Rare disease and pediatric studies present significant challenges, including country selection, site selection, and patient recruitment.
A thoughtfully-designed, well-executed feasibility study can help sponsors obtain a realistic assessment of the best path forward for developing and conducting successful global clinical trials.
Performing a thorough assessment of clinical trial feasibility is an important early step in initiating global clinical trials, particularly in rare disease or pediatric studies where country selection, site selection and patient recruitment each represent a significant challenge to successful clinical trial conduct.
A 2015 survey of 50 clinical development decision-makers in the U.S. and Europe revealed that identifying and setting up investigative sites were among the most difficult factors of conducting rare disease clinical trials. According to recent studies:
- Only six percent of all clinical trials are completed on time
- Eighty percent of all clinical trials fail to meet enrollment timelines
- Nearly 60 percent of all clinical trials that are terminated are terminated due to enrollment difficulties
Clinical trial feasibility helps in identifying potential obstacles to clinical trial conduct, enabling sponsors and study planning teams to proactively work with different countries, sites and investigators to develop processes and practices that mitigate risk and support study completion.
Investing in a robust feasibility process enables sponsors of rare disease or pediatric studies to obtain a realistic assessment of the capability of executing a planned clinical trial and the best path forward for developing a clinical research program. Feasibility assessments are critical tools, but they can be time-consuming and costly if they lack appropriate focus and direction.
A well-designed and executed feasibility study is the linchpin of a successful clinical research program. This white paper delves into three critical aspects of clinical trial feasibility with respect to rare disease or pediatric studies – country feasibility, site selection, and projection of patient recruitment – and offers