Patient-Focused Clinical Trial Design For Rare Diseases Like Duchenne Muscular Dystrophy

Any clinical program has its challenges: from recruiting patients, staying within timelines and handling the inevitable trial amendments, clinical researchers must manage all aspects of the study for the best chance of success. For clinical trials of rare diseases, these challenges can be amplified, particularly because the eligible patient population is so limited.

Contract research organizations (CROs) and clinical investigative sites are increasingly adopting a patient-centric approach to trial design to address some of the challenges inherent in clinical research. That special focus on both finding the right patients to enroll, and making it as easy as possible for them to participate in the clinical trial, can be a key differentiator between trials that meet their endpoints, and those that don’t.

This article highlights recent changes that are benefitting rare disease trials including multi-center trials, US Food and Drug Administration (FDA) and other regulatory bodies establishing programs aimed at incentivizing rare disease drug development and regulatory agencies now having smaller requirements for the number of patients in which a therapy must be tested before it can be considered for approval.