Pharmaceuticals Regulation

How Scott Gottlieb is transforming the FDA

The Economist has an article on the evolution of FDA regulation of pharmaceutical treatments.  Brining a drug to market costs billions of dollars.  FDA commissioner Scott Gottlieb aims to cut this cost by expediting the drug approval process in three key ways.

First, he wants the agency to rethink how much information the FDA demands at an early stage. For example, with better models of a drug’s toxicity, it could be tested in animals later in the process rather than at the outset.

Lowering development cost is not just a give-away to big Pharma; lowering drug development cost would incentivize more investment in R&D and likely help bring new drugs to market.

The second area where FDA is changing is considering allowing for adaptive trial designs. This approach has already been used to test some oncology treatments. Adaptive trial designs…

…may involve doing away with the traditional hard boundaries of the drug-testing process (known as phases 1, 2 and 3). Instead, trials constantly adapt by expanding and shrinking cohorts of patients depending on their response to treatment. This makes the process more efficient. The agency is also open to the inclusion of non-trial data (from wearables or patient records, say).

The FDA’s third area of reform is considering the use of surrogate trial endpoints. Treatments for oncology already use these surrogate endpoints. For instance, rather than measuring a drug’s effect on overall survival, many clinical trials for cancer treatment use surrogate endpoints like progression-free survival (PFS) or time-to-progression (TTP). My own research has shown the relationship between these surrogate endpoints and overall survival in the real-world for multiple tumor types. The FDA is considering more use of surrogate and biomarker endpoints for clinical trials in other diseases, such as Alzheimer’s.

Interesting developments and we will see how these play out in practice.


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