What if the medication that could improve or possibly save your life was just out of reach—available to others—but not to you? It’s a reality that for many patients with rare and difficult to diagnose conditions. They may have exhausted all available commercial therapies for their disease, they may not have access to a clinical trial, or there may not be efforts by the manufacturer to bring the drug to the Canadian market. In some cases, patients find themselves travelling to the United States or Europe to gain access.
It is important to note that clinical trials are the preferred and optimal way in which to gain access to an unlicensed medicine. Patients who qualify to enroll in trials can obtain access to investigational medicines through their participation in the trial. However, some patients cannot participate due to geographical limitations, ineligibility based on the patient enrolment criteria, or there simply may not be a trial available in Canada for a particular medicine.
In the event that enrollment in a trial is not possible, there are other pathways through which a patient can access a much needed medicine. The process of obtaining treatments that are not currently available through the normal channels can be confusing for many patients and their families. In this toolkit, we will introduce what access programs are, how they work, and what patients and their families can do to further explore access options, specifically in Canada.
What is “Named Patient Access” or “Compassionate Use?”
In most countries, regulations exist that allow for access to unlicensed medicines for patients with no alternative treatment options. Broadly referred to as named patient access or compassionate use, the names of these programs vary by country. For Canada, this narrow, request-based regulatory exemption is known as a Special Access Program (SAP), and it is intended to allow for a legal pathway to obtain access to an unlicensed medicine or medical device outside of the clinical trial or commercial setting.
The Special Access Program addresses individual patient need and does not have any provision to treat groups of patients. Therefore, physicians need to make an individual patient request to the country health authority, known as Health Canada, each time they encounter a patient who they would like to treat with an unlicensed medicine. The physician needs to gain individual approval on a per patient basis and further obtain approval for any re-supply of the medicine that is needed over the course of the treatment.
As background, Health Canada is the government department within Canada that is the equivalent to the United States Department of Health and Human Services. The Special Access Program, regulated by Health Canada, was established to provide healthcare practitioners discretionary access to drugs and medical devices that are unavailable in Canada. These could be medicines that:
• Are still in clinical development;
• Are not currently approved or commercially available;
• May never be commercially available, but may still offer benefit for certain populations; or
• Are approved in other countries but not Canada.
In Canada, and under the Special Access Program, an unlicensed medicine is permitted for use only under specific conditions. Generally, access to unapproved medicines is restricted to the treatment, diagnosis or prevention of serious or life-threatening conditions when conventional therapies have failed, are unsuitable, or are unavailable.
Medicines requested through the Special Access Program may not be available anywhere in the world or could be commercially available in one or more countries outside of Canada. It is important to note that in all cases, it is the drug manufacturer or drug wholesaler who must first agree to make the unapproved drug available, and neither is under any obligation to comply with a request from a healthcare provider. Further, Health Canada has the ability to reject a request for access to an unlicensed medicine even if the manufacturer has agreed to supply it to the healthcare practitioner for use with the patient.
For Patients in Canada
Heath Canada has established a Special Access Program (SAP) that permits importation of unlicensed medicines to treat patients with unmet medical needs. The program provides:
• Specific patient criteria to identify exactly which patients are eligible for access;
• Requirements for the manufacturer and the treating physicians; and
• Safeguards to protect patients since these medicines have not been approved for commercial use in Canada.
As previously stated, access to an unlicensed medicine through a clinical trial always is the preferred route. Therefore, the Special Access Program cannot be used in lieu of entering a clinical trial, and is to be utilized where clinical trial participation is not possible for the patient in need.
In all cases, the drug manufacturer or drug wholesaler has the final word on whether the drug will be supplied. Moreover, they have the right to impose certain restrictions or conditions on the release of the drug to ensure that it is used in accordance with the latest information available. For instance, the manufacturer may restrict the amount of product released, request additional patient information, determine charging requirements, and place conditions on shipping arrangements.
In addition, an authorization from Health Canada to import an unapproved drug is limited to a six month supply for chronic treatments. Subsequent access to the same drug for the same patient requires a renewal request submission to the program.
Special access to unlicensed medical devices is also restricted. Patients may access custom-made and unlicensed medical devices for emergency use or when conventional or approved devices have failed, are unavailable, or are unsuitable to provide diagnosis, treatment, or prevention. However, custom-made devices (i.e. devices created for a particular patient or use by a practitioner on a practitioner’s orders) are a special case.