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Fresh Recommendations For Rare Disease Trial Sponsors

 
• By Daniel Chancellor

Best practices to help sponsors design patient-centric studies, build trial awareness among important physician and health care professional groups, leverage advocacy groups to speed up patient recruitment, and ultimately gain a competitive edge in rare disease trial strategies.  

Rare disease awareness
Balloons released for rare disease day • Source: Alamy

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...

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