While individually rare, orphan diseases collectively are actually quite common, with an estimated 350 million sufferers worldwide. Since the introduction of the Orphan Drug Act in the US more than 30 years ago, followed by legislation in Japan in the 1990s, and the EU Regulation on Orphan Medicinal Products in 2000, the number of orphan designations has skyrocketed. There are currently around 566 orphan drugs in development, encompassing many diff erent therapeutic areas. Despite the strong financial and regulatory incentives introduced by the legislation, drug development programmes for the treatment of rare diseases still face many challenges. These include the small number of patients available, poor understanding of the disease, difficulties associated with trial design, and challenges with patient enrolment and retention. This paper discusses some of the practical approaches that can be taken to facilitate drug development in this challenging field.
Coralie is Clinical Project Manager (CPM) at VCLS. She has 10 years of experience in management of international clinical studies, in adult and pediatric populations, for common and rare diseases. She led cross-functional teams to achieve study milestones, ensuring successful planning, conduct, completion and follow-up of clinical trial activities, within budget and timelines, in compliance with corporate SOPs, ICH/GCP and local regulatory requirements.
Jean-Luc Béjot, MD, MBA, has over 29 years of international experience in clinical development and in medical affairs in the life science industries. His main experience and expertise lie in early stage (phases I and II) clinical development, and he has been exposed at various depth to many steps of the medicinal products life cycle (medicinal chemistry, pre-clinical, clinical, marketing, licensing).View Profile
Emmanuel has significant experience in the management of international clinical studies (pre, post-market. Device/feature validation, research studies) from protocol writing to report.View Profile