US and Europe Collaborate on Treatment of Rare Diseases

The working group was created as part of an objective to reinforce collaboration between the international agencies to address a variety of issues including biosimilars, orphan drugs, and patient safety. To collaborate on a regulatory approach to the development of treatments for rare diseases, the agencies plan on exchanging information on the design of clinical trials in small populations and the use of statistical analysis methods; the selection and validation of trial endpoints; preclinical evidence to support development programs; the design of post-marketing studies; and risk management strategies for long-term safety issues with medicines for rare diseases.

The cluster plans on having monthly teleconference meetings jointly chaired by FDA and EMA.

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