ABSTRACT
Driven by the intersection between an urgent need for molecules to treat life-threatening diseases and the escalating resources required to develop innovative medicine, platform trials have been created to advance the most promising molecules and novel combinations with the greatest effect sizes, in order to provide more value to society. While precision medicine can match the right patient to the right drug, it may significantly increase the ratio of screened to enrolled patients within trials, providing challenges as to ways to capably prosecute development programs. If designed effectively, a platform trial can serve as an engine to generate a robust set of data to inform developers on where to differentially invest resources across their pipeline. Designing a successful platform trial requires the understanding of the potential for Type I and II errors, frequent and transparent communication and negotiation among developers, and potentially reduced autonomy in order to deliver a mutually-agreed upon trial among sponsors. Collaboration between developers and key stakeholders, timely engagement with payers on the value of trial outcomes, greater incentives in support of transformational medicines, and appropriate risk taking are needed to enable the success of drug development in a cost-constrained environment. Foundational to this innovation is the platform trial, which has the potential to significantly accelerate innovation and reduce uncertainty.
