Today, there are multiple treatments for almost every type and stage of breast cancer, leaving patients—and the insurers who cover them—at times with nearly a dozen options to consider. The same goes for certain cardiovascular diseases and a growing list of other ailments. Little wonder, then, that research comparing the effectiveness of various therapies appears to be an idea whose time has come. On August 1, the U.S. House of Representatives passed legislation that would establish a comparative research center to conduct, support, and synthesize research comparing “the full spectrum” of treatments against each other, head to head. As of this writing, the Child Health and Medicare Protection Act with Section 904 containing the research provision still awaits action by the Senate.

 

Swiftly and fairly quietly, a wide range of Congressional advocates, the Medicare Payment Advisory Commission, the Institute of Medicine roundtable, and others have come to surprising agreement on the need for such a center and on many of its main characteristics. The concept, in one form or another, has been endorsed by the presidential campaigns of Senator Hillary Clinton (D-NY), Senator Barack Obama (D-Illinois), and former Senator John Edwards (D-NC).  

 

Nonetheless, the overriding concern of many stakeholders remains: Can such an entity conduct research with the fairness sufficient to ensure credibility, and without becoming merely a cover for cost controls? According to the legislation passed by the House, the comparative effectiveness center would be housed within the Agency for Healthcare Research and Quality (AHRQ), a part of the Department of Health and Human Services already authorized by the 2003 Medicare Modernization Act to conduct comparative effectiveness studies. It would be supported by federal appropriations in the first three years, with gradually increased funding up to $110 million a year in 2010, still less than the cost of a single F-22 jet. To keep it safe from the whims of the annual appropriations cycle in Congress, the center eventually would be funded by assessments on Medicare and private health insurance. That’s in light of the fact that there is a history of professional and industry organizations pressuring Congress to reduce or kill funding for AHRQ and other technology assessment agencies when their analyses touched on financial interests.

 

Proponents emphasize that the center would not do cost-effectiveness research and would not make decisions on payment coverage. But concerns persist. As Sean Tunis of the Center for Medical Technology Policy recently told a conference of biotech executives, “You don’t want the evidence for efficacy to be determined by the payers.” 

Today, there are multiple treatments for almost every type and stage of breast cancer, leaving patients—and the insurers who cover them—at times with nearly a dozen options to consider. The same goes for certain cardiovascular diseases and a growing list of other ailments. Little wonder, then, that research comparing the effectiveness of various therapies appears to be an idea whose time has come. On August 1, the U.S. House of Representatives passed legislation that would establish a comparative research center to conduct, support, and synthesize research comparing “the full spectrum” of treatments against each other, head to head. As of this writing, the Child Health and Medicare Protection Act with Section 904 containing the research provision still awaits action by the Senate.

 

Swiftly and fairly quietly, a wide range of Congressional advocates, the Medicare Payment Advisory Commission, the Institute of Medicine roundtable, and others have come to surprising agreement on the need for such a center and on many of its main characteristics. The concept, in one form or another, has been endorsed by the presidential campaigns of Senator Hillary Clinton (D-NY), Senator Barack Obama (D-Illinois), and former Senator John Edwards (D-NC).  

 

Nonetheless, the overriding concern of many stakeholders remains: Can such an entity conduct research with the fairness sufficient to ensure credibility, and without becoming merely a cover for cost controls? According to the legislation passed by the House, the comparative effectiveness center would be housed within the Agency for Healthcare Research and Quality (AHRQ), a part of the Department of Health and Human Services already authorized by the 2003 Medicare Modernization Act to conduct comparative effectiveness studies. It would be supported by federal appropriations in the first three years, with gradually increased funding up to $110 million a year in 2010, still less than the cost of a single F-22 jet. To keep it safe from the whims of the annual appropriations cycle in Congress, the center eventually would be funded by assessments on Medicare and private health insurance. That’s in light of the fact that there is a history of professional and industry organizations pressuring Congress to reduce or kill funding for AHRQ and other technology assessment agencies when their analyses touched on financial interests.

 

Proponents emphasize that the center would not do cost-effectiveness research and would not make decisions on payment coverage. But concerns persist. As Sean Tunis of the Center for Medical Technology Policy recently told a conference of biotech executives, “You don’t want the evidence for efficacy to be determined by the payers.” 

Although few have resorted to the “R word” (rationing), Scott Gottlieb, M.D., a former FDA deputy commissioner, recently warned in the Wall Street Journal that the government’s interest in controlling healthcare costs might trump other considerations. The aim for this center, he wrote, “is to arm government actuaries with data that proponents hope will provide ‘scientific’ proof that expensive new drugs are no better than their older alternatives.” And that “as the government begins tying its own payment decisions to the results of its own studies, there’s a great temptation to selectively interpret data and arbitrarily release results,” he said.

 

Michael Mussallem, CEO of Irvine, California-based medical device maker Edwards LifeSciences, agrees. “If these kinds of studies can turn into opportunities to deny patients access to care, then we think that is a big negative. We believe that they should be used to help make clinical decisions, not payment decisions.” 

 

Mussallem also says that the medical device industry supports the concept of comparative effectiveness research but worries that the metrics will skew in favor of dramatic leaps, while their business model is based on the concept of incremental improvement. Device makers have similar concerns that the timeline necessary to prove the value of a new device may not match an arbitrary timeline imposed by comparative effectiveness researchers.

 

Randy Burkholder, associate vice president of the Pharmaceutical Research and Manufacturers of America (PhRMA), says that his organization favors government-supported comparative effectiveness research “when it is done right.” Pointing to examples of policies elsewhere in the world, he makes it clear that “done right” does not mean replacing physicians’ decision-making with the distant mandates of a centralized government body.

 

Nonetheless, the costs borne by citizens and third-party payers loom as an incentive for gaining a better idea of which treatments work better than others. The Organization for Economic Cooperation and Development reported that the United States spent $6,401 per capita on healthcare in 2005, the highest in the world and twice as much as many other developed countries. And yet, according to Census Bureau rankings, the U.S. has lower life expectancy than at least 22 other countries and higher infant mortality that at least 27 other countries. Evidently, some of those healthcare dollars are not being spent wisely. As United Health Care’s Reed Tuckson noted at a recent conference of biotech executives, “healthcare is the only industry in which innovation drives up the cost.” Describing his own industry’s role, he said, “There are extraordinary misuses of assets within the system. We have to play the role of policeman to root out the ‘garbagey stuff.’”

 

Gail Wilensky, a health economist who is a former head of the Health Care Financing Administration (now known as the Center for Medicare and Medicaid Services), and perhaps the center’s leading advocate, adds, “We simply cannot continue for the next 20 or 30 years to have an increase in spending on healthcare that’s 2 to 3 percent faster than the growth of the economy in real terms.”

 

Currently, most money for biomedical research goes toward understanding basic biological processes and mechanisms. According to the Institute of Medicine roundtable, this research feeds the growing pipeline of possible treatments while sometimes crowding out studies that might determine what works best for either individuals or groups. The panel cites breast cancer as a case in point. Not long ago, breast cancer had only one treatment, radical mastectomy. Now, according to the IOM roundtable, “rapid and encouraging” growth in diagnostic approaches and treatment has reached the point at which the range of possibilities “is substantially outstripping the ability to understand what is best for a given individual.”  

 

Wilensky says studies supported by the center would look at both new and existing treatments for which there are alternative interventions, targeting primarily those that use the most money or affect the most people. She explains, for example, that there are few alternative ways to repair a broken hip, so it’s not a likely candidate for this research. But “there is a lot of variation in terms of how cardiovascular disease is treated, how back pain is treated, how some orthopedic problems are treated.” Some studies, Wilensky foresees, could be carried out by the entities currently doing this type of work. These include academic medical centers, freestanding research institutions, private companies, and even the NIH.

 

The House-approved bill underscores that the research should be insulated from inappropriate political and stakeholder influence; that research methods should be scientific, transparent, publicly documented, and available to all stakeholders; and that the prioritization and conduct of research, as well as the development of conclusions, should be transparent. The bill mandates that the center will be overseen by a commission of up to 17 members, named by the Comptroller General (head of the Government Accountability Office, an arm of Congress) in consultation with the appropriate Congressional committee chairs. The commission would set research priorities; monitor the use of the center’s trust fund; identify research methods and standards of evidence to be considered; and review and approve methodological standards and updates to such standards developed by the center. The commission would also recommend policies for public access to data, “while ensuring the information produced from research involved is timely and credible.”

 

Commission members are to include clinicians, patients, researchers, third-party payers, and consumers of federal and state beneficiary programs. The membership must include expertise in epidemiology, health services research, bioethics, decision science, and economics. And it must include at least one clinical researcher who does research on behalf of the pharmaceutical or device manufacturers.

 

But even if cost comparisons are not to be a component, some expect comparative effectiveness research to provide more value for dollars. Peter R. Orszag, director of the Congressional Budget Office, gave comparative effectiveness a stamp of approval that has been denied to other proposals. In a letter to Congress, his agency estimated that the center’s work, as configured in the legislation, would reduce healthcare costs slightly for the nation as a whole over its first 10 years. The studies would likely save Medicare and other programs money, he said, but not quite as much as would be expended on the research. In later years, Orszag suggested, the savings would increase as the body of data accumulated.

Understanding how a full-blown effort would affect the marketplace is incredibly complicated, says Jeffrey Lerner, Ph.D., president of ECRI Institute, a nonprofit healthcare research organization that has done comparative effectiveness research for 40 years. “If the health system moved much more toward doing these kinds of comparisons,” he says, “I think there would be a very large effect on how technologies enter the healthcare system.”

 

Despite widespread agreement that such research is needed, building consensus on specifics will remain difficult, in part because comparative effectiveness results almost always identify “a loser”—or at least a “less effective” procedure, drug, or device. And none of the center’s architects kids themselves that it will be a simple structure to build. 

 

PhRMA’s Burkholder points out that proponents of the research have taken many of their ideas from the United Kingdom’s National Institute for Health and Clinical Excellence (NICE) and a similar program in Australia. Those programs are worrisome to pharmaceutical manufacturers, who argue that NICE has delayed use of innovative treatments for years waiting on cost-effectiveness studies and that it has denied patients medication based on cost-effectiveness. 

 

What’s more, manufacturers have warned Congress that this kind of evidence can be misused as a foundation for one-size-fits-all coverage or payment policies on medications or other treatments. And this at a time when the medical world is showing that one size most certainly does not fit all—from patient experience to research to emerging molecular medicine.

 

In voicing the special concerns of the manufacturers of medical devices, Edwards LifeSciences’ Mussallem argues that device innovation evolves fairly continuously and that “the effectiveness of a particular product often depends on both the healthcare professional’s training and experience with the product.”

 

With comparative effectiveness research, however, “you essentially have to stop the music and measure your effectiveness at a point in time,” he says. It’s a process, Mussallem worries, which could cause valuable technology to become unavailable, because it did not test well against another treatment at a particular moment.

 

Mussallem emphasizes that transparency and input from industry is going to be critical: “Because it is possible for these studies to be misguided and to use the improper time frame or not be focused on the particular specifics of the condition and the disease.”

 

He calls for a process that not only encourages early input, but also has a review period at the end of the study, allowing for a possible challenge, for example, by industry or by medical societies. He, too, worries that the information might lead to a “one-size-fits-all approach to medicine,” as opposed to a marketplace where there are a number of choices for individual patients.