How patient organizations are driving rare disease drug development
Patient organizations in rare disease are no longer waiting for pharma to act, and a growing number now sponsor their own trials. In this Clinical Leader conversation, Craig Lipset and Dr. Derk Arts explain why the math forces it: the industry approves roughly 50 new medicines a year while researchers characterize 150 to 250 new rare diseases. They cover how small populations can still generate real evidence, why patients should govern their own data, and how master protocols can cluster related conditions under one design. A practical look at an efficiency lever that has been hiding in plain sight for clinical-development leaders.