On October 16, the FDA will host a public meeting to discuss its recently announced Rare Disease Innovation Hub, an initiative designed to expedite the development and approval of new treatments for patients with rare diseases. Cure Xchange Challenge winner Every Cure plans to attend the meeting to contribute ideas for making the hub a success, says CEO and co-founder Grant Mitchell, MD, MBA.
Every Cure’s mission is to use artificial intelligence to match existing drugs with diseases for which there are no good treatments. Most of the estimated 10,000 rare diseases have no approved treatments, so Every Cure’s work will no doubt intersect with the FDA’s initiative to promote innovation in rare disease.
The FDA’s hub is largely aimed at streamlining the development of new products that address rare diseases by improving collaboration between the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER), and undertaking other initiatives to advance biomarker development, innovative trial designs and more.
The Rare Disease Innovation Hub comes at a time of huge growth for companies working in the field. One estimate puts the market size for rare disease treatments at $205 billion and growing at a compound annual rate of 9 percent through 2032. And investors are supporting rare disease research at record rates. For example, rare disease startups CRISPR Therapeutics, EnGene Holdings and Larimar Therapeutics raised a combined $630 million from investors earlier in 2024.
Mitchell spoke recently with Cure about his hopes for the Rare Disease Innovation Hub and his experiences as part of Cure Xchange Challenge.
This conversation has been edited for length and clarity
Cure: What are some of the main barriers to innovation in rare disease that you hope the FDA’s initiative will help resolve?
Mitchell: I always think about this from the lens of drug repurposing. Why hasn't drug repurposing happened at scale? One reason is that no centralized database exists that aggregates biomedical data in a way that could be useful for identifying repurposing opportunities.
The Rare Disease Innovation Hub could be a nexus for all the patient advocacy groups, research foundations and key opinion leaders in rare disease. We hope that would lead to initiatives aimed at compiling and ultimately sharing data in a way that would benefit the rare disease community as a whole. We’re thinking a lot about what role we can play to encourage that data aggregation.
Cure: Incentivizing drug developers to pursue innovations for diseases that affect a small number of patients has been challenging. How might the FDA’s hub address this and how would that benefit Every Cure?
Mitchell: There's a lack of profit incentives around advancing generic drugs with no intellectual property into rare diseases with few patients. If this effort at the Rare Disease Innovation Hub starts to highlight the gaps and incentives for drug repurposing for rare disease, that would be a benefit.
If it ultimately leads to programs that encourage incentives, we'd be a huge advocate of that. Incentives could include voucher programs for discovering a new use for an existing drug, or extended exclusivity periods. We don't know if that's in the remit right now to be advocating or recommending those kinds of incentives, but we hope that at the very least the activities of the hub point to the need for incentivizing innovation in rare diseases.
Cure: Part of the FDA’s effort is aimed at advancing novel endpoints in clinical trials and innovative trial design. What do you hope to see there?
Mitchell: When we think about ensuring patient access to drugs in the world of repurposing, we don’t often include FDA approval, because so difficult to get there. We think more in terms of updating guidelines. If we can update guidelines and get buy-in and trust from the physician community to prescribe a drug to patients who will benefit, then that's great.
That being said, if the Innovation Hub promotes innovative trial design efforts, that would enable us to match drugs with diseases more efficiently. For example, basket trials would allow us to test multiple drugs and multiple diseases in the same trial.
Cure: What is Every Cure’s plan for the public meeting that the FDA is holding on October 16?
Mitchell: We are attending and we hope to elevate drug repurposing. They haven’t made any explicit statements yet on drug repurposing, so we hope to promote the idea that it should be a key part of their strategy to address rare disease.
Cure: How has winning the Cure Xchange Challenge benefitted your company?
Mitchell: We were so excited to win the Cure Xchange Challenge because it put us right at the center of the ecosystem in New York City around drug development, and particularly data-driven drug development. We really value just being in the building, sitting next to all the other organizations that are here, and having highly productive conversations with other organizations that are working on different problems.
For example, Insilico Medicine is developing novel drugs using AI, which is different from repurposing drugs. But where we can learn directly from each other is from the fact that we're both dealing with massively parallel analysis, and millions of potential leads to pursue. So being able to talk to a fellow CEO who is dealing with that same sort of framework of a problem, but in a slightly different way, and cross-pollinating our ideas and experiences has been really energizing and beneficial. We will continue to be part of this ecosystem for the long term.