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Draft Guidance Document for Industry and Practitioners on the Special Access Program (SAP) for Drugs

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Organization: Health Canada

Published: 2019/05/10

This guidance document is being distributed for comment purposes only.

Draft date: 2019/05/10

Foreword

Guidance documents are meant to provide assistance to industry and health care professionals on how to comply with governing statutes and regulations. Guidance documents also provide assistance to staff on how Health Canada 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 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 document, 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.

Table of contents

1. Introduction

1.1 Purpose/overview

Health Canada’s Special Access Program (SAP) administers the “SaleFootnote 1 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 2 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.

1.2 Scope and application

This guidance document is intended to clarify the mandate, intent and scope of the SAP and outlines:

  • the process to request a drug that cannot otherwise be sold or distributed in Canada for a medical emergency
  • the reconsideration process prior to the issuance of a denial
  • the responsibilities of the practitioners and manufacturers, in the process
  • the personalized service for urgent life-threatening or end-of-life situations, and
  • the conditions to be met to request drug pre-positioning by manufacturers and its process

For the purposes of this guidance document, "drugs" include pharmaceuticals, biologics, radiopharmaceuticals, and natural health productsFootnote 3. It excludes medical devicesFootnote 4, veterinary drugsFootnote 5 and active pharmaceutical ingredients (APIs)Footnote 6. Furthermore, while the term “treatment” is used throughout the document for how the drug is used, it also includes drugs for the diagnosis or prevention of a serious or life-threatening condition in a patient.

The draft Guidance Document should be read in conjunction with the proposed regulations pre-published in Canada Gazette, Part I.

1.3 Policy objectives

To ensure that requests for special access to unauthorized drugs are received, processed and decided upon effectively, consistently, and in accordance with Part C, Division 8, sections C.08.010 and C.08.011 of the FDR.

1.4 Policy statements

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.

1.5 Background

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.

2. Overview of the Regulatory Framework

Sections C.08.010 and C.08.011 of the FDR, provide the Minister with discretionary authority to issue a letter of authorization that:

  • (i) authorizes the sale of a quantity of a new drug to a practitioner named in the letter of authorization for use in the emergency treatment of a patient (or patients) under their care, or an unknown patient(s) at the time of the request (often referred to as “future use”)
  • (ii) authorizes the sale of a quantity of a drug that has been previously authorized through the SAP on at least one occasion and the drug is approved for the requested use by the United States Food and Drug Administration or the European Medicines Agency, and
  • (iii) authorizes a manufacturer to pre-position a product in Canada in anticipation of Health Canada receiving and authorizing a request from a practitioner for a drug to be used for a medical emergency

The Regulations do not expressly define the term “medical emergency” however, Health Canada has interpreted this term as “serious or life-threatening conditions when conventional therapies have failed, are unsuitable, or unavailable in Canada.”

“Conventional therapies” are treatments that are widely accepted and used by most health care professionals using authorized drugs that have been approved for an indication in Canada either through a new drug submission or an extraordinary use new drug submission. Conventional therapies also include treatment with medical devices when widely accepted and used by most health care professionals as well as procedures that are not subject to Health Canada approval (for example:  surgery, radiotherapy, etc.).

“Unavailable” is interpreted as a conventional therapy that is not marketed in Canada, or a conventional therapy for which marketed alternatives have been exhausted.Footnote 7

Several factors are considered in the determination of what is a serious or life-threatening condition in a patient. Such factors include but are not limited to:

  • the need for hospitalization or in-patient admission/care
  • the risk of adverse pregnancy outcome
  • the risk of persistent or significant disability in the absence of treatment
  • the prognosis of the condition

2.1 Sale of a new drug for a medical emergency

Under sections C.08.010(1) of the proposed regulatory amendments, the Minister may issue a letter of authorization allowing a manufacturer to sell a drug to a requesting practitioner to treat one, or several patients in a medical emergency.

The Minister’s authority to issue a letter of authorization is discretionary, meaning that the Minister may authorize or deny access to a drug based on the information supplied by the practitioner and other information that the program may have in its possession.  This discretion is carefully exercised by considering all information provided, the nature of the medical emergency, and the extent to which the information submitted in support of the request is credible and relevant to a specified medical emergency.

Practitioners filing a request must be entitled under the laws of a province to treat patients with a prescription drug, and must be practicing their profession in that province as per the definition of practitioner in Part C of the FDR, Division 1, section C.01.001. That is, “a practitioner is a person who (a) is entitled under the laws of a province to treat patients with a prescription drug, and (b) is practising their profession in that province”.

2.1.1 Information requirements to be submitted with new drug requests

Subsection C.08.010(1)(a) requires a practitioner to submit information and details about the medical emergency, the data in the possession of the practitioner with respect to the use, safety and efficacy that support the decision to prescribe this drug, the civic address to which the drug is to be shipped, and any additional information the Minister may request in respect of the medical emergency or the safety and efficacy of the new drug in relation to the medical emergency. 

2.1.1(a) Information about the medical emergency – Patient(s) may be known or unknown at the time of request

The practitioner must provide sufficient information on the condition of the patient to justify that the condition is serious or life-threatening, identify what treatments have been considered or tried and how they have either failed, are unsuitable for the patient, or are unavailable on the market.

In such cases where the patient is unknown at the time of the request, the practitioner must provide a rationale as to why the drug is needed in anticipation of a medical emergency for the treatment of a specified condition or disease.

2.1.1(b) Information about the use, safety and efficacy of the drug

The practitioner must provide information on the use, safety and efficacy of the drug being requested. This may include but is not limited to data/references and/or resources in the practitioner’s possession that support the decision to prescribe the drug, articles from medical literature, clinical protocols, investigator brochures, foreign prescribing information, etc.

As per the proposed subsection C.08.010(1.01), a practitioner is not required to submit information about the use, safety and efficacy of the drug for a specified emergency, if the drug has been authorized on at least one previous occasion, and the following conditions are met:

  • the European Medicines Agency or the United States Food and Drug Administration has authorized the new drug to be sold without terms or conditions in its jurisdiction to address the same medical emergency, and
  • the Minister has not cancelled the drug identification number of a Canadian drug for which insufficient evidence supported the drug use, or an order has been issued due to potential risks to the injury to health (as per paragraphs C.01.014.6(2)(b) or (c) or subsection C.01.014.6(3))

2.1.1(c) Shipping address

The practitioner must provide the civic address to which the new drug is to be shipped and specify whether the drug is being shipped to their office or a pharmacy. Both the practitioner’s name and address and, if applicable, the pharmacy name and address must be provided.

The practitioner remains responsible for accounting for quantity received and reporting the results of the use of the drug.

2.1.1(d) Any additional information the Minister may request respecting the medical emergency or the safety and efficacy of the new drug in relation to the medical emergency

Access to a drug under the medical emergency provisions of the FDR represents an individualized approach that takes into account not only the information about a drug, but also the individual health profile of a patient. The threshold for evidence required to support a request for a drug is comparatively lower than that required to support a clinical trial application or a drug submission for market authorization, where data on safety and efficacy are presented and assessed at the population health level. When the data submitted is not sufficient to allow the Minister to make a final decision regarding the issuance of an authorization, the Minister may request additional information.

2.2 No SAP authorization for a new drug that is or contains a restricted drug

A "restricted drug" as defined in Part J of the FDR, is not eligible for authorization by the Program in accordance with subsection C.08.010 (1.1).

Section J.01.001 in Part J, Restricted Drugs, of the FDR a restricted drug is defined as:

  • (a) “any substance that is set out in the schedule to this Part or anything that contains the substance; and
  • (b) cocaine (benzoylmethylecgonine) or any of its salts, or anything that contains such a substance, except a drug in dosage form, as defined in subsection C.01.005(3), that has a drug identification number assigned to it under Division 1 of Part C or that is authorized for sale under Division 5 of Part C, and except cocaine (benzoylmethylecgonine) or any of its salts, or anything that contains such a substance, that is to be or that has been compounded by a pharmacist in accordance with or in anticipation of a prescription.”

2.3 Reporting requirements by the practitioner

As per subsection C.08.010(1)(b), the practitioner must:

  • (i) provide a report to the manufacturer of the new drug and to the Minister containing the results obtained following the use of the drug to address the medical emergency, including information respecting any adverse drug reactions observed by the practitioner, and
  • (ii) account to the Minister, on request, for all quantities of the new drug received by him/her.

If the practitioner had the drug shipped to a community pharmacy on his/her behalf, he/she remains responsible to account for the quantities received.

2.4 Request to manufacturer that sells a drug to a practitioner to provide information

Drugs authorized through these proposed regulations are not marketed to the Canadian public as they have not been reviewed for market authorization in Canada, or have had market authorization in Canada, but have been discontinued from the market. As per the proposed subsection C.08.011(2) and C.08.011.1(4), the Minister may require a manufacturer who received a letter of authorization to sell a new drug to a practitioner to provide Health Canada with a copy of any report submitted to a foreign regulatory authority that has authorized the sale of the drug in its jurisdiction, regarding the safety, efficacy or quality information of the new drug.

2.5 Request from a manufacturer to pre-position a drug in Canada

The proposed subsection C.08.011.1(1) provides the Minister with the authority to authorize a foreign manufacturer to import a drug into Canada for pre-positioning in advance of an authorization to a practitioner requesting the drug. 

In requesting a letter of authorization from the Minister to pre-position a drug, a manufacturer is to specify the quantity of drug they wish to import, identify a Canadian importer who is the holder of an establishment licence in Canada and who will pre-position their drug in a Canadian facility, provide the civic address of the facility where the drug is to be stored in Canada; and any other information the Minister may require for the purpose of issuing an authorization.  This includes a rationale as to why the product needs to be pre-positioned.  The manufacturer would need to explain the urgency of having the product in Canada including if the drug requires timely administration to a patient.

The Minister may refuse to issue an authorization if the manufacturer has not met all requirements i.e., the quantity of drug requested to be pre-positioned exceeds the amount that is likely required to address potential medical emergencies; the establishment licence of the importer does not contain the same category as the drug to be imported; the manufacturer has not supplied the information requested by the Minister under the proposed subsection C.08.011.1(a)(v); or the information requested by the Minister regarding the medical emergency or the safety and efficacy of the drug in relation to the medical emergency does not support the issuance of an authorization to pre-position the drug.

2.5.1 Request to manufacturer that has been issued a Letter of Authorization for pre-positioning to provide information

As per the proposed subsection C.08.011.1(1)(4), the Minister may require a manufacturer who has been authorized to pre-position a drug in Canada, to provide Health Canada with a copy of any report that was submitted for the purpose of evaluating the safety, efficacy or quality of the new drug, to a foreign regulatory authority that has authorized the sale of the drug in its jurisdiction.

2.5.2 Importer Requirements

Importers must have been inspected and assessed as being in compliance with the full requirements of Divisions 2 to 4 of the FDR for their drug establishment licence (DEL).  DEL holders must comply with these Regulations if importing for the purposes of pre-positioning and further comply with any applicable terms and conditions that are included on their licence. Fees under the Fees in Respect of Drugs and Medical Devices Regulations are applicable.  If a drug is a controlled substance, the DEL holder must comply with the rules under the Controlled Drugs and Substances Act including those relating to dealer’s licences.

Any application for a new DEL, or amended DEL, will be subject to a regular Good Manufacturing Practices (GMP) inspection for those activities against the full requirements of Division 2-4 of the FDR. A DEL is only issued if the full requirements of Division 2-4 have been met.

However:

  • The importer is not required to have the relevant foreign buildings listed on either its DEL’s foreign building annex or active pharmaceutical ingredient annex.
  • The importer is not required to hold a DEL authorizing sterile dosage forms to conduct activities with respect to sterile drugs.
  • The importer is not required to hold a DEL authorizing the specific dosage form, class of the drug.

Subsection C.08.011.2(2) proposes the following relevant sections of Divisions 2 to 4 to apply to the importation of a drug as it relates to storage and distribution under the pre-positioning provisions of the regulations:

  • (a) sections C.02.003.1 and C.02.004 require that the drug is stored and kept in sanitary conditions by the licensee
  • (b) section C.02.006 requires the drug be stored under the supervision of personnel who, having regard to the duties and responsibilities involved, have had technical, academic and other training as the Minister considers satisfactory in the interests of the health of the consumer or purchaser
  • (c) subsection C.02.012(1) requires the importer maintain a system of control that permits complete and rapid recall of any lot or batch of the drug that is on the market; and a program of self-inspection
  • (d) sections C.02.013 and C.02.014 requires quality control and specific measures regarding quality control
  • (e) section C.02.015 as it applies to the storage and transportation of the new drug by the holder of an establishment licence
  • (f) subsection C.02.021(1) as it applies to the storage of the new drug by the holder of an establishment licence, i.e., maintaining all records and evidence of the storage of the drug in dosage form that are required to be maintained for a period of one year after the expiration date of the drug unless the person’s establishment license specifies some other period
  • (g) subsection C.02.022(1) requires record retention of sales of each lot or batch of the drug, for one year after the expiration date of that lot or batch unless their establishment licence specifies some other period to enable recall measures
  • (h) section C.02.023 iterates specific requirements when dealing with a complaint including retaining records of results of any investigations, contact information, and record retention for a period of one year after the expiration date of the lot or batch of the drug
  • (i) subsections C.02.024(1) and C.02.025(1) includes requirements for record retention of the results of the self-inspection programs for a period of three years, and require a sample of the drug be retained in Canada one year after the expiration date of the drug
  • (j) section C.03.013 prohibits importation of a drug that is derived from animal tissue unless the tissue is obtained from a healthy animal free from infectious disease, and
  • (k) section C.04.001.1 as it applies to the storage of the new drug by the holder of an establishment licence, i.e., prohibits the distribution of a drug unless it has been stored according to the Division

2.5.3 Distribution of the pre-positioned drug to a practitioner

As per the proposed subsection C.08.011.3(1), new drugs that have been pre-positioned can only be distributed to a practitioner by the importer once the foreign manufacturer of the drug has received a letter of authorization from the Minister that permits the sale of a quantity of the drug to a named practitioner under section C.08.010(1). The quantity distributed cannot exceed the quantity specified in the Letter of Authorization.

2.6 Effect of a Letter of Authorization

An authorization allows a manufacturer to sell the quantity of drug identified in the authorization for the specified use, i.e., to a practitioner for use in a medical emergency or to an importer for pre-positioning products in Canada. The authorization is valid until the quantity of drug identified in the authorization is depleted, or expired.  For an authorization to pre-position, if the drug expires before stock is depleted, the authorization is no longer valid.

The sale of the quantity of drug identified in an authorization is exempt from the Food and Drugs Act (FDA) and the Food and Drug Regulations (FDR). Drugs can only be distributed and/or sold for the use stated in the authorization. Also, drugs authorized under these provisions are prohibited from advertisement under C.08.002 of the FDRs and section 3 of the Food and Drugs Act.

3. Guidance for Implementation

3.1 Information for Practitioners Filing a Drug Request

A practitioner may file a request for an individual patient, multiple patients with the same serious or life-threatening condition, or future patient(s) in anticipation of a medical emergency for example, a drug needed in an emergency room and the patient is unknown at the time of the request.  In deciding to request an unauthorized drug, a practitioner assumes the liability and responsibility for the use of that drug.

Prior to filing a request, practitioners are encouraged to contact individual manufacturers to confirm the availability of a drug and to obtain the most recent drug information such as prescribing information and other data that supports the use of the drug.

When submitting a request, practitioners must provide the information required to support a request for the drug to be used in a medical emergency.  The practitioner should also specify the date when administration or dispensing of the drug is scheduled or anticipated. 

All requests must include the following information:

Contact Information
Contact information must be provided for the practitioner. When the requesting practitioner is not the person completing the form, the full name and position (e.g. Pharmacist, Nurse, Resident, etc.) of the individual completing the form must also be provided. An email address, telephone and fax number is to be provided including an area code and extension (if applicable), where the practitioner or contact person can be reached should further information or follow-up be required.  Other information to be included is the civic address of where the drug is to be shipped.  For example, a drug may be shipped to hospital pharmacies, practitioner’s office, a nuclear medicine department, a blood bank or a community pharmacy.
Patient(s) Information

The practitioner must specify the name of the new drug and information concerning the medical emergency for which the new drug is required. Specific information about the patient(s)’s medical history including conventional therapies that have been considered, ruled out and/or have failed or that are unsuitable and/or unavailable to achieve an adequate response for the specific patient(s) must be provided.  The practitioner must also indicate why the requested drug (e.g. mechanism of action, drug class, dosage form) is the best choice for the patient(s).

All rationales should be patient specific. In cases where additional pages are added, pages must be numbered appropriately.

Patient Unknown
In cases where the request is for the future use of a drug to treat a patient whose identity is unknown (e.g., a drug needed in an emergency room) the practitioner must provide a rationale as to why the drug is needed and the clinical circumstances under which it would be used.
Quantity of Drug Requested

The quantity of drug requested must be specified, such as the precise number of tablets, vials, etc. for each patient. The total quantity, i.e., the sum of the quantities for all patients, must also be specified. The request will be returned if the amount of drug required is not clearly stated. The quantity of drug authorized will depend on the nature of the medical emergency and the patient(s)’s medical condition.

To initiate a Special Access Request, practitioners are to complete the Special Access Request (SAR) Forms which can be downloaded from the Health Canada website.

3.1.1 Practitioners Filing a Request for treatment of a patient (or patients) in a Medical Emergency (Special Access Request - Form A)

The Special Access Request (SAR) - Form A, is to be used when the practitioner is requesting access to a drug for immediate use or use of the drug in the short term for one or multiple patient(s).  A clinical rationale must be provided for each patient indicating the patient’s medical condition, which alternatives have been tried or considered, and why the requested drug is the best choice for the patient(s). 

3.1.1.1 First time request to access a new drug for a new patient

Drug Information and References regarding use, safety and effectiveness

References to information that pertain to the requested use of the drug must be submitted.  This includes data/references and/or resources in the practitioner’s possession, with respect to the use, safety and efficacy that support the decision to prescribe the drug.  Reference should be made to the most recent information available that is relevant to the requested use being sought. Information may be from sources such as medical literature, clinical protocols, investigator brochures, foreign prescribing information, etc.  Either provide a complete citation including journal/article titles, author(s), volume, issue, date, and page information, or a copy of the references.

3.1.1.2 Request to access a drug previously authorized through SAP for the same medical emergency for a new patient

For certain drugs that have been previously authorized by the Program, the SAP only requires the practitioner to submit information as outlined in section 3.1 of this guidance, (i.e., clinical rationale to support the request, contact information, and the quantity of drug requested). The SAP does not require the practitioner to submit information on the use, safety and efficacy for the medical emergency if:

  • the request is for the same medical emergency as that previously authorized by the Program, and
  • the drug has been authorized for that use by the European Medicines Agency (EMA) or the United States Food and Drug Administration (US FDA) to be sold in those jurisdictions without terms or conditions

Should information on the use, safety and efficacy of the drug change, the Minister may ask the practitioner to provide information on the use, safety and efficacy of the drug for use in the medical emergency (as per section 3.1.1.1). Failure to provide that information could lead to issuance of a denial.

Information on the use, safety and efficacy of the drug may change due to the drug’s safety profile changing, regulatory actions taken by the EMA or the US FDA, or the addition of certain conditions or restrictions regarding the use of the drug.

Form A or B can be used to request drugs that have been previously authorized by the Program for a specific patient, or an unknown patient(s) and do not require the submission of safety, efficacy and use data.

3.1.1.3 Repeat requests for same patient(s), same medical emergency

Form A is also used for repeat requests. As the SAP has previously reviewed the information provided by the practitioner on the safety and efficacy for the requested drug and determined that the drug is suitable for authorization pursuant to the Program, repeat requests may reference the original data provided and do not require the practitioner to resubmit the drug information and references.

The practitioner must provide information on the patient(s)’s condition since treatment was initiated, including a rationale for continued access. The practitioner is to describe the patient(s)’s response to the drug relative to the initial treatment goal(s), and provide a rationale for requesting continued access. The practitioner also must attest that there have been no changes to the information provided in the practitioner’s previous request regarding the patient(s).

Note: Each time a renewal is requested, if there is a change in the patient(s)’ medical condition, the practitioner must update the patient(s)’ current medical state and ensure it is well described.

3.1.2 Practitioner filing a request for treatment of an unknown patient in a future medical emergency (Form B - Future Use Request)

The SAR for Future Use - Form B, is to be used to request access to a drug that is required on-site within a facility, in anticipation of patients presenting with a medical emergency.

There are circumstances where Canadian institutions, in anticipation of a life-threatening emergency, require certain unauthorized drugs be available on-site. Such institutions include emergency rooms in hospital settings, zoos, first responders i.e. fire and police departments, and workplaces where hazardous materials are handled. The SAP considers “future use” requests from practitioners who wish to access a supply of a drug in anticipation of an urgent future use need. For a future use request, the practitioner needs to include a clinical rationale as to why the drug is required on-site in advance of a medical emergency rather than requesting it on an as needed basis for specific patients. The practitioner is to explain under which conditions the drug will be used.

3.1.2.1 First time request to access a drug for future use in an unknown patient

Using Form B, refer to section 3.1 and 3.1.1.1 of this guidance for information that is to be provided with the request.

3.1.2.2 Request to access a drug previously authorized through SAP for the same medical emergency for future use in an unknown patient

Using Form B, refer to section 3.1 and 3.1.1.2 of this guidance for information that is to be provided with the request.

3.1.2.3 Repeat request to access a drug for future use in an unknown patient

Using Form B, refer to section 3.1 and 3.1.1.3 of this guidance for information that is to be provided with the request.

3.1.3 Personalized Service for urgent Life-threatening or End-of-Life Situations

A personalized service is available to physicians treating patients in urgent situations due to a critical or terminal illness.
A 24-hour direct service line will continue to be available to physicians on a triage-based approach (i.e., severity of condition and immediate need of treatment).

This service provides physicians with the option to access a Health Canada health care professional and will allow direct communication between Health Canada and physicians.

It is important to note that manufacturers still have the final decision on whether the drug will be supplied to the physician and whether any restrictions or conditions will be imposed on the release of the drug.

3.1.4 Practitioners wishing to transfer drug to another patient

Practitioners wishing to transfer the supply of drug to another patient must receive authorization from the SAP and the manufacturer prior to doing so. Therefore, a practitioner must submit a request by filling out Section E of Form A. The practitioner must specify the request number under which the initial supply was authorized and indicate where the supply is being transferred and to whom. Also, the practitioner is required to specify the amount of drug being transferred.

3.2 Information for manufacturers filing a pre-positioning request

The Program may allow foreign manufacturers to pre-position drugs in Canada that may be requested through the SAP to allow for a shorter shipping time to practitioner. This is particularly important for medical emergencies that require the timely administration of the drug.

For a foreign manufacturer to be authorized to pre-position a drug in Canada, it is proposed that the Canadian importer must have a drug establishment license (DEL) that meets the proposed requirements as described in section 2.5.2 of this guidance. Pre-positioning drugs in Canada is intended for drug establishment license holders conducting such activities as part of their regular business.

3.2.1 Manufacturer filing a request for pre-positioning (Form D - Pre-positioning Request)

Using Form D, the foreign manufacturer is to identify who will import and hold the pre-positioned product in Canada by providing the importer’s contact information, DEL number, shipping and storage facility address, and the quantity of drug to be pre-positioned.

Manufacturers must provide a rationale as to why the product should be pre-positioned in Canada. A pre-positioned drug cannot be distributed to a practitioner unless a Letter of Authorization has been issued to a manufacturer authorizing the sale of the drug to a named practitioner.

The SAP will consider authorizing pre-positioning requests based on the nature of the emergency and urgency for the drug to arrive to the patient for use.

3.3 Practitioner reporting requirements

3.3.1 Adverse Drug Reaction reporting

Practitioners are required to report to the manufacturer and to the SAP on the use of a drug and any adverse drug reactions (ADRs) encountered. 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 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 the reaction is fatal or life threatening. ADRs should be reported using the Council for International Organizations of Medical Sciences (CIOMS) forms and sent to the SAP (please refer to section 3.5 of this guidance for contact information).

3.3.2 Practitioners filing a follow-up report (Form C – Follow-Up Information)

Practitioners must report on the outcome experienced by the patient using the drug by filing Form C. Reporting should be on a patient by patient basis. Information to be provided includes whether or not the dose of the drug was altered, how the patient responded to treatment with the drug, and whether or not the patient experienced any serious and/or unexpected adverse reactions. Unexpectedness should be determined against the Investigator’s Brochure and/or the foreign prescribing information.  In the case where a serious or unexpected adverse reaction occurred, the practitioner should refer to the ADR report that was submitted, and if one has not been submitted, the practitioner must submit an ADR report as per section 3.3.1 of this guidance.

Practitioners who wish for their patient to continue treatment with the drug, must complete section D of the form and provide the patient’s response to the drug relative to the initial treatment goals when submitting their request.

3.4 Proposed requirements for manufacturers to submit foreign reports

Prior to authorizing the sale of a drug to a practitioner, or authorizing the pre-positioning of a drug in Canada, the proposed regulations allow the SAP to request from a manufacturer a copy of any report that was submitted, for the purpose of evaluating the safety, efficacy or quality of the drug, to a foreign regulatory authority that has authorized the sale of the drug in its jurisdiction. This information will be used by the Program to ensure there is no change in the drug’s benefit/risk profile which would inform the SAP’s decision-making for authorization.  Furthermore, it will allow the Program to inform practitioners about any conditions of sale or restrictions with use of the drug.

3.5 Sending request forms and reporting forms to Health Canada’s SAP

Pending the availability of an electronic system, completed forms should be faxed, or sent by mail to:
Special Access Program
Health Canada, Tunney's Pasture
Address Locator 3105A
K1A 0K9
Tel: 613-941-2108
Fax: 613-941-3194
E-mail: hc.sapd-pasm.sc@canada.ca

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.

3.6 Receipt of request by SAP

Most requests are processed within one working day of receipt. However, given the mandate of the Program and the potential volume of requests received, requests are triaged to ensure that more urgent matters take precedence over less urgent matters. For example, requests for blood products and certain antibiotics are given priority.

Upon receipt of a request, the form is reviewed to ensure that it is complete, legible, signed and dated by a practitioner.

The SAP proceeds to assess the information that has been submitted to support the need for a drug to be used in a medical emergency for a patient or multiple patients. 

3.6.1 SAP review of Special Access Request (SAR) form and supporting information

In deciding whether or not to issue an authorization, the SAP considers if the data supporting the request is credible and supportive of the need for a drug for the identified emergency.

The SAP assesses requests from practitioners on a case-by-case basis, and in addition, undertakes the following risk management activities when considering requests:

  • emphasizing that marketed alternatives should always be considered and/or tried before considering the use of unauthorized drugs
  • recommending alternative mechanisms, such as clinical trials, to provide emergency access to unauthorized drugs
  • encouraging the exchange of information about drugs released through the SAP between manufacturers, practitioners and the SAP
  • coordinating the dissemination of drug advisories, developed in conjunction with the manufacturer, for Healthcare Professionals respecting new information regarding drugs available through the SAP
  • reviewing documentation supporting emergency use of a non-marketed drug prior to its first release through the SAP
  • working with the manufacturer to gather and document information about a drug, its development and regulatory status, and
  • ensuring practitioners have access to current and relevant information respecting a drug available through the Program.

Each request represents a unique set of circumstances and is supported to varying degrees by information provided by the practitioner. In addition to the SAP’s overarching risk management activities, consideration of a request takes into account and balances the following factors to ensure that a medical emergency exists and there is credible data to support the request:

  • Seriousness of the condition - description of the condition for which the drug is requested and why it represents a medical emergency
  • Clinical status of patient - description of current clinical status of the patient, including prognosis
  • Other therapies tried and/or ruled out - summary of marketed therapies that have failed, have been considered, ruled out or are unavailable
  • Prior patient experience with the drug - summary of a patient's past experience with the drug, including evidence of efficacy and adverse drug reactions
  • References provided with request
    • References must be for the most recent information that is relevant to the medical emergency ranging from:
      • prescribing information/package insert from the jurisdiction where the drug may be marketed
      • references to data from the literature outlining the results of randomized controlled trials
      • references to data from the literature outlining the results of non-randomized trials
      • case series and individual case reports from the literature and/or;
      • unpublished reports
  • Other data the SAP may require – Any additional information in respect of the medical emergency or the safety and efficacy of the new drug in relation to that medical emergency.
  • Data available to the SAP
    • medical literature, treatment guidelines, investigator's brochures, information obtained from the manufacturer, clinical trial reports, etc.
    • consultations with expert reviewers within Health Canada, and
    • confirmation of the Canadian and international development /regulatory status of the drug
  • Availability of clinical trials - determine if enrollment in clinical trials is an option for an individual patient.

When assessing the information provided, the SAP may, at any time prior to making a decision, request additional information from the practitioner to allow appropriate consideration of the request.  The SAP may further consult with expert reviewers within the Department to seek additional information and may further confirm as needed, the Canadian and international development and regulatory status of the drug.

The SAP verifies that there are no marketed alternatives in Canada. In assessing the information provided with the request, the SAP determines if additional information is needed, for example, regarding the patient’s condition to confirm whether or not the known and potential benefits associated with the use of the drug outweigh the known and potential risks of the drug, prior to considering the issuance of a letter of authorization.

3.7 Special considerations

3.7.1 Drugs that have received a negative decision to a drug submission filed with a regulatory agency

The SAP will consider requests for drugs that have received a negative decision (i.e., Notice of Deficiency-Withdrawal (NOD/W), or Notice of Non-Compliance-Withdrawal (NON/W)) following the review of a drug submission filed with Health Canada or another regulatory jurisdiction.  Prior to issuing an authorization through SAP, the Program will:

  • Verify if the manufacturer agrees to disclose the concerns raised by the relevant regulator to the requesting practitioner(s).
  • Have the manufacturer draft a letter to requesting practitioners that includes the main concerns from the withdrawal letter.
  • Have the relevant review bureau at Health Canada verify that the concerns are well described regarding the negative decision issued for a Canadian drug submission.

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.

3.7.2 Marketed drugs with compliance actions in Canada

The SAP will consider authorizing access to drugs following compliance actions such as cancelling or suspending licences or market authorizations, provided that:

  • the drug is considered to be medically necessary for the treatment, diagnosis or prevention of a serious or life-threatening condition
  • the manufacturer is willing to publicly disclose the reasons for regulatory action
  • there are no other dosage forms of the drug on the market that would be considered a reasonable alternative
  • there are no other drugs or therapies that would be considered reasonable alternatives
  • a clinical trial is inappropriate under the circumstances for gathering new or confirmatory evidence of the safety and efficacy of the drug

3.7.3 Drug shortages and discontinued drugs

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 when:

  • the drug is considered to be medically necessary for the treatment, diagnosis or prevention of a serious or life-threatening condition
  • the manufacturer is willing to disclose the reasons for the shortage or discontinuance of the drug
  • there are no other dosage forms of the drug on the market that would be considered a reasonable alternative
  • there are no other drugs or therapies that would be considered reasonable alternatives, and
  • In the case of a drug shortage:
    • the manufacturer demonstrates that extraordinary efforts have been made to avoid and manage the shortage such as inventory control, rationing etc.
    • Other options to manage the shortage have been ruled out
    • The scope of the shortage is operationally manageable by the SAP

3.8 Issuance of an authorization

Following consideration of the SAR, the SAP will either authorize or deny the request. Should the SAP be satisfied with the information provided by the practitioner, the Program will issue a letter of authorization to a manufacturer allowing the sale of the requested drug and quantity to the named practitioner, or allow the importation of the drug for pre-positioning purposes.

Authorization letters are sent to the manufacturer and a copy to the practitioner.

3.8.1 Foreign manufacturers receiving a Letter of Authorization: (1) to sell a drug to a practitioner; or (2) for pre-positioning products in Canada

(1) Letter of Authorization to Sell to a Practitioner

  • The SAP issues a Letter of Authorization to the manufacturer and a copy to the requesting practitioner. The manufacturer is authorized to sell the identified quantity of drug for the specified use as per the authorization.
  • Manufacturers 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 substance. In addition, Health Canada's Office of Controlled Substances must issue an Import Permit to the importer. This permit allows the drug supplies to be shipped to Canada without contravening the Controlled Drugs and Substances Act (CDSA). If a drug is a controlled substance, the DEL holder must comply with the rules under the CDSA including those relating to dealer’s licences.
  • Manufacturers should clearly display the Letter of Authorization with other related documents, such as export permits, to facilitate clearance by the Canada Border Services Agency (CBSA).
  • Manufacturers should also maintain complete and accurate records of all transactions in a manner that permits rapid response to specific requests.

(2) Letter of Authorization to Manufacturer to Import a Drug for Pre-positioning in Canada

  • The SAP issues a Letter of Authorization to the manufacturer. The Letter of Authorization only allows the manufacturer to pre-position the drug in Canada through an importer who holds a valid drug establishment license to perform such activities. An importer is prohibited from selling or distributing the drug that is pre-positioned in Canada until a manufacturer receives a Letter of Authorization to sell to a practitioner a certain amount of drug.

3.9 Refusal to issue an authorization to a foreign manufacturer to (1) sell a drug to a practitioner or (2) pre-position a drug in Canada

SARs that are denied will be returned promptly to the practitioner, or the manufacturer, with an explanation.

The SAP may refuse to issue an authorization to a practitioner if:

  • an alternative treatment is available in Canada
  • the clinical rationale and other required information does not support the use of the drug in the medical emergency, or
  • the application does not otherwise meet the regulatory requirements

Prior to the issuance of a denial of a request from a practitioner, an independent review of the denial recommendation is conducted. If necessary, direct contact with the practitioner to discuss the reasons for the potential denial is conducted. The practitioner is provided the opportunity to provide clarifying information or to request a reconsideration review of the request. The reconsideration process (section 3.9.2 outlines the steps undertaken prior to the SAP officially issuing a refusal to authorize a request (otherwise referred to as a denial)).

For requests from a manufacturer to pre-position a drug in Canada, the SAP may refuse to issue an authorization if:

  • the quantity of drug to be pre-positioned exceeds the amount that is determined by Health Canada to be required to address potential emergencies
  • the importer does not have a valid drug establishment license to import the drug, or
  • the drug is a restricted drug that is found in the section Part J Restricted Drugs of the FDR, or
  • the information requested by the Minister regarding the medical emergency or the safety and efficacy of the drug in relation to the medical emergency does not support the request to pre-position the drug

3.9.2 Reconsideration process for practitioner’s request for a drug

The main objective of the Reconsideration Process (RP) is a means to provide the practitioner with an opportunity to be heard when the SAP is considering issuing a Denial and an ability to request an independent review following the issuance of a denial recommendation. The RP includes two levels of evaluations: the review of denial recommendation and the reconsideration review.

3.9.2(i) Review of negative recommendation

The first step in the reconsideration process is the review of a negative recommendation resulting from the screening and evaluation of a request with the supporting information. A negative recommendation is reviewed by a health professional who was not involved in the original negative decision. The health professional conducts a full independent review of the request and the supporting information. Once the independent assessment is complete, and if the health professional determines that the information provided supports an authorization, a final decision is made and a Letter of Authorization is generated for the request. If the health professional cannot make a decision based on the information provided, the requestor is contacted and specific information is solicited from the requestor to facilitate the decision making.

Any solicited information provided is reviewed by the health professional. If the information supports the authorization of the request, a Letter of Authorization is generated and the request is considered closed.

If the information does not support the authorization, the health professional will contact the requestor to discuss the recommendation to deny the request. If the requestor does not have any further supporting information to provide and accepts the recommendation to deny the request, the SAP will issue a denial, or the practitioner can withdraw the request.

3.9.2(ii) Reconsideration review of a recommendation to deny

In the event that an applicant is not in agreement with the evaluation and the recommendation by the health professional to deny a request, he/she may request a reconsideration of the case. At this stage, the SAP would solicit any additional supportive information for the request from the applicant. An independent review of the totality of the information would proceed.

The Reconsideration Review creates the opportunity for the requestor to have an independent reconsideration evaluation prior to any final decision made by the SAP. The outcome of the independent evaluation is provided to the requestor as a final decision, which may be a denial with supporting information for the decision, or may result in a Letter of Authorization.  A requestor always has the option to withdraw their request.

It should be noted that following a denial, a practitioner can resubmit a new request, should additional information become available.

3.10 Amendments to a Letter of Authorization to sell a drug to a practitioner or to pre-position a drug in Canada

The SAP should be contacted immediately if errors are found on a Letter of Authorization for pre-positioning a drug in Canada, or for selling the drug to a practitioner, to ensure the Letter is amended and the information is accurate for the purposes of importing the drug into Canada.

Corrections and amendments can be done administratively, such as typographical errors in the spelling of the drug name, manufacturer name, etc. The SAP should be made aware of such errors in a reasonable time frame. If the practitioner wishes to request a new or different route of administration; a different indication, or increased quantity of drug to be sold/imported from what he initially requested, a new request form is required.

3.11 Drugs authorized through the SAP that receive market authorization in Canada

Drugs authorized through the SAP may undergo clinical trials or submission review as a new drug. In the event that a drug submission is under regulatory review, the drug continues to be authorized by the SAP until the review is complete. Following regulatory review and the issuance of a Notice of Compliance (NOC), some manufacturers may market their drug on the Canadian market the day they receive their NOC whereas others may wait.  SAP drugs that are under regulatory review are added to a “Watch List”. SAP is informed that a submission is under regulatory review by manufacturers supplying their drug through the SAP and informing the Program about the status of their drug in Canada; or by seeking this information from the drug Review Bureaus of Health Canada. The SAP monitors this “Watch List” on a weekly basis to anticipate issuance of a Notice of Compliance (NOC). Close to the review target date, the SAP contacts the manufacturer to request information on the company’s transition plan and anticipated launch date.

Generally once a manufacturer receives an NOC and marketed supply is available, access via the SAP is no longer needed. The SAP can consider on a case-by-case basis a transition time until the marketed drug is available (generally no more than 90 days). In such situations quantities considered for authorization by the SAP would generally be adjusted to reflect the launch date provided by the manufacturer. Regular updates from manufacturers on review status and marketing developments facilitate effective transition.

In determining if access via the SAP is needed until the marketed drug is available, the following guiding principles are considered:

  • Patient safety is a priority. Therefore, once a marketed alternative is available, patients should access the marketed drug since it will be compliant with Good Manufacturing Practices and its benefit/risk profile has been established.
  • Once a drug receives market authorization, access through the SAP should be limited to meet an unmet medical need with a supply up to 90 days. In exceptional circumstances, continued supply will be considered to meet an unmet medical need should the marketed alternative not be medically suitable for the patient.

Practitioners are instructed to contact the manufacturer should they require more information on the plans for marketing the drug in Canada. Practitioners may consult Health Canada’s website to view the list of drug submissions under review, entitled “Drug and health product submissions under review (SUR)”Footnote 9.

3.12 Hours of operation

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 10, an On Call service is available.

3.13 Return of unused stock

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.

4. Advertising

In accordance with section C.08.002 of the Food and Drug Regulations, advertising of unauthorized drugs accessed through the SAP is strictly prohibited. Furthermore, it is Health Canada’s position that advertising of an unauthorized drug is inconsistent with Section 9(1) of the Food and Drugs Act (FDA), which prohibits advertising a drug in a manner that is false, misleading or deceptive, or is likely to create an erroneous impression regarding its character, value, quantity, composition, merit or safety.

Health Canada encourages the maker of a drug that is on SAP to not engage in activities deemed to promote a drug to healthcare professionals or the public. Health Canada uses its policy document entitled “The Distinction Between Advertising and Other Activities” (henceforth “Distinction”) to distinguish between activities intended to promote the sale of a health product and non-promotional ones. If a message regarding a health product is deemed promotional, it is subject to the advertising provisions of the FDA, the Controlled Drugs and Substances Act, and associated Regulations.

Appendix A - Glossary

Terms

Adverse drug reaction (ADR): as defined in section C.05.001 of the FDR, means any noxious and unintended response to a drug that is caused by the administration of any dose of the drug.

ADR reports: a summary of the patient's unexpected adverse drug reactions, as defined below, to the drug. For the most part, ADRs are only suspected associations however, a temporal or possible association is sufficient for a report to be made. Reporting an ADR does not imply a causal link, rather it is a precautionary measure.

Conventional therapy: a treatment that is widely accepted and used by most health care professionals using authorized drugs that have been approved for an indication in Canada either through a new drug submission or an extraordinary use new drug submission.  Conventional therapies also include treatment with medical devices when widely accepted and used by most health care professionals as well as procedures that are not subject to Health Canada approval (for example: surgery, radiotherapy, etc.).

Unavailable drug: a conventional therapy for which the drug is not marketed in Canada, or a conventional therapy for which marketed alternatives have been exhausted. This includes a drug in shortage.

Medical emergency: serious or life-threatening conditions when conventional therapies have failed, are unsuitable, or unavailable.

Notice of Compliance (NOC): a notification, issued pursuant to paragraph C.08.004(1)(a) or C.08.004(3)(a), indicating that a manufacturer has complied with sections C.08.002 or C.08.003 and C.08.005.1 of the Food and Drug Regulations. Notices of Compliance are issued to a manufacturer following the satisfactory review of a submission.

Notice of Deficiency (NOD): If deficiencies and/or significant omissions that preclude continuing the review are identified during the review of a submission, a NOD will be issued.

Notice of Deficiency - Withdrawal (NOD/W): When the response to a NOD is received, a new Screening 1 period (with an associated performance target) begins. If during the screening process, the response to a NOD is found to contain unsolicited information, is incomplete or deficient, the response to the NOD will be rejected and the submission will be considered withdrawn without prejudice to a refiling. A NOD-Withdrawal Letter will be issued by Health Canada.

Notice of Non-compliance (NON): After the comprehensive review of a submission is complete, a NON will be issued if the submission is deficient or incomplete in complying with the requirements outlined in the Food and Drugs Act and Regulations.

Notice of Non-compliance - Withdrawal (NON/W): When the response to a NON is received, a Screening 2 period begins (with an associated performance target). If during the screening process, the response to a NON is found to contain unsolicited information, is incomplete or deficient, the response to the NON will be rejected and the submission will be considered withdrawn without prejudice to a refiling. A NON-Withdrawal Letter will be issued by the responsible Health Canada Directorate.

Practitioner: as per section C.01.001 of the FDR, a person who is entitled under the laws of a province to treat patients with a prescription drug and is practicing their profession in that province.

Pre-positioning a drug: a drug that is placed in a facility in advance of the SAP receiving a practitioner request for that drug.

Serious adverse drug reaction: as defined in section C.05.001 of the FDR, means an adverse drug reaction that requires in-patient hospitalization or prolongation of existing hospitalization, causes congenital malformation, results in persistent or significant disability or incapacity, is life-threatening or results in death.

Special Access Request (SAR): a standard form used by the SAP to facilitate the request procedure. Practitioners fill out the SAR with the necessary information and submit it to the SAP.

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