Developers of medicines for rare diseases in children should consider the possibility of testing their products in multi-company, multi-product studies, according to a joint proposal by regulators in the US and the EU.
Multi-Company, Multi-Product Clinical Trials On The Cards For Rare Pediatric Diseases
Industry is guardedly optimistic about a new joint proposal from US and EU regulators for companies to test their drugs for rare pediatric diseases together in single multi-product studies.
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Reporter and editors from the Pink Sheet, Medtech Insight and HBW Insight consider the impact of the layoffs at the FDA on its drug, device and consumer products divisions, as well as the so-far limited public reaction from industry.
A lower asking price from BMS and more insight into the benefits Breyanzi offers have convinced the health technology assessment institute, NICE, to reverse its rejection of the one-off treatment for large B-cell lymphoma.
While CSL Behring has successfully negotiated reimbursement contracts for its one-time gene therapy Hemgenix in several European countries, it says there are still “barriers to innovative contracting solutions” in some member states that need to be addressed.