December 20, 2013
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Contact: SAP Drugs
Our file number: 13-119188-196
This document provides guidance on access to unauthorized drugs through the SAP and clarifies the mandate, intent and scope of the Programme. It outlines the process to be followed when requesting a drug through the SAP, as well as the information required to comply with Sections C.08.010 and C.08.011 of the Food and Drug Regulations.
This document has been updated as a result of amendments to the Food and Drug Regulations published in Canada Gazette Part II on June 19, 2013. The Regulations Amending Certain Regulations concerning Prescription Drugs (Repeal of Schedule F to the Food and Drug Regulations) provides for the repeal of Schedule F and incorporation by reference of a list of prescription drugs. This regulatory amendment comes into effect on December 19, 2013.
As a result of this amendment, a number of existing Guidance Documents have been identified that make reference to Schedule F and the regulatory process for assigning prescription status. Due to the replacement of Schedule F with the Prescription Drug List and the replacement of a regulatory process with an administrative process, the identified Guidance Documents required updating.
The Document Change Log has been added to reflect the changes.
Questions or concerns related to the Guidance Document for Industry and Practitioners - Special Access Programme for Drugs should be directed to:
Guidance documents are meant to provide assistance to industry and health care professionals on how to comply with the policies and governing statutes and regulations. Guidance documents also provide assistance to staff on how Health Canada=s mandates and objectives should be implemented in a manner that is fair, consistent and effective.
Guidance documents are administrative instruments not having force of law and, as such, allow for flexibility in approach. Alternate approaches to the principles and practices described in this document may be acceptable provided they are supported by adequate scientific justification. Alternate approaches should be discussed in advance with the relevant program area to avoid the possible finding that applicable statutory or regulatory requirements have not been met.
As a corollary to the above, it is equally important to note that Health Canada reserves the right to request information or material, or define conditions not specifically described in this guidance, in order to allow the Department to adequately assess the safety, efficacy or quality of a therapeutic product. Health Canada is committed to ensuring that such requests are justifiable and that decisions are clearly documented.
This document should be read in conjunction with the accompanying notice and the relevant sections of other applicable guidance documents.
|Change||Nature of and/or Reason for Change|
|1) December 19, 2013
Revision in Appendix A
|Changes were made to the document to reflect an amendment to the Food and Drug Regulations that replaced Schedule F with Prescription Drug List.|
To ensure that requests for special access to unauthorizedFootnote 1 drugs are received, processed and decided upon effectively, consistently, and in accordance with sections C.08.010 and C.08.011 of the Food and Drugs Regulations.
Health Canada is authorized under the Food and Drugs Act to regulate the safety, efficacy and quality of therapeutic products, including drugs (pharmaceuticals, radiopharmaceuticals, biologics and genetic therapies), 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 enrolment in a clinical trial, Health Canada may allow an exemption from the Food and Drugs Act and its Regulations to permit the saleFootnote 2 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 Special Access Programme (SAP) considers requests from practitioners for access to unauthorized drugs for treatment, diagnosis, or prevention of serious or life-threatening conditions when conventional therapies have been considered and ruled out, have failed, are unsuitable or unavailable. The regulatory authority supporting the programme 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 access is granted, the practitioner agrees to report on the use of the drug including any adverse events 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 should be limited until that review is complete. Manufacturers should anticipate exceptional demand for a drug and, where possible, incorporate open-label or compassionate access clinical trials into their development plans to meet the needs of patients who might be ineligible for enrollment in other pivotal trials. Drugs accessed through the SAP do not undergo the scrutiny of a benefit-risk assessment provided within the regulatory framework applied to new drug submissions or clinical trial applications. Accordingly, authorization through 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.
This guidance document is intended to clarify the mandate, intent and scope of the SAP and outline:
For the purposes of this guidance document, "drugs" include pharmaceuticals, radiopharmaceuticals, biologics and natural health productsFootnote 3. It excludes medical devicesFootnote 4, veterinary drugsFootnote 5 and active pharmaceutical ingredients (APIs)Footnote 6.
The regulatory authority to permit the sale of unauthorized drugs for a medical emergency was established in 1966 through an amendment to the Food and Drug Regulations. For many years, this authority was initially administered by the Emergency Drug Release Programme (EDRP) within Health Canada's former Health Protection Branch. The original purpose of the EDRP was to provide access to unauthorized drugs for medical emergencies on a case-by-case basis. In the 1990's, an internal evaluation of the EDRP found that the program was increasingly being used as a means to obtain broad access to drugs that were in the later phases of clinical trials or in the new drug submission review process. Consequently, the Programme's interpretation of the term "medical emergency" was expanded to include serious or life-threatening conditions and the EDRP was renamed as the Special Access Programme (SAP).
Requests are received by the SAP from practitioners seeking authorization for the sale of an unauthorized drug for their patient(s). Following careful consideration of the request, the SAP may either authorize a manufacturer to sell a drug to a practitioner, request additional information from the practitioner or deny the request.
The SAP undertakes the following risk management activities:
Information pertaining to the management of individual requests is outlined in Section 4.
The SAP reviews and tracks all Adverse Drug Reaction (ADR) reports submitted by either the practitioner or the manufacturer. In the case of a serious and unexpected ADR, the SAP will contact the manufacturer and recommend that information available on the drug be updated accordingly. The SAP may also contact the practitioner in the event of serious and unexpected ADRs.
The practitioner initiates a request and ensures that the decision to prescribe the drug is supported by credible evidence. Such evidence is usually available from the manufacturer in the form of an investigator's brochure, prescribing information from another jurisdiction, or a publication in the medical literature.
It is recommended that practitioners provide their patients with information about the drug's potential risks and/or benefits as well as alternative therapies available. It is also recommended that practitioners seek informed consent from their patients.
The practitioner is responsible for reporting on the results of the use of the drug in the medical emergency, including any adverse drug reactions encountered. The practitioner must also, upon request, provide an accounting for all drug supplies received.
Following authorization of a request by the SAP, the manufacturer is responsible for deciding whether or not to sell the drug. A manufacturer is under no obligation to sell an unauthorized drug and the SAP cannot compel a manufacturer to do so. A decision to invoice for a product authorized by the SAP rests with the manufacturer. Manufacturers are responsible for determining price, if any, and may consult the Patented Medicines and Pricing Review Board (PMPRB) in this regard if necessary.
The manufacturer may impose conditions on the sale of a drug to ensure that it is used in accordance with the latest information available. For instance, the manufacturer may restrict the amount of the drug sold, request further patient information, or offer a protocol for the use of the drug. Manufacturers are also responsible for providing all relevant information, such as an Investigator's Brochure, to requesting practitioners.
Foreign manufacturers are responsible for ensuring that they meet the regulatory requirements of their own country with respect to the export of drugs to Canada, especially in the case of a controlled drug. In addition, Health Canada's Office of Controlled Substances must issue an Import Permit to the manufacturer. This permit allows the drug supplies to be shipped without incident into Canada and ensures that all appropriate authorities are so notified.
Manufacturers should clearly display the SAP Letter of Authorization with other related documents, such as export permits, to facilitate clearance by the Canada Border Services Agency (CBSA).
Manufacturers are expected to ensure that significant new information respecting the safety, efficacy and quality of drugs released under the SAP is made available to practitioners and the SAP expeditiously. Should new information about a drug become available in other jurisdictions, this information should be vetted through the SAP prior to communication with practitioners.
To initiate a Special Access Request, practitioners must complete one of the following Special Access Request (SAR) Forms.
The Special Access Request (SAR) Form, Form A, should be used when the practitioner is requesting patient specific access to a drug for one or multiple patient(s) when required for immediate use or in anticipation of use in the short term.
The SAR Form for Future Use, Form B, should be used to request access to a drug is required on hand in anticipation of patients presenting with a medical emergency. The practitioner should include a clinical rationale as to why it is required on hand as opposed to requesting it for specific patients.
Both forms and their associated instructions may be accessed and downloaded from the Health Canada website.
Completed forms should be faxed, or sent by mail to:
A cover sheet is not required for forms sent by facsimile. Telephone requests should be reserved for life-threatening situations requiring immediate attention. By telephone, practitioners should be prepared to provide all of the required information using the form as a guide.
To place a request outside of the SAP regular office hours (please refer to Section 5), the On Call officer should be contacted.
The On Call officer can be reached by calling the regular business line (613-941-2108) and pressing 0. The officer will either answer directly or return the phone call within 20 minutes. The officer will determine and discuss how the request will be processed. If authorization is granted, the officer will endeavour to contact the manufacturer immediately or before the next business day. While many manufacturers have on-call services, not all are equally accessible. In circumstances where a manufacturer does not offer an On Call service, processing of the request may be delayed until the next business day.
Practitioners should submit a completed SAR Form to the SAP the following day.
Most requests are processed within 24 hours of receipt. However, given the mandate of the programme and the volume of requests received, requests are triaged to ensure that urgent matters take precedence over less urgent matters. For example, requests for blood products and certain antibiotics are given priority. Screening includes ensuring that: all sections of the form are complete; the information provided is legible; a quantity of 6 months or less is requested; the practitioner has provided their license number, and the request is signed and dated. Once a request is screened, it is forwarded to an officer for review.
Consideration is the process by which the SAP decides whether authorization is appropriate and justified. Each request represents a unique set of circumstances and is supported to varying degrees by information provided by the practitioner. Consideration takes into account and balances the following factors (Figure 1. Request Consideration Matrix) to ensure that an emergency exists and there is credible data to support the request:
The SAP will consider requests for drugs that have received a negative decision (ie, NOD/Wor NON/W) following the review of a drug submission by Health Canada or another regulatory jurisdiction provided that:
These steps ensure that requesting practitioners and their patients are aware of all relevant information respecting the drug required to make an informed decision about its use.
The SAP will consider authorizing access to drugs following compliance action provided that:
In circumstances where a drug is in short supply or is discontinued from the market, the SAP will consider authorizing access to an alternative source of an otherwise marketed drug in circumstances where:
Following consideration of the SAR, the SAP will either authorize or deny the request. Authorized requests are sent by facsimile to the manufacturer and a copy to the practitioner.
SARs that are denied are returned promptly by fax to the practitioner with explanation. The SAP may also contact the practitioner by telephone to discuss the reasons for denial and if the practitioner may have any recourse such as submitting a new request with additional information.
The SAP operates 24 hours a day, 365 days a year. Regular business hours are weekdays from 8:30 am to 4:30 pm Eastern Standard Time. Outside of regular business hours and during statutory holidaysFootnote 7, an On Call service is available.
Practitioners agree to report to the manufacturer and to the SAP on the use of a drug and any adverse drug reactions (ADRs) encountered. The use of a drug should also be reported by practitioners using the "Patient Follow-Up Report" form found on the Health Canada Website. Reporting should be on a patient by patient basis.
The SAP has adopted the International Conference of Harmonization (ICH) guidelinesFootnote 8 to be followed for ADR reporting in regards to what should be reported and the associated timeframes. Specifically, the practitioner shall inform the SAP of any serious unexpected adverse drug reaction within 15 days after becoming aware of the information if the reaction is neither fatal nor life threatening and within seven days after becoming aware of the information if it is fatal or life threatening. ADRs should be reported using the Council for International Organizations of Medical Sciences (CIOMS) forms and sent by facsimile to the SAP (please refer to section 3 for contact information).
Reports from use other than through the SAP, both national and international, should not be reported.
Consistent with the conduct of clinical trials in Canada, it is recommended that the practitioner maintain all records for a period of 25 years, in a manner that permits rapid retrieval if necessary. At any time the SAP may request that practitioners account for all quantities of drugs received under the auspices of the SAP.
The manufacturer is required to maintain complete and accurate records of all SAP transactions in a manner that permits rapid response to specific requests to verify the distribution of drug supplies to practitioners.
The SAP maintains electronic and paper records of all Letters of Authorization and Denial issued and all paper records of authorized and denied requests. In addition, the SAP keeps electronic records of requests that are returned as incomplete.
As a general rule, unused supplies of a drug should be returned to the manufacturer. Indeed some manufacturers require and enforce this policy. However, practitioners may request that unused supplies of a drug be transferred to a new patient by submitting a SAR and indicating the quantity to be transferred.
In accordance with section 3 of the Food and Drugs Act and section C.08.002 of the Food and Drug Regulations, advertising of unauthorized drugs accessed through the SAP is strictly prohibited.
The term "unauthorized" used throughout the document implies that sale of the drug has not commenced, pursuant to C.01.014 or that the product has been discontinued or removed from the market pursuant to C.01.014.6 and C.08.006 of the Food and Drug Regulations.
According to the Food and Drugs Act, "sell" includes offer for sale, expose for sale, have in possession for sale and distribute, whether or not the distribution is made for consideration.
The Natural Health Products (NHPs) finds its authority under the Natural Health Products Regulations, however an amendment to the regulations permits sections C.08.010 and C.08.011 of the Food and Drug Regulations to apply to NHPs (Regulations Amending the Natural Health Product Regulations (Special Access), SOR/2004-119, May 11, 2004).
The Medical Device Bureau administers its own Special Access Programme and has its own Special Access Regulations contained in the Medical Devices Regulations. Information on how to access a medical device through the Programme is available on the Health Canada website
The Veterinary Drugs Directorate finds its authority under sections C.08.010 and C.08.011 of the Food and Drug Regulations and administers a similar programme called Emergency Drug Release (EDR). Information on the Veterinary EDR is available on the Veterinary Drugs Directorate website.
Active Pharmaceutical Ingredients (APIs) for pharmaceutical compounding are subject to the requirements of the Food and Drug Regulations, Division 1A - Establishment Licensing and Division 2 - Good Manufacturing Practices (GMP).
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E2A: Clinical Safety Data Management: Definitions and Standards for Expedited Reporting