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Comparative Effectiveness Research and Beyond

Paul G. Barnett, Ph.D., VA HSR&D's Health Economics Resource Center

Despite great advances in medical knowledge, the effectiveness of many health services is unknown. Comparative effectiveness research can close this gap and improve the quality of U.S. health care. The value of this research is widely appreciated. Effectiveness evaluations are almost universally employed by public and private health plans to determine what new technologies they will cover.1

Comparative effectiveness research may not answer all decision makers' questions, however. An effectiveness study can determine both benefits and adverse effects of an intervention, but this does not reveal whether the health improvement outweighs the possible harm. Even with clear evidence that a new test can more effectively detect disease, other evidence is needed to know if early detection confers a net benefit. Data from many sources must be linked.

Using CEA to Extend Comparative Effectiveness

These types of limitations in comparative effectiveness research can be overcome by methods used in Cost-Effectiveness Analysis (CEA).2

Recently, FORUM spoke with Al Perry, Director, VA Central California Health Care System, regarding the challenges facing VHA Senior Leadership Teams in the field. Perry described five challenges:

  1. Finding the most effective among constantly evolving treatment techniques, programs, equipment, and drugs.
  2. Meeting the challenge of newly eligible Priority Group 8s, and of 'victims' of the economic downturn.
  3. Delivering health care services to returning OEF/OIF Veterans, particularly those with mental health care needs and those living in rural areas.
  4. Delivering increasingly expensive services under tight budget constraints.
  5. Addressing ever increasing expectations for measurable quality and outcomes.

HSR&D research topics of interest to leadership in the field include: provider behavior, comparative technology, inpatient vs. outpatient treatment particularly for mental health, settings and approaches to women's health, and solo vs. team or group treatment.

CEA values health outcomes using a measure of morbidity adjusted survival called the Quality Adjusted Life Year (QALY). This measure can also be applied to comparative effectiveness research, to trade off benefits against harm. The medical decision modeling methods employed in CEA can be used in comparative effectiveness research, to link effectiveness findings to studies of long-term health outcomes.

Despite its versatility, CEA is not nearly as well regarded as comparative effectiveness by U.S. decision makers. This may be because they do not understand the uses of CEA, because they feel that its methods are unreliable, or because findings have not been relevant to their particular setting or time-horizon.3

Other reasons why CEA may not be used include political opposition from drug and device developers, and unwillingness of Americans to concede that effective but expensive treatments cannot be provided if benefits are modest.

As Dr. Almenoff points out, new technology accounts for much of the increase in health care costs. CEA can help determine if innovations yield sufficient value to justify their cost. CEA methods have been standardized for more than a decade and applied to hundreds of innovations. CEA is widely used in other countries, where it is among the criteria used to make coverage decisions.

VHA: The Challenge to Lead in Comparative Effectiveness Research

The Veterans Health Administration (VHA) is well positioned to be a U.S. leader in applying both comparative effectiveness and cost-effectiveness research. VHA is a globally budgeted, national system, with the long-term responsibility for the health of a well-defined population. Research is integrated with VHA care. VHA utilization and cost data are the envy of other health care plans.

Social value judgements about vulnerable or especially deserving plan members need to be incorporated into decisions based on CEA. This type of review has been important to the acceptance of CEA in other countries.4

Veterans are a well-organized constituency that deserves to participate in VHA coverage decisions.

Researchers must do a better job of learning the needs of health care decision makers. (For an example, see the concerns of medical center director Alan Perry in the side bar). We must clearly articulate our methods. Our studies must be more relevant and timely. We can shorten our response time by developing models of care for major diseases in anticipation of future coverage decisions.

Every household understands that resources are limited and that choices must trade off value against cost. Comparative effectiveness is just the first step on a path to greater efficiency. Cost-effectiveness analysis can help us get the best possible outcomes from the available health care budget.

References

  1. Garber, AM. Evidence-based Coverage Policy. Health Affairs (Millwood), 2001; 20(5): p. 62-82.
  2. Russell, LB. The Methodologic Partnership of Effectiveness Reviews and Cost-effectiveness Analysis. American Journal of Preventive Medicine, 2001; 20 (3 Suppl): p. 10-2.
  3. Neumann, PJ. Why Don't Americans Use Cost effectiveness Analysis? American Journal of Managed Care, 2004; 10(5): p. 308-12.
  4. Gold, MR, Sofaer S, and Siegelberg T. Medicare and Cost-effectiveness Analysis: Time to Ask the Taxpayers. Health Affairs (Millwood), 2007; 26(5): p. 1399-406.

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