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Centers for Medicare & Medicaid Services

View Public Comments for CED Public Solicitation

Etheredge, Lynn
Rapid Learning Project
George Washington University
Thank you for the invitation to comment on Medicare's CED policies. I believe it is essential to learn as much as possible, as soon as possible, about important new medical technologies and their best uses. A national CED process can be a vital element of such a policy initiative. I have laid out a proposal in a study "A High Performance System for Comparative Effectiveness Research" that appeared in Health Affairs (Oct. 2010, 29:10 (1761-1767) that i will forward to the e-mail address below. For example, the article says "A Rapid-Learning System For New Technologies" The assessment of new medical technologies— defined broadly to include drugs, devices, biologics, diagnostics, and procedures—poses important challenges for a new comparative effectiveness research system. So far, the most important advance in furthering national capabilities for research into new technologies has been the FDA Sentinel Initiative. Building On The FDA Sentinel Initiative Launched in 2008, this program will eventually give the government access to up to 100 million patient records for surveillance of drug safety problems. It has been largely a reactive, safetyfocused mechanism that awaits the first signals of possible trouble and only then sets off a wider review of databases from health plans and other sources. But the risks of a wait-and-see approach are that it will miss important clues that can and should have been detected faster, and that the data sources will prove to be incomplete or noncomparable so that they cannot provide definitive answers. For rapid learning in comparative effectiveness research, the Sentinel Initiative should be expanded beyond its initial safety focus to systematically collect data on therapies’ effectiveness, starting when a new therapy first enters clinical practice. Indeed, one of the most important unanswered questions about new therapies is this:What are its benefits compared to those of existing therapies for real-world patients—including (for an individual patient) patients like me? A national comparative effectiveness research program needs an upgraded data collection system that will build on Sentinel’s capabilities and use a national set of metrics to compare the effectiveness of alternative technologies. The new system would aim to learn as much as possible— and as soon as possible—about the optimal use of new technologies. Even less is known about new technologies than about old ones. As the Congressional Budget Office (CBO) has pointed out, the health sector’s adoption of new technologies is a leading driver of health care spending and the federal deficit.4 Timing The period after a new technology is introduced is likely to offer a rich learning opportunity. During this stage, clinicians use new technologies on a much more diverse group of patients—and for more conditions and combinations of conditions—than are covered in the clinical trials. This contrasts with the premarket period, where the FDA allows studies on selected groups and illnesses. A larger and broader patient base will also create a richer data resource, allowing investigators to identify and study risks, benefits, and other issues for subgroups with greater confidence. In a landmark study that identified important preapproval knowledge gaps that research should fill in, Annetine Gelijns, Nathan Rosenberg, and Alan Moskowitz note: “The history of medical innovation is replete with instances in which new indications have been discovered after drugs and devices have been introduced into clinical practice.” Their study reports, for example, that as of 1993, 90 percent of “blockbuster” drugs had important indications beyond those for which they were originally approved. By 1995, secondary uses accounted for more than 40 percent of their sales. The study found a similar pattern among medical devices.5(p694) Similarly, the National Comprehensive Cancer Network estimated in 2005 that 50–75 percent of all prescriptions for cancer therapies were “offlabel,” defined as the use of a drug for purposes other than those for which the federal government found proof of effectiveness and safety.6 By some estimates, more than half of prescription drug use is now off-label. For surgical procedures, the study by Gelijns and colleagues reported that only 4–13 percent of patients undergoing coronary bypass surgery would have met the eligibility criteria for the clinical trial that established its efficacy.5 A com- parative effectiveness research initiative for new technologies could fill in these kinds of knowledge gaps quickly. How The System Would Work A national learning system fornew technologies could work as follows. At the point of market approval or the start of Medicare payments—in the case of surgical procedures—HHS, in consultation with the private sector, would establish a national research plan for the technology or class of technology in question. The plan would specify metrics for comparing effectiveness; propose key questions about appropriate uses, effectiveness, and safety; create national reporting registries; and assign funding responsibility for studies. At the end of an initial period—for example, three years—expert panels would review the evidence and determine future research needs. Medicare, Medicaid, and private-sector payers would adopt common “coverage with evidence development” policies to require reporting of needed data from health care providers to answer key research questions as a condition for payment. To design and implement this comparative effectiveness research study system, the federal government should work with physicians, patients, researchers, the new Patient- Centered Outcomes Research Institute, health insurance plans, and pharmaceutical and biotechnology companies. In starting this new-technologies comparative effectiveness research initiative, the federal government should set research priorities such as new cancer treatments, new surgical procedures, and off-label uses of drugs.With hundreds of cancer drugs under development, it will be important to learn about these treatments as quickly and definitively as possible. Capturing off-label uses of new drugs by cancer patients will also be an important advance. The federal government does not require safety and efficacy studies for new surgical procedures, so there is often much uncertainty, even within the surgical professions, about their comparative benefits and risks. Surgical procedures also account for far more medical spending than do drugs. And patients face difficult choices between standard surgery, laparoscopic surgery, drug treatment, or lifestyle changes, and they deserve to be well informed. As noted in the article by Gelijns and colleagues, it is particularly important to learn quickly about secondary uses for new drugs, in part because they have been important sources for advancing the therapeutic arsenal, and also because of potential problems that may arise, such as higher death rates (as happened with Vioxx, a nonsteroidal anti-inflammatory drug that had to be withdrawn from the market after it had been approved). Physicians, patient groups, and the pharmaceutical industry should collaborate in these studies.