Introduction
The Need for Information To Improve Quality
Challenges Facing Clinical and Health Services Research
Policy Options for Supporting Research
Examples of Collaborative Efforts
What Is Needed Now
The Role of Clinical Trials in Quality Improvement
Conclusion
References
Thank you for the opportunity to address the Advisory Commission's Subcommittee on Quality Improvement Environment on the critically important issue of health care research related to quality improvement.
The day after tomorrow—after the Commission completes the final stretch of deliberations on the Consumer Bill of Rights—it will engage an equally important task. While it is certainly important that we protect consumers' access to health care, it is also important that they have access to health care that is worth having access to—high quality health care that will make a difference in their lives.
We can do a lot better for our patients. As was noted at a recent Institute of Medicine (IOM) conference, "some studies suggest that a fourth of hospital deaths may be unnecessary, one-third of some hospital procedures may expose patients to risk without improving their health, one-third of drugs prescribed may not be indicated, and one-third of abnormal lab tests have no followup." Also consider the recent work by other researchers, who have studied errors in clinical practice.
These conclusions, like that of many leaders in health care quality today, are that errors less often result from an individual making a mistake than from system-wide failures.
The improvement in health care quality will depend not only on traditional methods like excellence in education, continuing education, professional standards and principled practice behavior, as important as they are. It will also depend on our developing the science of clinical practice, on our taking a fresh look at ways of measuring and improving care, and on our holding both the practice of medicine and the practice of quality improvement to the same levels of scrutiny and evidence as we hold technical clinical innovations.
In a market-oriented health care system, we need information to inform choice at three levels—choice of plan, choice of provider, and choice of service. Without information that provides the evidence that a plan, a provider or a service will be beneficial, how can consumers or patients make informed choices? How can they make the market work for them? As Nan Hunter said at the Commission meeting last month, "Information plus choice equals power." That is the best bumper sticker for health care quality I could imagine. We do need to protect consumers' rights to access and choice, but we also need to assure that they have the information to choose wisely and that the options from which they choose provide high quality clinical care.
One of the perennial debates in this Nation has focused on the appropriate role of government in the market and in the economy. This has particularly been the case as markets and industries have gone through major changes—industry a century ago, financial markets three-quarters of a century ago, and so on. Today, as the health care industry undergoes its transformation toward more integrated systems of care and assumption of risk by provider groups and organizations, it is small wonder that we are debating the nature of that market, how consumers can use it most effectively, and the appropriate role of government in helping consumers benefit from the products and services the market has to offer.
As we think about the roles that government might play in assuring the quality of care in a market-oriented health care system, one in which information and choice are the keys to patients' access to quality health care, six roles come immediately to mind.
And it is this final role—a role that goes to the heart of what the Agency for Health Care Policy and Research (AHCPR) does—that brings me before you today.
Having spent a career in academic health centers, I joined the Clinton Administration and Donna Shalala's Department of Health and Human Services 7 months ago. My responsibility—as Administrator of the Agency for Health Care Policy and Research—is to help this relatively new agency—only 7 years old—fulfill its mission of generating and disseminating information that improves the quality of our health care system.
Another hat that I wear is that of Senior Advisor to Secretary Shalala on quality health care. AHCPR has been designated by Secretary Shalala as the Department of Health and Human Services' lead agency on quality of care. And as many of you already know, the responsibility of serving as liaison between the Department and the President's Advisory Commission is ours as well.
As the famous medical sociologist Renee Fox has written, there are two levels of uncertainty in health care—not knowing the facts about what is effective and not knowing whether anyone knows. AHCPR exists to fulfill the public responsibility of government to know—to provide the evidence that is needed about what works and doesn't work in health care practice and about how practice can be improved, resting on the evidence.
To accomplish our mission, we recognize the need to deal with the many challenges facing clinical and health services research today. Some of these challenges have perennial standing, but what is new is the rapidly changing organization of health care delivery, especially with the growth of health plans and the growth of integrated systems of delivering care.
In 1988, Andrea Kabcenell and I published an article entitled, "Organized Practice and the Quality of Medical Care" (Inquiry 1988; 25:78-89). We concluded that "the relationship between the organization of medical care and its quality transcends the question of the ownership of an organization and the distribution of its revenues. The increasingly organized nature of medical practice, whether for profit or not, offers both potential enhancements and barriers to the quality of care."
The trend toward organized practices has accelerated in the intervening decade, and the opportunity to use the changing nature of the industry to the benefit of the nation's health continues. As we wrote in 1988, "Instead of perceiving larger and more complex organizations as the destruction of high-quality medical practice, physicians can pool their professional power to ensure that the highest standards of professional responsibility are maintained." It is my belief in the opportunity that the changing nature of our industry offers for evidence-based practice, for information that guides choice, and for critical review and improvement of health care quality based on research that motivated me to accept Secretary Shalala's offer to work with her.
Even when confronting the shared goal of improving health care quality, health plans and researchers may approach the challenges differently. Plans may focus on the applied, on the immediately useful, on what translates into practice today. Researchers may seek the same, but are also tugged in other directions—to discover new insights, uncover new problems, and examine new solutions with rigorous and scientific inquiry. Each approach can contribute a great deal to the other, but the research enterprise requires certain key elements:
Together these factors make up the bricks and mortar, the infrastructure required for clinical and health services research. Without attention to each, we will not find shelter nor solution to the problems of underutilization, overutilization and inappropriate use of health care services.
In any economy there are public goods, those that anyone can enjoy whether he or she paid for them or not. One of the most important public goods in the health care economy is knowledge that emanates from scientific research, research that underpins the science of quality measurement and quality improvement. This research spans a continuum from the foundations of biologic research to clinical and health services research.
Research is empowering. Clinical research leads to better treatments. Measuring quality gives consumers and group purchasers the information required for choice. To compete on quality, health plans will want to apply the potential for improvements garnered from clinical and health services research to change practice. Put simply, clinical and health services research enables the health care market to work better.
In my view, it is the responsibility of government, in partnership with the private sector, to ensure that the science that underpins health care quality is available to those who follow us as clinicians, patients, managers and policymakers. It is particularly important to this Commission's agenda that one type of research—the design, testing and translation of measures of quality and methods of improving quality—be fostered. The improvement of quality will mature as the assessment of quality matures in a way that promotes effective, efficient, and reliable measurement, reporting, and improvement. Government can contribute to this partnership in at least five critical ways.
First, government supports and conducts basic research underpinning the science of quality measurement and quality improvement. Resting on the strong foundation of outstanding biologic research by our colleagues such as those at NIH, AHCPR supports health services research about the effectiveness and outcomes of care that serves as an essential building block for quality measurement.
Second, government can put science into practice by developing measurement tools and instruments and testing them on an ongoing basis to ensure their reliability, validity, and usefulness in improving the quality of health care services.
Third, as the work of this Commission shows, government can convene consumers, group purchasers, plans, insurers, researchers and others to work for the public good, such as the development of a core set of measures for comparing health plans. Government can ensure an open process and neutral ground.
A fourth, and unique, contribution of government is that the research, measures, and tools developed by us and our partners are in the public domain, available for all to use. By keeping measures and tools in the public domain, we raise the floor and level the playing field on which health plans, hospitals, and providers compete to improve quality. By funding research and publishing its results in the public domain, we encourage the sharing of best practices, that is, collaboration in a system of competition. By translating research into action, the products of scientific inquiry can be made available through appropriate methods of dissemination and distribution of research results.
A fifth major role of government is the implementation of quality measures within Government health programs. The Government is the largest purchaser of health care in the nation, accounting for more than 43 percent of health care dollars spent in this country, and is entrusted with the care of many of this nation's most vulnerable citizens. In the care that it purchases and the care that it provides, government can help to assure that the products of research are translated into action in the delivery of health care services.
A few examples of collaborative research activities may demonstrate ways in which this partnership can translate research into improved tools for quality improvement:
Still, with all of our efforts, we have only just begun. To build a robust research enterprise that will support continuously improving health care for Americans, we must tackle several research challenges. A short list might include, but not be limited to:
AHCPR recognizes the value of clinical trials in improving health care quality. We have the responsibility of evaluating medical technology for our colleagues at HCFA; our Evidence-based Practice Centers will provide valuable assessments of the evidence about clinical services that can improve the quality of care in areas of medical uncertainty; and we are responsible for supporting the U.S. Preventive Services Task Force. In each of these activities, we seek the best available evidence to help clinicians and patients make decisions about which services to use, when and for which patients.
We also use clinical trials methods in some of our own sponsored research. Although there are several types of research upon which we rely, we generally hold clinical trials as the highest form of evidence. In this way, clinical trials are the foundation of evidence-based practice in American medicine. The results of these research efforts have led both to breakthrough advances in diagnosis, prevention and treatment, as well as to cost-savings in the health care system.
Clinical trials do not always involve "experimental" therapy, nor is experimental therapy always studied using a clinical trial design. Clinical trials may also compare two existing, already approved treatments for relative efficacy or for their effectiveness and cost-effectiveness, and this is often the nature of the clinical research that we sponsor. We need, as a Nation, to ensure that there is adequate support for both clinical and health services research—research funded by the NIH, the Centers for Disease Control and Prevention (CDC) and AHCPR that will enhance health care quality. The activities of other Federal agencies also contribute to this knowledge base. This is an important element in the Nation's health research agenda.
But the difficult decisions for this Commission have not revolved around debates about whether clinical trials benefit society or hold promise for some individuals. The question has been: who should pay? And the answer is far from simple, even for those of us who are the strongest advocates for clinical trials as an important component of the quality improvement agenda. I will not take a position in this debate today, but I do want to urge that we clarify the terms of the discussion. I am convinced that a substantial part of what seems to be disagreement is, in fact, a difference in how the elements of the discussion are being framed.
What is a clinical trial? Typically, a clinical trial refers to a prospective, planned investigation involving a sufficient number of patients to evaluate an experimental intervention in comparison to a control and/or existing treatment(s). Usually, patients are randomized into the experimental or control arms of the study, hence the term "randomized controlled trial."
First, all trials are not equal. One might imagine, at one end of the spectrum, the sophisticated, multi-center randomized controlled trial of an important new clinical service (or several established ones) with Federal sponsorship by a research agency, reviewed and approved by a critical panel of scientific peers, approved by an Institutional Review Board (IRB) for ethical and procedural standards, conducted under the oversight of a panel for outside review of the trial's implementation, and committed to releasing its findings in a peer-reviewed journal. These are the standards used, for example, by many clinical trials sponsored by the National Cancer Institute or the Veterans Affairs Cooperative Studies Program. At the other end of the spectrum, the advocate of a controversial service might decide to collect some information to compare his patients with those treated with an alternative treatment. In between are randomized controlled trials sponsored by industry under strict guidelines for their design and conduct by the Food and Drug Administration (FDA), randomized trials that are conducted by clinicians without external support but with IRB approval, and trials that are not randomized but rather allow patients to select which arm of the study they will enter. Before deciding that entry into a trial should be afforded all patients, or that a trial's costs should always be covered, the types of trials that are being discussed should be more clearly articulated.
Similarly, the costs that are to be covered can range from minimal to monumental. In some trials, the patients are receiving standard care, but one intervention is added that will have little impact on overall costs (or could even make care less expensive). For example, pain management after surgery is unlikely to add substantial costs, and it would make little sense to deny coverage for the surgery on the grounds that the patient was in a clinical trial. In other instances, the principal intervention may be randomized, but the patient would have required care in any case, such as is often the case with the use of different combinations of chemotherapeutic agents for cancer patients. Costs might be increased, at least in the short term, by introducing a more expensive intervention, or by introducing a treatment when none would otherwise be available.
Which costs should be covered by the research sponsor and which should be covered by the party responsible for covering the costs of medical care? Almost all would agree that the research sponsor should cover the costs of research design, trial organization, randomization, data analysis, and reporting results. Most would agree that the third-party payer (the plan, the insurance company, etc.) should pay for standard care that is part of its package of covered services. But who should cover the costs of the intervention itself; the additional costs of hospitalizations, diagnostic tests or followup visits required by the protocol; the cost of complications of the experimental intervention (especially if those complications require services that would normally be covered); the costs of transportation or lodging required by the patient and family? I know personally how difficult it can be to identify and enumerate these costs, having spent several years before joining AHCPR carrying out costing and cost-effectiveness analyses of clinical trials. There is too little unbiased literature on the cost of clinical services in a clinical trial. This uncertainty clouds the debate about who should pick up the costs.
Adding to the complexity is the spectrum of possible payment arrangements. A clinical trial might be eligible for coverage at a particular hospital, at selected centers of excellence, or within a particular health delivery system. For example, through an Inter-Agency Agreement with the National Cancer Institute (NCI), the Department of Defense (DoD) offers patients the opportunity to participate in clinical trials for cancer. They can do so either in the direct care system of 120 military hospitals or, in limited circumstances, under the care of civilian providers reimbursed through TRICARE/CHAMPUS. Coverage is limited to Phase II and Phase III clinical trials for cancer. Under this arrangement, DoD pays for all medical care required as a result of participation in approved clinical trials, but NCI foots the costs of non-treatment research activities associated with participation in clinical trials (including, but not limited to "data collection activities, management and analysis of the data, salaries of the research nurses, and the cost of the investigational agents"). Similarly, the Veterans Administration (VA) has entered into an agreement with NCI. Again the VA would assume the costs of all medical care associated with the clinical trial, but the NCI would provide funding for the research costs of participation.
As these negotiated arrangements indicate, there are many components to cost sharing between the clinical trial research sponsor and the health plan or insurer. In 1996, Blue Cross and Blue Shield Association (BC/BS) established the Pediatric Cancer Network. Through centers selected on quality-based facility standards and on-site review, BC/BS beneficiaries can participate in cooperative, multi-institutional clinical trials for pediatric cancer. In the case of some medical devices, the implantation of a next generation pacemaker might be such a modest incremental change that a third-party payer might cover the investigational device itself. In still other arrangements, the health plan might pick up both the costs of research and clinical care rather than face adverse publicity, state benefit mandates for unproven treatments, or litigation.
These complexities suggest an important role for technology assessment in determining future coverage. When does experimental therapy become standard practice? One approach is to limit coverage to centers where ongoing data is collected to evaluate a service's effectiveness and complications outside a trial, such as has been done with heart transplant surgery. Another alternative, which maintains the requirement for randomization as well as evaluation, is to offer conditional coverage, such as coverage contingent on patients being entered into a clinical trial sponsored by an approved organization.
The Lung Volume Reduction Surgery trial is a case in point. Medicare beneficiaries can receive coverage for lung volume reduction surgery (LVRS)—an investigational procedure that removes damaged, non-functional lung tissue from patients in the last stages of emphysema—by entering a randomized trial. HCFA discovered that providers billed for LVRS using codes that are assigned to other types of lung surgery that are covered by the program. At the time, there was only one published article available, and it described the short-term experiences of only 20 patients. HCFA denied coverage for the procedure, but requested that AHCPR conduct a full technology assessment. Drawing upon published and unpublished data on 3,000 LVRS procedures from 27 hospital centers, the Agency found that "it could not reasonably be concluded at this time that the objective data permit a logical and scientifically defensible conclusion regarding the risks and benefits of LVRS." Based on AHCPR's findings and recommendations, the National Heart, Lung and Blood Institute of NIH in collaboration with HCFA have undertaken a multi-center, randomized clinical study of the effectiveness of LVRS. Medicare will cover the patient care costs of beneficiaries who participate in the study, and NIH will coordinate the clinical trial and pay for the research costs. In addition, AHCPR is funding a cost-effectiveness analysis of the LVRS trial.
Thus, the issue of financial support for clinical services that are provided in a clinical trial is not a simple matter. Are we discussing standard care for patients in a federally funded, peer-reviewed trial, or all clinical costs in a non-randomized comparison of two services by the advocate for one of them? We need a better nosology for the types of costs and trials that are being discussed, as well as more information about the magnitude of these costs given the different definitions that might be used.
To conclude, the President's Advisory Commission has a rare opportunity to signal the importance of clinical and health services research to improving the quality of health care in this nation. Research will enhance the quality of care that we can provide, and enable us to measure and improve the quality of care we do provide. I believe that few enterprises return so much for so little investment. But it will take just that—investment in our researchers, their institutions, and their work. As the Department of Health and Human Services' lead agency on quality, we look forward to the opportunity to respond to the recommendations that you will send to the President.
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Internet Citation:
Statement on Health Care Research and Quality Improvement. John Eisenberg, MD, Administrator, AHCPR. Before the President's Commission on Consumer Protection and Quality in the Health Care Industry, November 18, 1997. Agency for Health Care Policy and Research, Rockville, MD. http://www.ahrq.gov/news/stat1197.htm
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