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Challenges in Conducting Clinical Trials in Rare Diseases

Post thumbnail Challenges in Conducting Clinical Trials in Rare Diseases

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.


Published on: February, 2018
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    publication by

    Coralie Hutchinson thumbnail
    Coralie Hutchinson
    Associate Director Clinical Trial Submission and Operations
    Emmanuel Prades thumbnail
    Emmanuel Prades
    Senior Director, Medical Devices
    With over 20 years in Clinical development in diverse areas from pharmaceutical to medical device industry, Emmanuel has broad technical expertise in clinical trial regulations and associated country-specific requirements and has extensive experience in various trial phases, product types, indications, and geographical areas. He has extensive experience in strategic clinical decision meetings with various cross-functional activities for regulatory, safety, quality, and study organization. He also has experience in quality as a clinical QA advisor (audit, SOP writing, Team training).
    Jean-Luc Béjot, M.D., MBA thumbnail
    Jean-Luc Béjot, M.D., MBA
    Vice President, Corporate Strategy