isn't wonderful in B.C., but the centre of excellence really only covers things like anti-virals. I have patients who easily spend $2,000 to $3,000 a month on other medications they need. This is far more than they're getting through their disability. Very often patients have to make choices - for example, taking medication today for thrush at $10 a pill, or eating today.
So I think one has to really factor in the whole question of the other aspects of what allows a person to be healthy with HIV. There are good studies that show basic things like nutrition and housing are important, but we're spending way more on the drugs than we are on feeding these individuals.
The Chairman: Thank you very much, Dr. Voigt.
[Translation]
With this, we put an end to this morning's roundtable. With the help of witnesses, we are going to do a draft summary of the evidence presented at the roundtable, most likely, during the third week of February; then, we will table our report with the Standing Committee on Health and, later on, with the House of Commons.
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I would like to thank all our panellists. This discussion has been both interesting and enlightening. Thanks to each and everyone of you, Merry Christmas and Happy New Year.
Mr. Ménard has a motion to present to the sub-committee.
Mr. Ménard: I think that, like me, you recognize that the roundtable format has very positive results. This is the reason why I propose that we organize a similar roundtable on the specific subject of HIV research programs.
I'm not going to read my motion. I believe you all have it and that I have some support on the government side.
The Chairman: Mr. Ménard, we support the idea of authorizing our research staff to look at this issue and present us, in February or March, at the beginning of the next session, with a draft working plan.
Mr. Ménard: So, we agree that we have to get the scientific community together to establish the parameters of a research program, and that we cannot present a report if we don't know exactly what are the needs of that community. I agree that the research staff should present us with a work plan, and I draw your attention to the last part of my motion where it says that this roundtable should be organized before April 1996.
The Chairman: I hope that this deadline is not carved in stone. You understand that there is a third issue we have to examine, according to the request made unanimously by the House of Commons. It's the issue of poverty and discrimination against HIV/AIDS patients. We have to take this into account.
However, I agree we should look at the idea. We're going to ask our research staff to prepare something and if half a day or two half days were sufficient, it would be possible to include that in our working sessions. I think it should be done.
Mr. Ménard: Mr. Chairman, you sound reassuring.
Mr. Chairman: Thank you. Once again, I would like to thank our witnesses. I wish you a Merry Christmas and all the best for the coming year. Thank you very much.
The meeting is adjourned.