FDA Updates Guidance on Expanded Access to Investigational IND Drugs

The U.S. Food and Drug Administration (FDA) has updated its guidance for industry on expanding access to investigational drugs through an Investigational New Drug Application (IND). ). The guidance now includes new frequently asked questions on how expanded access should be implemented given new regulatory access and legal requirements through the 21st Century Cures Act (Cures Act) and FDA Reauthorization Act of 2017 (FDARA).

Although the guidelines remain largely the same as of 2017, the FDA has added new recommendations for Institutional Review Board (IRB) review, informed consent, and new requirements in the Treatments Act and FDARA to publicly share sponsors’ policies on evaluating and responding to expanded access requests, the agency said. .

“The manufacturer or distributor is required to include contact information, procedures for submitting requests for expanded access, general criteria for evaluation and response, expected time frame for acknowledgment of receipt of such requests and a hyperlink or another reference to the record in ClinicalTrials.gov that contains information about the drug’s availability under expanded access,” the FDA wrote.

New Expanded Access Questions

The new guidelines add several questions regarding safeguards for the use of expanded access, expanded access for individual emergency and non-emergency patients, specific information in informed consent documents, and how investigators can comply with the requirement to make their expanded access policy readily available to the public.

Responding to a question about existing safeguards in the use of expanded access to an investigational drug under the IND, the FDA said that licensed physicians are investigators who administer and dispense these drugs, and that themselves and the sponsors are responsible for complying with the requirements associated with expanded access. use, such as reporting adverse events and meeting informed consent requirements as well as obtaining IRB review of the use of expanded access for the investigational drug. Sponsors are responsible for ensuring investigators are qualified to administer the investigational drug and for maintaining accurate records that show effective use of the IND’s expanded access.

The guidelines also added a section on expanded access for individual emergency and non-emergency IND patients. For Non-Emergency Expanded Access, “FDA intends to allow waivers of the review and approval requirement at a convened meeting of the IRB for Expanded Patient Access INDs individual where the IRB Chairman or other designated IRB member gives consent before treatment begins,” the agency said, while an IRB should be notified of the expanded emergency access within 5 days of a patient starting an investigational treatment so that they can be reviewed by an IRB using its standard operating procedure. A waiver for INDs and intermediate and treatment protocols is “not appropriate” in the agency’s view, the FDA said.

The FDA clarified in another question that physicians are not required to obtain local IRB approval if they participate in an expanded access protocol if the entity they are working with has already obtained the IRB review and approval. “A physician associated with a facility should verify that the sponsor has obtained IRB approval for the protocol, and the physician should consult with their facility about their policy in these situations. Some institutions may require their physicians to also obtain approval from the institution’s IRB,” they wrote.

Informed consent regulations require statements that the study “involves research,” and the FDA clarified in the guidelines that it would also be appropriate to include this statement in informed consent documents for broader access. “As an alternative, and given that the drug used under Expanded Access is investigational, the FDA considers a statement in the informed consent document indicating that although the primary use of the drug is for treatment, the drug is investigational and the FDA has not determined that the drug is safe or effective for use in treating the disease or condition, to also meet the requirement,” they said.

The agency also provided more details on how to make its expanded access policy public. A manufacturer or distributor must post the policy at the start of a Phase 2 or Phase 3 study or 15 days after the investigational drug has received Breakthrough Therapy designation, whichever comes first.

“Posting the expanded access policy on its own website will fulfill the requirement to make the policy publicly available. If a pharmaceutical company or drugmaker develops multiple investigational drugs, it may have a general expanded access policy that applies to all applicable products and should make that general policy publicly available,” the FDA wrote. “However, if it has different expanded access policies for different investigational drugs, each expanded access policy must be made public with reference to the products to which the policy applies.”

New informed consent model

The new FDA guidelines add an informed consent model that investigators can use when treating a patient with an investigational drug under an IND under the Expanded Access Program. The agency noted that the model is not intended to replace the Federal Food, Drug and Cosmetic Act Where Code of Federal Regulations and investigators are still required to meet the requirements of these laws.

The informed consent template asks the investigator to provide the name of the condition, to state that no other FDA-approved treatment is available, to note the experimental nature of the product, to explain that the patient is participating voluntarily to the program and to write the contact details of the staff whom the patient can ask questions.

Investigators should also explain the potential benefits, risks, and duration of treatment. They should note that there are no other treatment options available, list all applicable procedures associated with the treatment, and provide instances where the investigator would discontinue treatment. Investigators should document all costs and potential injuries and explain how the patient’s health information could be used. The informed consent should contain any other important information relevant to undergoing the experimental treatment, such as what happens if investigators learn of new health benefits or risks, patient compensation for the treatment, and links to educational materials.


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