We Responded to the FDA’s Request for Information on Drug Repurposing: Here’s What We Said
June 10, 2026
Every Cure submitted a formal response to the FDA’s Request for Information (RFI) on Drug Repurposing for Unmet Medical Needs (Docket No. FDA-2026-N-4492). This RFI displays FDA’s growing interest and commitment to drug repurposing, and we took the opportunity to share what we’ve learned from years of working at the forefront of this field.
Below is a summary of the key themes from our submission and why we believe this RFI represents an important step forward for the future of drug repurposing. We’re excited to share our full comments once they are posted to the FDA’s public docket.
Why this RFI matters
It is an exciting time for drug repurposing. Seeing the growing momentum across the field, and the FDA taking a leadership role in advancing the conversation, gives us real hope. At Every Cure, we believe collaboration is essential to unlocking the full potential of existing medicines, and this RFI is an important step toward helping more patients benefit from therapies that already exist.
Fewer than 25% of recognized diseases have an approved treatment, leaving millions of people without effective options. Yet many promising treatment opportunities may already exist among drugs that are FDA-approved and readily available today. Diseases often share common biological mechanisms, meaning a drug developed for one condition may also help treat another. While scientific, regulatory, and economic barriers have made it difficult to advance these opportunities at scale, the growing attention to drug repurposing gives us reason for optimism. By bringing together patients, clinicians, researchers, industry, and advocacy organizations, this RFI creates an opportunity to identify practical solutions that can help more repurposed treatments reach the patients who need them. Every Cure was excited to add our voice to that conversation.
What we said
Our response covered all four topics the FDA asked about (priority disease areas, drug repurposing candidates, approaches to identifying repurposing candidates, and barriers and opportunities for drug repurposing).
On priority disease areas, we shared insights from our publication on quantifying unmet medical need (PLOS Medicine, 2026), which highlights our ability to score all 22,701 recognized diseases across criteria including patient suffering, quality of existing treatments, and access to care. We broadly agreed with FDA’s identified priorities: rare diseases, metabolic conditions, neurodegenerative diseases, women’s and men’s health, and substance use disorders. But we pushed further. Within each of these categories, unmet need varies enormously depending on what treatments already exist. We recommended that FDA prioritize at the disease level, not just the category level and that it explicitly account for commercial neglect, not just clinical severity. The highest-value repurposing opportunities are often not the diseases with the most scientific attention, but the ones where the science exists and nobody has the incentive to act on it.
On drug repurposing candidates, we submitted nine specific drug-disease pairs including candidates where we believe sufficient evidence already exists to support regulatory changes that could help expand patient access. These include DFMO for Bachmann-Bupp syndrome, lenalidomide/dexamethasone for Rosai-Dorfman disease, and lidocaine for breast cancer. To learn more about the repurposing opportunities highlighted in our submission and our other active repurposing programs visit everycure.org/portfolio.
On approaches to identifying candidates, we described our AI platform and the methods behind it. We recommended that FDA invest in shared biomedical data infrastructure, expand CURE ID beyond infectious diseases, and leverage the Sentinel System to screen for off-label use patterns that may signal repurposing opportunities. We also noted that this RFI is itself an act of structured crowdsourcing, and that FDA should build on that instinct by creating mechanisms to capture clinical knowledge that doesn’t make it into the published literature.
On barriers and opportunities, we were direct about what isn’t working and offered several actionable recommendations. Today, no single stakeholder is positioned to advance a repurposing opportunity from discovery to patient access. Clinical studies for off-patent drugs often lack sustainable funding, non-commercial sponsors face uncertain regulatory pathways, and even well-supported repurposed treatments can struggle to reach patients if coverage policies have not kept pace with the evidence. To address these challenges, we recommended clearer guidance for non-commercial sponsors, structured opportunities for early engagement with the FDA, stronger coordination between FDA and CMS on coverage and reimbursement, and sustained federal investment in generating evidence for high-priority repurposing opportunities. Together, these changes could help create a more reliable pathway for translating promising repurposing discoveries into treatments that patients can access and benefit from.
The bigger picture
Drug repurposing has the potential to transform care for millions of patients, but realizing that potential will require coordinated action across regulators, researchers, clinicians, funders, and patient communities. We believe the FDA’s RFI is an important step in that process, and Every Cure is proud to have contributed our perspective and experience.
Comment submissions close on July 13, 2026. We look forward to sharing our full submission once it is posted to the public docket.
