The Food and Drug Administration (FDA) published an eight-page guidance for IRBs handling expanded access to investigational products during the pandemic.

The FDA’s guidance, issued in June, explains how IRBs might review individual patient expanded access requests for investigational drugs and biological products during the COVID-19 public health emergency.

“We applaud the FDA for continuing to put out timely guidance as people are managing this,” says David Borasky, MPH, CIP, vice president of IRB compliance for WIRB-Copernicus Group in Princeton, NJ. “It reinforces guidelines already in place for research of this nature. It’s not unique to COVID; it’s a long-standing, individual patient expanded access to unapproved drugs for any condition. The FDA is getting a lot of requests from people who are not familiar with the processes or the mechanisms for getting this approved.”

The first half of the guidance speaks directly to IRBs and investigators, outlining what they need to do, he says.

“The FDA didn’t put this out to manage the supply lines,” he notes. “There were stories early on about problems with hydroxychloroquine, where people were snatching it up to stockpile for use. But there’s a difference between approved drugs used off-label vs. something that is not approved at all and is being made available through one of these pathways.”

For example, expanded access might apply to a drug that showed promise for fighting viral infections, but never made it through the study process. The product might still be available in some limited supply, and patients want to try it without waiting for a new clinical trial to begin for that product, he says.

“IRBs get involved to make sure informed consent is obtained, even though expanded access is not research,” Borasky says. “The IRB’s role primarily is to ensure there is adequate consent, and the person receiving the unapproved therapy understands that it is unapproved and is not proven to work for the condition they have.”

The FDA’s guidance includes three categories of expanded access investigational new drug applications (INDs), including:

  • Individual patient INDs. This includes emergency use INDs (e-INDs), and can be submitted to the FDA by a licensed physician as a new IND or by a sponsor of an existing IND as a protocol amendment. Requests for emergency individual patient expanded access does not require prior IRB review, but IRBs must be notified within five days of treatment with the IND. For non-emergency expanded access requests, IRBs must review and approve before treatment.
  • Intermediate-size INDs for somewhat larger patient populations.
  • Treatment INDs for larger populations.1

The FDA recommends a single IRB member review an expanded access submission for an individual patient if the physician requests a waiver from the full board review.

The FDA also recommends the IRB assess the risks and benefits for the patient, including reviewing a thorough patient history and treatment plan.

IRBs should ensure the patient’s informed consent for the expanded access product includes information about how the purpose is to diagnose or treat, rather than to investigate, Borasky says.

“This is the FDA’s best attempt at balancing the work that an IRB traditionally does in the expanded access context,” he explains. “The biggest role for the IRB is to ensure content is adequate and people understand they’re not getting an approved drug.”

IRBs also should review the request to ensure risks are minimized to what is reasonable when compared with expected benefits for the patient. A willing sponsor should provide the drug for that purpose, Borasky says.

REFERENCE

  1. Food and Drug Administration. Institutional review board (IRB) review of individual patient expanded access requests for investigational drugs and biological products during the COVID-19 public health emergency: Guidance for IRBs and clinical investigators, June 2020. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/institutional-review-board-irb-review-individual-patient-expanded-access-requests-investigational