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Compassionate Use of Unapproved Investigational Product

Compassionate Use of Unapproved Investigational Product

1. Purpose & Scope

The purpose of this clinical and medical controlled document (CMCD) policy is to set forth Pfizer standards for compassionate use involving the provision of unapproved Pfizer investigational products to patients outside of a clinical study. This policy applies globally with respect to compassionate use of Pfizer investigational products which are not approved for any purpose in the country from which the request originates or intended to be used.

Pfizer is committed to providing reasonable assistance so that seriously ill patients who have exhausted other appropriate treatment options may, under the conditions described in this policy and in accordance with applicable local law, have appropriate access to Pfizer investigational products before they are commercially available.

  • Compassionate use of unapproved Pfizer investigational products is permitted unless local laws/regulations prohibit such programs. When allowed, they must comply not only with the Pfizer standards of this policy, but also with the country-specific legal and regulatory requirements. These requirements may differ between countries, including in program name (e.g., Expanded Access to Investigational Drugs for Treatment Use (United States), Special Access Program (Canada), and Temporary Authorisation for Use (France)).
  • This policy generally does not cover requests for unapproved new dosage form versions of an approved product (e.g., a request for an unapproved intravenous formulation of a product which is a solid oral dosage form which has already been approved). However, decisions relating to such requests are governed by the same considerations as those presented in this policy in accordance with applicable legal/regulatory requirements. Accordingly, Pfizer evaluates compassionate use requests for new formulations on a case-by-case basis.
  • Pfizer-sponsored clinical studies, open label safety studies, and continuation phases of clinical trials are out of scope of this policy.

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2. Terms, Acronyms & References

Terms used in this Policy that are in the CMCD Global Glossary are hyperlinked upon first occurrence. Acronyms can be found in the CMCD Global Acronyms List. References can be found in the CMCD Global References List. All lists can be found on the eSOP portal.

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3. Policy Statements

Any use of unapproved Pfizer investigational products outside a clinical study covered by this policy must be in accordance with local laws and regulations governing such programs, as well as relevant standards including:

  • Pfizer Corporate Policies and Procedures and any applicable Pfizer CMCD standard operating procedures, including compliance with Pfizer's safety requirements for adverse event reporting and distribution of safety information.

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

  1. The investigational product is being studied under an appropriate regulatory authorization or all investigational studies of the product have been completed.
  2. The patient(s) for whom compassionate use is sought:
    • Is suffering from a serious disease or condition;
    • Has undergone appropriate standard treatments without success, no comparable or satisfactory alternative treatment/ prophylaxis vaccine is available, or no standard treatment/ prophylaxis vaccine exists for the disease or condition;
    • Is ineligible for participation in any ongoing clinical study of the investigational product, including lack of access due to geographic limitations; and
    • Meets any other relevant medical criteria for compassionate use of the investigational product, as established by Pfizer.
  3. There are meaningful human clinical data to support an assessment that the potential benefits to the patient outweigh the risks to the patient. In practice, this will generally occur at a point when Pfizer management has made a decision to seek marketing approval in one or more countries.
  4. Providing the investigational product for compassionate use will not interfere with development of the product.
  5. The physician(s) attending to the patient(s) for whom compassionate use is sought is properly licensed and fully qualified to administer the investigational product. They also agree to comply with:
    • All applicable country specific legal and regulatory requirements in relation to the request;
    • Any requirements in terms of medical criteria, safety reporting or other data provision which the relevant Pfizer team may require; and
    • International Conference on Harmonisation (ICH) Good Clinical Practice (GCP) principles, especially in informed consent, to ensure the intended patient(s) understands the risks associated with taking an unapproved investigational product where safety and efficacy may not be fully established.

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3.1. Other Considerations

When compassionate use requests are granted, relevant documentation (e.g., but not limited to, investigator brochure, labeling documentation, protocol) regarding the use and safety of the investigational product must be provided to the treating physician.

In addition to any country specific regulatory requirements, compassionate use programs need to be monitored, at a minimum, to ensure compliance with Pfizerýs standards for safety reporting in the use of an unapproved Pfizer investigational product.

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

3.2. Responsibilities

It is the responsibility of the Clinical Lead, in conjunction with Pfizer team members, including Medical, Worldwide Regulatory Strategy, Worldwide Safety Strategy and Legal representatives, to consider compassionate use requests on a fair and equitable basis that satisfy the criteria set forth in this policy.

The Pfizer team also needs to ensure that the physician(s) of the compassionate use program continues to receive any updates and other relevant materials regarding the investigational product, as well as be included on the distribution list for safety reporting.

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