By Deborah Borfitz 

May 29, 2012 | The role of cost and patients in comparative effectiveness research (CER) were among the themes of a Post-Approval Summit held earlier this month at Harvard Medical School*. So, too, was the agenda and funding priorities of the latest actor in the CER arena: the independent, multi-stakeholder Patient-Centered Outcomes Research Institute (PCORI).

PCORI, authorized by the Affordable Care Act, is getting $1.1 billion in government funds to advance comparative clinical effectiveness research. Standards guiding how this research gets conducted were in the final days of conception at the time of the Post-Approval Summit, according to keynote speaker Joe Selby, MD, MPH, executive director of PCORI.

The ambitious goal of PCORI is to give patients and clinicians the “exact information” they need at the moment of decision making, says Selby. “The focus is on clinical outcomes versus cost effectiveness or cost comparisons.” Its stated mission is “produce and promote evidence-based information that comes from research guided by patients, caregivers, and the broader health care community.” In short, research questions and methodologies get aligned with patient needs.

Patients are expected to be involved on the research teams of all PCORI-funded projects and will in fact be engaged as reviewers of proposed studies, says Selby. Doing so will ensure that practical questions get generated and a range of outcomes are considered, in addition to differences in effectiveness across various patient subgroups. The differences may fall along the lines of race and ethnicity, gender, age, genetic and molecular sub-types, or even quality of life preferences.

PCORI recently awarded $31 million worth of pilot grants to address methods for engaging patients in various aspects of research and the dissemination process. Selby says priorities for forthcoming primary PCORI funding are: comparing preventive, diagnostic, and treatment options; improving the health care system (notably, support of patient self-management); communicating and disseminating research (with an emphasis on shared decision making); and addressing disparities.

A fifth priority, to accelerate PCORI and methodological research, will require building a “sustainable infrastructure” for ongoing comparison of options and dissemination of evidence as well as facilitating the study of rare diseases, says Selby. Subsequent rounds of funding will be directed at research addressing how to routinely collect and electronically record patient-centered outcomes, engage patients in research, and involve clinicians in generating research questions and disseminating findings.

Selby says PCORI has already successfully solicited 300 patients that it will train to serve as reviewers of this year’s grant applications, due in by the end of July. Patient input will likely be limited to comments on engagement and patient-centeredness of research and questions, he adds.

The research agenda is focused on projects most likely to lead to health-promoting changes in practice, consider patient subpopulations, and include a variety of outcomes, says Selby. No specific conditions or treatments are being targeted for evaluation and grantees may include for-profit organizations, private entities, and partnerships. The research budget grows from $150 million this year to $300 million in 2013 and $550 million annually thereafter through 2019. The Department of Health and Human Services and the Agency for Healthcare Research & Quality (AHRQ) get an automatic 20% of the funding pie.

“I hope the focus is on patients not currently responding well,” says Selby. One conundrum is how to incorporate patient preferences for various outcomes and the uncertainty of imperfect markers into research that will guide decision making by individuals. “The truth is that most treatments [as well as health education and disease management approaches] don’t work for everybody.”

During an “evidence face-off” at the Post-Approval Summit, Selby clarifies that PCORI sees health care costs as increasingly important to consumers even as it takes the “prudent course” of concentrating on clinical outcomes. “Costs are more variable and negotiable, can change in a matter of minutes, and are local.” Out-of-pocket costs are also a “critical, explanatory variable that drives treatment choice and adherence, so we need to account for that in order to interpret comparative effectiveness analysis.”

The bigger issue, especially outside the U.S., is affordability, says Samuel Wagner, PhD, RPh, executive director of oncology, global health economics and outcomes research at Bristol-Myers Squibb. And that includes interventions of demonstrated value.

“In health care, there’s a lot of wariness when cost gets introduced into the message…even when we talk about it,” adds John Santa, MD, MPH, director of the Consumer Reports Health Ratings Center. “Most people think ‘more is better’ and it’s difficult to change that.”

Several panel members agree that a “trusted source” will be needed to help consumers understand how research is done and minimize treatment biases (i.e. surgery over drugs) in CER. Ultimately, specialty societies will be in the best position to help patients and clinicians decide which study results are “valid for whom under what circumstances,” says Robert Dubois, MD, PhD, chief science officer for the National Pharmaceutical Council.

At a workshop in July, PCORI will address the question of how best to contribute to the infrastructure for safety and patient-centered outcomes research, says Selby. Plenty of others players have been at it for some time now, including: AHRQ, the Food and Drug Administration, National Institutes of Health, and Office of the National Coordinator for Health Information Technology, plus the Veterans Administration, Institutes of Medicine, and numerous specialty societies.

* Post-Approval Summit, May 1-2, 2012, Harvard Medical School