Comparative Effectiveness Research — The New Imperative

Concern over accelerated health care costs in the United States has increased sharply in recent years. The per capita spending of gross domestic product (GDP) in the United States on health care is greater than any other developed country. In 2006, the United States spent $2.1 trillion, or 16 percent of GDP, on health care. This figure translates to $7,026 per person annually, but unlike other developed countries that provide near-universal coverage, 47 million Americans (15.8 percent) lacked health insurance in 2006.

One of the dominant drivers of rising health care costs is technology related changes in medical practice (38-62 percent). Technology is pushed out into the field at an alarming rate often before it is ready for national deployment. Other factors that are dramatically increasing health care costs include prices in the health care sector (11-22 percent), personal income growth (11-18 percent), changes in third party payments (10 percent), administrative costs (3-10 percent), and aging of the population (2 percent).1

At its current rate of increase, the rising cost of health care will be unsustainable in the future. As a result of these driving forces, researchers are examining a variety of approaches to controlling or decreasing health care costs.

One of the hot new areas in research that is attempting to make an impact on improving health care and controlling cost is comparative effectiveness. While there is no standard definition of comparative effectiveness as of yet, several definitions have been proposed by the Center for Medical Technology Policy, Congressional Budget Office, and the Institute of Medicine. The VA Office of Research and Development is using the following working definition:

Comparative effectiveness studies are studies that provide information on the comparative benefits and/or harms of two or more alternative choices for a given clinical condition, patient population, or health care system. These choices can involve medications, invasive therapies, non-pharmacologic treatments, diagnostic tests and strategies, models of care, or implementation strategies.

Given this working definition, the minimum criteria for comparative effectiveness studies include:

Different research methods are available for the study of comparing effectiveness of treatments. These methods include systematic reviews of existing research, analyses of claims records, analysis of medical registries, randomized controlled trials, and computer modeling. Each of these methods offers benefits and drawbacks.

Systematic reviews of research offer the easiest method by utilizing existing studies and synthesizing them to make additional comparisons. Analyses of claims records offer a more complex and time consuming method by utilizing existing sources of raw data. One advantage of this method is that it provides new information to resolve questions about treatments at a relatively low cost. One of the main difficulties with analyses of claims records, however, is that such analyses do not account for patient health status differences. Medical registries are developed to track patients with a similar disease or similar specific treatment.

Randomized controlled trials are the most definitive way to compare different treatments but are generally very expensive to perform and take a long time to complete. Computer models are programs that simulate the effects of different treatments on various populations. This method has been suggested as an alternative or an addition to clinical trials. Each of these analytical methods offers advantages and disadvantages in studying comparative effectiveness; these techniques should be customized depending on the research question, or combined to answer specific questions.

There is current debate on whether federally funded comparative effectiveness research should include consideration of cost effectiveness as well as clinical effectiveness. Some of the main arguments against including questions of cost effectiveness in comparative effectiveness studies are that cost structures vary across health care plans and, as a result, findings might not be generalizable to different plans or geographic areas. In addition, analysis of cost effectiveness might have a negative bias on the analysis of clinical effectiveness, leading to concerns that such analysis may result in restricted access to effective treatments. One advantage of considering cost effectiveness in federally-funded comparative effectiveness research is that it would encourage greater transparency and standardization in the methodologies used to determine cost.

The dominant driver of health care cost is the expanding medical technology arena where new modalities either fill a need for diagnosis and treatment, or replace older modalities that are cheaper. Newer technologies have a large impact on health care spending in the United States because there are few requirements that effectiveness be demonstrated before wide national implementation. Newer technologies also have the potential to increase applications where therapy might not even be effective.

The Veterans Health Administration research program offers an ideal home for studying comparative effectiveness. We are a large integrated health care system with an electronic medical record, Bar Code Medication Administration (BCMA) and provider order entry, strong pharmacy benefit, Technology Assessment Program (TAP) -- and outstanding researchers.

  1. Smith S, Heffler S, Freeland M. The Next Decade of Health Spending: A New Outlook. The National Health Expenditures Projection Team. Health Affairs (Millwood), 1999 Jul-Aug; 18(4):86-95.