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USFDA Guidance: IRB Review of Individual Patient Expanded Access Submissions & Peptide Drug Products

Earlier today (11 September 2023), The U.S. Food and Drug Administration (FDA) Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research and Oncology Center of Excellence released a final guidance "Institutional Review Board (IRB) Review of Individual Patient Expanded Access Submissions for Investigational Drugs and Biological Products" and "Clinical Pharmacology Considerations for Peptide Drug Products".


Expanded access also known as "compassionate use," refers to the use of an investigational drug 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.


There are three categories of expanded access submissions:

  • individual (also known as single) patient,

  • including for emergency use; intermediate-size for intermediate-size patient populations; and

  • “treatment” for larger populations

The purpose of this guidance is to inform institutional review boards (IRBs) and clinical investigators about the key factors and procedures they should consider when reviewing individual patient expanded access submissions, including reviews conducted by one IRB member, to comply with 21 CFR part 56 obligations.


Peptide drug products are a subset of biopharmaceuticals that harness the power of peptides, which are naturally occurring molecules made up of amino acids. The term peptide, refers to any polymer composed of 40 or fewer amino acids.


These drugs are designed to interact with specific targets in the body, offering the potential to treat diseases at their root cause. In contrast to small-molecule drugs, peptide products are relatively large and complex, so clinical pharmacology considerations are unique and important.


According to this guidance, the FDA recommends clinical pharmacology considerations for peptide drug product development programs, including hepatic impairment, drug-drug interactions (DDIs), assessing QTc prolongation risk, immunogenicity risk, and its impact on pharmacokinetics (PK), safety, and efficacy.


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