Economic Evaluation of Cost and Time Required for a Platform Trial vs Conventional Trials

Abstract

This economic evaluation compares the cost and time associated with platform trials vs conventional trials.

Proceedings of the 2022 NHLBI and OASH state of the science in transfusion medicine symposium

“…Clinical trials are needed which compare blood products, antifibrinolytics, and coagulation factor concentrates simultaneously to determine if the concurrent use of each type of therapeutic agent increases efficacy or worsens safety 24,25 . These research questions are ideally suited for adaptive trial designs that include platform trials to facilitate the study of combinations of therapies 26,27 …”

“…24,25 These research questions are ideally suited for adaptive trial designs that include platform trials to facilitate the study of combinations of therapies. 26,27…”

An Economic Perspective on Platform Trials—The Gift and the Curse

“…2 It is often claimed that one of the greatest advantages of platform trials is their potential to use a single infrastructure (eg, common centers, investigators, independent data monitoring committee, protocol, recruitment procedures, database, and so forth), thus offering certain administrative and logistic advantages that further translate into a greater economic efficiency. In their article, Park et al 3 compared the cost and time of setting up, conducting, and analyzing a platform trial with other development strategies, including a series of independent randomized clinical trials. The authors demonstrated that, via simulations informed by the famous Systemic Therapy in Advancing or Metastatic Prostate Cancer: Evaluation of Drug Efficacy (STAMPEDE) trial, the cost and time of planning and conducting a platform trial is larger than that of a single trial but smaller than other sequential development options including several trials.…”

“…The authors demonstrated that, via simulations informed by the famous Systemic Therapy in Advancing or Metastatic Prostate Cancer: Evaluation of Drug Efficacy (STAMPEDE) trial, the cost and time of planning and conducting a platform trial is larger than that of a single trial but smaller than other sequential development options including several trials. Even though in the absence of publicly available data on the cost and setup time of clinical trials, the authors had to resort to a survey of experts to inform their analysis, the article illustrates that, when the intention is to generate efficacy data on several treatments, it is more efficient in the long run to do so via a platform trial. Otherwise, if, for example, the read-out of a specific treatment is likely to condition the investigation of the other experimental treatments, then a more staggered approach might be preferrable.…”

“…While the article from Park et al deals with economic aspects pertaining to the design, setup, and analysis of the trial, supplementary savings in cost and time can arise from the structure of the platform trial itself. First, the opportunity to receive 1 of several experimental treatments with a greater probability than receiving the comparator can be attractive for both investigators and participants and can boost recruitment.…”