Approved drugs are available for just 5% of the approximately 7,000 rare diseases identified, many of which are life threatening or limiting. The pharmaceutical industry is making inroads through R&D, despite the unique challenges facing clinical trials for rare diseases, not to mention often navigating without regulatory precedents and an uncertain commercial outlook. Progress can therefore feel slow despite the priority that many drug companies place on these patients and their unmet needs.
Rare diseases are so-called because of the low number of prevalent patients, with long-standing definitions being fewer than 200,000 people...
Read the full article – start your free trial today!
Join thousands of industry professionals who rely on In Vivo for daily insights
- Start your 7-day free trial
- Explore trusted news, analysis, and insights
- Access comprehensive global coverage
- Enjoy instant access – no credit card required
Already a subscriber?