[medscape] As healthcare reform legislation grinds its way through Congress, 2 articles published online January 6 in the New England Journal of Medicine (NEJM) advocate for one of its touchiest provisions — comparative-effectiveness research (CER).

In theory, CER sounds like a calm, academic subject: evaluate different treatment options for a given illness — drug A vs drug B, or drug A vs surgery — and determine which does a better job of reducing morbidity and mortality. You also can go a step further and compare these treatment options in terms of risks or cost-effectiveness: Does drug B outperform drug A by a 2% margin but cost 3 times as much? Experts say such research is in short supply, leading to poorer clinical outcomes and runaway costs.

However, talk of government-sponsored CER pushes hot buttons in medicine and American society alike, being called “rationing” and “government takeover of medicine.” For proof, consider what happened when the US Preventive Services Task Force announced last November that, based on the scientific evidence it weighed, it no longer recommends mammograms for women aged 40 through 49 years. The task force also recommended that women aged 50 years and older no longer receive annual mammograms but, instead, get them every other year. Public outcry and pushback from several medical societies and expert groups like the American Cancer Society swayed Senate Democrats to rewrite their pending healthcare reform legislation to guarantee mammogram coverage.

If Congress enacts healthcare reform, more such medical recommendations could roil Americans. That’s because reform bills passed by the House and Senate (which have yet to be reconciled) call for the creation of a CER entity that would question the value of many trusted procedures and treatments. These provisions come on top of economic stimulus legislation passed in early 2009 that pumps $1.1 billion into CER and establishes a new federal bureaucracy to manage it. The government would not conduct CER itself by and large but would instead fund the work of academic investigators.

NEJM Authors Bolster Support for CER Individual physicians and medical societies approach CER with varying degrees of enthusiasm, cautious support, and downright fear and loathing. The 2 recent NEJM articles seek to bolster support for this controversial discipline. Two professors at Weill Cornell Medical College, New York City, write in an article titled “Health Care Reform and the Need for Comparative-Effectiveness Research” that CER is “physicians’ first line of defense against blind cost containment.” Furthermore, it could spur drug and medical device manufacturers “to develop products that really matter.” “I think CER is the physician’s best friend,” coauthor Alvin Mushlin, MD, professor and chair of the Department of Public Health at Weill Cornell, told Medscape Medical News. Similarly, the other NEJM article, titled “Comparative Effectiveness and Health Care Spending — Implications for Reform,” warns that without a shift to best-bang-for-the-buck services identified by CER, cost-cutting alone could produce worse health outcomes. “If we can induce hospitals and health plans to improve efficiency and not just cut costs, then health costs in the United States will come down and outcomes will improve,” write coauthors Milton Weinstein, PhD, a professor of health policy and management at the Harvard School of Public Health, Boston, Massachusetts, and Jonathan Skinner, PhD, a professor of economics at Dartmouth Medical School, Hanover, New Hampshire. Proposed Government-Sponsored CER Would Be Transparent The American Medical Association (AMA) and several other medical societies believe that the federal government can play a valuable role in CER. After all, the cost of this research deters drug and device manufacturers from studying how their products fare in head-to-head competition with others; plus, they may be afraid to tell the world that their product is second-best, according to AMA President J. James Rohack, MD. The government would not operate under these constraints. Furthermore, government-sponsored CER would be a public, transparent enterprise. In contrast, said Dr. Rohack, private payers that conduct CER to determine what they’ll cover don’t always reveal how they’ve reached their decisions — the proverbial, and hated, black box. “You don’t know if they’re just trying to maximize profits or if science backs up their coverage determination,” Dr. Rohack told Medscape Medical News. Accordingly, the AMA and its medical allies have mostly supported provisions in healthcare reform legislation that make the federal government a player in CER. The House bill establishes a Center for Comparative Effectiveness Research within the Agency for Healthcare Research and Quality in the US Department of Health and Human Services. Findings from this center could not be construed as mandates regarding treatment, coverage, or payment, but some experts interpret the legislation as giving the center the ability to make recommendations. In contrast, the Senate bill would create a nonprofit, independent Patient-Centered Outcomes Research Institute that is confined to only publishing its findings and is explicitly prohibited from issuing even recommendations. The AMA prefers the Senate approach because it’s more emphatic about denying a CER body any policy-making clout. The association also prefers an independent body. In contrast, the CER center in the House bill would be a fiefdom within Department of Health and Human Services, which also operates the Medicare and Medicaid programs. There’s fear in some quarters that a CER group embedded in the federal bureaucracy would be vulnerable to political bullying, with scientific integrity sacrificed on the altar of budget-cutting. However, an independent body also comes with risks, said Neil Kirschner, PhD, a senior associate in regulatory and insurer affairs for the American College of Physicians (ACP), a strong advocate of government-sponsored CER. “The public-private group that the Senate wants could be unduly influenced by the private sector,” Dr. Kirschner told Medscape Medical News. “The ACP hasn’t taken a position on which approach is right.” Dr. Kirschner said the ACP would like government-sponsored CER to freely factor in cost-effectiveness data. “The Senate bill makes this more difficult,” he said. “The House bill is more silent on the use of cost.” Cost-effectiveness, he added, doesn’t necessarily mean adopting the least-expensive treatment. “Sometimes the most expensive treatment may be a better value by helping a person live much longer,” he said. Give Physicians Scientific Findings, Not Recommendations Both the ACP and the AMA agree that a government-sponsored CER body should forgo recommendations, much less mandates, on how physicians should practice medicine. For one thing, private insurers and government programs like Medicare are tempted to turn such recommendations into binding policies that may arbitrarily deny patients coverage for needed care, explained the AMA’s Dr. Rohack. “Look at vaccines,” Dr. Rohack told Medscape Medical News. “The federal Advisory Committee on Immunization Practices recommends what vaccines should be administered, and insurers key off that to determine what they’ll pay for.” The fracas over mammograms last year occurred at the intersection of recommendation and insurance coverage, noted health-policy analyst Dennis Smith from the Heritage Foundation, a conservative think tank. “The essential benefit package in the [original] Senate reform bill was supposed to include preventive services recommended by the US Preventive Services Task Force. So if you didn’t meet the [task force] criteria, you wouldn’t get your mammogram paid for.” Dr. Rohack also pointed to the problem of inflexible application of CER. A study that identifies the best way to treat a particular medical condition may have excluded patients older than 65 years, for example. A payer may then decide to cover that treatment, but not for anyone older than 65 years, he said. It’s sufficient, said Dr. Rohack, to give CER findings to physicians and patients and let them make the final decision on medical care. “Physicians by training try to use evidence-based science to do what’s best for patients,” he said. “If there is a gray zone, they’ll rely on history, experience, and local practice styles. And there are gray zones.” Dr. Rushlin at Weill Cornell Medical College agrees with Dr. Rohack that merely publishing CER findings without adding recommendations will benefit healthcare. “We need to get started incorporating information from CER into the public debate on healthcare. It can be a very positive step in the right direction,” he said. “When you put the evidence on the table, it illuminates the discussion. It doesn’t eliminate the discussion. That’s healthy.” Rationing With a Light Touch? Despite all the restrictions in the House and Senate healthcare reform bills, government-sponsored CER alarms some physicians, such as the American Association of Physicians and Surgeons. An opponent of reform legislation, the society states on its Web site that CER “will provide the rationale for rationing” and suggests that this discipline is a reincarnation of managed care. Sen. Thomas Coburn, MD (R-OK), wrote in the Wall Street Journal last month that CER is one reason why “seniors will die sooner” if Congress passes the Senate’s version of healthcare reform. In other countries, such as the United Kingdom, Dr. Coburn wrote, CER panels amount to rationing panels. Dr. Weinstein and Dr. Skinner acknowledge in their article that Americans have less of a stomach for explicit, top-down rationing of healthcare resources than other countries in which CER has a longer history. The authors point out, however, that Americans appear more amenable to rationing when it’s based on free-market price decisions, such as whether to pay a higher copayment for more expensive medication. With that tendency in mind, Dr. Weinstein and Dr. Skinner suggest several ways to coax patients and providers to veer toward cost-effective care without getting heavy-handed about it. Physicians and hospitals could receive higher compensation when they perform more cost-effective services, and lower compensation when they choose more wasteful services. Likewise, consumers purchasing insurance could pay a higher premium for a plan that would give them immediate access to higher-cost medications, for example, without first having to try less expensive ones — read generics — to see whether they work. N Engl J Med. Published online January 6, 2010.