Highly Efficient Clinical Trials (HECT)
Randomized clinical trials (RCTs) are the gold standard for establishing the safety and efficacy of health interventions. However, they can be expensive and time-consuming—and the results can be hard to generalize.
New techniques allow us to optimize trial design to obtain better results in less time and at a lower cost. Adaptive trials rely on a process of modifying the design during the course of the trial based on interim analyses of the data.
This application helps investigators explore a wide range of trial designs, including adaptive and platform designs, to maximize the efficiency and impact of the trial.
By exploring a wide range of trial parameters, investigators can compare various adaptive designs in terms of cost and operational characteristics such as power. Possible adaptations include adjusting the randomization ratios according to intermediate results as well as stopping an arm or terminating the trial due to superiority or futility. The application helps investigators consider a variety of stopping criteria under the comparison type that best suits the primary research questions. In addition, exploration of a platform design where a trial arm may be added later during the course of the trial is facilitated.
Common challenges in global health research, include but are not limited to, large number of proposed interventions, small effect sizes and uncertainty about the baseline risks. In presence of uncertainty about the initial assumptions, planning a conventional RCT is extremely challenging and can result in under-powered trials and high rates of false positives.
Information that becomes available during the course of a trial can be used to reassess the validity of the assumptions that the trial design is based on. Under a conventional RCT, however, modifications to the design are not allowed. Adaptive clinical trials, on the other hand, leverage interim results to modify the trial. By facilitating a variety of flexible designs, HECT, allows the researchers in Global Health to plan efficient trials with a higher success rate.