Bethesda, MD—Implementing and effectively using comparative effectiveness research (CER) and personalized medicine (PM) will require time and better communication among the research community conducting these types of studies and with the public that will be impacted by them, according to participants in a panel discussion on the societal implications of CER and PM held during the ECRI Institute’s Comparative Ef fectiveness and Personalized Medicine: An Essential Interface conference.
For David Meltzer, MD, PhD, Associate Professor in the Department of Medicine at the University of Chicago, the flood of additional information provided by CER and PM may not necessarily be helpful in a system where individuals are disconnected from paying costs. For him, aspects of healthcare reform—increased patient costs and copayments, bundled payments, and accountable care organizations—will have the beneficial effect of reconnecting patients with costs, thereby realigning incentives. CER will be essential for delineating the benefits and costs of care, and in cancer, PM will determine the highcost, low-value treatments that should not be used by most patients.
Frances Miller, JD, Professor of Law at Boston University School of Law and Professor of Public Health at Boston University School of Public Health, also emphasized that innovations in CER and PM are inextricably linked to reimbursement. Describing that linkage as the “800-lb gorilla,” she wondered how to get the public to accept that link. She also expressed concern that the potentially ambiguous concepts of CER and PM may go over the heads of the American public, especially given the current climate surrounding health reform.
“Everyone here to a greater or lesser extent accepts the value of comparative effectiveness and the promise of personalized medicine,” she said, referring to those attending the conference. Among the larger American population, however, “the level of accurate information is shockingly low.” Given the threat of healthcare reform repeal and challenges to the individual insurance mandate, Dr Miller said, “I’m worried.”
Also concerned was Gail Wilensky, PhD, Economist and Senior Fellow at Project Hope. Noting the attempts to paint CER as anti-individualized and intended to prevent people from receiving therapies from which they might benefit during the healthcare reform debate, Dr Wilensky pointed out that the current system does not provide adequate information on who is likely to benefit, particularly from complex, expensive therapies. Examining how conditions might be treated in varied groups is precisely the direction that PM and CER should be taking the country, she argued. This will get treatments beyond the “on average” approach.
Reframing the terminology—think “patient-centered outcomes research” rather than CER—is one of several ways suggested by Dr Miller to foster CER and PM among the general public and keep the debate about these approaches rational. In addition, pointing out that similar types of research have long been conducted (ie, whether clinical trials for drugs and devices or research in the Veterans Administration setting) may also be helpful. Finally, she urged attendees to control the agenda and momentum, emphasizing that patient-centered medicine and effectiveness data will mean better care for individuals. She acknowledged, however, that this is a tough message to get across in the face of all the wellfinanced opposition that’s out there.
For Dr Meltzer, CER and PM will help researchers get smarter about which studies will be valuable and what treatments may be obsolete, but only if the research is conducted in the real environments where care takes place. In addition, questions about the role of the public and private sectors in CER have created uncertainty in both those domains.
For many, the specter of randomized clinical trials (RCTs) hangs over CER. Many stakeholders will only be convinced by data from an RCT, Dr Wilensky suggested, but she is hopeful that statistical approaches to data collection and analysis will gain traction as part of CER. J. Sanford (Sandy) Schwartz, MD, Leon Hess Professor of Medicine and Health Management and Economics at the Leonard Davis Institute of Health Policy and Economics, echoed the need for new approaches during the question session, saying “what we’re doing now is fundamentally different.” We need to figure out new methodologies and policies to support CER, he said, and this will take time. Although legislators will expect data tomorrow, he pointed out, “it’s going to take 3 to 5 years before data start coming out, if we’re lucky.”
On the public side, Dr Wilensky suggested that the concepts of value-based reimbursement and value-based insurance will be essential in fostering public acceptance of CER and PM. In addition, plain speaking from researchers would be helpful—as she put it, “for goodness sake, stop talking about QALYs and DALYs [disability-adjusted life years] when you’re out in public.” For Dr Miller, tiered formularies offer an example of how the public might come to accept these new approaches. Pointing out that there has not been too much resistance to tiered formularies because they are taking us in a lower-cost direction, we may see tiered pricing as well.
Better communication among the research community is also essential to furthering CER’s use, according to session moderator Sean Tunis, MD, MSc, Founder and Director of the Center for Medical Technology Policy. Improving the discourse will depend on recognizing common themes and achieving mutual understanding within the community that does this work. “We have to recognize that there are really dramatic differences in perspective among the players in such areas as priority setting and methods, and we can’t continue to paper these over,” he said.
One other potential confounder to greater use of CER and PM may arise from planned cooperation be tween the US Food and Drug Administration and Centers for Medicare & Medicaid Services regarding approval and coverage. For Dr Wilensky, it is important to keep the functions of the 2 agencies separate, because coverage is very different from reimbursement. “CER is a reimbursement tool and not a coverage tool,” she posited. Dr Tunis sees parallel review as a good step, saying that from a product developer’s standpoint, it is not feasible to conduct 2 sets of required studies.
For now, Dr Meltzer wondered if anything will change. The ascendance of CER and PM is a slow continuation of a process that will create ambiguity before it creates clarity and saves money. And given the many difficulties to implementation raised during the session, Dr Tunis summarized that the real challenge will be “to reconcile different public policy objectives while retaining the scientific integrity that is required.”
