Rare disease advocates are descending on Capitol Hill this week armed with the simple, yet important message to refrain from being penny-wise and pound-foolish.
Rare Disease Advocates Pushing Against Penny-Wise, Pound-Foolish Government Cuts
In the wake of cuts at the FDA, NIH and CMS, rare disease advocates are reminding Capitol Hill that some cuts can hinder progress, rather than eliminate waste.
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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.
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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.
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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.
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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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Japan adds four new pediatric vaccines, including for MMRV and norovirus, to a list of priority vaccines eligible for assistance in regulatory processing.
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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.
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The Clinical Trials Regulation was “supposed to harmonize” requirements in the EU, but instead it is giving some countries the chance to get ahead by offering faster approval timelines, notes Telethon’s head of regulatory affairs.