ABSTRACT
Platform trials are increasingly being used in drug development. Factors contributing to this phenomenon include a better understanding of the biologic underpinnings of disease, the desire of multiple stakeholders to conduct development more efficiently, and the limited pool of patients available for enrollment in clinical trials. Regulators are taking an increased interest in these trials as they seek to play a more active role in guiding development as partners, not just as passive reviewers of data. The availability of regulatory tools to allow acceleration of development and review, including the possibility of filings with phase 1/2 data in some cases, has also contributed to an increased interest in these trials. Regulators are interested in the flexibility that these trials provide, while making sure that the rigors of trial design and analysis are maintained in order to assure interpretation and use for regulatory purposes. In oncology, platform trials are being used to validate novel endpoints, to assess safety and efficacy in a tumor pathology agnostic manner, and to potentially address questions relevant to the post-marketing setting. Regulators are also interested in partnering with multiple stakeholders to expand the use of platform trials to other disease areas such as rare diseases, inflammation/immunology, and infectious diseases. Special regulatory considerations arise when platform trials are used to address questions regarding novel combination therapies, drug-diagnostic development, and pediatric development. Future considerations include the need to explore novel statistical approaches that enable transitioning from exploratory to confirmatory studies, data management and prioritization, how to better leverage platform trials for development of biologics and combinations, and the role of platform studies in an overall platform development paradigm.
