The outlook for drug development to treat rare diseases looks encouraging, offering hope to the 300 million people globally facing the significant challenges of these often-overlooked condi
For many years, orphan drugs for rare diseases have been a draw for pharma companies, offering high prices, swift regulatory reviews, and often extended marketing exclusiv
Leela Barham uses the very latest data – running up to November 2023 – to take stock of NICE’s Single Technology Appraisal (STA) recommendations on orphan drugs versus non-orphans.