September 14, 2012
Sherine Gabriel, MD, MSc
Chair
Methodology Committee
Patient-Centered Outcomes Research Institute
1828 L St., NW, Suite 900
Washington, DC 20036
Dr. Joe Selby
Executive Director
Patient-Centered Outcomes Research Institute
1828 L St., NW, Suite 900
Washington, DC 20036
Dear Drs. Gabriel and Selby:
The Association of Clinical Research Organizations (ACRO) appreciates the opportunity to comment on the draft report of the Patient-Centered Outcomes Research Institute (PCORI) Methodology Committee.
ACRO represents the world’s leading CROs, which employ more than 75,000 research professionals who operate in more than 100 countries. Each year, our members conduct in excess of 11,000 clinical studies involving nearly two million research participants. Approximately 10 percent of this research is categorized as Phase IV post-approval studies, including a substantial amount of comparative effectiveness research, health technology assessments, outcomes research and the like.
With this broad perspective, we will focus on comments on four areas:
1) Where the Methodology Committee adheres to its statutory directive to develop methodological standards for comparative clinical effectiveness research and provide a translation table, it has produced a workmanlike document to guide those institutions and organizations with which it will contract for research services;
2) The Methodology Committee has far exceeded its statutory mandate in this draft report. Establishing research priorities and a research project agenda is the clear and sole purview of the Board of Governors;
3) The draft report includes an inherent underlying presumption that PCORI will be funding investigator-initiated research. This is in contravention of PCORI’s authorizing legislation which clearly envisions the Institute contracting for research services to carry out its established agenda, not making research “grants”;
4) The Methodology Committee’s assertion of a “trust problem” is outside the scope of the Committee’s duties and is not supported by any evidence.
Methodological Standards and Translation Table
Chapters 6, 7 and 8 of the draft report, which outline the translation table and research methods, comprise the foundation of the Methodology Committee’s work and, by statute, the extent of the Committee’s mandate.
In general, the Committee has provided a comprehensive overview and analysis of research methods relevant to patient centered outcomes research, including, importantly, the need to include subgroups in research projects.
We are also pleased to see the Committee acknowledge that flexibility of methods will be needed in each research project, as, by definition, the goals and objectives of the research will determine the specific methodology employed.
Similarly, the Committee’s desire to promote “crosscutting” and innovative research methods should be commended as comparative clinical effectiveness research with this high level of patient engagement is unprecedented. The use of adaptive and Bayesian trial designs will clearly come into play within this new research paradigm.
ACRO has no further specific comments on research methodology at this point as we see this as an evolving exercise. We look forward to lending the expertise that resides within our member companies to the Committee and to provide representatives to any expert panels that may be convened by the Institute.
The Statutory Authority of the Methodology Committee
ACRO is troubled by the Methodology Committee’s grossly exaggerated view of its statutory mandate in the report.
“We have interpreted this legislation to mean that the PCORI Methodology Committee’s role to advise PCORI on the best methods for promoting its agenda – in prioritizing research, establishing a specific research project agenda, reviewing research proposals, supporting infrastructure for PCOR, improving the conduct of PCOR, and furthering the implementation of PCOR to help patients receive optimal outcomes.”
No. The specific statutory functions of the Methodology Committee are to develop and update methodological standards for research and to develop a translation table. These two functions are the limits of the Methodological Committee’s charter.
The job of “prioritizing research, establishing a specific research project agenda, (and) reviewing research proposals” is the clear role of the Board. The Methodology Committee, which is largely unaccountable to Congress or the public, cannot subsume these duties.
A final Methodology Report must redefine the role of the Methodology Committee to acknowledge the limits on its responsibilities as specified by Congress.
Contracting vs. Grant Making
PCORI’s enabling statute uses the term “contract,” or a derivative of the word, 19 times; the word “grant” does not appear at all.
Yet throughout the draft Methodology Report, the Committee refers to “grant applications”, “grant funding”, “grant submissions” and “grant applicants”.
This is more than a semantic difference. “Contract” presumes that the Institute establishes a well-defined research project agenda and solicits proposals to carry out those projects with specific methodological approaches, budgets, timelines and deliverables.
Conversely, “grant” implies that the Institute will entertain investigator-initiated research proposals that may or may not match the priorities established by the Board. PCORI was not established to duplicate the work of the National Institutes of Health or the Agency for Healthcare Research and Quality. In fact, PCORI was established purposefully to not duplicate the mission or approach of NIH and AHRQ. PCORI is simply not a grant making body; it was clearly established to “contract” for research services.
We suggest that any presumption of investigator-initiated research must be stricken from a final Methodology Report and the Board must adopt a clear contracting process.
“A Trust Problem”
The draft Methodology Report contains a section titled “Problems that PCORI Hopes to Address.” Frankly, we do not believe this section is at all relevant to the work of the Methodology Committee and are unsure why it would appear in the draft report.
That said, to the extent any of these “problems” exist, they would be largely addressed through the execution of a well-designed research project agenda.
Most disturbing is the discussion of “a trust problem” in this report. There is an important fundamental and entirely legitimate difference in the research approach taken by the biopharmaceutical industry when developing a new drug and that taken to study comparative effectiveness. The study objectives and endpoints are entirely different.
Further, for a Committee comprised of researchers to make such assertions of manipulation or bias without any supporting evidence is highly problematic and calls into question the objectivity and integrity of the Committee.
This discussion of “a trust” problem” impinges on PCORI’s credibility as a research organization by exposing its own potential bias. This discussion is beneath the research standards to which PCORI should adhere and should be removed from the final version of the report.
ACRO appreciates the opportunity to provide comments to the draft Methodology Report and we welcome the prospect of working with the Board and Methodology Committee to assist PCORI in carrying out its important agenda.
Sincerely,
John J. Lewis
Vice President of Public Affairs