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Implementing Evidence |
PERSPECTIVE
A Clinical Research Strategy To Support Shared Decision Making
| Abstract |
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Greater use of evidence in health care decision making has highlighted the limited quantity and quality of evidence for many decisions. To make informed decisions, patients and physicians depend on valid evidence about benefits, risks, and costs of alternative treatments. Policymakers can also use this information to make promising new technologies available sooner, while ensuring adequate evaluation. The clinical research enterprise can be expanded with simple trials and registries. A more systematic effort to produce this evidence will entail establishing research priorities, improving research methods, expanding the research infrastructure, and engaging new funds.
As Americans become more engaged in shared health care decision making, patients and their physicians must have reliable information on the risks, benefits, and costs of the alternatives from which they must choose. Also, as the rate of national spending continues to rise much faster than inflation and wages, the importance of using resources effectively and efficiently becomes more urgent.1 High-quality information is essential for making decisions that maximize the health benefits derived from available resources.
Examples of common questions that must be answered daily by patients and physicians include the following: Would it be better to begin drug therapy for mild to moderate osteoporosis, or begin a regimen of diet and exercise? Which specific order or combination of drugs is associated with the lowest risk of long-term complication of diabetes or hypertension? Will a topical compound help a chronic leg wound heal more quickly than standard wound care would? Would obtaining a positron-emission tomography (PET) scan in addition to computed tomography (CT) and magnetic resonance imaging (MRI) for staging a particular cancer influence the recommended treatment regimen? Will a surgery have better short- and long-term results if done using minimally invasive techniques instead of the standard open surgical approach? What are the long-term benefits and risks of various interventions to treat prostate enlargement or prostate cancer? Clinical research that is explicitly designed to answer common questions like these has been referred to as "practical clinical research."2
The recent problems with hormone replacement therapy (HRT) and rofecoxib (Vioxx) underscore the fact that patients, physicians, and policymakers have a common interest in the generation of reliable comparative information on the risks and benefits of various health care choices, and the importance of increasing the capacity of the clinical research enterprise to produce this information quickly and efficiently.
It would be natural to assume that all such questions are systematically identified and answered as part of U.S. clinical research activity. And, in fact, many similar questions are addressed through publicly and privately funding clinical research. However, for many critical issues, adequate information is not available. There is no public or private organization whose mission is to determine which of the many critical clinical research questions need to be answered first and to expand the capacity to answer them. Without a focused effort to address this problem, we are unlikely to obtain the maximum benefits potentially available from new and existing medical technologies.
In this paper we call for a systematic effort to increase the supply of simple, real-world prospective clinical trials and registries. We also describe how the Centers for Medicare and Medicaid Services (CMS) is using its policy-making authorities and resources to increase the supply of reliable evidence to assist decisionmakers—patients, physicians, and policymakers—in making informed health care choices.
| Medicare Strategies To Improve The Supply Of Evidence |
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 Top Medicare Strategies To Improve... What Needs To Be...Editor's NotesNOTES |
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Medicare has a number of policies in place that help promote the development of better evidence for decisions. For clinical trials sponsored by the National Institutes of Health (NIH) and several other federal agencies, Medicare will pay all routine costs of care for beneficiaries enrolled in those trials. The CMS applies a relatively broad interpretation of "routine costs," paying for all clinical costs for these patients unless the service would clearly have been provided only to patients participating in the clinical trial. Under its Investigation Device Exemption (IDE) policy, Medicare also will pay for experimental devices that are designated by the Food and Drug Administration (FDA) as being similar to already proven devices (so-called category B devices) and that are being studied in the context of trials expected to lead to FDA approval.
Medicare has also applied its national coverage authority to provide access to new technologies that are promising but that still entail important unanswered questions about risks, benefits, and costs. Medicare is required by law to provide coverage only for medical interventions that are considered "reasonable and necessary." The purpose of this statutory requirement is to ensure that Medicare funds are spent on services that are likely to improve the health outcomes of beneficiaries. During the past five years, there have been major improvements in the scientific basis of coverage decision making within Medicare.3
Certain experimental interventions may be considered reasonable and necessary (and therefore covered by Medicare) only when they are provided in the context of additional protections provided in clinical research studies. The most important example is the National Emphysema Treatment Trial (NETT), which took place from 1996 to 2003 and evaluated the risks, benefits, and costs of lung volume reduction surgery (LVRS) in patients with severe emphysema.4 NETT was cofunded by the NIH and the Agency for Healthcare Research and Quality (AHRQ), with all clinical costs paid by Medicare. The study produced critical information about the impact of surgery on quality of life and mortality, identified a subgroup of patients for whom surgery was harmful, and provided strong evidence that helped to identify which specific patient characteristics were associated with reduced mortality and increased quality of life. NETT provided crucial data to patients and clinicians who were considering this procedure, and Medicare used the results to make coverage available to all patients for whom there was a reasonable chance of improved survival or quality of life.
In several recent national coverage decisions, Medicare has linked coverage of innovative diagnostic and therapeutic technologies to their provision in the context of clinical studies.5 This approach was applied to coverage of carotid stents when used in a postmarket study required as a condition of FDA approval. Medicare also decided to cover the costs of PET scans for patients with suspected dementia when they are enrolled in a large, community-based, practical clinical trial. The CMS is working with the Alzheimer’s Association, PET scanner manufacturers, AHRQ, and clinical and methods experts to design and implement such a trial. Most recently, Medicare has proposed to link expanded coverage of implantable cardioverter defibrillators (ICDs) with submission of data to a clinical registry. The primary intent of this registry is to gain additional information about the risks and benefits of the technology outside the context of formal randomized controlled trials (RCTs) and to provide data that may better define which patients benefit most from the device and whether specific provider characteristics are associated with better procedural outcomes.
These examples illustrate how Medicare can make coverage decisions that are faster and broader than would otherwise be possible, by linking coverage to the prospective gathering of important clinical data. Even in the absence of definitive evidence proving benefit, promising innovative technologies can be covered in the context of studies that will demonstrate whether and how the technology works best.
However, the approach is not yet systematic, and additional attention and resources will be required if the amount of information available to decisionmakers is going to be better matched to the number of important health care decisions that need to be made. With the recent doubling of the NIH budget and massive private investment in biomedical research and product development, the pace of innovation has increased, and many experts expect dramatic breakthroughs in the coming years. Additional attention will be required to develop the information necessary to use these advances effectively and efficiently.
| What Needs To Be Done Next? |
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| TopMedicare Strategies To Improve...What Needs To Be...Editor's NotesNOTES |
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There is an urgent need to increase the capacity to conduct simple, real-world, prospective clinical studies to efficiently provide reliable data on the risks, benefits, and costs of existing, new, and emerging technologies. Practical clinical trials and registries can provide some of the needed information, and these methods can produce data that complement what is derived from traditional RCTs. Traditional trials are essential to translating biomedical discovery to clinical use and are the optimal approach to providing new knowledge about the mechanism by which diagnostic and therapeutic interventions may produce clinical benefit or harm. But traditional RCTs and the infrastructure that supports them cannot supply all of the necessary evidence. Funds for clinical research are finite, and traditional clinical research sometimes moves more slowly than the pace of technological development and the public demand for technology. To avoid the continuation of the status quo, in which technologies and services are adopted with little useful information about their clinical value, a strategy is needed to collect data more rapidly and efficiently.
To increase the production of better evidence for decisionmakers, it will be necessary to focus on four main areas: (1) setting priorities based on a systematic process for identifying information gaps; (2) developing simpler, less expensive methods for evaluating effectiveness; (3) building the capacity to efficiently conduct clinical research into the care delivery system; and (4) exploration of nontraditional sources and mechanisms to pay the clinical and research costs for these studies.
Priority setting. In the current approach to setting priorities for clinical research, decisionmakers’ information needs are sometimes an important consideration. What is missing is a systematic process through which important gaps in knowledge are identified, from the perspective of those stakeholders who must decide how to use health care services: patients, clinicians, payers, purchasers, and policymakers.
Technology assessments, clinical practice guidelines, and systematic reviews are excellent sources from which to identify critical research gaps. Each of these analytic activities begins by identifying questions for which decisionmakers need answers, and subsequent review of existing evidence that is relevant to those decisions generally demonstrates that the quality of evidence is suboptimal. It also becomes possible to identify the specific studies required to fill those information gaps. The studies could be reviewed by patient advocacy organizations, clinical experts, methodologists, product manufacturers, and policy-makers to determine (1) which questions would be the highest priority to address and (2) the fastest, most efficient approach needed to provide adequately reliable answers. AHRQ has made much progress in establishing priorities for comparative effectiveness studies within the context of implementing Section 1013 of the Medicare Prescription Drug, Improvement, and Modernization Act (MMA) of 2003. Building on this work, AHRQ, the CMS, and the Institute of Medicine are developing a priority-setting process for practical trials and registries, with special attention to establishing a transparent process that identifies critical gaps in knowledge from the perspective of those who make clinical and policy decisions.
Methods development. It is also critically important to develop consensus on methods for conducting simple, inexpensive clinical studies that provide answers that are sufficiently reliable to guide decisions. Practical clinical trials, registries, and other simplified approaches to clinical research have been successful in the United States and abroad; they have provided considerable useful information at relatively low cost. These studies offer the potential advantages of rapid recruitment of large numbers of patients producing data that reflect results of care as it is provided beyond the confines of clinical research settings. However, practical trials and registries may provide data that are less detailed and reliable than the data derived from traditional RCTs, and many important questions cannot be adequately studied using these methods. In exploring how best to design and implement practical trials, registries, and other real-world prospective studies, it would be useful to review the strengths, weaknesses, and contributions of similar studies that have already been conducted.
For these studies to be affordable and relatively simple to conduct, efficient methods for human subject review, patient recruitment and consent, and collection of baseline and follow-up data also must be developed. The potential to randomize group practices or practice sites within larger organized delivery systems may offer a viable approach to conducted large, simple studies.
Infrastructure. To complete more trials and registries faster and at low cost, a research infrastructure must be designed and maintained for this purpose. It must be constructed to allow simple prospective studies to be conducted in busy clinical practices where clinical research is not a primary focus. It is extremely expensive and inefficient to develop a capable network of investigators, study site coordinators, data collection systems, procedures to enroll and track patients, and quality assurance mechanisms for each new clinical research project.
Wider adoption of interoperable electronic medical record (EMR) systems will greatly simplify implementation of real-world clinical studies, and adoption of these systems, along with local data networks, will be expanding rapidly as a result of the efforts of the Office of the National Coordinator for Health Information Technology. Electronic administrative data already provide an inexpensive means of obtaining data on resource use, economic endpoints, and some clinical outcome information, and the CMS is refining strategies to use these data to better evaluate new pilot programs and policies. The CMS is also collaborating with the FDA to use Medicare claims data for postmarketing surveillance, looking for major adverse events associated with adoption of newly approved medical devices. Similar work assessing drug safety could be possible using data gathered in the context of the new Medicare drug benefit.
As part of the NIH Roadmap, the NIH has been working to identify and link existing clinical research networks and to equip these networks with informatics tools that will increase the efficiency of data collection, protocol preparation, institutional review board (IRB) management, and adverse-event reporting.6 If these research networks become engaged in practical clinical trials and registries identified by the priority-setting strategies described earlier, they could represent a tremendous contribution to the U.S. clinical research capacity.
Among the existing research networks that could be useful for practical trials and registries are the primary care research networks funded by AHRQ and by the American Academy of Family Practice. The health maintenance organization (HMO) research network, an alliance of nearly two dozen large managed care organizations, all with functional EMRs, is well equipped to begin real-world studies on a wide range of clinical topics. Although these networks have had limited experience in conducting clinical trials, they represent an existing infrastructure that may be an important future resource.
Funding. New sources of funds will be required to support practical clinical research, and the financial responsibility will likely need to be shared by all stakeholders. Linking coverage of medical technologies to enrollment of patients in practical trials or registries represents sizable new funding for the costs of these experimental interventions. In conjunction with simplified methods for conducting trials and registries, this approach can accelerate the dissemination of promising innovations and generate better information for future patients and clinicians. This approach is also useful for proven technologies when additional research would help better define the patient factors that influence the benefits and risks of the intervention. The impact of linking coverage to prospective clinical studies would be increased with the participation of other public and private payers and purchasers.
While linkage of coverage to participation in clinical research helps cover the costs of the interventions being studied, it will be necessary to identify additional resources. Assuming that progress is made on simplified and less costly methods, particularly using the existing infrastructure of clinical research networks and organized systems of care, these costs may be modest in comparison to the clinical costs being supported by Medicare, and perhaps other payers that choose to adopt this approach. Product sponsors may choose to support research costs to facilitate rapid completion of the necessary studies, which could lead to a rapid increase in use of the technology if important benefits are demonstrated through the studies performed. It is also anticipated that payers, purchasers, medical professional organizations, and disease-specific philanthropies would all be potential funders of practical research under appropriate circumstances.
Such solutions will be needed for health care decisionmakers to make informed choices toward the goal of improving health and to get good value for available health care dollars.
| Editor's Notes |
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| TopMedicare Strategies To Improve...What Needs To Be...Editor's NotesNOTES |
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Sean Tunis (sean.tunis{at}cms.hhs.gov) is chief medical officer and director of the Office for Clinical Standards and Quality, Centers for Medicare and Medicaid Services (CMS), in Baltimore, Maryland.
The author thanks Mark McClellan for numerous contributions to the concept presented in this paper.
| NOTES |
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| TopMedicare Strategies To Improve...What Needs To Be...Editor's NotesNOTES |
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- P.B. Ginsburg, "Controlling Health Care Costs," New England Journal of Medicine 351, no 16 (2004): 1591–1593.
[Free Full Text] - S.R. Tunis, D.B. Stryer, and C.M. Clancy, "Practical Clinical Trials: Increasing the Value of Clinical Research for Decision Making in Clinical and Health Policy," Journal of the American Medical Association 290, no. 12 (2003): 1624–1632.
[Abstract/Free Full Text] - S.R. Tunis and J.L. Kang, "Improvement in Medicare Coverage of New Technology," Health Affairs 20, no. 5 (2001): 83–85.
[Free Full Text] - T.V. Carino, S. Sheingold, and S.R. Tunis, "Using Clinical Trials as a Condition of Coverage: Lessons from the National Emphysema Treatment Trial," Clinical Trials 1, no. 1 (2004): 108–121.
- CMS, "Medicare Coverage—What’s New?" 21 October 2004, cms.hhs.gov/coverage/8g.asp (24 October 2004).
- National Institutes of Health, "NIH Roadmap," nihroadmap.nih.gov (24 October 2004).
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