PMC’s Suggestions to PCORI’s Methodology Committee

The September 14, 2012 (11:59 PM ET) deadline for submitting public comment on the Draft Methodology Report recently published by the Patient-Centered Outcomes Research Institute’s (PCORI) Methodology Committee quickly approaches.

We encourage you all to submit comment as the revised version will be considered for adoption by the PCORI Board of Governors at its November 2012 public meeting in Boston, Mass.

PCORI was designed to do something new and very different – to define how practical, feasible, and useful patient-centered research is to be conducted and the legislation authorizing it directed the methodology committee to address the role of molecular and genetic variation in comparative effectiveness research. With this bold goal outlined, the Personalized Medicine Coalition (PMC) issued its comments to Dr. Joe Selby and Dr. Sherine Gabriel, the Executive Director and Chair of PCORI on September 13, 2012. You can find a copy of that letter here.

Citing our prior work with the Lewin Group, PMC stressed the importance of PCORI moving beyond conventional CER analysis, noting that “comparative effectiveness research (CER) studies to date have rarely accommodated the collection and reporting of genomic, behavioral, environmental, and other individual patient differences.”

To ensure that the tenets of personalized medicine outlined in the authorizing legislation are upheld, PMC reiterated our call for creation of a PCORI Advisory Panel on Personalized Medicine and Innovation and for establishment of standards for updating studies and analyses based on changes in the science or evidence base. We also are making recommendations to address:

1. Heterogeneity of Treatment Effectiveness Standards: Recommend that either a separate section on standards for biomarkers and genetic variables be added to the report or an additional section on heterogeneity of treatment effectiveness be developed to guide the use of biomarker data in patient-centered outcomes research.
2. Diagnostic Test Study Standards: Develop a new paradigm for collecting and analyzing evidence by recommending variations in new clinical trial design, using patient registries to identify respondents, analyzing archived biospecimens, and implementing retrospective analysis of laboratory data.
3. Stakeholder Engagement: Release a table outlining the categories of specific comments received in all future comment submission periods with an explanation of how PCORI addressed the comments as a way to re-emphasize the importance of stakeholder engagement.

PCORI Announces Comment Period; Feedback Critical to Ensure Quality Research

The Patient-Centered Outcomes Research Institute (PCORI) announced this week the public comment period for its landmark draft Methodology Report, which proposes standards for the conduct of patient-centered outcomes research.  While the draft report came out last month, the comment period closes on Friday, September 14, 2012.

The Personalized Medicine Coalition (PMC) has a long history with PCORI starting from when the legislation creating it was being drafted.  We advocated for recognition of personalized medicine in the conduct of comparative effectiveness research, which was incorporated into the legislation.  PMC’s methodology committee is charged with ensuring that the research funded by PCORI examines, among other things, research question responses based on patient molecular subgroup.

As part of the comment period announcement, PCORI stated that feedback received during the comment period would be reviewed for potential incorporation into a revised version of the report. The revised version will be considered for adoption by the PCORI Board of Governors at its November 2012 public meeting in Boston, Mass.  We have previously urged PCORI to be explicit in how the public comments are considered and incorporated. That, and other institutional development, is essential for the proper functioning of the organization.

The current report does not adequately address personalized medicine as an important factor in improving health outcomes through patient-centered outcomes research methodology. PMC will comment on the methodology report, and we urge our member organizations and others to do the same.  Active input from the community is required so that the research will live up to the high expectations that we have for it.

PMC Nominee Dr. Freda Lewis-Hall Selected for PCORI Board of Governors

In June, PMC nominated a slate of individuals representing the diversity of our coalition including representatives from patient advocacy groups, hospitals and health systems, the pharmaceutical and diagnostics industries, and public health agencies for appointment to the Patient-Centered Outcomes Research Institute (PCORI) Board of Governors.  We were pleased that the Government Accountability Office (GAO) included Pfizer’s Chief Medical Officer Dr. Freda Lewis-Hall, a PMC nominee with expertise in and a commitment to personalized medicine, as a member of the PCORI Board of Governors.

But while we thank GAO for selecting a group of individuals for the PCORI Board of Governors who are committed to improving the health of the American people through research that will, we hope, improve outcomes, we also recognize that our work is not finished. We must continue to advocate for personalized medicine both with the PCORI Board of Governors and eventually with its Methodology Committee, to ensure that comparative effectiveness research considers population subtypes, individual variation, and personalized medicine in general.

 The 21-member PCORI Board of Governors, established by the Patient Protection and Affordable Care Act, is tasked with assisting patients, clinicians, purchasers, and policymakers in making informed, evidence-based, health decisions.  It will identify research priorities, carry out a research agenda, and receive support from a Methodology Committee and expert advisory panels.

We were disappointed that the newly selected members of the PCORI Board of Governors did not include more individuals with specific expertise in personalized medicine, in particular, individuals representing small, innovative diagnostics companies.  Small diagnostics companies, which develop new tests for detecting disease, are central to the field of personalized medicine and will play a necessary and important role in the research PCORI will conduct. We hope that the yet-to-be-named Methodology Committee will include more representation from this important sector.  

The members picked today by the GAO include the 19 members below, as well as the Director of the National Institutes of Health and the Director of the Agency for Healthcare Research and Quality, or their designees:

• Freda Lewis-Hall, Pfizer
• Eugene Washington, MD, UCLA
• Steven Lipstein, MHA, BJC Health Care
• Christine Goertz, Palmer College of Chiropractic
• Sharon Levine, MD, Permanente
• Ellen Sigal, PhD, Friends of Cancer Research
• Harlan Weisman, MD, Johnson and Johnson
• Robert Zwolak, MD, Dartmouth-Hitchcock
• Lawrence Becker, Xerox
• Arnold Epstein, MD, Harvard/Brigham and Women’s
• Andrew Imperato, AAPD
• Robert Jesse, MD, Department of Veterans Affairs
• Grayson Norquist, MD, Dept of Psychiatry and Human Behavior,  University of Mississippi Medical Center
• Debra Barksdale, UNC Chapel Hill School of Nursing
• Kerry Barnett, The Regence Group
• Allen Douma, AARP
• Leah Hole-Currty, JD, Washington State HTA program
• Harlan Krumholz, MD, Yale University
• Richard Kuntz, Medtronic