Changing How Drugs Are Reviewed, Approved, and Reimbursed: Stories of [In]-Flexibility

“Regulatory flexibility” is such a common buzzword among sponsors and health agency officials that it is sometimes hard to step back and reflect on how difficult it can be to change one's mind.

This week, the Pink Sheet offers stories of agencies trying to adjust their approaches to do things faster and better, as well as an account of a country being warned it needs to do the same.

Why’s it important? 

The FDA is talking with cell and gene therapy staff about the importance of review timeline alignment for products accepted into the Commissioner’s National Priority Voucher program, CBER’s Katherine Szarama said.

Why’s it important? 

Approval of the Barth syndrome drug elamipretide marks a rare instance where a novel drug received accelerated approval even though the required postmarketing study was not underway. Stealth has until March 2026 to begin enrolling its confirmatory trial.

Why’s it important? 

Drug companies are being urged to review their internal processes to ensure compliance with new requirements in the EU’s variations guidelines, which take effect in January 2026.

Why’s it important?

The French government has been drafting the new social security financing bill, which will likely have implications for the French pharmaceutical industry with regard to drug pricing and spending.

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Why’s it important?

The US FDA's long-awaited draft guidance could help streamline dose-finding studies for the fast-growing class of radiopharmaceutical therapies.

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