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IlIE: NEEI) FOR CLINICAL INVESlIG~ION TO CONSIDER THE ISSUES OF aF~tE ASSAM John E. mug me Ordinary progress ~ the ad~t of basic biologic Irk is a proud a~li~nt, perhaps Me At distinguished of our era. me exertion of ~i~ of me nab of human biology arm the Onion of disease, and the Assyrian growth of a ~aaDar t ~ ologic ca ~ city to antenrene in biologic pr ~ , has fostered the rapid proliferation of treatment theories on how to improve patient well-being. But, in contrast to the well established policies and mechanisms for promoting biomedical science, support for the evaluative clinical sciences the measurement sciences used to test the validity of clinical Theories concerning the prevention, diagnosis, and treatment of disease--has been inconsistent and unsystematic. With the exception of the evaluation of new drugs, there is no mechanism in place to assure that major clinical theories are systematically evaluated. The nature of innovation in The pharmaceutical industry tw here research proceeds in a reasonably orderly fashion Frau the bench to human experimentation), lends itself to regulatory approaches to est~hlishinq proof of efficacy. _ ~ ~ ~ _ The regulatory legislation of the 1960's was followed by a massive investment by the drug industry in the evaluation of new drugs, an investment that is recovered from patient dollars through the price of drugs. by contrast, the investigation of efficacy for other treatments where regulatory requirements have not been imposed is unsystematic, and that which is undertaken is largely on The basis of investigator-initiated grants with funds coming fern general revenue tax dollars. The amount of money devoted to randomized clinical trials for drugs--estimat^d at about $1.5 billion in 1987--oontrasts sharply to the amount allocated to all other types of treatment theories, and exemplify the current imbalance in investment in outcomes research. For example, the outcome research program of the National Center for Health Services Research and Health Care Technology Assess meat disco below, received an appropriation of only $6 million dollars in FY 1989. The immediate consequence is an increasingly inadequate scientific basis for clinical decision-making. The basic prc~abilities for The outcomes of specific treatments are often unknown or in contrctversy and patients and physicians face urrYcessary uncertainty in choosing among alternative treatments. The last few years have seen the rapid proliferation of medi~1 theories concerning the best way to treat chest pain caused by impeded blood flow in the artery that feeds the muscle of The heart. Some physicians recommend surgery~the well-known coronary bypass operation. Others recommend coronary artery 82

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ar~ipplas~the insertion of a Balloon cashew' And the heart's art~y, With is then eyed to rue the correction. Still others new dry treatment. Day; abaft me relative value of these different Days rage In clinical medicine, but becalm - the at are not ~yst~ti~ally Dry, the Sty caulk be sexy. mere are new ways to treat arthritis of the hip arm knee. C,ne approach involves the surging relay: of the hip or knee joint, and for physicians and patios Do choose this method there are a number of alternative ways of ac~npli~hing the replan. mere are many Choices, but no consensus on which approach is best for the patient. mere is also considerable disagreement about when, An the natural history of the Hinge, the operation should be planned, if at all. men- differenoes On op ~ on translate into c06~1y differences in the ramp at which various services are performed in different parts of the country. m e evaluation of the outcomes of clinically different approaches to treating common medical conditions such as back pain, pneumonia and gastro~nt=~tinal disease, is Perhaps the most neglected area of all. In many . _ _ communities in this Scantly, physicians Favor One Outpatient setting for treating Chew- patients, while in other cc~munities, the Standard of practice favor the use of hospitals. Similar uncertainties an] controversies about correct practice exist concerning the value of intensive care units. The secondary consequences are large variations in combs and utilization among apparently similar populations treated by different (but equally qualified) physicians. For example, per capita expenditures under the Medicare program for residents of Boston were about 80 percent higher than for residents of New Haven. Hider rates of hospital izations for chronic Melissa Envisions accounts for Huh of ye differences in e~rxliture. As variations such as these became Ore widely known outside of the profession, they are a scarce of growing m bares ~ ant to Physicians and an invitation for knot containment agencies to intervene directly in the practice of medicine. m e need is for a broad extension of the mandate to evaluate medic interventions to include the full spectrum of relevant treatment, clinical theory and outcomes. ISe mandate must be expanded to include diagnostic and therapeutic procedures an] established drugs used in novel ways, as well as the use of hospitals and intensive care units compared to their 83

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alternatives. In addition to traditional ~ of r~rtali=, morbidity and Physiologic or biochemical parameters; of cx*c~ne, good ~ ~~ ~ good ~ of ~t~ arx} how treatment affects Sheen. It also d~s on functional seaIs ark ~ali~ of life ~ . = ^~- ~ are ~ irk in ~ asingly important as, more and more, The abate objectives of many med;~1 interventions is the reduction of symptoms and improvement in well-being. A well conceived cutccmes research program will also take advantage of newer methods for evaluating symptoms and functional sbatt~C; use large claims dat~hacPc for establishing the probabilities for outcomes; use decision analysis to test med;~1 theory as part of the assessments undertaken to esbablis;h the scientific and ethical feasibility of conducting clinical trials. It will also invest in the development of new methods for evaluating outcomes, particularly for ~~.c~ in non-experimenta~ situations, such as the comparison of death rages among hospitals or treatments. For these comparisons, well tester methods for adjusting severity are needed. For epidemiologists and clinicians to have confidence in this research, it must be formally peer reviewed and be part of public sector science. It will also invest in research into ways for improving drug evaluation strategies, particularly methods for comparing drugs to treatments, such as surgery where the chic double blinded randomized clinical trial are difficult and sometimes unethical to conduct. Extending the mandate to evaluate efficacy and effectiveness presents a challenge to res arch scientists, funding agencies, and policy makers. In contrast to the situation for new drugs, other treatment innovations often arise within the context of everyday practice as part of the problem solving process. The uses now made of hospit=1s and ICUs, as well as many surgical operations are good examples. It would not be easy to subject such practice conventions to formal regulation, simply because there is no clear distinction between "regular" practice and innovation. Moreover, because the innovators often are individuals or members of small groups, and Their prc~ucts are not generally patentable, they have little capital an] little incentive to invest their own rescurocs in outcomes evaluation. The result of a serious effort to regulate innovation would ~lmn=~ ~-rtA;nlv he She stifling of innovation. The failure of the current investigator-initiated grant mechanism to meet the challenge of outcome assessment suggests, however, that a new approach is needed. The National Center for Health Services Research is initiating a program which provides one model of how a research program might be built. me program uses the extramural grant mechanism to fund assessment teams that accept responsibility for evaluating alternative treatment Theories for specific common dismay= conditions (for example, prostatism or stable angina). The interdisciplinary teams, coupes l of clinical investigators, epidemiologists, practicing physicians, and others with relevant skills in the evaluative clinical sciences, are 84

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radial by the tens; of they grant award to keep track of treab~t innovations In heir area of r~ponsib;1i~r are undertake for all treatises the equivalent of phase I are phase II aunts raw ruin for urns. n~ on Heir ants, the ~ are to identify priority clinical trials (phase III studies) that are need, making their reccmmendations on a periodic basis. They are also responsible for conducting phase IV studies. Funding for the program is frees patient dollars- Frau the Medicare trust fund. Priorities are according to common conditions for which at least one current treatment arm poc=-=c particularly high casts and/or risks for Medicare enrollees. About 15 teams will be necked to cover the majority of surgery and medical hospitalizations. Table 1 gives an example of the priority areas under consideration for this program. The program provides a non-regulatory model for assuring that systematic e~videno. of efficacy an] effectiveness is developed. How the information on efficacy and effectiveness is used will depend on other mechanisms, agencies or incentives. The goal of outcomes research is better clinical science--to establish the facts and t=~t theory not to mate specific regulatory determinations. Many sources of funding and various research programs contribute to outcomes research. In FY 1987, the National Institutes of Health spent about $420 million on clinical trials. The Food and Drug Administration and the drug industry contribute at least $1.5 billion to ass essrents. But the lack of regulatory mandate means that many important priorities--such as those listen in Table 1, are left unattended. The peculiar need for a public program responsible for the systematic rationalization of The scientific basis of clinical theory suggest the need to designate a single agency with responsibility for setting the baseline outcomes research agenda for assuring that systematic attention is given to The important unanswered scientific questions, particularly for 'big ticket diseases." Evaluations under this agency must be done in a teeny and ongoing way. The National Center for Health Services Rr search and Health Care Technology Assessment is a good candidate. It is situated within the orbit of government-sponsored, peer-reviewed science, and historically this agency has been responsible for much of the methodologic research that now makes a concerted effort to evaluate outcomes possible. Its study section members represent the various disciplines that constitute the evaluative clinical sciences. One outstanding problem is that the level of expectation and acknowledgment of mission needed from policy makers for NC9SR to accomplish this mandate is insufficient. The current level of fin~;n~ a:; mill ion in REV lOS2c~ ;c Iran ^ ~ ~ A- ---- ~4 ^- --a- -a ~~- The budget for an acceptable program is $200 million. Achieving consensus that NC9SR can accomplish this mission requires assurances that it can grow rapidly as a credible scientific institution. The National Institutes of Health has the rescuers and the experience to assure the rapid and orderly growth of an agency with this mission. At the same time, the agency 85

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must rain responsive to the As of patients and policy male; for _ that a = at priorities are At and that the core group of scientists Hose ~ are situated In the evaluative clinical sciences are mobilized. It Is bone to consider carefully the option of Are the National Cater it the Non. 86

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Surest Eli Conditions or I1~ for Erase I art I] As~nts leer Me National Cents for Him Senric~es ~3~ are H~th Core T~lagy As~nt's Patient Are P~r~ E0gram (S. 2181) Corx~ition Stable Angina Unstable Angina Arteriosclen~ Causing Sake P~i~ibem1 Vascular Disease lens Exaction ~1 Stories Arthritis of the Hip are Knee None C;onditions of the Uterus Prostatism Air, Nose & throat editions Herniated Disc Acute and Chronic M~di~1 Conditions: Treatment Controversies Eypass Surgery vs Angiqplasty vs Drugs Eypass Surgery vs Angiqplasty vs Drugs Endarterectomy vs Drugs Eypass Surgery vs Angiaplasty vs Medical Management Surgery (by type) vs Watchful Waiting Surgery vs Stone ~i~ vs M~dir~1 Management vs Watchful Waiting Surgery (by type) vs Medical Management Surgery (by type) vs Hormone Treatment vs Watchful Waiting Surgery (by type) vs Angiqplasty vs Drugs vs Watchful Waiting Surgery (by type) vs Various Drugs Surgery vs Various Medic Treatments Back Pain/Strain Hospitalization vs Amkulatory-base] Care; Gastroenteritis ICU vs Usual Ward core Respiratory Disease Heart Disease 87

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