The ‘white paper’ released by the New England Healthcare Institute (NEHI) is likely to get a serious look from officials charged with developing priorities for use of the fund, potentially raising concerns among those who fear that pharmaceutical and medical device manufacturers will have too much influence on the research agenda. But Brandeis University Professor Stuart Altman, who was not involved in developing the white paper and who has strongly endorsed comparative effectiveness research funding, said that while the institute has “a strong industry presence,” it is “a pretty balanced group.”
The document emphasizes that comparative effectiveness research, dubbed ‘CER’ in the field, should identify the relative clinical effectiveness of medical treatments rather than focus on cost. But the paper also implicitly acknowledges that the cost of treatments is likely to be studied in some way in at least some of the studies, urging that to the extent cost is considered, “the debate should focus on long-term value, not simply short-term cost of innovations.”
NEHI boasts that it “brings together diverse perspectives from the health care community across the country — including patients, payers, providers, universities, hospitals and not-for-profit institutions, and for-profit companies and associations.” In addition to the Pharmaceutical Research and Manufacturers of America (PhRMA) and the Advanced Medical Technology Association, its 70 or so members include the Harvard Medical School, Tufts Health Plan, Blue Cross Blue Shield of Massachusetts, the Friends of Cancer Research, and the National Organization for Rare Diseases, formed to spur research on drugs for patients with rare diseases.
The paper is the focus of a meeting to be sponsored by NEHI in Washington April 22 in which federal officials will participate as panelists. They include Agency for Healthcare Research and Quality Administrator Carolyn Clancy, Thomas Valuck, chief medical officer at the Centers for Medicare and Medicaid Services Center for Medicare Management, and Robert Ratner of the Institute of Medicine. The institute must prepare a report by June 30 advising federal officials and lawmakers on what the priorities should be for federally funding of comparative effectiveness research. Ratner heads the team writing the report. Various NEHI members also will make presentations at the event.
Lawmakers eager to bring down spending growth in the United States look to CER studies as a way to guide insurers on what treatments and procedures to cover. Medicare coverage decisions will not be made on the basis of the research findings, however. But some CER enthusiasts note that the findings will be available to private insurers in determining their coverage decisions.
The NEHI paper, prepared under a grant from PhRMA and through support from “diverse member organizations,” according to NEHI, cautions that CER research should not interfere with the introduction of new products. The CER process should allow “reasonable access to new products and processes that realize their true value only after a period of post-market utilization over time,” NEHI says. It warns that “‘on/off’ coverage mandates based on CER results could eliminate access to valuable interventions that may ultimately benefit patients.”
The white paper emphasizes that treatments often yield their true value over time through a process of trying various uses. “Frequently, medical technologies approved for use in one indication find new or different uses in other indications as a result of repeated, off-label utilization and the resulting experience gained by clinicians,” it notes. “New technologies frequently find their best, highest value use only in combination with other products or procedures, often as a result of utilization with varying combinations of treatments carried out over time.”
Studies that focus on clinical effectiveness are most likely to have an impact and allow access to new technologies, the paper argues. “A clinical effectiveness goal is more likely to provide a period of utilization by patients and physicians that will prove an innovation’s highest and best value.”
“Given the controversy over cost effectiveness research, findings from clinical effectiveness studies are more likely to influence medical decision making quickly,” it adds.
The paper also notes that “there is a widespread presumption that CER studies should entail a direct, head-to-head comparison of discrete items, such as drugs and devices. However the cope of CER analysis can be broader,” the paper says, adding that “many experts suggest that CER studies should be broader in order to compare the different choices that physicians and patients face in the daily practice of medicine.”
Thus, “the CER research agenda should encompass studies of the broader issues of health care practices, organization and delivery in addition to studies of medical technologies.” Narrow analyses “focused on comparing individual drugs or devices has the potential to more negatively impact innovation, particularly in medical technologies.”
Funds also should be spent to develop ways of comparing treatments other than randomized clinical trials, the paper says, arguing that they do not capture the way drugs, devices and other treatments are used in less controlled conditions in the real world. In addition, “different research methods are needed to conduct research on innovations in the practice, organization, and delivery of health care.”
Does the paper tilt too much toward the financial interests of drug and device companies or research institutions that receive a lot of funding from those companies? NEHI Executive Director Valerie Fleishman said in an interview that “we were very careful to bring everybody to the table” in developing the white paper. “Our feeling is that yes, cost-effectiveness could be included” if it isn’t used to say yes or no to coverage and it is used as part of a larger analysis that looks at the long term value of a treatment including its impact on quality of life. “This needs to be done in a transparent way where there is a dialogue about this,” she said.
Fleishman said, “we’re not saying never look at drugs or devices” but the research agenda also needs to look at systems of care, treatment protocols, and care delivery processes in order to get costs under control. “At the end of the day….the biggest bang for the buck is…in the whole area of practice variation,” she said.
Altman of Brandeis credited the NEHI effort for trying “to push the ball forward and in a positive way,” but noted that the debate over CER is ducking the tough issues of using findings for coverage and pricing decisions. Those decisions will “probably be put off until four or five years from now,” he said.