Leuchter discusses FDA’s new pilot program.
The FDA recently launched a new pilot program to encourage the development of new drugs for rare diseases. Dr. Bruce Leuchter, CEO of Neurvati, spoke with Pharmaceutical Executive about this new program and the impact it could have on the industry.
Bruce Leuchter: We’re most interested in actionable opportunities that meet a certain threshold for likely success in subsequent development. We have the benefit of stepping back and looking across their assignments and, as the case may be, rare disease in neuroscience is a pretty relevant topic. We find ourselves organizing around our communities where the sorts of things that have been put forward in the single trial path by FDA are incredibly useful for the work we do. It is often the case where the back and forth with FDA is very accretive to what we do as a company and our efforts to collaborate with them and use them as a thought partner.
This program provides the ability to engage more frequently, have a dialogue, and be more fluid. This is an opportunity to really collaborate, which is how we see the relationship with the agency. These diseases are difficult, rare neurological diseases and patient populations are heterogeneous. It’s hard to view all patients as being equal in terms of how they present clinically, so the ability to think about the pharmacodynamic effect that drugs are having on patients and triangulating around what you’re seeing clinically in a single study with additional supporting information from the preclinical work is really important. This is especially true given the small number of patients that are participating in these studies and the very high medical need and morbidity rates. There are a lot of different factors that one needs to think about in the rare disease land, and many of them are conducive to the kind of fluid dialogue and constructive partnership that we have with FDA.
PE: How will this impact the Pharma industry from a financial perspective?
Leuchter: I think it was 1995 or so when the confirmatory evidence thesis came into play. The terminology that’s been used over time has changed a little bit but the notion that you can engage FDA in an accelerated process to deliver drugs to patients in rare disease and how to navigate the regulatory path has been a top of mind for drug developers for some time. The question marks around exactly how that plays out are what created some confusion historically, and the efforts here to clarify that and provide more definition is always a good thing.
It’s a good thing for drug developers because there’s a clear road map and that level of clarity, eliminating variables and question marks is reassuring to investors. When there is clarity from the agency, that reduces conceptual and theoretical risk in the investment thesis. We found that that being able to clarify FDA’s stance and being able to think about well-controlled advocacy data that can be supported by confirmatory data is helpful across all constituencies. That includes FDA, who is motivated to work with us to achieve success for patients and caregivers. They certainly benefit from more clarity, we benefit from more clarity, and investors benefit from more clarity. I would also add the patients and the caregivers the patient advocacy organizations benefit from that level of clarity because oftentimes in these rare disease indications, you’re working pretty closely with patient advocates and they are messaging to patients and caregivers the process that companies are undertaking to achieve approval and treat that rare disease.
PE: What will new policy and regulatory strategies will emerge due to the FDA pilot program?
Leuchter: Whether it’s a privately held, small cap, or all the way up the largest of the large cap biotech companies, this pilot program is universally applicable. There are more resources in almost every case on the large cap side of the of the ledger, and a leaner, more nimble team on the private or small cap side of the ledger, but they’re both leveraging the same accelerated path. Everyone’s got to think about delivering on a well-controlled study that shows definitive efficacy. Everyone needs to organize around whatever clinical work was done before that in an open label context and then start thinking about the data that’s generated to support the well-controlled confirmatory study. That’s true across the board. There may be there may be parallel paths that are pursued by large pharma companies, depending on the therapeutic modality and the disease state.
The strategy is pretty similar and that does create a level playing field. The ability to conduct these studies in the way that we’ve been describing creates a level playing field ,which I think is helpful. A lot of the time, the work that’s being done by a privately held or small cap biotech doesn’t matter to the large caps because those are also the partners with which entities engage with when they think about strategic transactions. Then there’s been regulatory correspondence and having a more dedicated path helps them think about the ways in which they might partner around these assets, which streamlines everything. That’s a good word to describe a lot of this. It’s a streamlining of what is in almost every case a challenging development path, given the uniqueness of the industry.