The US Food and Drug Administration wants rare disease drug sponsors to know very early what questions need to be answered about potential surrogate markers in order to avoid confusion at the application review stage.
New Translational Science Team Created In US FDA’s CDER To Aid Rare Disease Drug Development
The group is intended to help rare disease drug developers answer all the necessary questions about surrogate endpoints and markers before the application review cycle.
More from Rare Diseases
• By
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.
• By
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.
• By
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.
• By
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.”
More from Pink Sheet
• By
Pharmaceutical companies are being encouraged to reach out to NICE in relation to its HTA Innovation Lab, which provides a sandbox environment in which the health technology assessment body can test new methods of evaluating “innovative and disruptive” therapies.
• By
The Brazilian medicines regulator will also offer more clarity on the requirements for radiopharmaceuticals that are exempt from registration.
• By
South Korea announces planned revisions to a government scheme to designate "innovative" biopharma companies amid allegations of "discrimination" against foreign firms.