Limited patient populations that result in small study sample sizes is typically seen as the key difficulty associated with the development of therapies for rare diseases. Other hurdles often present when studying rare diseases include uncertainties around disease history, target patient profiles, and the existence of appropriate comparators. Further complications may arise when developing novel therapies and disease targets where the choices of the most appropriate primary endpoint(s) is unknown or not well established.
In this webinar, we will provide examples of clinical development issues and possible solutions from studies in rare diseases, including rare cancers.
- Natasa Rajicic, Executive Advisor, Strategic Consulting