Hiring, not reorganization, is the answer to rare disease expertise issues at the US Food and Drug Administration’s Center for Drug Evaluation and Research, Director Patrizia Cavazzoni said.
Rare Disease Challenges At US FDA: CDER Director Says More Staff, Not Reorganization, Will Fix Issues
Rather than changing the agency's structure to create more focus on rare diseases, Patrizia Cavazzoni wants funding for people to do the necessary work.

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Not all companies will be able to access joint scientific consultations under the EU Health Technology Assessment Regulation, but success is still possible for those that engage with national agencies early on, says EUCOPE’s Alexander Natz.
Payers and health technology assessment bodies in the Netherlands, Germany and Italy are either unwilling to use real-world data in assessments or cannot due to their existing frameworks, say representatives from Gilead Sciences and Autolus Therapeutics.
Companies have until 17 March to respond to an EU consultation on a new strategy that will seek to simplify the regulatory framework and make it easier for innovative small and medium-sized enterprises to “access the capital they need” to scale up in the bloc.
Center for Biologics Evaluation and Research Director Peter Marks expects no fundamental changes in support for FDA initiatives and said the Rare Disease Innovation Hub, which promotes cross-center collaboration, is “consistent with what we're hearing in the current environment.”
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