Establishing PCORI’s Advisory Panel on Clinical Trials

A well-designed clinical trial is one of our most reliable tools for determining the comparative effectiveness of various options for preventing, diagnosing, and treating disease. But it’s not always straightforward to achieve the best trial design and identify topics where clinical trials are preferable to less costly studies.

These issues will be among those considered by our new Advisory Panel on Clinical Trials, formally empanelled today by our Board of Governors. Due to their importance and complexity, clinical trials are one of two topics for which PCORI’s establishing legislation specifically requires an advisory group. (The other topic is rare diseases.) Our new panel has 10 members with diverse experiences in the healthcare and research communities to provide counsel on PCORI’s strategies for supporting clinical trials.

A Partner to the PCORI’s Methodology Committee

A large part of the advisory panel’s focus will be on supporting our methodological work by providing expertise in the selection, design, and implementation of trials. The use of clinical trials in patient-centered outcomes research (PCOR) can offer unique challenges. PCOR trials address questions patients commonly face and take place in clinical practice, rather than research, settings. Also, they usually compare two or more interventions, rather than an intervention and a placebo. Other new approaches to the conduct of clinical trials, such as allowing the trial to evolve in response to information accruing within it, can be useful for PCOR.

The advisory panel will complement PCORI’s Methodology Committee. The panel has a smaller scope that will allow it to delve more deeply into clinical trial practice and consider the barriers to designing successful trials. For instance, in an activity outside of the scope of the Methodology Committee, the panel can examine trials PCORI is funding in its growing research portfolio and advise us on strategies for implementing the standards set forth in the committee’s Methodology Report.

Going forward, the panel can provide additional oversight, after PCORI’s merit review process, on proposed clinical trials to confirm that PCORI is investing in study designs that are likely to provide trustworthy information useful to patients and those who care for them. The panel will also provide insight into how PCORI’s portfolio of clinical trials aligns—in topics and methods—with those of other research institutes.

This information, from the panelists’ broad views, will be helpful to PCORI’s staff and Board of Governors in determining what types of clinical trials PCORI can support for improving patient care.

Different Perspectives

The advisory panel will also bring new perspectives to our methodological discussions. While the Methodology Committee is made up of research specialists appointed by the Comptroller General of the United States, the advisory panel is a more diverse group selected through an open application system. According to the panel’s charter, at least two panelists must be patients, caregivers, or representatives of patient advocacy organizations.

We received over 230 applications to serve on the panel, which were reviewed according to specific criteria by PCORI staff. The reviewers considered each applicant’s familiarity with relevant issues, ability to communicate with constituencies in the healthcare community, and overall potential to contribute to PCORI’s mission.

The strong response we received led to a slate of panelists who possess a variety of strengths, backgrounds, and areas of expertise. In addition to representatives of patients and caregivers, the advisory group includes individuals with methodological expertise or experience in conducting trials and an expert in ethical issues of trials. The panelists come from medical and research centers, a university, a pharmaceutical company, an association, a consulting group, a nonprofit education network, and the Food and Drug Administration.

Getting to Work

The panel will meet for the first time on May 1 and begin the work of addressing the various challenges to conducting high-quality clinical trials that provide information patients and front-line clinicians need to make better-informed decisions.

With the support of the Methodology Committee and with your continued feedback, we expect the Advisory Panel on Clinical Trials to provide insights and recommendations that will improve the quality of clinical trials that PCORI funds, leading to more relevant and useful findings.

Hickam is Program Director for Clinical Effectiveness Research