Development programs in rare diseases are becoming more common and a major focus for small biotech and pharmaceutical companies. A crucial consideration in clinical study and protocol design in these indications is the choice of control group used to assess the efficacy of an intervention. Standard study designs and control are often not feasible due to limitations in the number of prospective patients as well as ethical considerations. Non-standard study designs, including studies relying on external historical control groups, can be used to evaluate the efficacy of an intervention. This webinar will discuss:

• Factors to consider when evaluating the use of an external control in a planned clinical study
• Appropriate analysis and interpretation of externally controlled studies
• The critical role of externally controlled studies in supporting marketing applications for orphan/rare disease products.