Key Factors for Compassionate Use

The FDA published a draft guidance for institutional review boards (IRBs) and clinical investigators regarding the key factors and procedures IRBs should consider when reviewing individual patient expanded access submissions (IPEAS), including reviews conducted by a single member of the IRB.

Compassionate Use

Expanded access refers to the use of an investigational medicinal product (IMP) when the primary purpose is to diagnose, monitor, or treat a patient’s disease or condition rather than to obtain the kind of information about the drug that is generally derived from clinical trials (see also FDA's draft guidance on Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers). This pathway is sometimes called "compassionate use" and allows a patient with a serious or immediately life-threatening disease or condition to gain access to an IMP when there is no comparable or satisfactory alternative therapy. In this case, the potential patient benefit justifies the potential risks to the patient. An individual patient expanded access request can be submitted to FDA by a licensed physician as a new investigational new drug application (IND) or by a sponsor of an existing IND as a protocol amendment, either on an emergency (e.g., a situation that requires a patient to be treated before a written submission to the IRB can be made) or non-emergency use basis. For non-emergency IPEAS, prior IRB review and approval is required before the treatment begins.

Key Factors to Consider

FDA recommends that IRBs:

  • Establish procedures to review a non-emergency IPEAS if the physician requests a waiver of the requirements for review by the full IRB. These procedures should reflect the information that the IRB deems relevant for a single IRB member review and should include procedures designed to ensure that the member documents the decision to concur or not concur with the treatment.
  • Focus the review of an IPEAS on assessing the risks and benefits for the patient involved. The information reviewed by the IRB must be adequate to assess whether risks to the patient have been minimized and that such risks are reasonable in relation to anticipated benefits. Such information should include:
    - The proposed daily dose, route, frequency of administration & duration of planned treatment; criteria for discontinuation of treatment; and planned dose modifications for adverse events
    - The planned monitoring for adverse events, response to treatment, and changes in clinical status, as well as proposed modifications to the treatment plan to mitigate risks to the patient
    - The key details of the patient’s history, including diagnosis and summary of prior therapy (including response to such therapy); the reason for request, including an explanation of why the patient lacks other therapeutic options; and information regarding a patient’s relevant clinical characteristics (such as comorbid conditions and concomitant medications) that is necessary to assess the potential for increased risks of the drug
    - A summary of known risks of the drug

For more information please see FDA's guidance Institutional Review Board (IRB) Review of Individual Patient Expanded Access Submissions for Investigational Drugs and Biological Products.

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