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Abstract:
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The Orphan Drug Act of 1983 defined rare disease as a disorder or condition that affects less than 200,000 persons in the United States. Clinical trials in rare diseases can be extremely challenging for several reasons, including limited number of previous trials, limited natural history data, limited number of patients, limited understanding of the patient response towards treatment and limited understanding of the disease leading to difficulty in selection of appropriate endpoint. In addition, for most rare diseases, it can be challenging to conduct a well powered trial to detect the treatment effect. Oftentimes, a number of drugs are used for a rare disease despite limited information about the efficacy of these drugs. Hence, it is important to find efficient approaches that incorporate individual patient data using robust statistical methods and flexible and innovative study designs to especially observe disease progression and improvement. This talk will begin with an overview of the statistical challenges in rare disease, sharing some perspective from the recent FDA guidance and then discuss the role of some innovative methods for rare disease.
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