Comment from Duke-Margolis Institute for Health Policy

The Duke-Margolis Institute for Health Policy is pleased to see this Request for Information from the U.S. FDA on Drug Repurposing for Unmet Medical Needs. Over the past four years, Duke-Margolis has been exploring policy solutions to unlock the full potential of our existing drugs to meet unmet patient needs. [Response to Question 1] Drug repurposing is an often overlooked but potentially impactful tool for bringing new treatments to patients quicker and at a lower cost than new drug development. For this reason, several leading use cases have been identified for drug repurposing, including pandemics/public health threats, rare and ultra-rare diseases, neglected tropical diseases, and other areas with unmet need and lack of traditional market incentives. However, drug repurposing may also be an impactful tool for many chronic diseases, and we have tried to remain disease agnostic in much of our policy work and recommendations. [Response to Question 4] Duke-Margolis’s drug repurposing policy analysis has focused primarily on the barriers and opportunities for repurposing generic drugs – or those drugs which no longer have market exclusivity. One barrier we explored is the lack of financial incentive for the pharmaceutical industry to repurpose generic drugs, including conducting trials and updating the drug’s label. Working with the Market Shaping Accelerator and their team of economists, we proposed the design of pull incentive mechanism that could be implemented by the US government (e.g., CMS, NIH, or VA) to incentive drug developers to pursue drug repurposing. We also looked specifically at the regulatory barriers and opportunities, including the potential role of the FDA in advancing generic drug repurposing. In our white paper, included as an attachment, we present a framework of potential solutions that range from actions that can leverage existing programs to those that would require statutory or structural changes. A few leading recommendations include: •Programming — The introduction of a program offering advice to nontraditional developers (e.g., academic institutions, nonprofits) on research protocols and evidentiary needs to obtain regulatory approval/attract a manufacturer partner (potentially a NCATS/FDA joint effort), including workshops, individual meetings, and guidance documents. •CURE ID Expansion — The linkage of CURE ID with a government funding partner to pull out promising candidates and prepare research protocols to meet regulatory standards (may include matchmaking conferences). •Notice and Comment — A pathway that allows nontraditional developers to submit evidence through a Citizen Petition which can then be reviewed by FDA upon review, FDA can publish a Federal Register Notice if reviewers determine there is substantial evidence for effectiveness. •New Regulatory Pathways —The creation of a “Labeling only” 505(b)(2) pathway (Reboot Rx proposal) OR a “Repurposing Evidence Pathway” for nontraditional developers to submit data directly to the FDA. •New Federal Initiative — The creation of a new large-scale government initiative (e.g., BARDA) to advance drug repurposing for unmet medical need.