Drugmakers, advocacy groups ask FDA to clarify expanded access draft guidance

Regulatory NewsRegulatory News | 06 January 2023 |  By 

Pharmaceutical companies and research advocacy groups are asking the US Food and Drug Administration (FDA) to clarify some of the provisions of its recently updated expanded access draft guidance. Commenters inquired about the agency setting a response time frame for responding to expanded access waivers, when children qualify for expanded access drugs and expanded access to medical devices.
In November, FDA published the updated draft guidance for when investigational new drugs (IND) can be used under expanded access. It includes new frequently asked questions about how expanded access can be implemented based on new regulatory access and statutory requirements under the 21st Century Cures Act (Cures Act) and FDA Reauthorization Act of 2017 (FDARA). (RELATED: FDA updates guidance on expanded access for investigational drugs under IND, Regulatory Focus November 2 2022)
Drugmakers Bayer and Novartis were among the stakeholders who submitted comments to the draft guidance.
Bayer asked FDA to make it clearer that under certain circumstances, the agency will grant a waiver for non-emergency expanded access to drugs where an institutional review board (IRB) does not need to review the drug’s use but rather its use can be waived by the IRB chair or designee. The drugmaker also asked FDA to state explicitly that it plans to give a decision on the waiver within 15 days of the request.
“To avoid a potential delay in treatment, add a timeframe for FDA response to the waiver request,” Bayer wrote.
The German multinational pharmaceutical company also asked that at the conclusion of treating individual patients under expanded access, a summary of their results should be allowed to be included in the IND annual report.
While FDA provides examples of when expanded access may be used, such as when a drug has been withdrawn for safety reasons but may still be beneficial to certain patients, and to address drug shortages, Novartis has asked the agency to provide more examples of when drugs can be used under expanded access.
Several research organizations also submitted comments to the docket, including the International Society of Gene and Cell Therapy (ISGCT).
The ISGCT noted that FDA has proposed allowing expanded access to INDs for intermediate-sized patient populations where there is preliminary evidence the drug is effective. However, the group notes that patient populations with rare diseases typically have much smaller cohorts and has asked FDA to clarify its position.
“In many patient populations of rare diseases, a ‘standard’ clinical trial is much smaller than typical,” said the ISGCT. “What would FDA define as ‘intermediate size’ for a disease or condition that affects an already small number of patients.”
The GE2P2 Global Foundation also submitted comments to the docket and asked FDA to elaborate on its position on when children may qualify for drugs under expanded access. The group said that the agency may be setting an arbitrary limit to when children can qualify for such drugs when it states that children 7 years of age and older may have the ability to consent.
“We recognize that children and young persons are among patients requesting and benefitting from expanded access to investigational drugs, medicines and therapies,” said GE2P2. “Depending on state law, children and young persons generally do not enjoy any informed consent rights. But clearly, the protections provided by robust consent in adults in expanded access situations are not less important to young persons – indeed such protections may be more important.”
The group asked FDA to be more explicit about its assent process for allowing children access to expanded access drugs. It also asked the agency to address the issue in the agency’s recent draft guidance entitled, “Ethical Considerations for Clinical Investigations of Medical Products Involving Children.”
The Transforming Expanded Access to Maximize Support and Study (TEAMSS) collaborative asked the agency to look beyond drugs in their guidance.
“Although the guidance states it applies to investigational drugs and biologics, we encourage the agency to continue to develop these documents for a wider range of products,” said the group. “There continues to be a significant need for guidance for [expanded access] to medical devices through the Center for Devices and Radiologic Health (CDRH), which is not covered in this draft guidance document.”
TEAMSS also said they are concerned about FDA’s emphasis on single-patient protocol submissions to existing INDs, particularly when the drug is in active development. The group said the focus puts physicians and institutions in a difficult position because it may delay securing expanded access documentation which may lead to noncompliance; lack of time frames and waiver status, which may delay the start of an institutional review process; and may lead to logistical burdens for intermediate-sized patient populations.


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