Methods to Generalize Results from Clinical Trials

One of PCORI’s goals is to improve the methods that researchers use for patient-centered outcomes research. PCORI funds methods projects like this one to better understand and advance the use of research methods that improve the strength and quality of comparative effectiveness research.

What is the project about?

Clinical trials test how well new treatments or programs work for a specific group of people, such as people with diabetes. But people who take part in a clinical trial may not represent everyone from that group. For example, people in the trial may be healthier than people who aren’t in the trial. To address this issue, researchers have developed statistical methods that apply results from one or more clinical trials to the larger group of interest. But these methods don’t work well when data are missing. Data may be missing:

• Within a trial, for example, when patients stop taking part in the trial
• When researchers have not collected data about patient traits from patients in the trial

In this study, the research team is developing methods to apply results from clinical trials with missing data to the larger group of interest.

How can this project help improve research methods?

Results may help researchers apply results from clinical trials to the larger group of interest.

What is the research team doing?

First, the research team is creating new methods for applying the results of clinical trials with missing data to the larger group of interest. Next, the team is testing the new methods on data that mimic real patient data. Finally, the team is applying the methods to six trials of programs for youth at risk for HIV.

Research methods at a glance