The US FDA may create a pool of rare disease specialists to enhance advisory committee expertise when relevant applications are under review.
US FDA May Create Rare Disease Expert Pool For Advisory Committee Reviews
FDA's Peter Stein calls idea "useful"; rare disease experts would temporarily serve on standing advisory committees when needed.
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The hub’s goals of efficiency, centralization and coordination fit with the Trump Administration’s current focus for the FDA, Amy Comstock Rick, director of strategic coalitions, told the Biopharma Congress.
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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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Israel has introduced a new framework to expedite the marketing approval of medicines, make the country a more attractive destination for drug registration and help reduce drug prices through market competition.
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Stay up to date on regulatory guidelines from around the world with the Pink Sheet’s Guidance Tracker. The complete Global Pharma Guidance Tracker, with sortable and searchable listings going back to 2014, is available online.
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US-based Colossal Labs has claimed to bring back the dire wolf from extinction. While the science is not as dramatic in India yet, cell and gene therapies are making progress as treatments for cancer and other diseases. Pink Sheet takes a look at what is driving CGT success and growth