EMA Dismisses Claims That RWD Is The ‘Wrong Remedy’ For Drug Development Challenges
The European Medicines Agency has responded to an analysis that argued that generating evidence for decision making in health care using observational real-world data, and thus potentially replacing randomized controlled trials, “is the wrong remedy for the challenges in drug development.”
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EMA Report Highlights Parallel Regulatory/HTA Consultations Among Achievements
A report from the EMA summarizing the mid-term achievements of its Regulatory Science Strategy to 2025 covers health technology assessments and a range of other topics.
Upstaza: First Gene Therapy For Ultra-Rare Disorder AADC Deficiency Receives English Funding
The single-dose gene therapy for aromatic L-amino acid decarboxylase in children is to be made available on the National Health Service following a confidential commercial deal between NHS England and PTC Therapeutics.
‘Gene Silencing’ Drug Oxlumo Wins English Funding After Alnylam Improves Discount
Health technology assessment institute NICE has reversed its provisional rejection of Oxlumo for treating the rare disease, primary hyperoxaluria type 1.