The Role of Other Stakeholders in the Clinical Research Enterprise
Myrl Weinberg, C.A.E.
National Health Council
Now that this workshop has given us a better understanding of the different stakeholders’ views, which are legitimate from their perspectives and from the standpoint of their organizations’ mission and goals, it is even more imperative that we look for unique solutions that we can work together to apply. We need to think creatively about new ways to address some of the clinical research issues that have been raised.
It is important not to view the stakeholders and the patients as distinct, potentially conflicting, parties. In fact, patients are the ultimate stakeholders of clinical research. A habit ingrained in many of our infrastructures and organizations is to give patients information, and perhaps survey them once in a while, but not to enter into an interactive and respectful dialogue. All decisions about plans, structures, what is paid for and how it is paid for must involve consumers/ patients/employees from the very beginning. Simply “keeping them happy” is not appropriate because if they are not involved and educated about the nuances within health care, they may be happy with the wrong things. If we do not involve them up front, they will be involved but perhaps in ways that are not the most effective or appropriate. They may become more and more aggressive and
demanding, and the process will not be as productive as a cooperative dialogue would be.
The important role of the consumers should be borne in mind during this part of the workshop as we hear from representatives of voluntary health agencies, the device industry, and the Agency for Healthcare Research and Quality. Panelists from each organization will describe their organization’s relationship with the Clinical Research Enterprise, define the organization’s contribution to the enterprise, and discuss the organization’s needs and concerns related to the research arena.
THE ROLE OF VOLUNTARY HEALTH ASSOCIATIONS IN THE CLINICAL RESEARCH ENTERPRISE
John Stevens, M.D.
Vice President for Extramural Grants, Research Department
American Cancer Society
The American Cancer Society (ACS) has a single purpose, which is to eliminate cancer as a major health problem, and it accomplishes that goal through a variety of programs. The organization is present in 3,400 communities around the country and represents the interests of cancer patients, cancer survivors, and their families. The Clinical Research Enterprise has been critical to the progress that has been made in the cancer arena. Cancer is foremost among the health concerns of the American public, and the disease costs the nation about $180 billion a year.
From the perspective of the American Cancer Society, funding for the Clinical Research Enterprise continues to be a high priority. The clinical research enterprise also faces a barrier in dissemination. New therapies and standards of care must be disseminated throughout the health care delivery system in order for them to achieve the goal of improving care.
The Clinical Research Enterprise has been critical to the progress that has been made in the cancer arena.
In addition, many patients have limited access to the fruits of the Clinical Research Enterprise. The new therapies produced by clinical research do not reach enough patients. Barriers to receiving the high quality care that the country can produce include educational barriers, financial barriers (including inadequate insurance coverage for cutting-edge care), and barriers within the health care delivery system that may make accessing high quality care difficult. This is especially true in access among medically underserved communities and populations.
For many cancer patients, all of these barriers may apply. In addition to increased funding for the Clinical Research Enterprise, the American Cancer
Society supports the dissemination of the information learned through clinical research through public health programs, and through increased access to public and private insurance programs, increased access to high quality care at community health centers, adequate coverage to ensure quality care throughout the continuum of cancer care, increased coordination of health care delivery systems to improve dissemination of high quality care, and quality assurance for care.
Other obstacles facing the Clinical Research Enterprise include ensuring the protection and privacy of participants in research involving humans. Improving elements of the drug development process to ensure that new standards of care are available for patients as efficiently as possible is also important.
The ACS has its own research program, which ranges from basic to clinical and applied, including epidemiology, psychosocial research, behavioral research, health policy, and health services research. Advocacy for more research funding is a very important role of the ACS. The ACS has a strong advocacy effort in Washington, D.C., where it joins with other interested organizations under an umbrella called One Voice Against Cancer Together. Representatives from the organizations visit Congress to advocate for increasing the NIH budget and the budget of the National Cancer Institute. The ACS also advocates at the Centers for Disease Control (CDC) for additional funds so that the fruit of this research can be translated into community application. An example would be the breast and cervical detection cancer programs, where the CDC gives block grants to the state health departments.
Consistent with our mission, we have traditionally funded about 80% of what we call basic research in our own program. About 20% is “nonbasic” or applied, which ranges from translational research to investigation at the other end of the spectrum—cancer control, small clinical trials, prevention trials, behavioral trials, and health policy. The ACS is shifting that balance so that it will become 50/50 over the next few years, not by removing funding from basic research, but by adding more resources to the applied side.
The ACS is also very interested in educating the public, the patients, and the professionals as to what is available in state-of-the-art detection, prevention, and treatment. The organization has a 24-hour 800 number in at least two languages for providing information to patients or their families, or to whoever is interested in calling. That number receives about a million calls a year. The ACS also has its own website.
The ACS advocates for high-quality care for all cancer patients, primarily through efforts at the state and federal levels. It also advocates for coverage of the cost of the research component of clinical trials. Further, it advocates for all efforts that will result in decreasing disparities and outcomes among the various patient groups. Not everyone has equal access to health care or equal outcomes in this country. Not only are there differences in how state-of-the-art health care is practiced by different professionals in the U.S.A., but there are also huge
regional differences in how health care is administered and practiced. That issue is another area that needs special attention.
John Graham IV
Chief Executive Officer
American Diabetes Association
The mission of the American Diabetes Association is to prevent and cure diabetes and to improve the lives of all people affected by diabetes. Its vision statement is, have we made an everyday difference in the quality of life for people with diabetes? The ADA is unique among voluntary health agencies in that it is actually two organizations in one, which is a benefit. It is the professional society for the 20,000 health professionals who specialize in diabetes— everyone from PhD scientists to practicing clinicians, to nurses and dieticians, to exercise physiologists and pharmacists. It is also the advocate for the more than 16 million Americans who have diabetes. We have banished from our language the term ‘diabetic.’ We use the term ‘people with diabetes’ because we believe that people are people and not necessarily diabetics. We do not use the term ‘patient,’ because it has a victim connotation. Instead we use the term ‘people who have a chronic disorder,’ with which they struggle every day.
The ADA has three critical roles in clinical research. The first is advocacy. The ADA advocates not only for increased dollars for NIH and CDC but also for increased access to care for people with diabetes. The organization wants to ensure that quality care is reimbursed, for the provider as well as the patient. Without reimbursement, quality care does not take place.
The ADA funds clinical research in four general areas. First, the organization identifies important clinical questions, important clinical trials, and federal trials. Second, it guides prospective donors to clinically important areas relevant to their funding interests. Many people with wealth are interested in discussing where they can best channel their resources. Generally, the larger the gift, the more direction the donor would like to exercise in the use of that gift. Third, the ADA identifies qualified investigators, both established and new, and funds their work in answering important questions about diabetes. Finally, the organization monitors the progress of research on diabetes. The ADA is not the “biggest player on the block,” but it has a unique niche in helping to inspire and train young scientists and investigate areas that might not receive funding otherwise.
The ADA facilitates the translation of diabetes research into clinical practice through several vehicles. It conducts the largest meeting on diabetes in the world annually and provides a forum for the discussion of basic and clinical research. It conducts postgraduate courses for practicing clinicians in various cities throughout the year. It also holds clinical conferences on such topics as reducing cardiovascular mortality and morbidity in diabetes and understanding the effect
of diabetes on cardiovascular disease. The ADA publishes three professional journals: one in basic science, one in clinical science, and one for allied health professionals. A fourth is a consumer publication for people with diabetes that provides information about the latest advances in pharmaceuticals, treatments, and care. The publication helps them engage in a dialogue with their physician or health care provider about what these advances mean for their treatment.
Several ADA committees play a role in clinical research. A professional practice committee develops diabetes care guidelines. The ADA has about 2,000 recognized education programs around the country in diabetes—office-based, hospital-based, and plan-based. It also has a provider recognition program, which is still in the development stage. The program recognizes providers who exercise best-practice performance measures in caring for people with diabetes.
The ADA is setting up an expert committee on the diagnosis and classification of diabetes and the role that impaired glucose tolerance plays. The incidence of diabetes is rising in epidemic proportions, and impaired glucose tolerance appears to be a growing factor. The association is currently examining several questions regarding the relationship between impaired glucose tolerance and diabetes: At what point do we diagnose diabetes? At what point do we diagnose impaired glucose tolerance? Is impaired glucose tolerance a separate disease? The answers to these questions have significant ramifications, such as potentially changing the number of persons with diabetes from about 16 to 20 million to 40 to 45 million. The association also prepares many clinical guidelines and position papers, such as the ADA clinical practice guidelines, which are used internationally. It helps support screening for type 2 diabetes and is preparing to release evidence-based principles and recommendations on nutrition. So the association plays a significant role in the Clinical Research Enterprise.
Needs from the perspective of the ADA include several issues. First, the business case needs to be made that prevention is important, but the cost benefit needs to be proven. It is commonly known that the onset of diabetes, particularly type 2, can be delayed, if not prevented, by the practice of appropriate health behaviors. Second, the business case needs to be made that diabetes care makes sense, i.e., that good-quality care will reduce future costs, not only for the private payer system but also down the line for the Medicare system. Third, there is a crying need for behavioral and outcomes research. If we could learn what behavioral interventions cause people to modify their behavior, we could make tremendous strides in treatment. Fourth, we need to know how to reach effectively those populations that are dispropor
The business case needs to be made that prevention is important . . . that good-quality care will reduce future costs, not only for the private payer system but also down the line for the Medicare system.
—John Graham IV
tionately afflicted with diabetes. Investigating this question requires a great deal of money, and that investment has not been forthcoming.
Finally, to put forth a more provocative viewpoint, we are all participants in a capitalistic society. It is interesting that we do not provide incentives for people to take better care of themselves. The health benefits that employers pay are simply an extension of the compensation plan. We give incentives for making more “widgets” and for selling more products. In the workplace we give incentives for people to become more efficient but not for people to take better care of themselves. Why don’t we? Why not give people tax credits for taking better care of themselves? People receive tax credits for child care. Companies receive tax credits for investing in research and development. Why not give people tax credits for taking care of their health? The long-term payoff might be a significant step forward.
PRIORITY SETTING IN BASIC AND CLINICAL RESEARCH
Robert Califf, M.D.
Division of Cardiology, Department of Medicine
It is remarkable how unified the key messages were in the first part of this workshop. For all the doom and gloom projected, we need to consider that people are actually living longer and feeling better than ever. The rate of improvement, not just in survival but in disability-free survival, is growing at nearly an exponential rate in this country. Although we have identified the key diseases and causes of disability, in terms of morbidity and mortality, to be dealt with, we still have concern that the efforts of the research community are misaligned relative to the priorities of purchasers and payers.
This morning’s panelists brought up the need for comparative evaluative research. Almost none of that type of research is being conducted. Why is this so, and what can be done about it? It should be remembered that the randomized clinical trial is only about 50 years old. Today’s technology, with its history of only a decade or so, has made randomized clinical trials possible in a way that did not exist in earlier decades. Randomization of large numbers of subjects, and collection of pertinent data, is simple now. With technology no longer a limitation, the research structure has to catch up.
Two research models exist for conducting research: the new and the old. The old model is the so-called basic research model. It is hypothesis-driven and investigator-driven. It involves people chasing ideas in a “selfish” way, which is a good thing for basic research and is the only way to do it The largest pharmaceutical companies in the world, which invest $4 billion or $5 billion in research, are realizing that it does not work well to simply tell scientists: “We will put you at a desk and your work will be to discover a treatment for diseases A, B, and C.”
Basic science works by people pursuing their curiosity. Under these conditions all kinds of interesting things happen.
Evaluative research depends on a good structure, and the research priorities should be set . . . by those who understand the health care priorities.
The new model is evaluative research, which is entirely different. Evaluative research depends on a good structure, and the research priorities should not be set by individuals but by those who understand the health care priorities. When we confuse one model with the other, we end up with a mess on both sides. Telling basic scientists how to do their research does not work well. Turning evaluative research loose to hypothesis-driven mechanistic research produces research that is not aligned with our priorities for health care. One nuance is that research on the methodology of conducting evaluative research is basic research and should be hypothesis-driven. This type of research appears to be completely unfunded in this country.
A reason that not many researchers are conducting evaluative research is that there is great difficulty sustaining a career in it. There is no sustained funding for this kind of work. Everyone shares in the blame for the deficiencies in our research structure. Those who train practitioners—medical schools, nursing schools—are way behind in research training. Medical students and nursing students are not being trained in how to function in a collaborative, evidence-driven environment. Medical students still spend two years in basic sciences and two years in clinical apprenticeships. If you make rounds today in most of our medical schools and ask why a certain treatment is being used, you will hear a mechanistic answer, despite the fact that we do not know the exact mechanism by which most of the highly effective treatments work.
The last thing that medical products companies want to do is comparative research. At the last count, there were 80,000 sales representatives in the U.S.A. Choosing to turn to a sales force rather than putting a product to the test is quite reasonable from a business perspective. It is just not necessarily good for the public health. One point regarding government: the government has a good policy on clinical trials coverage, but that policy is not being enforced, and the elderly are having trouble participating in clinical trials as a consequence. This problem is solvable, and the government needs to step in.
The role of the press needs to be part of the research agenda in this country. The power of the press is enormous today, and people in the research community are afraid to deal with the press. They are afraid to study the actions of the press for fear that criticism may make the press turn on them. The least of the problem is the patients. We have no trouble getting people to volunteer for clinical trials. Nearly all people who participate in clinical trials are delighted to be part of the studies, and their feedback is almost uniformly positive.
In conclusion, we have a fragmented model for the kind of research being
considered today, and it needs to be put together. If the NIH, with all its resources, channeled money into an infrastructure that could leverage the participation of the payers and the medical products companies and identify top research priorities, comparative evaluative clinical research could be performed. When it is performed for reasons that are forced, such as comparing TPA with streptokinase or other easily citable models, it is effective. If we spend some money building an infrastructure within the current funding limits, and we create a model where people work together—not through hypothesis-driven models, but by identifying priorities and then putting the infrastructure to work (the NCI is close but has not yet reached this point)—we can do much to solve the problems that have been brought up today.
PRIORITY SETTING IN HEALTH SERVICES RESEARCH
Dennis Scanlon, Ph.D.
Associate Professor, Health Policy and Administration
Pennsylvania State University
Two recent reports issued by the Institute of Medicine’s Committee on Quality of Health Care in America, To Err Is Human: Building a Safer Health Care System (2001) and Crossing the Quality Chasm: A New Health System for the 21st Century (2001), suggest some serious problems in quality in health care in the U.S.A. The U.S. health care system might harm patients, or even kill patients, and is not living up to its potential. This realization comes at a time when health care premiums for employers and purchasers are predicted to rise by about 13% in the next year, and there is no downward trend in sight1.
The U.S. health care system might harm patients, or even kill patients, and is not living up to its potential.
To illustrate the magnitude of those concerns, a report in an unpublished study by the Midwest Business Group on Health and the Juran Institute estimated that the direct cost of poor quality and medical errors is $1,800 per employee per year, while the indirect costs, which include lost work days and productivity, are $500 per year for a worker with an average salary of about $32,000.2 These costs create a serious situation for those who purchase care and those who provide care. We must ask ourselves, why is there poor quality? The Institute of
Medicine reports provide insight, stating that many barriers are not the result of the incompetence of providers or inferior technical skills, but are due instead to lack of well-integrated and coordinated systems and processes for delivering health care to patients.
We still live in a paper world in health care. That reality creates the probability of errors, problems, and diminished continuity and coordination of care, which translate into poorer quality. The increasing rate of scientific development makes the cognitive decision-making task of providers much more difficult than years ago. Tools may exist for assisting providers in making decisions and helping patients make decisions that meet their preferences. However, our understanding of the impact of various interventions for coordinating patient care, improving quality, and eliminating waste and medical errors is still in its infancy. The evaluation of various interventions toward these ends, including alternative organizational and financing arrangements, should be a high priority for the Clinical Research Enterprise. Purchasers, payers, and researchers all have a role in this endeavor. In many cases the effort involves creating systems and changing culture. The irony is that at the same time that we consider these factors as potential solutions, the health insurance market is moving in the opposite direction, away from systems and away from creating organizational culture.
We still live in a paper world in health care. That reality creates the probability of errors, problems, and diminished continuity and coordination of care, which translate into poorer quality.
The fastest growing form of health insurance is the preferred provider organizations (PPO). There is a move toward having employers and purchasers more or less absolve themselves from purchasing decisions and shifting these decisions to the individual consumer level. That situation is somewhat ironic. In considering additional areas for research, we see clearly that the goal from a purchaser perspective and an employer perspective is to focus on cost, health outcomes, satisfaction, and labor market outcomes. Some of those factors are easier to measure than others. For example, costs are no doubt easier to measure than health outcomes, including health status, morbidity, and mortality. The labor market outcomes such as workplace productivity are very difficult to measure. A trend for health care purchasers is the use of so-called “value-based purchasing,” where they are involved in either working with or—as some might describe it—working against plans or providers in some cases to help drive the quality improvement process.
We need to understand whether partnerships between plans and purchasers, or between providers and purchasers, might lead to quality improvement. An example comes from some data presented by Dr. Ron Kessler of Harvard University in a recent paper. Dr. Kessler’s study examined the prevalence of chronic condi
tions in employees and the relationship between these conditions and work impairment and disability days. This research is important because purchasers and employers are particularly interested in workplace productivity. Indeed, there might a place for research that attempts to link health status with productivity and to understand the relationship between the two. Programs might be developed that would help to improve not only health status but also workplace productivity.
Researchers can assist the progress of the Clinical Research Enterprise by working with purchasers and health plans to evaluate the impact of activities, collaborative programs, and interventions.
As the recipients of health care dollars, health care providers—including health plans, hospitals, and physicians— play a prominent role in the allocation of health care resources and the quality of care received for those dollars. Research is needed on how providers can partner with purchasers to achieve creatively the objectives outlined in Dr. Kessler’s research. Improving quality of care requires measurement, action, improvement, and remeasurement. This concept is demonstrated by Don Berwick’s work in the Institute for Health Care. To improve quality, we need to educate providers and organizations, and we need to develop systems for engaging in measurement, acting on that measurement, and translating action into improvement.3,4 What is the role of researchers in this process? Researchers can assist the progress of the Clinical Research Enterprise by working with purchasers and health plans to evaluate the impact of activities, collaborative programs, and interventions. Not only do we need traditional researchers with experience in conducting clinical trials, but we also need health services and social science researchers for this effort.
Many programs that require evaluation are in real-world settings, for example, as part of employment-based benefit programs or government-sponsored health insurance. In these cases, analytic techniques may be needed to account for nonrandom sample selection. Unlike typical patient trials, many of the creative interventions must involve changes in organizational structure and the use of contractual incentives, requiring the expertise of social science-based research. Specific examples of research suggestions include the following:.
Studies that demonstrate effective techniques for improved quality and value, including the reduction of waste and medical errors
Studies that evaluate the potential synergy between improved health sta
tus and workplace productivity, including creative interventions for achieving these goals
Studies that demonstrate the cost-effectiveness or return-on-investment of population health interventions, including comprehensive disease management programs and that identify effective incentives for encouraging and differentially compensating high-quality care
Studies that view employees and dependents of employer-based purchasers as populations to be studied over time
Research that focuses on medical education training, including studies that evaluate or design mechanisms for training physicians and other clinicians to practice evidence-based medicine and to evaluate not just the effectiveness but also the cost effectiveness of treatments, and also the ability to synthesize scientific findings and incorporate their meaning into practice.
Lawrence E. Shulman, M.D., Ph.D.
National Institutes of Health
Selecting research priorities presents an enormous challenge and raises important questions. The first is, who will determine these priorities? Just the “selfish” applicant, as Dr. Califf said, or participants in consensus conferences? A second set of questions involves the burden of disease. What aspect is most important —mortality or morbidity? Which diseases are most important? Do we choose to study cancer over arthritis or aging or whatever? We have to make those particular choices. A third issue is the huge amount of data that are needed for setting public policy, for administration, and for other types of decision making. A fourth challenge is setting priorities according to the type of clinical research to be conducted. The definition of clinical research worked out at the Graylyn summit exercise (AAMC, 1999), for example, has nine different categories. We need to select priorities not only among these categories but also within each one, and this task poses difficulties. How do we choose, for instance, between therapy on the one hand and prevention on the other? Or between translational research and health services research?
Taking the broadened outlook of one who was more or less freed by semi-retirement, Dr. Shulman stated that all these things are good. He noted, for example, that all nine categories were mentioned in the talk on combating diabetes.
HEALTH SERVICES RESEARCH IN VOLUNTARY HEALTH ASSOCIATIONS
Mary Woolley of Research!America began a discussion of the health services research conducted by voluntary health associations by responding to the list of needs presented by John Graham of the ADA, which largely involve perform
ing more behavioral and outcomes research. She asked if she was correct in assuming that, in the ADA’s venture capital mode of funding research, the association addressed some of the needs that John Graham laid out. She suggested that this approach could be promoted more vigorously so that even more dollars flow into those important areas.
Enriqueta Bond of the Burroughs-Wellcome Fund asked whether the voluntary organizations are also viewing the emerging area of health services research as a major need for advancing the management of particular chronic diseases, and whether they are also advocating for dollars in this area. Has this area been a focus in the past? Might it be a larger one in the future?
John Graham replied that the ADA has become involved in outcomes research and has found some synergistic relationships. For example, the association is working with Pacificare in examining the effect of the presence of a nurse case manager in a wide diabetes practice on outcomes for people with diabetes in that practice. Outcomes research is very expensive and also very long term. It is not as glamorous as basic science. Providing a critical mass of funding that will attract researchers who will answer important questions about behavior and outcomes should considerably enhance the treatment of people with chronic disease.
Providing a critical mass of funding that will attract researchers who will answer important questions about behavior and outcome should considerably enhance the treatment of people with chronic disease.
—John Graham IV
John Graham said that the ADA has a very simple message and admitted that the association has not perfected it and could use help in getting it out. Myron Genel of Yale University suggested that the message should be simply that we need to find out what works, because that is the only way people understand health services research. He also reiterated that truly good evaluative science is expensive and requires long-term research. He lamented that as a nation we are not providing anywhere near the money needed for this type of research.
Outcomes and health services research is also an area of focus for the American Cancer Society, stated John Stevens of the ACS. He noted that the society is shifting its portfolio so that it devotes 50% of its research funding to basic research, because understanding the fundamentals of most diseases is the key to dealing with them in the long run. The other 50% is for applied research, which ranges from pre-clinical or translational research to psychosocial, behavioral, prevention, cancer control, community projects, health services, and outcomes.
An important question for the ACS is, how do you change the behavior of health care providers? Historically, one means of change has been patients’ demand for a particular type of care from their physicians. Patient advocacy is one reason that mammography and the pap smear have become widespread in most segments of the population in this country. These examples demonstrate the
importance of patient education in driving behavioral change in health care providers. Pharmaceutical companies are very successful at this approach through television advertising. Advertisements appear for every new product, and soon thereafter the physicians begin writing prescriptions. Either the physicians are influenced directly by the advertisements, or the patients ask for the products.
Another issue brought out by John Stevens is reimbursement of the health care provider’s time for the efforts taken to provide behavioral or preventive messages. In some cases specific reimbursement is not provided for counseling against a negative health behavior. The problem then is, why would physicians spend much time providing that counseling if they know they will not be reimbursed? Unless there is reimbursement, it is very difficult to implement a procedure.
THE ROLE OF THE DEVICE INDUSTRY IN THE CLINICAL RESEARCH ENTERPRISE
Susan Foote, J.D.
Medical Technology Leadership Forum
The Medical Technology Leadership Forum is a medical technology think tank that brings together a broad range of representatives from what is called the medical technology community, which includes physician organizations, university and research centers, health plans, device firms, bioengineering organizations, and patient groups. While the leaders from the various member organizations may have different perspectives and different incentives, they share a common goal—to contribute to public policy solutions to issues of concern to the medical technology community. This model is commendable because bringing together people who are well-meaning and involved in parts of a particular problem leads to creative thinking.
It is critical that the other participants in the Clinical Research Enterprise understand and appreciate the unique characteristics of the medical device industry and the innovative products that the industry produces. The notion that there is a distinction between engineered technologies, drug technologies, and procedures is becoming blurred in light of the innovations in genomics and new biology and in information technology applications for therapies. The device industry is a completely different industry than it was just 5 or 10 years ago.
The cost of investing in clinical trials is an important issue with many confounding viewpoints. On one hand, it is argued that the device industry should pay for the research because device firms recover the value of the investment in clinical trials when they sell the products. On the other hand, many on the side of the device industry argue that the differences between drugs and devices need to be considered:
Devices are more dependent on physician/operator skills than most drugs.
Innovation in devices is highly iterative, with accumulation of smaller innovations as distinct from unique chemical compounds.
Product life cycles can be very short (18 months), and patents often confer little protection from competition.
Clinical trial costs vary, and can be very high for some devices being studied (e.g., implanted defibrillator, artificial organ), and may cost many thousands per unit as opposed to the cost of one pill.
Although there are many large device firms (e.g., Medtronic, St. Jude Medical, Siemens, General Electric), 80% of the firms are very small.
If a drug or device company invests in clinical trials to obtain evidence for either the payers or the FDA, others tend to discredit those trials just because they are industry-funded.
A Catch-22 in funding is an important issue in industry’s investing in clinical trials. If a drug or device company invests in clinical trials to obtain evidence for either the payers or the FDA, others tend to discredit those trials just because they are industry-funded. The incentive for industry to fund the trials is to obtain data so that the approval required to sell the product will be forthcoming. The catch is, if those studies are biased, who will fund the studies and provide the information? Why can we not provide the right incentives, or design the right forums, so that industry performs studies that meet the test? Millions, maybe billions, of dollars are invested in trials sponsored by the product producers. If that money is being wasted –that is, if many of the studies are flawed—we have a problem, and it does not involve more resources. Instead, it involves trying to deploy our resources in a more constructive way in order to get value for that investment.
Other issues are timing-based. We are challenged on the device side because of the incremental nature of engineered innovations; i.e., the product life cycle of many innovations is very short and getting shorter. The short product life cycle of our own computers, for instance, helps us understand the problem facing the device industry. Considering the rapid advancement in technology, how long can you wait, or should you wait, for the trials, data, and development that are generated on a much slower time schedule than the innovation cycle of 12 to 15 months for a product?
There are structural issues, too, such as concern about conflict of interest. As the device industry and the drug industry have worked more closely with universities, there is interest from the private sector and government to redesign the conflict-of-interest rules. The Medical Technology Leadership Forum is not clear yet about how those rules should evolve. Nevertheless, the rules could pose an enormous barrier unless they are carefully drawn.
Issues of evaluation tie in to the costs of clinical trials and their credibility. What criteria do that the payers want in order to evaluate a new technology?
What standards of evidence are appropriate, and what does it mean to evaluate a study? It is difficult to have a coherent context in which to review the benefits of a new technology when the standards of evaluation are evolving as rapidly as the technology. The industry itself, in the aggregate, has some responsibility for the problems in the environment of evaluation. The politics of Medicare coverage have been intense for more than 25 years. Speaking from a medical technology perspective, we do not have a clear sense of what standards must be met. In the absence of clear standards, well-meaning people will invest a great deal of money to obtain a great deal of data that will not be well received, and none of us is well served by that situation.
THE ROLE OF THE AGENCY FOR HEALTHCARE RESEARCH AND QUALITY IN THE CLINICAL RESEARCH ENTERPRISE
Francis Chesley, M.D.
Office of Research, Review, Education and Policy
Agency for Healthcare Research and Quality
The mission of the Agency for Healthcare Research and Quality (AHRQ) is to support and conduct research that will improve health outcomes, quality of care, and cost and utilization of health care services. Along the spectrum of clinical research, the AHRQ is a federal funder of health services research. We heard earlier today that it would be valuable, across the funders of clinical research, to have a reasoned approach to setting priorities in clinical research. At AHRQ, we believe that two reports from the IOM—To Err is Human: Building a Safer Health Care System (2000) and Crossing the Quality Chasm: A New Health System for the 21st Century (2001)—point us in important directions, in terms of focusing clinical research on the issue of patient safety and looking at the system of care in the U.S.A. in an empirical way. The reports also force us to think about improvements in the system of care so that we deliver the best care possible to the most people.
We believe that two reports from the IOM—To Err is Human: Building a Safer Health Care System (2000) and Crossing the Quality Chasm: A New Health System for the 21st Century (2001)—point us in important directions.
Translating research into practice is a major priority for the agency. We also need a smart and capable cadre of researchers to conduct the research that we are talking about today. We see as very important the continued funding of not just the clinical research at large, but also the research produced by the next generation of researchers—clinical health services researchers and epidemiologists. AHRQ funds research and also conducts research. An important role we play is
that of brokers—brokers of collaborations and partnerships, both public and private. The Evidence-based Practice Centers are one example. The National Guideline Clearinghouse, where we work with the American Medical Association and the American Association of Health Plans, is another. The issue of many clinical guidelines on the same topic is what drove the agency to seek out and work with partners.
An important role we play is that of brokers—brokers of collaborations and partnerships, both public and private.
We focused some of our research on the basis of the IOM report To Err is Human: Building a Safer Health System (2000). Last year, patient safety was a major focus for the agency. Researchers across the country received $50 million to examine and address issues related to patient safety. Perhaps most importantly, we looked to fund Centers of Excellence. The CERTs program is an example of a Center of Excellence that examines therapeutic agents. We have funded Centers of Excellence in patient safety and in training. We fund practice-based research networks in which nursing and physician networks actually do research with participants in clinical practice settings. It is that kind of research which will translate more broadly. We also focus on issues of health care disparity in terms of both outcome and delivery of care. The researchers are probably not the best ones to disseminate the results of that research. They probably do not receive funding for dissemination, and it may not be a priority for them. We do know, from research, what kind of mechanisms work best for disseminating information. We know from the pharmaceutical industry that there are effective ways to disseminate information to practitioners. It is fair to say that these methods do work in certain settings. Learning from that example, and figuring out how to be smart about disseminating information to those who need it —individuals as well as systems— is a need that might emerge from our introspection into the Clinical Research Enterprise.
TRANSLATIONAL BLOCKS AND PRACTICE GUIDELINES
Participants launched into a discussion of practice guidelines and their role in translating clinical research into practice. Elaine Larson of Columbia University School of Nursing started the discussion by emphasizing the importance of clinical practice guidelines as a mechanism or model for translating basic, and then applied, research into clinical practice. The National Guideline Clearinghouse has well over 1,000 clinical practice guidelines. For seven years, Larson has chaired the committee for CDC that
There is very little external validity, i.e., little evidence as to how well these guidelines will work when widely applied nationally or globally.
writes the practice guidelines for infection prevention in health care facilities in the U.S.A. The committee has struggled during the entire seven years with how to assess the impact of the guidelines. Although the guidelines are very rigorously written and are based on randomized clinical trials and good epidemiological data, there is very little external validity, i.e., little evidence as to how well these guidelines will work when widely applied nationally or globally. The committee will soon issue a new guideline that will revolutionize certain aspects of infection control. A tremendous cultural change and much systems work will be needed to change practice, attitudes, and values. Elaine Larson warned that we are not prepared for that kind of research. We are prepared in terms of how the research should be done but not in terms of how to get people to change.
When we talk to institutions about partnering with us in assessing impact, we are told that they have only a small role in that endeavor. To reiterate a question asked earlier, how do we transform the research and professional culture into a team sport? Those of us in the Clinical Research Roundtable need to get beyond the rhetoric about being a team and take action to determine what kinds of efforts, recommendations, practices, and perhaps research at the systems level are needed to bring about that team culture.
Robert Califf of Duke University mentioned that every practitioner knows that the current guidelines apply to only a small part of his or her everyday professional life. In cardiology we have solid evidence for a few factors that affect major outcomes. There is a very nice correlation between mortality rates and guideline compliance. The most important elements of the guidelines could be pared down to perhaps 10 processes of care for which there is good evidence of a relationship between process and outcome. Actually, probably fewer than 10 are broadly applicable to almost everything we do.
John Graham of the ADA noted that attaining the culture of a team sport is very difficult and requires a paradigm shift. He pointed out that certain disorders and diseases need to be treated in an acute manner that lends itself to the “cowboy mentality,” and that this mentality might even be preferred in treating those diseases. Other, more chronic, diseases such as arthritis and diabetes lend themselves to ongoing management. Although not every individual responds in the same way to care, in diabetes care following certain performance measures leads to certain outcomes. Health systems and employers need to be willing to pay the costs and should require the implementation and enforcement of that kind of system.
We have not figured out, however, how to measure the impact of the reports and other documents on clinical care.
Francis Chesley of the AHRQ stated that the AHRQ has evolved away from traditional guidelines and toward evidence reports, in the scheme of what is actually an evidence-based practice approach to clinical medicine. AHRQ will not do research unless it has a partner, public or private, to address three or four
key questions that will inform their clinical practice or their approach to clinical practice. If there is no evidence to answer those questions (and often there is not), the process is stopped. The evidence report is not a clinical practice guideline; instead, it represents a start for an organization or a group of practitioners who are federal partners, who then take the answers to those questions and move that knowledge into their practice environment. At AHRQ we have learned that if the creation of the document does not involve persons who are likely to use it once completed, there is less likelihood that the document will have any real impact. We have not figured out, however, how to measure the impact of the reports and other documents on clinical care. One focus for the agency this year will be to fund research that examines how to translate research into practice and measure the impact of that work.
The goal of the session on the role of other stakeholders in the Clinical Research Enterprise was to examine how voluntary health associations, academic institutions, research organizations, and the medical device industry contribute to the Clinical Research Enterprise and what they need from the enterprise to better promote health and health care (see box).
Representatives from voluntary health organizations (the American Cancer Society and the American Diabetes Association) stated the mission of their organizations, described their research and advocacy efforts, and discussed their educational programs for professionals, patients, and the public. Representatives from academic institutions (Duke University and Pennsylvania State University) spoke about the emergence of evaluative research, which moves away from individual, hypothesis-driven basic research toward collaborative research with a social science base. A former Institute director from the National Institutes of Health pointed out the challenges involved in setting research priorities nationally. A representative from the Medical Technology Leadership Forum emphasized the unique characteristics of the medical device industry and the challenges the industry faces in funding research, obtaining timely research results, and evaluating those results. A representative from the Agency for Healthcare Research and Quality described the mission of the agency and spoke about its role as a broker of public and private collaborations and partnerships. Finally, participants discussed the need for a paradigm shift that would transform the current research and professional culture into a team effort.
Highlights of Session on the Role of Other Stakeholders in the Clinical Research Enterprise
What can voluntary health associations, academic institutions, research or ganizations, and the medical device industry contribute to the Clinical Re search Enterprise?
What do these organizations need from the Clinical Research Enterprise to better promote health and health care?