Health Canada has established a Special Access Programme (SAP) for physicians to apply for access to unlicensed medicines. The premise of the program is to allow for compassionate release of unlicensed products in special circumstances, usually for emergency release in serious or life threatening conditions where convention treatments have failed, unsuitable or unavailable. While the program is well intentioned, there are limitations and considerations if one decides to follow this route of access.
The mandate of the Special Access Program is to consider requests for medications that are not marketed in Canada, that they have not received a Notice of Compliance (NOC). These medications may be pharmaceuticals, radiopharmaceuticals, biologics or natural health products. They may also be drugs under regulatory review in Canada or drugs withdrawn or discontinued from the Canadian market. Drugs in shortage, needed for emergency preparedness or orphan drugs may also be accessed via the SAP as well.
Here are Top 5 things to know:
- Inadequate Efficacy Data or Lack of Incentive – a new medication may not be approved for use because the clinical trials are still ongoing and that, the efficacy of the drug may not be fully established. On the other hand, medications may have robust efficacy data but they may not be available in Canada because the market is too small for the investment by the manufacturer to bring the drug into the Canadian market.
- Inadequate Safety Data – If there isn’t enough efficacy data, safety data is also lacking. In addition, the safety profile of a new medication often requires post-marketing surveillance at a population level. If the medication is only available on a limited basis via SAP, it may not have sufficient experience to establish an accurate safety profile. Having said that, the SAP would approve an unlicensed medicine to the practitioner with the understanding that he or she agrees to report any adverse drug reactions to the manufacturer.
- Unregulated Pricing of Medicines – A licensed prescription product is regulated by PMPRB – Patented Medicine Pricing Review Board which applies a number of guidelines and principles to the pricing of a new medication. One of the criteria is that the list price must be consistent with the median price in seven comparators (France, Germany, Italy, Sweden, Switzerland, United Kingdom and United States), rendering the pricing not to be excessive or unreasonable. However when a product is available through the SAP, the manufacturer will not be required to subject the pricing via this Pricing Review Board. In some cases, the manufacturers may choose to release the medication free of charge, on a compassionate basis. However for a new medication that is still at the investigation phase via clinical trials, they may choose to charge a price that isn’t regulated by any means.
- Lack of Conventional Reimbursement Assistance – By the same token, a product that is only available through the SAP may be expensive and difficult for the patient to afford. Unfortunately, it will not be considered for reimbursement assistance via the usual channels (e.g. private insurance coverage, Trillium Drug Programs) because it is an unlicensed medication.
- Unregulated Manufacturing Practices – When an unlicensed product is authorized for sales via the SAP, the medication is exempted from all aspects of the Food and Drugs Acts and Regulations. This means that the usual mandatory requirements do not apply to ensure the usual quality standards of the final product (e.g. not required to meet the Good Manufacturing Practices or other mandatory labeling requirements). If the medication is currently available via clinical trials, it may be required to follow good clinical practices. Having said that, most reputable manufacturers usually ensure these unlicensed products still meet some quality standards even though they are not mandated by the Food and Drugs Acts and Regulations.
For more information about the Special Access Programme, click here for the official website.
Note that to apply to the Special Access Programme for an unlicensed product, the application must be completed by a physician to provide all supportive information and / or argument for use. If the product is approved, it must be delivered to either a hospital or a physician office (e.g. not a retail pharmacy).
Do you have any experience with the Special Access Programme that you would like to share?
Thank you for reading my post.