BALTIMORE—The health reform legislation that established a new entity to facilitate and fund comparative effectiveness research (CER)—the Patient-Centered Outcomes Research Institute (PCORI)—has opened the door to transforming clinical trials by drawing patients and the public into research in unprecedented ways. But PCORI also faces major challenges in accomplishing its mission (see box).
The opportunities and the challenges were discussed at a symposium here on transforming oncology through CER. The meeting—the first annual National Leadership Summit on CER Priorities, Methods and Policy—was convened by the Center for Medical Technology Policy (CMTP) in partnership with the Angiogenesis Foundation.
Acting US Comptroller General Gene L. Dodaro appointed PCORI's 19-member governing board in October, the majority of whom are in academia or nonprofit organizations but also included are the directors of the National Institutes of Health and the Agency for Healthcare Research and Quality.
“I would like us to present to PCORI some guidelines on where to get started on CER and oncology,” said CMTP Founder and Director Sean Tunis, MD, MSc, a former director of the Office of Clinical Standards and Quality and Chief Medical Officer of the Centers for Medicare and Medicaid Services (CMS).
Dr. Tunis told OT that he plans to provide a CMTP white paper to PCORI by the end of the year with some basic insights on setting research priorities. He said he chose cancer as the symposium topic because it touches so many lives. “We want to try to bring together a strategic framework,” said Dr. Tunis. “This is a place to begin the conversation.”
IOM Identified 7 Priority Areas
In a report on CER, the Institute of Medicine (IOM) has already identified seven high-priority areas for CER studies in the field of oncology, including: localized prostate cancer, ductal carcinoma in situ (DCIS), imaging strategies for cancer patients, genetic and biomarker testing, diagnostic use of upper endoscopy, interventions to reduce health disparities and comprehensive care coordination programs (OT, 8/10/09 issue).
By definition, CER aims to transform health care to a system firmly rooted in evidence-based medicine, one in which health care outcomes are translated into practice. Health reform legislation—the Patient Protection and Affordable Care Act, enacted in March 2010—calls for CER to be done in “real-world” settings.
Thus, speakers here said, once the public understands what CER is all about, ideally PCORI can involve patients and the public in clinical research in new and more active ways.
Involve Patients & Advocates Upfront
For example, involving patients and advocates in clinical trial design upfront “offers a really exciting opportunity” to increase clinical trial participation, said Scott Ramsey, MD, PhD, Director of Cancer Outcomes Research at Fred Hutchinson Cancer Research Center.
Currently, he said, “We really don't crack that three to five percent participation rate; trials are failing for lack of recruitment.”
Susan Brown, MS, RN, Director of Health Education at Susan G. Komen for the Cure, agreed that when patients are invited to participate in the clinical research enterprise (such as helping to develop educational materials and sitting on institutional review boards), it can pay dividends: “Invite them early and often,” she said. “CER won't be successful without patients. Patients know they aren't doctors or researchers, and they won't try to be.”
Another speaker, Amy Bonoff, MBA, Co-Chair of the National Breast Cancer Coalition (NBCC) Quality Care Advocate Advisory Committee and a breast cancer survivor, said that in a 2009 survey done by the NBCC, survey respondents ranked CER as the most important part of health care reform. She said that educated consumer advocates participating in CER can help to move cancer research beyond incremental improvements in cancer care to major advances.
“Patients are an extremely rich resource that is hardly ever used,” added cancer survivor Deborah Collyar, founder of the Patient Advocates in Research (PAIR) international network. “We [advocates] have a connection into patient communities.” She said PCORI has an opportunity to fund innovative research that does not follow the “myopic models” of some previous US research.
Susan Love: ‘Democratize Research'
“I think we need to democratize research....I hope CER will help us do more relevant, exciting trials,” said Susan M. Love, MD, MBA, President and Medical Director of the Dr. Susan Love Research Foundation. “My answer is to go directly to the public...engage them in the process. The public is willing and ready to participate.”
In October 2008 Dr. Love launched the Army of Women campaign, aimed at recruiting one million women who are willing to consider participating in research to find the cause and help identify ways of preventing breast cancer.
“We have the ability to involve patients who have not traditionally participated in clinical trials,” said Ethan M. Basch, MD, MSc, an outcomes researcher at Memorial Sloan-Kettering Cancer Center and Chair of ASCO's Clinical Practice Guidelines Committee and a member of ASCO's CER task force. “Involving the public has many beneficial downstream effects.”
SEAN TUNIS, MD, MSC,...Image Tools
Also at the meeting was Otis Brawley, MD, Chief Medical Officer of the American Cancer Society and Professor of Hematology and Oncology at Emory University School of Medicine. Asked by OT if he believes PCORI's role in CER will help to increase clinical trial participation, he, said, “I'm not sure that is going to happen; I hope it will happen.”
Dr. Brawley, who gave a keynote luncheon address on prostate cancer, said a segment of the public still has to overcome its lingering fear of clinical research, rooted in highly publicized abuses of the past such as the Tuskegee syphilis trial.
Symposium participants agreed that effective educational strategies for the public and physicians about the potential benefits of CER will be essential to its success.
Challenges for the Patient-Centered Outcomes Research Institute
In addition to the need for effective education about CER, the following challenges emerged from the symposium discussion:
* What mix of methodologies in studies should be pursued (systematic reviews, observational research, and randomized trials)? Sharon Murphy, MD, Institute of Medicine Scholar-in-Residence, said she is concerned about CER methodology, such as subgroup analysis. “We will need bio-banking; that is not cheap—not in your dreams,” she cautioned.
* What is the appropriate role of regulatory agencies, especially the US Food and Drug Administration? Jeff Allen, PhD, Executive Director of Friends of Cancer Research, raised this issue in his remarks. (Ellen Sigal, PhD, Friends of Cancer Research Founder and Chair, has been appointed to the PCORI board).Janet Woodcock, MD, Director of FDA's Center for Drug Evaluation and Research, has proposed a federally funded national clinical research infrastructure that would increase CER by encouraging community-based physicians and their patients to participate in clinical trials. Dr. Woodcock points out that the current clinical trials system is already at full capacity and cannot absorb large amounts of CER without resource diversion.
* How will PCORI be integrated with personalized medicine? Several speakers raised this issue, including Dr. Murphy and Ms. Collyar. Speakers said they are worried that subgroup populations may be overlooked in CER as part of a cost-cutting strategy. In fact, said Ms. Collyar, some patients are concerned that the “c” in CER stands for “cost” effectiveness rather than “comparative” effectiveness.
* How will PCORI set priorities for CER? The IOM report on CER priorities provided a starting point, but speakers said it was just a start.
* How can PCORI make the most of its research allocation? Its funding is estimated at $650 million per year in the near future—a relatively modest amount when compared with the funding of institutes within the NIH.
© 2010 Lippincott Williams & Wilkins, Inc.