Abstract:
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Platform trials and Master Protocols provide an efficient approach to accelerate drug development in a wide range of therapeutic areas. Within a platform trial, we are focused on a common disease area and interested in investigating numerous current and emerging therapies for the disease of interest. A key aspect of platform trial design is establishing a master protocol with a set of global rules for how patients are treated and what actions will be taken on the different arms/therapies in terms of when success and/or futility will be declared. Concurrently, therapy-specific appendices are established that provide local rules that are unique to that therapy. One main challenge that emerges when designing a platform trial is trying to find a balance between the flexibility that is allowed in the therapy-specific appendices while still maintaining synergy and efficiency across the arms. Through the design of a Platform trial in Amyotrophic lateral sclerosis (ALS), we will highlight the key features and advantages of platform trials as well as common concerns and challenges.
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