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Terms of reference for the EMA/FDA cluster on rare diseases

The primary goal of this rare disease cluster is to share scientific evaluation of various aspects of drug development for rare diseases.These aspects include selection and validation of trial end points, potential trial designs in small populations, opportunities for regulatory flexibility (approval supported by other than two adequate and well controlled studies and/or use of a novel endpoint), determination of safety populations, evaluation of pre-clinical data needed to support human trials, and design and conduct of post-marketing studies especially in the cases of accelerated approval (FDA) and conditional/exceptional approval (EMA) or breakthrough designation (FDA) and PRIME designation (EMA). The main mechanism to achieve these goals will be regularly scheduled teleconferences, including individuals from review divisions and therapeutic teams, for exchange of information and experiences.

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