Why Rare Disease Drug Developers Need Regular Interaction with the FDA
Despite accelerated pathways for the development and approval of rare disease therapies, significant challenges remain for companies wishing to bring new treatments to market. Mallory Factor, CEO of IntraBio, recently testified at a U.S. Senate Subcommittee hearing and argued that the programs in place today fail to address the needs companies like his have for timely and early interactions with the U.S. Food and Drug Administration when developing therapies for diseases afflicting small populations of patients. We spoke to Factor about his recent testimony, the issues companies like his face, and what he’d like to see done to address the problem.
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6 年Yes! Time has come for a reality check to factor in risk/benefit analysis and QOL endpoints rather than just risk analysis when it comes to rare and horrific, deadly diseases. And more collaborative, frequent, “kitchen table” strategizing with the super smart FDA team members will get meaningful beneficial treatments to patients dying for help. This of course requires funding to The Agency via conviction by the senate. As we say in the CNS space, this is a no brainer. Or, we can do this. It’s not brain surgery.