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?
Patients with chronic or long-term health conditions often need to try different treatments before they find one that works for them. To help patients and doctors decide the order in which to take treatments, researchers use sequential, multiple assignment, randomized trials, or SMARTs. In SMARTs, patients take more than one treatment, but they take them one at a time. Treatment order, or sequence, varies across patients, and researchers assign patients by chance to a treatment sequence.
But current SMARTs don’t account for when patients prefer a certain treatment. Patients with a treatment preference may not enroll in the study if they aren’t guaranteed their preferred treatment. If they do enroll, they may be less likely to stay in the study. To address this concern, researchers can use partially randomized patient preference trials, or PRPPs. In these studies, some patients can receive their preferred treatments. Combining PRPPs with SMARTs could help researchers recruit and retain patients in studies. But questions remain about how best to design PRPP-SMARTs. Also, few methods exist for analyzing data from PRPP-SMARTs.
In this study, the research team is developing methods to design PRPP-SMARTs and analyze data from them.
How can this project help improve research methods?
Results may help researchers when designing and analyzing PRPP-SMARTs.
What is the research team doing?
The research team is developing new methods for analyzing data from PRPP-SMARTs. The methods take into account patients who choose their treatment and patients who researchers assign by chance to treatment. The team is comparing the new methods with existing methods. The team is also developing software to help researchers plan PRPP-SMARTs. The software helps researchers decide how many patients to include in a study, how many patients can choose their treatment, and how often they can do so.
Patient advocates, clinicians, and researchers are providing input on the study.
Research methods at a glance
|Goal||To develop statistical methods that increase the efficiency of PRPP-SMARTs|
Methods development, simulation, guideline development, software development
*All proposed projects, including requested budgets and project periods, are approved subject to a programmatic and budget review by PCORI staff and the negotiation of a formal award contract.