Health Canada’s Special Access Program (SAP) administers the “SaleFootnote of New Drug for Emergency Treatment” in Part C Division 8 of the Food and Drug Regulations (FDR). These regulations provide authorities that allow drugs unauthorizedFootnote for sale in Canada to be requested by practitioners for the treatment, diagnosis or prevention of patients with serious or life-threatening conditions in circumstances where all marketed alternatives have been exhausted and sufficient evidence supports the specified use. This guidance document is intended to provide assistance to practitioners and manufacturers who use the SAP by explaining the regulatory information requirements and conditions to be met depending on the nature of their request.
Health Canada is authorized under the Food and Drugs Act (FDA) to regulate the safety, efficacy and quality of therapeutic products, including drugs (pharmaceuticals, biologics and radiopharmaceuticals), natural health products and medical devices. Prior to market authorization of a drug, access is usually limited to clinical trials sponsored by a manufacturer or research organization, and authorized by Health Canada through a clinical trial application. On those occasions when a drug is not available through enrollment in a clinical trial, Health Canada may allow an exemption from the FDA and the FDR to permit the sale of an unauthorized drug for a medical emergency.
Special access by Canadian health practitioners to unauthorized drugs is intended for serious or life-threatening conditions where conventional therapies have failed, are unsuitable, or are unavailable either as marketed products or through enrollment in clinical trials. Emergency access should be exceptional and where possible, open label or compassionate access trials should be incorporated into drug development plans to meet the needs of patients not eligible for enrollment in other pivotal trials.
The regulatory authority supporting the SAP is discretionary and a decision to authorize or deny a request is made on a case-by-case basis by taking into consideration the nature of the medical emergency, the availability of marketed alternatives and the information provided in support of the request regarding the use, safety and efficacy of the drug. If an authorization is granted, the practitioner agrees to report on the use of the drug including any adverse drug reaction encountered with such use and, upon request, account for all quantities received.
The SAP is neither a mechanism to encourage the early use of drugs nor is it meant to circumvent clinical development of a drug or regulatory review of a submission for marketing. Access to any drug through the SAP should be limited in duration and quantity to meet emergency needs only. In the event that a drug submission is under regulatory review, access will continue to be limited until that review is complete and the drug is marketed.
Drugs accessed through the SAP do not undergo the scrutiny of a benefit-risk assessment that is part of the regulatory framework applied to new drug submissions or clinical trial applications. Accordingly, an authorization through the SAP does not constitute an opinion that a drug is safe, efficacious or of high quality. Furthermore, an authorization through the SAP does not compel a manufacturer to sell a drug.
Prior to selling a drug in Canada, a manufacturer is required to submit information on a drug’s safety, efficacy and quality and to comply with the provisions of the FDA and the FDR, including obtaining market authorization from Health Canada. The FDR require that manufacturers of new drugs submit substantial evidence of a new drug’s clinical efficacy and safety for the purpose and under the conditions of use recommended. This type of evidence can only be obtained through the conduct of clinical trials in humans. It is a manufacturer’s decision on whether market authorization for a drug will be pursued in Canada. As the development of a drug and its market authorization can take a substantial number of years, it may be many years before a drug is available to patients. Therefore, a regulatory mechanism is needed to allow drugs to be made available for medical emergencies when the drug is not available in Canada and other therapeutic options have been exhausted.