The FDA and a breakthrough for drug repurposing efforts
Finding a path for off-patent drugs to reach patients for new uses
On Monday, the FDA issued a Request for Information on drug repurposing to address unmet medical needs. The agency is asking the public to identify FDA-approved drugs with compelling evidence for new uses — particularly in metabolic disease, neurodegenerative conditions, women’s and men’s health, substance use disorders, and rare diseases — where, in the agency’s own words, “there appears to be limited commercial incentive” to pursue approval.
In February, CASPR published HILT, our proposal for a NIH High-Leverage Trials Program that focuses on this problem. With support from Coefficient Giving, we’ve since been lobbying for HILT-style approaches to the market failure of off-patent medicines, working with members of Congress, HHS, FDA, and others. Things are moving faster than we had ever hoped. This new FDA RFI is a breakthrough for this field.
The bulk of the FDA RFI concerns the identification of drug candidates. Reboot Rx, EveryCure, Cures Within Reach, and others have built strong pipelines, and the agency now has a formal channel to receive them. That is a huge win.
But the RFI also goes further. In Scenario 2, the FDA explicitly asks for drug-disease pairings where preliminary clinical signals exist but the evidence is not yet sufficient for a determination of efficacy. That is precisely the gap HILT was designed to fill. CASPR will be submitting suggestions based on an ROI framework of impact for patients and cost-savings, as described in HILT.
A pipeline of repurposing candidates is only as useful as the mechanism that funds the trials needed to turn them into approvable evidence — and no such mechanism currently exists. CASPR will submit a comment focused on the barriers question: what is blocking successful repurposing when no commercial sponsor will pay for the necessary trial, and what could the FDA and its federal partners do about it? The short answer is the missing market. The longer answer is what we propose in HILT: a mechanism to identify high impact opportunities and drive funding for phase 3 trials.
The comment window closes June 11. If you’re working on repurposing and want to coordinate on submissions, please get in touch.
The Scenario 2 framing is the part that matters most, and it is also the hardest. The candidate-identification problem is largely solved, since EveryCure, Reboot Rx, and others already generate more leads than anyone can fund.
The real bottleneck is that most preliminary signals, observational or in-vitro, do not survive a properly powered trial, so a funding mechanism like HILT also needs a triage layer that ranks signals by how likely they are to replicate. Otherwise the missing market just gets filled with trials that confirm the null.
With due respect to your efforts, the current HHS leadership has repeatedly shown it rejects excellent science and promotes exactly those “ideas” most untethered to reality
It fires expert boards that are skilled and expert in evaluating treatments. It rams through policies like candy-flavored products engineered to addict children. It systematically destroys programs oriented to diseases afflicting certain subsets of the population and has fueled “influencers” in the public who claim unsupported anecdotes outweigh serious research
In the hands of an administration not corruptly driven by dogma, politics and outright fraud, HILT and other efforts would deserve fair consideration.
In this one, I fear you are only amplifying its worst instincts and will further legitimize its destruction of one of America’s bright spots in a system that hugely over-spends on placebos and gets the expected drop in healthspan
Please consider the “Law of Second Best” and find a way that your good-will efforts can do good instead of harm