2
The Role of Payers in the Clinical Research Enterprise

INTRODUCTION

Sean Tunis, M.D., M.Sc.

Workshop Co-Chair

Director, Coverage and Analysis Group

Office of Clinical Standards and Quality

Centers of Medicare and Medicaid Services

and

Allan Korn, M.D.

Workshop Co-Chair

Senior Vice President and Chief Medical Officer

Blue Cross and Blue Shield Association

Two separate but distinct themes have emerged during the first part of the workshop regarding issues faced by purchasers, and each has different implications for the Clinical Research Enterprise. One theme is the notion of following best practices, following clinical guidelines, and reducing variations in care when the best thing to do is actually known. For example, treating heart attack patients with aspirin, with beta blockers, is the right thing to do, and yet this treatment is not given consistently. The central issue is how to translate that research into practice. This issue in turn has implications for determining what sorts of research studies and interventions the Clinical Research Enterprise ought to be conducting and figuring out what barriers exist to improving quality of care, changing physician behavior, monitoring variations in care, and so forth.

The other theme is how to improve care when the best thing to do is unknown. It is not known, for example, whether new-generation antibiotics lead to similar or better outcomes for patients with chronic obstructive pulmonary disease or emphysema exacerbation than the old inexpensive generic antibiotics. A head-to-head study of any novel antibiotic versus Bactrim or amoxycillin has not



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2 The Role of Payers in the Clinical Research Enterprise INTRODUCTION Sean Tunis, M.D., M.Sc. Workshop Co-Chair Director, Coverage and Analysis Group Office of Clinical Standards and Quality Centers of Medicare and Medicaid Services and Allan Korn, M.D. Workshop Co-Chair Senior Vice President and Chief Medical Officer Blue Cross and Blue Shield Association Two separate but distinct themes have emerged during the first part of the workshop regarding issues faced by purchasers, and each has different implications for the Clinical Research Enterprise. One theme is the notion of following best practices, following clinical guidelines, and reducing variations in care when the best thing to do is actually known. For example, treating heart attack patients with aspirin, with beta blockers, is the right thing to do, and yet this treatment is not given consistently. The central issue is how to translate that research into practice. This issue in turn has implications for determining what sorts of research studies and interventions the Clinical Research Enterprise ought to be conducting and figuring out what barriers exist to improving quality of care, changing physician behavior, monitoring variations in care, and so forth. The other theme is how to improve care when the best thing to do is unknown. It is not known, for example, whether new-generation antibiotics lead to similar or better outcomes for patients with chronic obstructive pulmonary disease or emphysema exacerbation than the old inexpensive generic antibiotics. A head-to-head study of any novel antibiotic versus Bactrim or amoxycillin has not

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been done. The lack of such comparison studies marks a huge failing of the Clinical Research Enterprise. In this part of the workshop, payers will examine how they are affected by each of these issues and will explore their relationship with the Clinical Research Enterprise. WHAT PAYERS NEED FROM THE CLINICAL RESEARCH ENTERPRISE Eric Book, M.D. Chief Medical Officer Wellmark Wellmark Blue Cross Blue Shield is a mutual insurer, predominantly in Iowa and South Dakota. The company insures roughly half of the population in Iowa and about a third of the population in South Dakota. As a payer, Wellmark is “caught between a rock and a hard place” in these times of double-digit inflation in medical costs. Purchasers ask payers what they are going to do about this problem. Payers do have some control over the unit cost but not much control over volume. Payers have two main questions concerning new products that to come to market: Why are we not paying less? Why are we not being given consideration with regard to the investment that we have already made in the development of the products and procedures being brought to market? These inquiries lead to the next question: What do payers expect to get from the enterprise? The short answer is structure and discipline or, said another way, a demonstration that the dollars are being spent wisely. Wellmark is receiving a greater number of requests from members to fund experimental or new procedures or interventions. These requests often concern terminal illnesses or those for which conventional care has not worked. The company does not want to be pushed into providing coverage, either by public opinion or by the courts, when a treatment has not been proven, has not demonstrated a cost-effective outcome, or has had deleterious effects on patients. An example is autologous bone marrow transplant for breast cancer. The company does not want to be pushed into providing coverage, either by public opinion or by the courts, when a treatment has not been proven, has not demonstrated a cost-effective outcome, or has had deleterious effects on patients. —Eric Book What payers want from the Clinical Research Enterprise is evidence of disciplined and robust management. They would like some individual or some entity to be accountable for the “big picture.” They need assurance that the focus of research is appropriately prioritized and managed for cost-effective outcomes.

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George Isham, M.D. Medical Director and Chief Health Officer HealthPartners Payers appreciate the current product of the Clinical Research Enterprise in terms of the new treatments and new procedures that help our members and patients obtain better health care and attain better health. What payers need are products and methods that improve the care of patients when they are sick, improve the overall health of individuals when they are well, and help prevent disease. They need to know from the enterprise what works and what does not work, with emphasis on what does not. Organizational research, such as that in the business schools, needs to be incorporated into the Clinical Research Enterprise. Two recent reports by the Institute of Medicine’s Committee on Quality Health Care in America, To Err is Human: Building a Safer Health System (2000) and Crossing the Quality Chasm: A New Health System for the 21st Century (2001), eloquently call for a system of care. Health care itself is a system of individual solo entrepreneurs and practitioners who need help in making that transformation. Payers need more research on how to be a system of care, and the Clinical Research Enterprise can make the appropriate linkages. Payers are also concerned with how to transform the culture of existing medical practice from a profession-centered, largely individual activity to something resembling a team sport that focuses on patient needs. Payers need to know from the enterprise what works and what does not work, with emphasis on what does not. —George Isham The culture of the individual medical professional has brought much that is positive to the health of this country over the last century, but the country is now facing the limits of some of the professional precepts. More research is needed on how the professional culture interacts with technology and how medicine can be transformed into a different culture. More needs to be known about population-based community methods of improving individual and community health. Knowledge of community preventive health is lacking. Few research funds are channeled to health promotion and disease prevention relative to the funds spent for treatment. If money is chemotactic, it is drawing most of the attention to the sick end of the spectrum. Although effective treatments are needed and appreciated, some money and attention must be drawn to the other end of the spectrum. Payers are also concerned with how to transform the culture of existing medical practice from a profession-centered, largely individual activity to something resembling a team sport that focuses on patient needs. —George Isham The research community needs ob

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jective measures for evaluating which research projects are of high quality and which are not. The information needs to be much more transparent than it is today, and it should be available to the public as well as to payers and purchasers. We need to know which trials are safe and which are not, and which research is subject to conflict of interest. The research community also needs a broader perspective on the ethics involved in funding research than is customarily given to researchers. One issue of importance to researchers is the ethics of competing for money at any price relative to other social goods such as affordable health care and equity of distribution of health across the population. These are the ethical issues with which payers wrestle in trying to provide affordable, cost-effective care; yet they seem to be “off the table,” in large part, for many individual researchers. In Minnesota, Wellmark has seven methods of providing preventive care to children and adolescents. These methods are promoted by the federal government through the Early and Periodic Screening, Diagnosis, and Treatment Program; through the state Medicaid program’s local program; through the American Academy of Pediatrics, which has one view; through the American Academy of Family Practice, which has another; through the consortium of health practitioners that have come together in the Institute of Clinical Systems Improvement, which has yet another; and through other organizations. It is ironic that although the federal government has done well in funding the research on preventive services for children and adolescents through its task force on clinical preventive services, sponsored by the Agency for HealthCare Research and Quality, much of the information does not find its way into the requirements of the programs that provide health care to children in Minnesota. Whenever possible, federal and state governments should work towards unified requirements. There ought to be some research into how to create better governmental mechanisms for balancing science with other competing interests. Other important research areas are differential characteristics of populations of poor, underserved children; the frequency of their needs and diseases; and the influence of standards of care relative to the influence in well-off and well-insured populations. Reed Tuckson, M.D. Chief Medical Officer United Healthgroup United Healthgroup is among the largest of the national health care insurers. One of its major business units is a company called Ingenix, which, among other things, conducts clinical research. A view from the payer perspective is that currently the Clinical Research Enterprise is not focused enough. Greater leadership is needed to help coordinate and focus research activity in a climate of limited resources. The Clinical Research Roundtable is a critical forum for pro

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viding this leadership. The members of the roundtable should be very vocal in putting forward a relevant and focused point of view. The time is right. The bioterrorism issues before us demonstrate the need to integrate epidemiology and disease surveillance and connect the data with identification of people at risk. In turn, those data must be connected with the recognition of appropriate therapy; long-term compliance; efficient use of resources; coordination across disciplines; and coordination among employers, health plans, and government. The moment has arrived to bring all of these components together. Greater leadership is needed to help coordinate and focus research activity in a climate of limited resources. —Reed Tuckson Payers need researchers to decide which studies they should devote their resources to exploring, and they need leadership in that regard. How will they know whether the resulting improvements are worth the effort? What is the process by which they make those choices and those decisions? They do not have a vehicle in place that allows that analysis. The process of prioritizing and funding research projects today is not systematic. Too often research is guided, for example, by what a junior faculty member at “Obscure University Number Three” wants to examine. In addition, payers need to know how new interventions or therapies compare with those that are already available. Government needs to do a much better job of leadership and of standardizing protocols across different agencies—for example, protocols for mammography or breast self-examination for women at various ages. The organization of clinical research needs to be enhanced. A specific need is to increase the speed with which products are delivered to the market. The current infrastructure does not deliver its products quickly, and it is inefficient from a cost standpoint. The cost structure is caught up in all kinds of competing needs, with much waste. What is needed is better integration of the discovery, development, and commercialization of new interventions. This integration should provide feedback that leads to better and more precise use of newly introduced technologies. The process of prioritizing and funding research projects today is not systematic. Too often research is guided, for example, by what a junior faculty member at “Obscure University Number Three” wants to examine. —Reed Tuckson The integration needs to include studies of actual use of these technologies by physicians and patients, and that feature has to be built into the model. Often it seems that new products are introduced, and then we wait to see what happens later. Payers want these features to be built in as a continuum. The reality of chronic disease must be recognized, and the coordination of multidisciplinary teams that are centered on the needs of the patient must be encouraged. The

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research infrastructure needs to be modeled on that new reality. The incorporation of clinical research into practice is still a major challenge. The organization of clinical research needs to be enhanced. A specific need is to increase the speed with which products are delivered to the market. —Reed Tuckson Payers need much better sources and integration of information that include performance data. Much more emphasis must be placed on the life-long learning of clinicians, so that they can incorporate these data and are able to use them. Much more attention must be given to the rewards for using the data, whether the rewards come through Continuing Medical Education, board certification, or board recertification, or whether the reward is becoming a “five-star doctor.” Similarly, consumers must have information that informs them about what they should be asking for in their health care coverage. They need to know how to make decisions about choice, not only with regard to elements in their plan, but also with respect to choice of hospitals and physicians on the basis of some sense of evidence of performance. The methodology currently available for determining quality at the level of the individual physician has is inadequate. Employers are beginning to ask the health plans to make individual physician ratings available on the employers’ Internet sites. Employers need leadership from the profession, through health services research and other valid measures, to determine how people can be informed legitimately about quality and be fair to physicians. Robert McDonough, M.D. Medical Director for Quality Management Aetna U.S. Healthcare Aetna U.S. Healthcare is a large national private health insurer that has about 18 million members. In addition to health benefits, Aetna offers dental benefits, vision benefits, life insurance, disability benefits, global health- and other health-related benefits. Its top clinical research priorities are to identify interventions, particularly at the health plan level, that are effective in improving health outcomes and in making more efficient use of health resources (by reducing costs while maintaining or improving health outcomes). Few clinical studies compare medical technologies of known effectiveness to one another. . . . New devices are often introduced into the market with little evidence of efficacy. —Robert McDonough Health plans can play a role in encouraging dissemination of medical technologies of proven safety and effectiveness. To accomplish this objective, they

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need studies that evaluate new medical technologies, and studies that new technologies with older established technologies. Few clinical studies compare medical technologies of known effectiveness to one another. In the absence of direct comparative studies, it is difficult, if not impossible, to determine which of several medical interventions are safest and most effective for a particular indication. Safety and efficacy studies tend to focus on new technologies, and the comparative effectiveness to older established technologies has often been overlooked. Private research funding has focused on clinical studies of new, patentable medical technologies such as new drugs and medical devices. This focus leaves out many developments, such as new surgical procedures and physical therapy maneuvers that do not involve a patentable technology. Private research funding for that kind of research is lacking, yet it is important research. Payers need health services research that examines methods of organizing the health delivery, both to increase the efficiency and to improve the organization of care. —Robert McDonough Food and Drug Administration requirements for devices are much weaker than those for drugs. New devices are often introduced into the market with little evidence of efficacy. They often are promoted heavily, and pressure for coverage is enormous. Health plans try to hold the line and insist on evidence of effectiveness. Frequently that evidence is not forthcoming, and at some point comes the realization that the intervention is not effective at all. Evidence-based clinical practice guidelines and consensus statements have often been used by health plans in making coverage decisions. Cost-effectiveness analyses have only been applied to primary clinical preventive services. Payers have not reached the point where they perform cost-effectiveness analyses to decide to cover new technologies that are proven to offer a clinically significant benefit. Cost is only taken into account in coverage decisions where there are two or more equally effective medical interventions for a given indication. Payers need health services research that examines methods of organizing the health delivery, both to increase the efficiency and to improve the organization of care. Chuck Cutler, M.D. Chief Medical Officer American Association of Health Plans Research on the effectiveness of prevention is an example of the kind of research that is helpful to health plans. A fair amount about the cost-effectiveness of preventive care services is known. Virtually all health plans have put a substantial amount of energy into improving preventive care services, even though many of these services do not provide cost savings to the health plan.

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Payers should look more broadly at the research topics and examine not only whether something works but what its benefits are—benefits not just in terms of medical care cost savings but also to society. A question that has been raised is whether the funding of clinical research is sufficient. While it is difficult to answer that question, there is always more valuable research to be done and other equally worthy activities to fund. One clear problem that emerges is the prioritization of clinical research. The goal of the Clinical Research Enterprise should be to improve the health of the American people. At the present time, it appears that politics and traditional drive some of the research endeavors, and the political process also drives some of the funding. It is not clear how the rest of the funding priorities are decided. Bringing a more transparent, rational decision-making process to the funding process would be an improvement. The Institute of Medicine report Crossing the Quality Chasm suggests a prioritization for research activities that is focused on disease burden, and the research activity should be structured in the manner that would be most likely to decrease the disease burden. In order to decide what services to cover and recommend, health plans need information about what works and what does not. Health plans also provide an infrastructure to support improvements in care that is otherwise lacking in the American health care system. They have a large interest in clinical trials. To some degree, they also provide an infrastructure to support improvements in care that is otherwise lacking in the American health care system. So they need to know where to invest their energies to gain the greatest improvements in outcomes for the populations they serve and which interventions will produce the best results. Currently under-funded [areas of clinical research are] operational research, information technology research, research on how to get people to change their behaviors in a clinical setting, and how to provide the appropriate information systems and other supports in a clinical setting. —Chuck Cutler Clinical research endeavors have significant safety problems. Payers have been concerned that insufficient safety controls are in place in the Clinical Research Enterprise and that many patients participating in clinical research are subject to avoidable risks. The clinical research enterprise needs to assure adequate oversight of clinical trials to protect patient safety. About 15 years is needed to put innovations that are proven to be effective into practice. Health plans need a better understanding of how to move these innovations into practice and how to be part of the Clinical Research Enterprise. That effort to translate research and innovation into practice would include areas that are currently under-funded, e.g., operational research, information technology research, research on how to get people to change their behaviors in a

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clinical setting, and how to provide the appropriate information systems and other supports in a clinical setting. The research community could learn much by examining how industries have improved their operations. The health care community seems to be focused parochially on clinical research and has continued to follow a traditional research agenda. We need to know more about behavioral factors in care. We need to understand what the behavioral barriers are. We can address access barriers where they exist, but there are other barriers that we do not understand. Health literacy barriers are problematic, and additional research in this area is needed. Appropriate incentives are needed to encourage providers and researchers to investigate how to translate research into practice. The main way in which information is currently disseminated is through traditional venues, e.g., professional journal publication. Research is needed on how to improve the delivery of innovations that we know are effective, and information about these innovations needs to be disseminated through new, more effective means. To protect patients, we also need more rigorous standards for innovations that do not require FDA approval, such as surgical procedures. Devices, even with FDA approval, are loosely evaluated and may not be safe or effective. Finally, an important issue is patient-centeredness. More patients will be making decisions about what kind of care they receive. They already are making those decisions. Right now, physicians see patients who bring in stacks of printouts from the Internet about new therapies and factors that they should consider. Payers need measures and information that patients can use to evaluate their care. Similarly, few measures exist for physicians and others to use for evaluating their performance. Most physicians in practice probably measure very little in the population they serve, other than through the data that they receive from health plans or perhaps from hospitals. The situation needs to change, and payers need guidance from the Clinical Research Enterprise as to what measures are important, what will bring the greatest improvement in the health of the American people, and what support to provide to physicians to promote these changes. We depend on the Clinical Research Enterprise to develop, test, and evaluate new interventions to determine if they are safe and effective as well as how they compare to existing interventions. These interventions should include the widest range of services including not only new technologies, but educational, counseling, and other services as well. WHAT PAYERS ARE WILLING TO CONTRIBUTE TO THE CLINICAL RESEARCH ENTERPRISE Robert McDonough, M.D. Health plans can contribute to the Clinical Research Enterprise by directly funding research. An example of an effective partnership that has produced

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worthwhile results is Aetna’s Academic Medicine and Managed Care Forum, which was founded by Aetna to foster a closer working relationship between academic medicine and managed care. Participants now include 53 of the nation’s top medical institutions, medical societies, major employers, federal agencies, private foundations, pharmaceutical companies, and medical professional organizations. The forum provides an arena where participants can collaboratively influence the delivery of high-quality medical care through the forum’s three principal components: working groups, research funding, and semiannual meetings. The forum includes a Quality Care Research Fund, initiated in 1997 with a $15 million commitment from Aetna. Between 1997 and 2000, over $26 million was awarded for research. The types of studies that have been funded by the forum are described on its website, www.academicforum.org. Health plans help put clinical research findings into practice through disease management efforts, provider education initiatives, and other programs. Health plans also disseminate clinical research findings and evidence-based guidelines to physicians. Aetna distributes tens of thousands of continuing medical education monographs to its physicians, and it provides financial incentives to physicians to participate in continuing medical education. It distributes clinical practice guidelines that are based on evidence-based guidelines of medical professional organizations, and preventative structure guidelines that are based on the work of the U.S. Preventive Services Task Force. Aetna also has its InteliHealth professional website, which provides constant flow of information that is available to all physicians, not just its participating physicians. Health plans help put clinical research findings into practice through disease management efforts, provider education initiatives, and other programs. —Robert McDonough Aetna disseminates health information to its members through its member health education program, InteliHealth website, and its Informed Health nurse help line. These services help empower consumers to obtain the information that they need for making health care decisions. Aetna develops evidence-based clinical coverage policies and uses the results of evidence-based research to make coverage decisions objectively. The tools that health plans use to help provide incentives for promoting evidence-based medicine are preauthorization, pre-certification, the pharmacy formulary, concurrent review, and retrospective review. Also, quality improvement programs involve measurement of adherence to clinical guidelines, feedback on the results, and provision of incentives to physicians. Clinical programs are used to identify and assure provision of appropriate evidencebased care to members with special health care needs. These are disease management programs that deal with specific diseases, case management programs

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that focus on people with critical illnesses, and maternity management programs. Health plans can also help implement the results of clinical research in clinical practice by encouraging enrollment of patients in FDA-approved clinical trials. —Robert McDonough Health plans can also help implement the results of clinical research into clinical practice by encouraging enrollment of patients in FDA-approved clinical trials. Aetna and many other health plans cover promising experimental treatments for patients with life-threatening illness who are being treated as part of a protocol in an FDA-approved clinical trial. Health plans may cover some costs of clinical trials besides those for life-threatening illnesses. Many health plans cover routine care costs for patients who are enrolled in clinical trials. Medicare and many health plans cover Category B investigational devices, which involve incremental modifications to established devices. Finally, health plans have an opportunity to create networks of care that can steer patients to centers that use evidence-based protocols and attain superior clinical outcomes. Aetna’s National Medical Excellence Program reviews institutions’ evidence-based protocols and clinical outcomes data to select those preferred centers for organ transplantation and for management of complex cases. Health plans are important consumers of research on clinical effectiveness, comparative effectiveness, cost effectiveness, and the effectiveness of the organization and delivery of health services. Health plans can support the Clinical Research Enterprise by funding clinical research directly and indirectly by using the tools available to them to promote the translation of clinical research results into clinical practice. Health plans have an opportunity to create networks of care that can steer patients to centers that use evidence-based protocols and attain superior clinical outcomes. —Robert McDonough There are arguments for both private (e.g., health plan) and public funding for research on interventions at the health plan level for improving efficiency and health outcomes. Health plans have an obligation to fund this research. But public funding should also be provided because the general public benefits from research on the organization and delivery of health care, and the results of this research may improve the efficiency of health care delivery and improve health care outcomes. Eric Book, M.D. Most Blue Cross Blue Shield plans provide benefit coverage for clinical trials. In October 1998, the Blue Cross and Blue Shield Association board passed a recommendation that member health plans pay up to the member’s benefit

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level for participation in a clinical trial, and 88% of Blue Cross Blue Shield members now receive this benefit. Another 7% to 8% receive coverage for clinical trial participation on request or on appeal. Once a new technology comes to market, has been proven, and becomes accepted, it tends to be priced much higher than the technologies that it replaces. The reason given is that the additional revenue funds research and development of these drugs and continuing research. Finally, a large amount of tax revenue is used for research. George Isham, M.D. Healthpartners, like many other payers, is directly involved in clinical research. Its research priorities are as follows: Heart disease, diabetes, and depression Obesity and its prevention Promotion of physical activity in the population Elimination of smoking Development of a system of prevention, health promotion, and clinical care Development of community and population approaches to improving the members’ health Development of methods for improving patient safety and avoidance of errors in care Applying methods of care known to be effective and eliminating unsafe and ineffective care RESEARCH PRIORITIES AND PRIORITY SETTING FOR PAYERS Myron Genel of Yale University School of Medicine began the discussion of research priorities by pointing out that many of the points made about translational blocks earlier in the workshop concerned the second translational block, putting clinical research into practice, rather than the first block, translating basic research into clinical research. He noted that in the first area, the traditional investigator-initiated research project has thrived in this country because it has created the ferment that allows great ideas to percolate. In contrast, it appears that this approach does not work in the second area. Other speakers have suggested that a more industrial model is needed at the second bottleneck to circumvent the disincentives that exist. At the first level, one cannot predict what research will be important, and too great a focus at that level might shut off the out-of-the-way idea that later proves to be fruitful. A more focused effort at the more pragmatic second-level bottleneck may be appropriate, however. Reed Tuckson of United Healthgroup mentioned that it is possible to focus

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the Clinical Research Enterprise and set priorities without inhibiting investigator-initiated research. From a national perspective, efforts should be focused on the largest disease burden. He suggested that there is a need to track research questions and their answers in an integrated way that puts all the pieces together from inception, rather than to look at the pieces individually and hope that somewhere along the line they will fall into a nice mosaic. Purchasers and payers understand the term “research” differently than investigators do. Purchasers and payers usually use the term to mean the application of analytic methods to solve business problems, whereas investigators use it to mean asking and answering questions or testing hypotheses. Sometimes those two meanings intersect, but sometimes they do not. —Lewis Sandy Lewis Sandy of the Robert Wood Johnson Foundation stated that purchasers and payers understand the term “research” differently than investigators do. Purchasers and payers usually use the term to mean the application of analytic methods to solve business problems, whereas investigators use it to mean asking and answering questions or testing hypotheses. Sometimes those two meanings intersect, but sometimes they do not. Lewis Sandy noted a paradox in the discussion: purchasers and payers have expressed a need for more information about what works and why, as well as a need for more outcome research, and have noted that the process of research is slow and laborious and does not fit their timeframes for decision making. Yet the laboratory for asking and answering such questions is the world of practice, change, and purchaser behavior, as well as the world of delivery systems, which purchasers and payers collectively represent. The RWJ Foundation funds, as does AHRQ and others, the work of many investigators who want to answer the kinds of questions purchasers and payers raise; yet investigators find that task challenging because the world is changing in a chaotic way that does not allow asking and answering questions or testing hypotheses. Sandy asked purchasers and payers: when you make changes in your system, would you consider using random assignment or quasi-experimental designs that would allow health services and outcome researchers to ask and answer questions and provide you with the answers that you say you would like? Chuck Cutler of the American Association of Health Plans mentioned that health plans do hypothesis testing of research ideas regularly, sometimes through formal investigation as in collaborative research with universities, and other times from a more formal practical business case analysis. Health plans are interested in questions such as the following: What are the causes of disease? What are the potential interventions? Are they successful or not? Health plans are interested in more than just a business research model. —Chuck Cutler

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Health plans are interested in more than just a business research model, however. A number of health plans are directly involved in research that is funded by NIH, AHRQ, the Veterans Administration (VA), and other agencies. In fact, more and more health plans are participating in research. A payer’s main purpose, as suggested by George Isham of HealthPartners, is to provide affordable health care to as many people as possible and, as a delivery organization, to provide what is known to be good health care as efficiently and effectively as possible with the highest satisfaction. The main purpose is not necessarily the discovery of new knowledge. Dr. Isham also noted that payers have tremendous opportunities for working together with researchers who are disciplined and trained in methodology, analysis, and study design and who are trying to determine how to deliver the care that is known to be efficacious. New methodologies are needed to answer these questions because the randomized clinical trial cannot always be used, particularly if the number of subjects is small. Reed Tuckson commented that the questions payers need to ask must represent the interests of many stakeholders and as part of the ultimate public good. Employers should not be expected to jeopardize their financial stability by paying for health care that is not effective. Instead they need to have their questions answered. Bright clinical researchers with expertise in addressing the relevant questions must take the lead in finding answers to these questions. Unless and until they do, measures may be imposed from outside the profession. That is not the way it should happen. There need not be a dichotomy; instead there can be a fluid integration between different interests. For example, United Healthcare sponsors a center for health care policy and research; while completely independent, the researchers live shoulder-to-shoulder with the business people who are trying to answer some of these questions. We know that relevant and practical research can occur in such settings. Employers should not be expected to jeopardize their financial stability by paying for health care that is not effective. —Reed Tuckson Al Reece, Vice Chancellor and Dean of the University of Arkansas College of Medicine, asked whether payers’ interests are so focused that the health benefits need to be short-term. Suppose that the benefits could indeed be long-term, he speculated. For example, some estimates by medical economists have suggested that the savings from osteoporosis prevention could exceed $300 million annually. Although the savings would not show up in the balance book for several years, it would be indeed be substantial. Chuck Cutler responded that payers need to know whether interventions are safe and effective, where they fit in the hierarchy of other interventions for the same disease, and what the cost implications are. The cost benefits of preventive

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care services must also be understood. Only a few preventive interventions offer cost savings. All of these have benefit, but the benefits come at some additional cost. Health plans have invested in increasing the use of many of these preventive interventions. Payers also need to understand the costs and benefits associated with disease management programs that help physicians do more than they can individually to provide preventive care and continuing care services for chronic diseases. George Isham noted that accompanying the decrease of uncompensated care is the increasing use of cost accounting mechanisms to justify the implementation of programs. In his view, going the accounting route totally is not an adequate way to decide which programs should be deployed and which should not. He called for consideration to be given to what social and other mechanisms can be instituted to benefit society as a whole in the long run, stating that a health care organization that invests in the health of its community will benefit in some fashion from its own efforts and those of its competitors. In the view of Eric Book of Wellmark, it is less important to payers that the outcomes are short-term or long-term and more important that they are tangible. If they are tangible, payers will support them and will do so with the full knowledge their members today may not be their members five years from now. TRANSLATIONAL BLOCKS Ken Getz began the discussion of translational blocks in research by asking for examples of instances in which translation of a medical intervention from research into practice actually went smoothly. Panelists were unable to offer any concrete examples. Chuck Cutler mentioned the use of beta blockers after myocardial infarction as a good example. Health plans and hospitals in some cases have installed systems to detect cases and examine prescription records. Persons not on beta blockers are identified for their physicians so they prescribe them appropriately. Advances in anesthesiology provide an example of identifying major threats to safety and designing industrial solutions. For instance, to eliminate errors, equipment for delivering anesthetic agents has different inputs for oxygen and the anesthetic agent. Other areas in which progress has been made are examinations for retinopathy, foot examinations, and hemoglobin A1c testing in people with diabetes. These efforts are most successful when they are value-added to physicians and when they are systematic. Information on these advances needs to be disseminated to individual physicians. [Translational] efforts are most successful when they are value-added to physicians and when they are systematic. —Chuck Cutler

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Reed Tuckson mentioned diabetes care as an example in which all legitimate expert opinions have been heard and organizations have worked together to develop integrated performance measures. The cooperating organizations range from the American Diabetes Association (ADA); to specialty societies for endocrinology, internal medicine, and family practice; all the way to health plans and the Joint Commission on Accreditation of Healthcare Organizations. Veronica Catanese of New York University School of Medicine gave the example of achieving normal glycosylated hemoglobin and pointed out that, although practice guidelines are well defined, the essential components for accomplishing this goal are not always in place. There are missing links in terms of how we enable the patients to achieve the goal. What is needed is behavior change, technology, counseling, and family support, among other factors. In response, Hugh Tilson of the University of North Carolina School of Public Health suggested that some partnerships outside the health care delivery system, in the public health system, might be useful in bringing about the desired change. The practical application of these initiatives can be interesting, replied Eric Book. As are many health plans, Wellmark is interested in reducing variation among physician practices. In Iowa Wellmark attempted to emulate the Minnesota model and to see if all major insurers and provider organizations could agree on a single guideline in a disease entity, and diabetes was chosen. They were very successful in agreeing on implementing a statewide guideline. The problem was that physician behavior was not substantially affected. A greater effect was achieved by working with the people with diabetes rather than with the physicians. When we focused directly on those with diabetes we started to see behavioral changes. Hal Slavkin of the University of Southern California School of Dentistry called for including a broader set of stakeholders if large gains are to be made in improving quality of life, reducing incidence of disease, and decreasing health disparities. He noted that we may miss a critical opportunity to include those who influence prenatal care and K-12 education in this country if we just use terms such as risk assessment, disease prevention, and health promotion. John Graham of the American Diabetes Association noted that the ADA is moving aggressively in the area of school lunches because it has noted that the food in the schools is deplorable. Not only does school food have low nutritional value, but the opportunity to buy food is entirely too prevalent. The issue of food in schools cuts across all disease entities. Although practice guidelines are well defined . . . there are missing links . . . behavior change, technology, counseling, and family support among other factors. —Veronica Catanese John Graham noted that the second key area is physical education. Once mandatory for all grade levels, and even for college students, it is now much less visible. Programs such as the President’s

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Council on Physical Fitness need to be reinstated; we are now paying a price for the lack of physical activity among the country’s youth, and the cost will continue to rise. The ADA is moving forward to work with the administration and the Department of Agriculture on those two issues. A discussion of these larger issues poses some risk of running well ahead of the evidence, warned Robert Califf of Duke University School of Medicine. He cited diet as an example of an area in which implementing broad public policies may turn out to be detrimental. For instance, the low-fat diet that has been promoted as beneficial for the heart, has been misinterpreted by the public, leading to a major increase in consumption of carbohydrate, causing obesity and diabetes. Allan Korn of the Blue Cross Blue Shield Association mentioned that the FDA has a good process for the adoption of new drugs. When pharmaceutical agents are proven to be safe and effective, they are rapidly adopted by health plans and made available. Where the system breaks down is the stampede to allow use of the drug for other conditions than those indicated in order to increase market share rapidly. This process can proceed smoothly, but if focus and discipline are not maintained, it can break down along the way. George Isham noted that the payer and purchaser community adopts guidelines developed by AHRQ, or policy announcements by Medicare or Medicaid in the states, but that many factors other than evidence and research come into play. Better models are needed for how the government can make linkages between evidence and practice. It is high time that the government stepped forward and took on this issue. As important as private purchasers have been in driving the use of good science and evidence over the last 10 years, the leading purchasers represent only a small sector of the great mass of private purchasers, more and more of whom are unaware of these issues and ineffective in driving change in local markets. David Rimoin of the Cedar-Sinai Medical Center, cautioned against assuming that once something is unambiguously demonstrated to be effective, it will be adopted. He described working in a system with 200 full-time physicians and 2,000 attending staff physicians who are fiercely independent “lonesome cowboys.” In this climate, information technology may be a key to the standardization of effective practices. Once a guideline received approval, it could become the standard and “roll out” through the electronic medical record and patient billing systems. If a physician did not agree with the standard, the physician would have to make an effort to document his or her reasoning. This system might initially be limited to hospitals because most outpatient facilities and physicians’ offices do not have this technology. Independence among physicians is an important issue, noted Reed Tuckson. If physicians decide that there really is no accountability for quality, and if the physician is not accountable for taking the lead, the question then becomes, who then steps up to the plate? Many employers in the room today, and other leaders

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around the country, are saying, if no one is going to step up to the plate, we will. They are demanding change. An example is “Leapfrog.” What are the boards going to do? What are the specialty societies going to do? Who will step up in the lead from the profession itself? Will it take the employers demanding that we all push this through? It is intellectually dishonest if we do not point out the issues, and if each of us—employers, purchasers, plans, hospitals, individual physicians, medical groups—do not make collective decisions about how we are going to behave and what our accountability is. Ideally, we need a collaborative effort and physicians should lead the way. Francis Chesley of the Agency for Healthcare Research and Quality noted in response that the government role is not sufficient and that partnerships are essential. The Agency for Healthcare Research and Quality, under its previous name the Agency for Healthcare Policy and Research, had the charge to bring clinical practice guidelines to the fore. As a result of that charge and leadership, that agency almost went out of existence. It is only through partnerships—and the AHRQ has them with the American Medical Association and the American Association with Health Plans—that we have actually been able to accomplish something in terms of bringing evidence-based information to the practitioner level. Purchasers are already interested in trying to hold physicians accountable at the individual level, stated Patricia Salber of General Motors Corporation. Purchasers are asking physicians to set up information technology systems so that the physician can monitor how he or she is managing patients. The aim is to encourage the physician to not only provide care to the individual patient but also manage the population that is his or her panel. David Scheinberg of the Memorial Sloan-Kettering Cancer Center described a system that the center established several years ago to try to deal with the issue of standardizing care among a captive group of physicians, all of whom are full-time employees salaried by the corporation. Every patient who entered the institution became a homunculus on a computer system, and all were tracked from the day they walked in to the day they left or died. Every laboratory order, every test, every operation, and every drug administered was tracked, the costs were monitored, and all data were tabulated for every physician and every patient. The system was an effort, at that time, to deal with costs and managed care issues. It turned out that, even in this extraordinarily well controlled, captive audience, every patient was an “N of one” and every patient had a variance. Although plans, drafted over two years, were instituted by the physicians to propose the appropriate and best standard of care for every disease encountered, hundreds of different pathways, mapping every possible pathway, were instituted in this computer, and yet there were still hundreds of variants. That result was in a very small microcosm. The idea of instituting such a system on a community or national level is a laudable goal, but very difficult to achieve, to say the least.

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CONSUMER DEMAND A discussion of the participation of consumers in health care and the resulting impact on payers was begun by Edward Campion of the New England Journal of Medicine, who stated that the locus of control seems to be shifting toward the more assertive patients and consumers. In many cases these consumers are basing their decisions largely on health information from the media, which is, at times, directed at them with financial incentives behind it. Consequently, what drugs are dispensed or what operation is performed depends not on consumers’ knowledge of results of randomized controlled trials or cost benefit analysis, but on patients’ perception of how these treatments will affect them. Reed Tuckson asserted that the real dilemma is how to address the public education system in America, which helps us train and educate people to understand the complexities underlying modern science. Another important dilemma is how to arm people with the information that they need to be able to give informed consent in the genomic era. The educational system, not the health care system, must deal with those enormous public challenges. It is encouraging that organizations such as the National Health Council are providing the beginnings of fundamental sets of questions and answers to people in all of their organizations, and are emphasizing that consumers are the captains of their own health care teams. These organizations are also recognizing that consumers have the right to make health care choices in the context of their relationships with their physician. The empowering role of the consumer is key, and the means of providing the information is important. Here the Internet plays a major role, although the racial and economic disparities in access to that technology must be recognized. It is clear, though, that we are making progress. Consumers must have access to information that allows them, across a continuum from the most interested to the most passive, to participate intelligently in their health care. What drugs are dispensed or what operation is performed depends not on consumers’ knowledge of results of randomized controlled trials or cost benefit analysis, but on patients’ perception of how these treatments will affect them. —Edward Campion George Isham noted that the more that consumers are faced with competing information and competing clinical trials in which to participate, the greater will be the need for transparency with respect to the Internal Review Board proceedings, the benefit of the research intervention, and the options available outside of the research trial. Consumerism is often equated to paying the bill, in the sense that there are more co-pays. We find that for more of our insurance and managed care products, purchasers and others are asking us to include more features such as co-insurance and deductibles in the product. This inclination is in direct conflict with the inclusion of all therapeutic elements for diabetes, which was brought

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up earlier in the workshop. Our thoughts used to be that if it works, it ought to be paid for in a broadly first-dollar coverage insurance product. Apparently many conflicting paradigms exist in the current marketplace as to what an insurance product is or is not, and how consumerism interacts with whether it is right to have consumers pay the bill. PATIENT PARTICIPATION IN CLINICAL RESEARCH An issue posed by John Gallin of the National Institutes of Health Clinical Center is how to encourage providers to refer patients to the Clinical Research Enterprise as study participants. Recruitment of patients for participation in clinical studies, whether natural history studies or clinical trials, is increasingly difficult. Patients no longer have the personal relationship with their physician that was prevalent 15 years ago, when nearly all patients knew their physicians, as opposed to only 6 in 10 patients today. Many patients consider their insurance carrier to be their provider, and many worry that they will lose their insurance coverage if they participate in a clinical trial. A common perception among patients is that they would have difficulty obtaining permission to participate in a clinical research activity. In response, Chuck Cutler noted that barriers to participation in clinical trials are multifactorial and that health plans do not present the greatest barrier. Other barriers are lack of physician awareness of clinical trials, lack of physician willingness to refer patients to clinical trials because the patients will then lose their relationship with the physicians, and patients’ unwillingness to be what they perceive as “guinea pigs” in clinical trials. He stated that he did not believe that any health plan would deny someone the opportunity to participate in a study funded by the National Institutes of Health (NIH), depending on the benefit design. SUMMARY The goal of the session on the role of purchasers in the Clinical Research Enterprise was to elucidate how the Clinical Research Enterprise can better serve payers as they strive to provide affordable health care coverage to as many people as possible, provide best evidence-based health care efficiently and effectively with the highest satisfaction, and contribute to health care research. Representatives from health plans (Wellmark Blue Cross Blue Shield, Health Partners, United Healthgroup, and Aetna U.S. Healthcare) and a representative from the American Association of Health Plans presented their views on what payers need from the Clinical Research Enterprise, how the enterprise has met payers’ needs, and what payers are willing to contribute to the enterprise (see box). The representatives of the health plan industry also discussed bottlenecks in translation of basic science into clinical practice and the translation of clinical

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guidelines into care, citing incidences of successful translation of medical interventions from research to practice. They examined the role of health care plans in improving the health care system and advancing the health of the community as a whole. They acknowledged the shift toward greater participation of consumers in their own health care and explored means for enhancing consumer education. Finally, they addressed the concern that health care plans may present barriers to patient participation in clinical trials.

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Highlights of the Session on the Role of Payers in the Clinical Research Enterprise What do payers need from the Clinical Research Enterprise? Payers depend on the Clinical Research Enterprise to develop the interventions that will improve the health of the populations they serve and society as a whole. Payers need to understand what works and what does not work in the care of patients, the prevention of disease, and the promotion of health. They need to know how to provide safer care and eliminate errors to keep members free from harm while participating in health care. Payers need new knowledge on how to transform health care into a more systematic effort and how to help professionals understand that they are part of this systematic effort. They need to give guidance on how to transform the culture of medical practice from a profession-centered, individual activity into a patient-centered, team activity. They need more insight into population-based community methods of improving individual and community health. Payers need to know from the enterprise which research projects are of high quality and which are not, and which research efforts are safe and which are not. They need to know which researchers have conflicts of interest and what those conflicts are. They need full disclosure of information regarding research funding to the public, potential research participants, and other stakeholders. Research is needed on the barriers to the delivery of or patient adherence to interventions or behavior change, such as health illiteracy, cultural competencies, insurance issues, financial issues, or transportation issues. How has the Clinical Research Enterprise met payers’ needs? The Clinical Research Enterprise provides the knowledge that helps payers understand that what they do for patients is helpful. It provides payers with innovations that improve care, prevent illness, and promote health. The Clinical Research Enterprise holds the potential to question existing practice and help over time to eliminate ineffective and even harmful practices. What are payers willing to contribute to the Clinical Research Enterprise? Payers play a role in encouraging dissemination of medical technologies of proven safety and effectiveness. They can also encourage the implementation of evidence-based guidelines and consensus statements in clinical practice. Payers have supported research activities both independently and by forming partnerships with medical institutions, medical societies, employers, federal agencies, private foundations, pharmaceutical companies, and medical professional organizations. Payers help put clinical research findings into practice through disease management efforts and provider-education initiatives. They also disseminate health information to their members through health education programs.