The US FDA is casting a spotlight on a new approach to the development of drugs for rare pediatric diseases in which companies would test multiple product candidates in the same clinical trial.
How To Conduct A Multi-Company Trial For Rare Pediatric Diseases
US FDA draft guidance suggests efficacy endpoints for testing multiple drug products in the same clinical trials; advances an FDA-EMA joint proposal promoting innovative approaches for development of Gaucher disease medicines.
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England’s health technology assessment institute, NICE, is looking to “reimagine” its evaluation process with the help of AI, rather than just using this technology to speed up its existing processes.
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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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While the pharma industry appears to be exempt from US tariffs imposed by President Trump, a member of the UK House of Lords says the details are unknown and warned that uncertainty “leads to less investment” in business as a whole.
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The EU Clinical Trials Information System has achieved primary registry designation in the International Clinical Trials Registry Platform in a move that is expected to reduce regulatory burden for companies and help them lower compliance costs by aligning with publication requirements in medical journals.
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The UK government has listened to industry concerns about high clawback rates under the voluntary scheme and will review it in June in a bid to resolve the issue and “move on to bigger and more important things,” health secretary Wes Streeting says.