The Role of the PCORI Methodology Committee

In the near future, the U.S. Comptroller General is expected to announce the appointees to the Patient-Centered Outcomes Research Institute’s (PCORI) methodology committee, which by law will assist the Institute with developing and improving “the scientific methods of comparative clinical research.” The methodology committee’s work will play a key role in the development of quality information that is used by patients, physicians, payers and other decision makers, so it’s important to understand the committee’s makeup and responsibilities.

Composition

According to the law, this standing committee will be composed of 17 members who include the directors of the National Institutes of Health and the Agency for Healthcare Research and Quality or their designees, and who “shall be experts in their scientific field, such as health services research, clinical research, comparative clinical effectiveness research, biostatistics, genomics, and research methodologies.”

Tasks

The purpose of this committee is to ensure that the comparative effectiveness research (CER) developed under PCORI’s watch is of high quality, which is why this group is charged with the development of methodological standards for research. The types of research include not only new or primary research such as randomized clinical trials, molecularly informed trials, and observational studies, but also systematic reviews or synthesis of existing research, and any other methodologies recommended by the methodology committee. The law states that, “such methodological standards shall provide specific criteria for internal validity, generalizability, feasibility, and timeliness of research and for health outcomes measures, risk adjustment, and other relevant aspects of research and assessment with respect to the design of research.” In addition, the law allows for the flexibility to include new information, data and advances in technology in ongoing research projects; calls for public input as methods are developed and updated; and ensures that patient subpopulations are considered in different kinds of research.

Another important task for the committee is the development of a translation table, which would serve as a reference tool to assist the PCORI Board of Governors in determining which research methods are best suited for a specific research question. A translation table helps to lay out the framework for developing research by first looking at the questions that decision makers need answered, and then determining what kinds of research can best address those questions. This places the decision makers at the center of the development of CER, rather than as a simply a recipient of research results. 

Reports and Deadlines

The committee has a short amount of time in which to complete its work—merely 18 months after the establishment so it is permitted to rely on outside assistance. The methodology committee “may consult and contract with the Institute of Medicine of the National Academies and academic, nonprofit, or other private and governmental entities with relevant expertise to carry out” its activities. 

As the methodology committee moves forward with its tasks, it must submit reports to the PCORI Board of Governors. The reports should include “recommendations for the Institute to adopt methodological standards developed and updated by the methodology committee as well as other actions deemed necessary to comply with such methodological standards.

Ongoing Methodology Development

A number of public and private sector organizations, such as the Agency for Healthcare Research and Quality (AHRQ), the Cochrane Collaboration, the International Society for Pharmaceoconomics and Outcomes Research, and NPC, among others, are actively engaged in work to advance the methodology for conducting comparative effectiveness research. This work includes developing new methods, garnering agreement on good research practices, and exploring the types of questions facing researchers, providers, health systems and patients.   

To date, there have been several initiatives resulting from those efforts, some of which were supported by NPC’s funding and participation. Those include the GRACE Principles (Good ReseArch for Comparative Effectiveness), which focus on observational studies; the PACE Initiative (Pragmatic Approaches to Comparative Effectiveness) for Bayesian and pragmatic trials; and guidance documents developed by the Center for Medical Technology Policy. In the future, NPC will continue to engage in thoughtful research and partnerships to help inform the advancement of CER. 

While much work is ongoing, it will require the research community to engage in dialogue with the decision makers who will need credible, reliable, and relevant CER in making treatment decisions. In the coming months, the yet to be named members of the methodology committee will be certain to play a key role in this ongoing discussion.   

(Source: Subtitle D--Patient-Centered Outcomes Research, SEC. 6301. PATIENT-CENTERED OUTCOMES RESEARCH, PL 111-148)