FDA Rare-Disease Guidance Brings Both Hope and Questions

Healthcare & Life Sciences Team Co-Leader Michael Werner was quoted in a Law360 article on the U.S. Food and Drug Administration's (FDA) draft guidance proposing a "plausible mechanism" framework for individualized therapies targeting rare diseases when randomized trials are not feasible. He noted the guidance aims to clarify how companies can show safety and effectiveness using evidence from small patient populations, while raising questions about eligibility, how the FDA will apply the framework in practice and what post-market data may be required. Mr. Werner also pointed to potential downstream implications for insurance coverage and broader life sciences market adoption as stakeholders evaluate therapies supported by limited clinical data sets.

"What regulated industry likes is predictability and clarity, and to the extent this guidance moves down that road, that'll be seen as a good thing," he said.

READ: FDA Rare-Disease Guidance Brings Both Hope and Questions