Sponsors developing drugs for rare diseases should pursue several strategies to assess safety, including gathering robust natural history data and using a concurrent comparator arm, the US FDA advises in a new draft guidance.
Rare Diseases: US FDA Gives More Guidance On Premarket Safety Evaluation, Historical Controls
Agency specifies several approaches to augment safety assessment in a new draft guidance, which also encourages participation by patients and their advocates in the drug development process.
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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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South Korea announces planned revisions to a government scheme to designate "innovative" biopharma companies amid allegations of "discrimination" against foreign firms.