Abstract:
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In many rare disease indications the optimal clinical development option is to employ an accelerated approach (accelerated approval, fast-track, priority, and breakthrough designation). Innovative statistical (and non-statistical) methods must be used to address these non-standard approaches in drug development. Statisticians therefore must be an integral part of the strategy behind clinical development planning. In this talk I will present and discuss examples where creative statistical methods have been used to support accelerated approval for diseases with substantial unmet medical need; including the use of historical control data, Bayesian analyses, and unique composite endpoints that may be more sensitive to detecting a treatment effect.
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