If healthcare reform legislation now before Congress becomes law, it will certainly contain a clearly articulated organization structure and a fairly generous flow of funds to develop and implement a robust comparative effectiveness research program.
The impetus behind the CER movement is two-fold. First, there is the realization that we have too little definitive empirical information about the relative efficacy of different therapies for the same conditions, or about the circumstances for which various therapies are the best choice. Second is the parallel realization that what little reliable evidence of comparative effectiveness exists is not well disseminated or translated into consistent clinical practice across geographic regions. As a result, there appear to be substantial unrealized opportunities to improve quality by developing and assuring the broad dissemination of better evidence about which therapies work and when, and accompanying opportunities to conserve resources and constrain spending by reducing the use of ineffective and inappropriate interventions. Development of electronic medical records and a broader health data transfer infrastructure (PPT) provide a critical enabling tool for this enterprise, and the growing acceptance of the personalized medicine movement creates a conceptual framework for efforts to assess the differential response of patient sub-groups to different therapies. The train is inexorably leaving the station, we know where it is going, and we appear to have decided to take the trip.
While the Senate (PDF) and House versions vary on the particulars, they each build on the CER provision of the Obama stimulus package — creation of a Federal Coordinating Council for Comparative Effectiveness Research with $400 million to fund research, train researchers, build a data infrastructure and translate research findings into implemented quality improvement initiatives. They each provide several hundred million dollars in annual financing; they each mandate attention to substantive research, data resource development, and translation and implementation of research findings, all in the name of better education of providers, patients and insurers with regard to the effectiveness of diagnostic, monitoring and therapeutic technologies and services; and they each specifically demand attention to differences among population groups. The stimulus commitment to CER signaled an aggressive beginning; the reform legislation will, if successful, make CER an increasingly vital part of the environment in which life science companies conduct their business. It just may, in time, create meaningful change in how companies develop products and business strategies. And if I’m right, CER will go a long way over time in leveling the playing field for new market entrants confronting larger, more established and better financed competitors.
Critics of the CER proposals emphasize a few standard fears:
- CER findings will be misused by regulators and insurers to eliminate coverage of viable therapies and restrict patient options;
- The CER enterprise will allow bureaucrats to get between doctors and their patients, improperly restricting the practice of medicine; and
- CER is a stalking horse for something that is far more pernicious — the use of formal cost-effectiveness analysis to base coverage decisions on the dollar cost of Quality Adjusted Life Years — thereby also discriminating against the old, the disabled and the terminally ill.
A careful reading of both surviving congressional bills reveals ample explicit protection on each of these fronts. The things that opponents say they fear the most simply will not happen.
A CER program managed by the Dept. of Health & Human Services’ Agency for Healthcare Research and Quality (as in the House bill) or by a newly formed independent not-for-profit corporation (as in the Senate bill), funded at a level approaching half a billion dollars annually, will do something more dramatic:
- It will change the nature of research on the effectiveness of healthcare interventions in a fundamental way, by introducing a substantial and credible publicly funded and independent counterweight to the privately funded, commercially driven research that has been the predominant mode of investigation of clinical effectiveness; and
- It will, through the translation and implementation process, introduce a similarly independent information dissemination and provider education process that will provide a readily accessible validity check for the marketing communications that have been so central to the clinical “education” of physicians and patients.
Today, the costs of clinical research and of a substantial sales and marketing infrastructure give large, long-established and resource-rich companies an enormous competitive advantage over potential new, small and resource-constrained competitors. Most clinical trials are sponsored, designed and executed by companies with a direct commercial interest in the outcome. This does not mean that they are necessarily corrupt or that their findings are invalid. It does mean that they are designed — within constraints imposed by the Food & Drug Administration and the insurer community — to test propositions that are of commercial value to the company and that much of the reporting of results is shaped by the choices made in trial design. Have you wondered why every drug-eluting stent developer is able to point to clinical data demonstrating the superiority of its own product? Might the answer lie in the subtleties of trial design and cohort selection? Do you think it is curious that nine years into the 21st century, we have no clear effectiveness studies to explicate differential responses to spinal fusion surgeries? Might it be because the surgeries are such a crapshoot that no technology company feels confident that they could produce an outcome supportive of its commercial interests?
An independent and well-funded CER program will design and implement studies that compare competing technologies, and will do so from a neutral perspective that is far more likely to yield meaningful results than is the corporate-driven research we’ve long relied on. In key priority areas, this effort will lessen the disadvantage borne by resource-constrained competitors, thereby leveling the playing field between large and small companies. And the information dissemination function built into the CER enterprise will similarly erode the advantage currently bestowed by a large marketing budget. Some of the questions that may actually help us choose therapeutic options more effectively will be asked and answered, and the answers will be communicated, through a neutral process of priority setting, study design, trial management, and outcomes dissemination. That would be a major change.
Edward Berger is a senior healthcare executive with more than 25 years of experience in medical device reimbursement analysis, planning and advocacy. He’s the founder of Larchmont Strategic Advisors and the vice president of the Medical Development Group. Check him out at Larchmont Strategic Advisors.
