New Comparative Effectiveness Center Should Ensure Evidence Quality – IOM

The Institute of Medicine says the government can boost the quality and usefulness of comparative effectiveness research by creating a single national program to build on existing efforts.

The Jan. 24 release of a new IOM report, "Knowing What Works in Health Care: A Roadmap for the Nation," comes as the idea of a national comparative effectiveness center continues to buzz with lawmakers in Washington as a means of reducing the amount the nation spends on healthcare.

Avalere President Dan Mendelson recently said that comparative effectiveness is "becoming an untouchable," meaning that it will be difficult to stop the comparative effectiveness momentum because it is hard to find anyone who will speak negatively of it. But he cautioned that conversation needs to focus on how comparative effectiveness is going to be used to reduce costs because more research in and of itself is not the answer.

Comparative effectiveness also is a major topic among insurers and has attracted significant lobbying dollars (¹ (Also see "Comparative Effectiveness, Generic Biologics Draw Insurers’ Lobbying Efforts" - Pink Sheet, 3 Sep, 2007.), p. 6).

Wilhelmine Miller, a health policy expert at George Washington University who was on the committee that wrote the report, said the recommendations are "complementary" to many of the proposals for a national center that have already surfaced on the Hill, but go further to discuss exactly how the research should be developed to assure health care decision makers can discern which evidence is valid in which situations.

The 280-page report, a work in progress since 2006, recommends that a new national program be created to set priorities for and fund and manage reviews of clinical effectiveness; standardize and invest in advancing the scientific methods underlying the reviews; and create voluntary standards to encourage groups that develop clinical guidelines or recommendations to use research that meets the program's standards.

The program IOM recommendations would be overseen by an advisory board appointed by the Secretary of Health and Human Services and comprised of public and private stakeholders. However, IOM did not recommend a specific organization take a leadership role in spearheading the research.

"The committee believes that the most pragmatic - and also the most promising - approach to establishing such a program is to build on current efforts," a prepublication copy of the report states. "In addition, private organizations that currently produce guidelines, such as professional societies and others, treasure their autonomy and would likely oppose efforts to reduce their role. Further, guidelines that have the imprimatur of a respected professional society are able to engender trust in end users."

Report Attune To Complexity of Research

Amid ongoing talk among policy makers of creating a national center for comparative effectiveness research, stakeholders have voiced a number of concerns about how such research would be applied to their products.

One of the biggest fears is that comparative effectiveness research will be used to determine which treatment is best on average, and deny access of products to individual patients that might benefit.

The IOM report underscores this complexity of synthesizing clinical evidence on a particular therapy without losing the nuances of patient population and other treatment variables.

"The art and science of systematic reviews requires expertise in both medicine and methodology to know where you can combine studies, where you have to distinguish their results because they're telling you something slightly different. So it's not a cut-and-dried technical exercise," Miller said in an interview.

It is also important to have a consistent source of funding to keep effectiveness reviews and resulting recommendations up to date, she noted.

Program Would Centralize Existing Efforts

One challenge the IOM report points to is the fact that there may not be enough researchers in the United States trained in the latest methodologies to conduct high quality systematic reviews. Miller noted, however, that by developing a national center to recognize the value of such work and provide funding to train more researchers, the capacity could be expanded relatively quickly.

Private groups such as the ECRI Institute and the BlueCross BlueShield Association's Technology Evaluation Center regularly perform systematic reviews of clinical evidence for medical technologies.

Miller pointed to the Agency for Healthcare Research and Quality's relatively new Effective Health Care program as another example from which the national program could build to produce information as a public good.

The AHRQ program has released comparative effectiveness reports on drug topics such as comparing oral medication for adults with type 2 diabetes, off-label use of atypical antipsychotic drugs and choosing antidepressants for adults.

Part of the advantage of having a national center would be to increase the scale and funding of the efforts. But of equal importance would be the centralized infrastructure, Miller said.

"Right now there's no single organization or national program that takes a perspective across payers, across age groups, across disease categories, types of services, to do this kind of work."

Cost Underpins Comparative Effectiveness

Miller noted that the IOM committee specifically set aside any discussion of what role cost-effectiveness research would play. "But clearly, effectiveness is one half of the equation in cost effectiveness," she said, adding that the systematic reviews could support cost-effectiveness analyses that payers or other stakeholders might want to do.

Meanwhile, the Congressional Budget Office released a ² report Jan. 31 reiterating its belief that comparative effectiveness research could help reduce overall growth in health care spending.

The CBO report, titled "Technological Change and the Growth of Health Care Spending," notes that if a statutory change allowed CMS to take comparative cost information into account, the agency could promote higher value care.

For example, CBO writes, if CMS tied payment amounts to the cost of the most effective treatments, hospitals would probably elect not to provide more expensive, but less effective, alternatives, having "the same practical effect as a coverage decision."

Another alternative would be to have patients pay for the additional cost of less effective procedures, CBO says.

Cost ultimately is going to have to play a role in comparative effectiveness research, Mendelson suggested during a Jan. 30 Avalere-hosted teleconference on comparative effectiveness.

"There is a lot of pressure coming from Congressional Democrats, particularly in the Senate, to find cost offsets for things that they want to do in the health care system [such as] covering children, expanding the nature of the various entitlement programs that we have and the like," Mendelson said.

Stakeholders expect to see a national comparative effectiveness program work its way into Medicare legislation in the first half of this year.

Proposals on the Hill so far have included funding original comparative effectiveness research as well, not just the systematic reviews that were the focus of IOM's report.

The House passed language last year in its CHAMP bill to create a comparative effectiveness center (³ (Also see "Benzodiazepine Coverage Among Medicare Provisions In Child Health Bill" - Pink Sheet, 30 Jul, 2007.), p. 18). On the Senate side, Finance Committee Chair Max Baucus, D-Mont., and Budget Committee Chair Kent Conrad, D-N.D., plan to introduce comparative effectiveness legislation early this year.

But it is going to take more than just generating research to find a benefit for comparative effectiveness, Mendelson said.

"Putting a commission together and creating more research ... it's kind of hard to see how just generating more evidence is likely to actually result in cost reduction over some period of time," Mendelson said.

He added that a key will be seeing that the research generated is actually being put to use. "There is a lot of evidence that has been generated that has simply not been adopted by medical care providers."

- Chloe Taft ([email protected]) & Gregory Twachtman ([email protected])