In addition to disease-specific barriers, therapeutic developments in rare diseases face many challenges, such as incomplete understanding of natural histories to inform trial designs, need for alternatives to randomized controlled clinical trials, requirement for more sensitive outcome measures to quantify diseases, limited access to resources – including funding – required to set up clinical trials, and difficulties of recruiting small patient samples to participate in trials.
Among the above barriers, patient recruitment and enrollment is probably the most challenging, as enrolling enough patients in order to generate statistically meaningful results from a trial can turn to be highly time, energy and money consuming. Such a challenge may be overcome by good trial planning and design from the outset. Involving patient groups at as an early stage as study design could be rewarding as this can help suit patient needs and ensure those that join a study do not withdraw prematurely.
Planning, design and delivery of clinical trials is rare diseases may also be very challenging in small, dispersed populations, from both logistical and statistical perspectives. Here are some insights that could help on conducting a clinical trial in a rare disease: