Comparative Effectiveness Research: Medical Practice, Payments, and Politics: the Need to Retain Standards of Medical Research

Comparative effectiveness research (CER), once only the scientific interest of clinical and health services researchers who compared medical treaments, now has tumbled into the public arena. ( 1 ) Facing the need for drastic improvement in our nation’s healthcare delivery, Congress and the Obama Administration are looking to CER to improve and broaden the use of treatments in a cost-effective way. Researchers, clinicians, professional societies, and policy experts have welcomed this, as they see CER as a scientifically rigorous way to select the most effective treatments for the benefit of patients and the public. On the other hand, while there has been support in the healthcare industry, apprehension has surfaced among those whose products would be subject to evaluation of effectivness, including pharmaceutical, biotech, and medical device companies, and among those who pay for treatments, including health plans, insurers, and large employers. Such concerns have translated into intense politics around CER—perhaps the best evidence that CER is truly likely to change practice and payment for medical care. This intensity also serves to remind us why, for CER, we must retain the usual standards and structures for medical research. Potential outcomes of CER include scientific knowledge, improved health, and financial impact. ( 2 ) In terms of science, across the spectrum of CER, from structured analyses of prior studies, databases, and registries, to the conduct of large clinical effectiveness trials, the scientific objective is rigorous reliable information about what treatments are best for what patients, and under what circumstances. Unless the conduct or public release of such research is compromised by poor quality or conflicts of interest, such information should have a direct positive impact on health. The economic consequences are less direct. For the nation, whatever the total costs of healthcare, CER should have a positive impact on cost-effectiveness—we would be spending healthcare dollars more wisely, on the most effective care, as concluded by detailed analyses by the Congressional Budget Office. ( 3–5 ) However, for those who sell treatments, the consequences are mixed. Pharmaceutical manufacturers may benefit financially because CER will compare drugs to not only other drugs, but also to medical devices and procedures, which could expand the conditions for which their drugs might be used, and thus would enlarge their market. However, some new on-patent drugs may be found to be no more effective than earlier off-patent versions already generically available at far less cost, and this could compromise sales of their most profitable drugs. Similarly, for medical device companies, profits could be reduced. Because currently FDA’s statute mandates less evidence of treatment benefit for medical devices than for drugs, a new requirement for rigorous testing of effectiveness would require extra time and money, and ultimately likely would show that at least some devices have undiscernable treatment benefits, which would curtail sales. These adverse effects on manufacturers’ profits are the other side of the coin that should result in greater costeffectiveness, which should be attractive to healthcare payers, including insurers, self-insured companies, and the government. Reliable well-accepted information on treatment effectiveness on which to base payment decisions would be very helpful. Moreover, that such information would be generated without insurers using their own funds, and without violating anti-trust rules against colluding with competitors about business decisions, but rather using public funds, is very attractive. However, the possibility that they may be mandated to provide access to treatments found to be effective and that their decision-making about payment would be potentially limited, based on such data, is of concern. These business concerns have generated political action. While expressing general support for CER, industry interests have conducted a two-pronged approach to alter CER legislation. First, they want to be sure that cost-effectiveness is not part of CER. Manufacturers fear impediments to sales of their most profitable products, and healthcare payers fear impediments to their healthcare coverage decisions. Manufacturers’ lobbyists have pressured Congressional offices involved in healthcare policy that have included CER in proposed legislation, insisting that cost-effectiveness analyses be specifically precluded from CER. Also, there has been a public campaign to raise fears about CER intended to influence Congress. For example, when in mid-February 2009 CER appeared likely to be part of the American Recovery and Reinvestment Act, commentary and editorials on Fox News, the Wall Street Journal, and other outlets, and commentators such as Lou Dobbs and Rush Limbaugh warned that CER would lead to government restrictions in treatments based on cost, and that old people would be denied all costly medical treatments under