Evaluating the Current State of Randomized Platform Clinical Trials

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A study recently published on JAMA Network Open found that a group of randomized platform clinical trials were often lacking in the reporting of adaptive features and results.¹ This type of trial design is becoming increasingly popular as it solves challenges commonly seen with randomized controlled trials (RCTs), which include a lack of flexibility, timeliness, and cost efficiency.

“Existing platform trials predominantly focus on evaluating drugs and tend to cluster in medical areas, such as oncology, COVID-19, and other infectious diseases. After the peak in 2020 with the arrival of the COVID-19 pandemic, the initiation of new platform trials has decreased,” the study authors wrote. “However, there has been a noticeable diversification of medical fields and interventions of platform trials over the past 5 years. This diversification encompasses areas such as neurology, dermatology, and general surgery, as well as the testing of behavioral, surgical, or dietary interventions.”

The authors analyzed a pool of 127 platform trials with a total of 823 arms, primarily in the fields of oncology and COVID-19. Accessible information such as the use of control arms, adaptive design, and adjustment for multiplicity was used to evaluate the trials.

Analysis of the data found platform trial features were often not reported in 48.0% of the selected trials. This includes no reporting on multiplicity adjustment for arms (77.2%) and statistical framework (29.1%). In addition, adaptive design features were only used by half of the studies. Results were available for just 65.2% of closed arms. For added context, the authors also found that there was a more than twofold increase in the initiation of new platform trials at the beginning of the COVID-19 pandemic and the number of platform trials since has declined.

While reporting for the randomized platform trials was found to be insufficient, the authors found that almost 80% of platform trial protocols were publicly available in some format. This is much higher than what previous research has found to be the case with traditional RCTs, according to the investigators.

“However, reporting of essential features [for randomized platform trials], such as adjustment for multiplicity, use of nonconcurrent control data, and criteria for dropping and adding new arms, was often unsatisfactory,” the authors wrote.

Looking forward, the authors aim to conduct a similar analysis. However, it will be a direct comparison of platform trials to RCTS. It will build off the results of this study, which is believed to be first to investigate key platform trial features, protocol and results availability, and the status of individual arms, according to the authors.

“In this systematic review, we found that platform trials were initiated most frequently during the beginning of the COVID-19 pandemic and appeared to decrease thereafter, with a trend toward more diversified medical fields and interventions. Despite the potential for complexity, most made use of only 1 adaptive feature, or none,” the authors concluded “The reporting of platform features, the status of trial arms, and the results of closed arms needs to be improved. Guidance and infrastructure are needed so that the status and results of individual trial arms can be reported in a timely manner (eg, adaptations of trial registries for platform trials) and so that decisions about the need for a platform design and its planning is optimized.”

Reference

1. Griessbach A, Schönenberger CM, Taji Heravi A, et al. Characteristics, Progression, and Output of Randomized Platform Trials: A Systematic Review. JAMA Netw Open. 2024;7(3):e243109. doi:10.1001/jamanetworkopen.2024.3109. https://jamanetwork.com/journals/jamanetworkopen/fullarticle/2816496