441-02551 (Health)

The Government of Canada is working to provide Canadians with earlier access to drugs intended for the treatment, prevention or diagnosis of serious, life-threatening or severely debilitating diseases or conditions. To that effect, Health Canada has policies in place, such as the Priority Review of Drug Submissions Policy and the Notice of Compliance with Conditions Policy.

The Priority Review of Drug Submissions Policy has been used as a mechanism to expedite the review of new drugs and new drug indications for the treatment of rare diseases, including heritable diseases, such as cystic fibrosis, Rett syndrome, spinal muscular atrophy, and others. This policy expedites the drug submission review time from 300 calendar days to 180 days. This policy applies to drug submissions that are filed to seek or update market authorization for a serious, life-threatening or severely debilitating disease or conditions. The submissions must provide substantial evidence of clinical effectiveness that the drug provides:

• effective treatment, prevention or diagnosis of a disease or condition for which no drug is presently marketed in Canada; or,
• a significant increase in efficacy and/or significant decrease in risk, such that the overall benefit/risk profile is improved over existing therapies, preventatives or diagnostic agents for a disease or condition that is not adequately managed by a drug marketed in Canada.

The Notice of Compliance with Conditions (NOC/c) Policy allows Health Canada to provide earlier market access to potentially life-saving drugs under a strict set of conditions. For example, sponsors of drugs authorized under the NOC/c Policy must agree to carry out additional clinical trials to verify the clinical benefit of the drug and must also undertake increased monitoring of the drug, among other conditions specified in the NOC/c. Consideration for NOC/c status is given to eligible drugs that have demonstrated promising clinical effectiveness in clinical trials. Once a sponsor provides Health Canada with satisfactory evidence of the drug's clinical effectiveness, the conditions associated with market authorization will be removed.

The approval of a new drug or a new drug indication for a currently marketed drug is reliant on receiving an application from the sponsor of the drug. In some instances, companies have chosen to file new drug submissions or revisions to drug indications to other jurisdictions before filing to Canada. In the absence of a submission from a sponsor, Health Canada cannot authorise a therapeutic product, regardless of whether the product is authorized elsewhere. When a sponsor decides that they would like to market their drug in Canada, they file a submission to Health Canada. Health Canada conducts a thorough review of the submitted information, evaluating the safety, efficacy and quality of data to assess the potential benefits and risks of the drug. If, at the completion of the review, it is concluded that the benefits outweigh the risks and that the risks can be mitigated, the drug is given marketing authorization.

In filing a drug submission in Canada, the Food and Drug Regulations require the provision of substantial evidence and do not exclude any particular type of evidence. The regulations require that a new drug submission includes detailed reports of the tests made to establish the safety of the new drug for the recommended purpose and conditions of use, and substantial evidence of the clinical effectiveness of the new drug for the purpose and the conditions of use recommended. 

Health Canada accepts clinical trial data, patient data (real-world evidence), and in vitro data in considering evidence of efficacy and safety of drugs. The merits of such data is considered on a case-by-case basis. In cases where the rarity of a condition or specific underlying mutations precludes conducting clinical trials with a meaningful number of patients, in vitro evidence may be used as a viable alternative for demonstrating drug efficacy. The acceptability of any evidence is based on factors such as scientific rigour used in producing the data, adherence to the principles of Good Laboratory Practice, and demonstration of clinical relevance.

Health Canada understands the particular needs and challenges of Canadians with rare diseases and has made a commitment to improve access to medications that treat these conditions.

Currently there are a number of regulatory mechanisms in place to support access to drugs for rare diseases, in addition to the aforementioned Priority Review and NOC/c policies.

One of the regulatory mechanisms in place is pre-submission meetings. Understanding that there are particular challenges associated with designing and conducting trials in populations with rare diseases, Health Canada encourages sponsors to request regulatory advice from the Department before filing a clinical trial application or New Drug Submission. This helps to ensure that clinical trials are designed in a manner that enables Health Canada to make efficient regulatory decisions.

Another regulatory mechanism is increased domestic collaboration. Recognizing the complex and often lengthy process of bringing a drug to market, Health Canada is increasing domestic collaboration through initiatives such as Aligned Reviews.This enables the Department to work with Health Technology Assessment (HTA) organizations, such as Canada’s Drug Agency (CDA) and Quebec’s Institut national d'excellence en santé et services sociaux (INESSS) to help reduce the time between Health Canada’s market authorizations and the HTA reimbursement recommendations.

A third regulatory mechanism in place is the use of foreign reviews. Foreign regulatory counterparts with a similar regulatory system to Health Canada provide a wealth of information to take into consideration when reviewing the safety, efficacy, and quality of health products. Increasing the use of reviews and decisions of trusted foreign regulators can expedite Health Canada’s reviews.

Another is the use of incentives for Market Authorization Holders (MAH). Health Canada is providing an eight-year period of market exclusivity in Canada through data protection provisions for innovative drugs. An additional six-month pediatric data protection extension is applied if an innovative drug sponsor includes (as per the applicable terms) in their submission the results of clinical trials that increase knowledge about the use of the drug in pediatric populations. Health Canada is also providing measures for small business fee reductions for drug submissions, to minimize the impact of fees on small businesses making their first submission to Health Canada.

Health Canada is also working diligently on a number of regulatory modernization initiatives and on international harmonization. The Department’s broader Regulatory Innovation for Health Products agenda aims to provide regulatory flexibility to support innovative research and health product development, including for drugs for rare diseases, and to help regulate the risks, benefits and uncertainties of more diverse and complex products. Health Canada is working extensively with international partners (such as the International Conference on Harmonization) to achieve greater regulatory harmonization worldwide and support faster access to safe, effective, and high-quality health products in Canada.

A sixth regulatory mechanism in place is the Special Access Program (SAP). Under the SAP, Health Canada considers and grants requests for access to drugs that are unavailable for sale in Canada, which are received from practitioners treating patients with serious or life-threatening conditions when conventional treatments have failed, are unsuitable or unavailable.

Additional details concerning Health Canada’s regulatory approach to orphan drugs for rare diseases can be found at Canada’s regulatory approach to drugs for rare diseases: orphan drugs.