YSPH Biostatistics Seminar- “Endpoint selection in clinical trials: when stakeholders disagree about what matters most”

NOTE: BIS 526 students are required to attend in person (47 College St., Room 106A). All others are requested to attend via Zoom.

Speaker: Christopher John Lindsell, Ph.D.

Title- “Endpoint selection in clinical trials: when stakeholders disagree about what matters most”

Abstract

One of the major decisions in a clinical trial is which endpoint to use. The endpoint reflects the single most important question the trial is intended to answer. The choice of endpoint drives many aspects of the trial, from how and when measurements are made, to how the trial data will be analyzed, and the results interpreted. In turn, the practical setting of the trial can constrain when and how measurements are made. The most important question for the same trial may also differ depending on the viewpoint of the stakeholder consuming the trial results and the intended policy or regulatory impacts.

During the Covid-19 pandemic, we designed two platform trials to answer the following overall questions: are there any existing therapies that have a known safety profile that could be repurposed to help mild to moderately ill outpatients with Covid-19 get better faster, and stay out of the hospital.

In this context, there are many different viewpoints as to the most important question. The balance between generating evidence fast enough about what might work and generating sufficient evidence to support a claim about what does work, and between a decentralized pragmatic trial deployed nationwide and a controlled clinical trial intended for supporting registrational claims. In this talk we will review the lessons learned about choosing endpoints in outpatient treatment trials for Covid-19, including model-assisted endpoints.