Chapter 2.5: Innovations in Clinical Trial Design: Lessons from the COVID-19 Pandemic

COVID-19 has shown that clinical trials need to be efficient, adaptable, and provide high-fidelity data that are actionable for public health officials and clinicians to deliver safe and effective interventions. Traditional fixed design trials, such as prospective randomized, double-blind clinical trials, need large sample sizes, are subjected to multiple study protocols for every interventional arm, take time to complete, and are expensive. In addition, they are not flexible enough to add or delete study arms during the course of the trial. Standard clinical trials are performed through sponsor-specific networks of clinical sites, with each trial and sponsor essentially competing with others for trial subjects to validate their therapeutic platform. Adaptive trials are more flexible and dynamically adjustable based on the accumulated results. This approach focuses on the best treatment, individual drug, or a combination among various options for a disease instead of focusing on the best single drug for a disease. Adaptive trials are often more efficient, cost-effective, and need fewer trial participants as futile treatment arms may be stopped early. Fewer patients may need to be randomized to a less promising treatment, and smaller study cohorts may be required to address the trial’s questions. Global health consortiums, governments, and multilateral institutions have the opportunity to collaborate and conduct adaptive trials, via established global clinical trial networks, to study and identify disease-outcome-driven interventions.