Medical Device Link.

The need to judge effectiveness

The prevailing sentiment among most health care and budget experts in Washington is that demands on federal resources have reached a critical point. The growth of entitlement spending has risen to a level where difficult decisions need to be made about the future of several critical health care programmes. These include Medicare, the federal health insurance system for senior citizens (www.medicare.gov); Medicaid, which gives coverage to the extremely poor (www.cms.hhs.gov/home/medicaid.asp); and the social security system. The rising cost of health care services together with greater federal spending on health care programmes has increased motivation towards evidence-based medicine. Many experts are promoting the idea of comparative effectiveness research (CER) as a solution to these problems.

The significant growth in the development of medical technologies over the past 20 years and, more importantly, the clinical benefits that have increased because of these developments means that people are now living longer, healthier lives. The influx of developments has resulted in more patients being treated and ultimately increasing costs. For example, patients have received more imaging and other diagnostic tests in the past decade than ever before.

Although these tests are successful in revealing medical conditions, payers are looking more closely at the utilisation rates to determine if therapies or tests are reasonable and necessary. Insurers, including Medicare, want to see a greater correlation between the increased allocation of resources and the benefits received. For Medicare, comparative effectiveness is seen as a viable solution to address this issue.

Difficulty in defining CER

CER is discussed as a methodology to produce information to help health care decision makers reach informed, evidence-based decisions. Most people agree that the research compares the “effectiveness” of two or more health care services or treatments. It evaluates outcomes resulting from different treatments or services and provides information on the relative effectiveness of treatments. However, specifics about the research and the definition seem to be sources of contention. In particular, the two areas that cause the most disagreement are the definition of “effectiveness” and whether or not costs should be included in CER.

The underlying structure for CER has been in place for some time through the Agency for Healthcare Research and Quality (AHRQ, www.ahrq.gov). With the economic stimulus package passed earlier this year, Congress moved to make CER a larger priority in overall health care reform. Specifically, Congress appropriated US$1.1 billion for the purposes of CER to be divided between the Secretary for Health and Human Services (www.hhs.gov), the National Institutes for Health (www.nih.gov) and AHRQ. In addition to the funding, the new law also established a Federal Coordinating Council, which will provide research priority recommendations to the Congress and Administration. The first report by the Council is expected in summer 2009.

Comparative effectiveness and cost

An important part of the medical technology commercialisation process is acquiring timely and adequate reimbursement for a product. In any market place, this means adhering to the competing and often different standards that are necessary to gain coverage from private and public payers of health care. Solely assessing clinical out- comes in CER is the preferred approach, and adding cost into the analysis has proven to be a controversial aspect of the methodology. In nations where a substantial portion of health costs are covered by private insurers, cost considerations have become the norm through the process of tech-nology assessments. It appears to have become more controversial to inject cost considerations into coverage determinations made by public payers of health care.

Government-financed health systems must make delicate judgements between wanting the appearance of adopting new, innovative technologies and having to control costs in a seemingly smaller pool of public resources. In many industrialised nations, CER has become a standardised process of reviewing and adopting new drug and medical technologies. For example, prior to 1993, most European countries did not formally require evidence-based outcome determinations in coverage decisions. However, as health care expenditures increased, most nations adopted some aspect of CER. Currently, most have a fully established comparative effectiveness component of coverage determinations or are developing one.

The comparative effectiveness model used in European countries may have difficulty in the United States (US) health care system. One unique aspect in the US is the strong emphasis placed on the patient–doctor relationship. Many believe that this relationship, in which the doctor will often recommend a product regardless of price, would be threatened if cost is considered. For example, the senior citizens’ Medicare health insurance system essentially prohibits costs to be considered in most coverage decisions. This limitation is based on the patient–doctor factor and the programme strives to ensure patients have reasonable access to new, innovative technologies.

Issues with the evaluation period

Problematic in any method of CER is the “horizon timeline” in which results are compared with cost. If a CER study is evaluating a short time period immediately after a treatment is initiated, it may only consider the initial costs without taking into consideration the long term savings. Savings such as reduced hospital or doctor visits and overall improved health may be lost if a CER assessment considers only the initial cost of a medical technology. This remains an issue that US policymakers must consider.

Thomas Novelli is Director of Federal Affairs and Policy, Medical Device Manufacturers Association, 1350 I Street NW, Suite 540, Washington, District of Columbia 20005, USA tel. +1 202 354 7171 e-mail: tnovelli@medicaldevices.org, www.medicaldevices.org