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    • Compassionate Use Policy
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Policy on Compassionate Use

1. Purpose

This policy sets forth Pfizer standards for compassionate use of Pfizer investigational products specifically the circumstances for the provision and treatment use of an unapproved Pfizer investigational product outside the scope of a Pfizer-sponsored clinical study or Pfizer-supported investigator-initiated research ("compassionate use").

As a general rule, Pfizer resources are directed toward conducting clinical studies to support regulatory approval of investigational products. Nevertheless, Pfizer is committed to making reasonable accommodations so that seriously ill patients who have exhausted other appropriate treatment options (including enrollment in available clinical studies) may, under the conditions described in this policy, have access to investigational products before they are commercially available. This policy is intended to strike an appropriate balance between the public interest in the thorough review and approval of new products and allowing access to investigational products that may be necessary to treat seriously ill patients who cannot be satisfactorily treated with commercially available products.

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2. Scope

This SOP applies to Pfizer colleagues within Worldwide Development (WWD), Safety and Risk Management (SRM), Worldwide Regulatory Affairs and Quality Assurance (WRAQA), Worldwide Medical, and the Pfizer Country Organizations (PCOs – Medical, Regulatory, Safety) in Worldwide Pharmaceutical Operations. It also applies to those parties with which Pfizer contracts (e.g., Contract Research Organizations [CROs] or consultants) who are involved in Pfizer-sponsored clinical studies.

This policy applies globally with respect to compassionate use requests for investigational products with active ingredients that have not been approved for any purpose in the country from which the request originates, or are otherwise not available in that country through normal commercial channels. For purposes of this policy, a compassionate use request is any request for the use of an investigational product outside the scope of a Pfizer-sponsored clinical study or Pfizer-supported investigator-initiated research.

Pfizer-sponsored open-label clinical studies are subject to Pfizer clinical study SOPs and do not fall under this policy.

This policy also does not cover requests for unapproved new dosage form versions of a commercially available product (e.g., a request for an unapproved intravenous formulation of a product which is a solid oral dosage form has already been approved). However, decisions relating to such requests are generally governed by the same considerations as those presented in this policy. Pfizer recognizes that in some cases, the risk/benefit analysis may favor compassionate use of new formulations at an earlier stage of clinical development than would be the case for investigational products that have not been approved for any purpose. Accordingly, Pfizer evaluates compassionate use requests for new formulations on a case-by-case basis.

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3. Policy

Pfizer considers compassionate use requests only when all of the following criteria are met:

1. The investigational product is being investigated under an appropriate regulatory authorization or all investigational studies of the product have been completed;
2. Pfizer is pursuing or intends to pursue marketing approval for the investigational product in at least one country;
3. There are meaningful human clinical data to support an assessment that the potential benefits to the patient outweigh the risks to the patient (generally such data is not available until the beginning of Phase III of the clinical development process);
4. The patient for whom compassionate use is sought:
   • Is suffering from a serious disease condition;
   • Has undergone appropriate standard treatments without success (or no standard treatment exists for the patient's condition);
   • Is ineligible for participation in any ongoing clinical study; and
   • Meets any other relevant medical criteria for compassionate use of the investigational product, as established by Pfizer.
5. A physician attending to the patient for whom compassionate use is sought agrees to:
   • Direct the administration of the investigational product under a Pfizer-approved protocol;
   • Provide, at a minimum, information on any serious adverse events associated with the use of the investigational product, along with any other clinical data considered by the study team to be necessary under the circumstances;
   • Conduct the compassionate use study under appropriate ethical standards, including obtaining review by an Institutional Review Board/Independent Ethics Committee (IRB/IEC) and obtaining appropriate informed consent and authorization to disclose data from the subject;
   • Maintain treatment records for an appropriate time period after administration of the investigational product and agree to make treatment records available for inspection by Pfizer and regulatory authorities, as requested.

Pfizer recognizes that in some emergency situations, applicable regulations and ethical standards may permit compassionate use in the absence of a formal protocol or prior to review by an IRB/IEC. In such cases Pfizer may expedite the consideration of the specific request, and may waive the requirements pertaining to the protocol and prior IRB/IEC review to the extent that such a waiver is permitted by applicable regulations and standards.

In general, where permitted by local regulation, compassionate use treatment should be discontinued when the product becomes commercially available in that location.

Geographic limitations generally do not factor into consideration of a compassionate use request. However, Pfizer may consider geographic limitations as effectively rendering a patient ineligible for a clinical study, and therefore eligible to be considered for compassionate use, especially in cases in which the rationale for compassionate use is particularly compelling.

A patient may also be assessed as effectively ineligible for a clinical study if the patient has already received and failed to respond to the active comparator used in a randomized study.

Compassionate use requests are decided on a fair and equitable basis in accordance with the principles set forth in this policy. Other non-clinical factors, such as the identity of the proposed subject or the requestor, must not play a determinative role in the consideration of a compassionate use request.

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4. Responsibilities

It is the responsibility of the relevant study team, including Medical, Regulatory Affairs and Legal representatives, to consider compassionate use requests that satisfy the criteria set forth in this policy. If the team cannot reach a consensus, the final decision rests with the clinical lead.

Exceptions to this policy may be granted only after a recommendation by the relevant study team and written approval by appropriate senior management in consultation with Legal.

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