The ethics of expanded access research 241 12 ♠ Yet IRBs must be careful not to unnecessarily inhibit expanded access data. They should not balk at requiring patients to agree to expanded access data collection as a condition of accessing an investigational drug, as this is no different than what is required of trial participants who may seek personal benefit from enrollment. Relatedly, IRBs reviewing plans for EA data collection may need to ensure that data are rigorously collected; other review bodies, such as scientific review committees, may also need to become involved to help maximize what can be learned from expanded access, as they often are in other types of research. Conclusion Expanded access does not fit squarely in the mold of either research or conventional clinical care. The value of expanded access data is limited compared with traditional trials, but it can provide a meaningful addition over other RWE sources, particularly for rare diseases. Companies seeking expanded access data should facilitate its collection, while FDA should mandate reporting and provide guidance to IRBs. Research goals for expanded access should not be overstated, nor should they supersede treatment goals at the heart of the expanded access pathway, but given current trends, ethical issues in expanded access data collection must be recognized and addressed.
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