Designing Platform Trials
A platform trial, a type of Master Protocol, is an experimental infrastructure to evaluate multiple treatments and/or combinations of treatments in heterogeneous patient populations. Unlike traditional clinical trials, in platform studies not all interventions are included or even known at the beginning. This approach allows us to answer the question of what the best treatment option is for a given disease in a single perpetual trial by allowing interventions to enter and leave at different time points.
Although difficult to administer, platform trials are ideal for use during a public health emergency, when rapidly identifying cost-effective new therapies is a priority. For example, the benefits of fluvoxamine, an inexpensive drug currently approved as an antidepressant, for treating COVID-19, were recently confirmed thanks to a platform research study, the TOGETHER Trial, which included key design and implementation contributions from Cytel researchers.
In the final webinar of Cytel’s Master Protocol webinar series, Dr. Kyle Wathen, Vice President of Scientific Strategy and Innovation at Cytel, presented on platform trials and lessons learned from hands-on experience working on many approaches, from single sponsor to cooperative group organized platform trials. Although, there is no best approach for design and organization of master protocols, there is value in understanding various alternatives. Dr. Wathen offers a brief overview of such options focusing on team compositions, and recommendations for optimal approaches across various scenarios.
The Biostatistics Perspective
Designing and implementing a platform trial requires several interactions, and biostatisticians should focus on the long-term strategy, rather than a single trial. They have the onus to guide the team to understand how decisions in any one area can impact the results. The statistical considerations and potential improvements need to be carefully evaluated via simulation.
OCTOPUS - Optimize Clinical Trials On Platforms Using Simulation – is a project that was released during Dr. Wathen’s time at Jannsen, R&D. It is a flexible framework for simulating platform trials and requires the knowledge of R to efficiently utilize the power of OCTOPUS. In the webinar, Dr. Wathen shares the key features of the OCTOPUS package and provides examples of Platform simulations.
To learn more about Platform Trials and its various approaches, watch the on demand webinar.
About the Author of Blog:
Mansha Sachdev specializes in content creation and knowledge management. She holds an MBA degree and has 11 years of experience in handling various facets of marketing, across industries. At Cytel, Mansha is a Content Marketing Manager and is responsible for producing informative content that is related to the pharmaceutical and medical devices industries.