Drug repurposing: FDA extends comment period on addressing unmet medical needs (updated)

The RFI forms part of broader FDA and HHS efforts, like Project Renewal and the Make Our Children Healthy Again Strategy Report, to strengthen the use of repurposed drugs for the treatment of chronic disease, including through reliance on published literature, real world data, and emerging tools such as AI.

Drug repurposing RFI: Scope and policy context

Announcing the RFI, FDA emphasized that “[d]rug repurposing can make better use of available scientific data to deliver effective treatment options for patients in need.” The initiative is not a regulatory change but rather draws on existing tools that FDA intends to use more systematically to update labeling when supported by sufficient evidence or encourage drug repurposing, including:

• Project Renewal, an initiative in which FDA collaborates with external oncology experts to review public literature and update older oncology drug prescribing information and labeling to ensure it is clinically meaningful and scientifically up to date.
• The Best Pharmaceuticals for Children Act (BPCA), which incentivizes the study of drugs for pediatric indications when information relating to the use of those drugs in children may produce pediatric health benefits.
• The MODERN Act, under which FDA can require labeling updates for generic drugs where the reference listed drug (RLD) has been withdrawn for reasons other than safety or effectiveness and no unexpired Orange Book patents or exclusivities remain, and the approved labeling no longer reflects current scientific evidence, regulatory requirements for content or format, or accepted clinical practice.

FDA’s priority areas and evidentiary focus

FDA seeks input from patients, clinicians, researchers, and other stakeholders on both priority disease areas and specific candidates for repurposing. While FDA identified an initial set of focus areas, it also invited stakeholders to suggest additional priorities.

Priority disease areas identified by FDA include metabolic diseases, neurodegenerative conditions, women’s and men’s health conditions, substance use disorders, and rare diseases. FDA is also seeking drug candidates across the evidentiary spectrum—from those that may already satisfy the substantial evidence standard without additional trials, to those with only preliminary clinical or pre-clinical signals of efficacy.

Who should pay attention

Although the RFI is directed broadly at patients, clinicians, and researchers, several categories of industry stakeholders have a direct regulatory or commercial interest in engaging.

• Manufacturers of generics with withdrawn RLDs. The RFI is part of FDA’s effort to build an evidentiary record that could trigger the MODERN Act’s labeling update process. Manufacturers of generics within the MODERN Act’s reach should assess their product portfolios and monitor submissions to the docket closely.
• Oncology product holders. Project Renewal is an ongoing program that FDA is already extending to non-oncology disease areas. Companies with older oncology products not yet touched by Project Renewal, and companies with products in adjacent therapeutic areas, should take note.
• Rare disease and pediatric sponsors. Rare diseases are an identified priority area. The BPCA framework can be used to incentivize new pediatric uses of approved drugs, and FDA has flagged NIH’s pediatric drug program as a complementary tool. Sponsors whose products have meaningful off-label use in rare or pediatric populations could see FDA activity on their assets regardless of whether they initiate it.
• Companies with approved products that intersect FDA’s priority areas. Even companies with no plans to pursue a new indication should be aware that commenters may publicly nominate and discuss repurposing candidates. A company that learns through the docket that its product is being studied for a new use, or that a competitor’s product is, should factor that into its regulatory and commercial planning.
• Business development and pipeline teams. Docket submissions from academic institutions, patient advocacy organizations, and competitors may surface repurposing candidates and supporting data with competitive significance. Companies evaluating older off-patent assets for in-licensing or acquisition should factor FDA’s stated repurposing priorities into their diligence.

The comment deadline presents a time-sensitive opportunity to shape FDA’s evolving repurposing priorities and evidentiary expectations.

If you are considering submitting comments, or have questions about drug repurposing strategies, labeling updates, or related regulatory pathways, feel free to contact either of the authors of this alert or the Hogan Lovells attorney with whom you regularly work.

Authored by Elizabeth Jungman and Ashley Grey.