US FDA will continue to tie orphan exclusivity to uses or indications for which the drug was approved. Attorneys say this buys agency time to sort out complex policy and legal questions and continue to seek legislative fix. Additional litigation is also possible, as happened in Depomed case.
The US Food and Drug Administration has decided not to adopt the Catalyst court ruling limiting the scope of orphan drug exclusivity and instead will continue to tie exclusivity to the uses or indications for which the drug was approved rather than to the entire disease. The move leaves questions about the agency's policy unresolved and will likely lead to further litigation.
The agency announced in a Federal Register notice scheduled for publication on 24 January that it would set aside...
Artificial intelligence tools used for clinical research should be developed with input from patients and focus on their needs and abilities, FDA experts said.
Office of Therapeutic Products Director Nicole Verdun and her deputy Rachael Anatol were escorted out of FDA headquarters on June 18. Disagreements over CAR-T regulation and Capricor’s DMD treatment may be to blame.
As Chief Medical and Scientific Officer, Vinay Prasad will advise the FDA Commissioner on cross-cutting issues, provide strategic leadership and be a public face of the agency.
Highlights from Day Two of the BIO International Convention include BIO officials raising concerns about Trump Administration policies, the future of ACIP, an interview with BI's head of global business development, and FDA Commissioner Martin Makary's view of the FDA-industry relationship.
The FDA will use electronic health records to “get eyes” on drugs immediately after approval, Commissioner Martin Makary told the DIA 2025 Global Annual Meeting. He also wants to reduce development time with better communication and new approval pathways.
Center for Biologics Evaluation and Research Director Vinay Prasad said Commissioner Martin Makary wants to release previously inaccessible FDA documents, but previous commissioners have made the same pronouncements only to make little progress.
FDA employees are concerned that while the budget authority total proposed for fiscal year 2026 meets the user fee appropriations' triggers, the FDA could have trouble meeting allocation triggers without cuts to non-review work.
