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Focus on Regulation

FDA Simplifies and Clarifies Expanded Access Program

Last week, FDA announced via a blog post simplifications and clarifications to its expanded access program.  Under FDA’s expanded access program, physicians may request that patients with a serious condition receive treatment with an investigational product when there is no therapeutic alternative.  In particular, FDA made three announcements:

  • FDA has decided that physicians requesting individual patient expanded access will no longer need review and approval at a convened IRB meeting where a majority of the members are present. Instead, FDA intends to allow for a waiver of this requirement if physicians obtain approval from the IRB chairperson or another designated IRB member.  FDA says this change is intended to address delays that may result from the full IRB board not meeting routinely.  In accordance with this change, FDA has updated its guidance documents on Individual Patient Expanded Access Applications: Form FDA 3926 and Expanded Access to Investigational Drugs for Treatment Use – Questions and Answers.
  • FDA clarified that sponsors of expanded access INDs are required to report an adverse event as a suspected adverse reaction only if there is evidence to suggest a causal relationship between the drug and the adverse event. FDA says this clarification is intended to address reluctance by companies to provide investigational drugs for expanded access due to potential uncertainty about how FDA will handle adverse event data.  In making this clarification, FDA stated it recognized that it may be more difficult to determine the cause of an adverse reaction with patients receiving expanded access, because they are being treated outside of a controlled clinical setting.  As a result, they may have more advanced disease than clinical trial participants, be receiving other drugs at the same time, and have other diseases.  FDA updated its guidance on Expanded Access to Investigational Drugs for Treatment Use – Questions and Answers in accordance with this clarification.
  • Finally, FDA announced that the Expanded Access Navigator – a directory for companies to submit links to their expanded access policies and contact information – would now include information from FDA’s Rare Diseases Program. FDA said this change was intended to help promote expanded access to treatments for rare disorders.

FDA promises that “[m]ore simplifications and clarifications” to its expanded access program will be forthcoming.

If you have any questions about FDA’s expanded access regulations, please contact one of the authors of this alert or the Hogan Lovells attorney with whom you regularly work.