Assessing Medical Technologies (1985) / Chapter Skim
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Appendix B: Selected Papers
Appendix B: Selected Papers
Pages 490-564
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From page 490... ...
Goodman* This guide is intended to help a reviewer evaluate reports of comparative experimental clinical trials.
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(1982) examined 67 clinical trials published in four prominent medical journals in 1979-1980 for 11 important aspects of trial design and analysis (e.
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(1984) repeated the study on 84 clinical trials published in 1 vear in six journals, and they found that 58 percent were clearly reported, 5 percent were ambiguously mentioned, and 37 percent were not reported at all.
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From page 493... ...
. The major subjects addressed in this guide are · basic descriptive material · sample size · selection of patients · random allocation · blinding · treatments compliance withdrawals/loss to follow-up treatment complications tabulation of outcomes statistical methods and analyses power These aspects of clinical trials should be closely examined before one combines the results of smaller trials, or generalizes the results of trials to other populations.
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From page 494... ...
Patient number and treatment Controls Group 1 ASSESSING MEDICAL TECHNOLOGY Group 2 Group 3 e. Placebo described Yes No Unclear na 7.
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From page 495... ...
To the extent that study patients are not selected at random from a well-defined population (not to be confused with random allocation to treatment groups) , doubt may exist as to whether trial findings may be generalized to that population, as well as to others.
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Random Allocation Random allocation of patients to treatment groups is a major bias-reducing technique in controlled clinical trials. The observed results of a trial may be affected (biased)
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If the trial results are to be considered for use in combination with other trial results, the significance of known prognostic factors by treatment category may be important in deciding whether to do so. Blinding Blinding is a major bias-reducing technique in clinical trials.
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Examples are pre- and postvagotomy measurements of gastric acid output in therapeutic trials of peptic ulcer. Blood or urine levels of ASSESSING MEDICAL TECHNOLOGY an active agent may also be used to measure compliance.
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In the analysis of the data gathered in any clinical trial there are certain minimal procedures that are indicative of quality. These include, but are not limited to, significance of major endpoints, confidence intervals, and regression or correlation analyses.
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1983. Size of clinical trials.
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1983. Bias in treatment assignment in controlled clinical trials.
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From page 502... ...
as a complex process requiring a broad comprehensive base of data in order to permit evaluation of shortand long-term, intended and unintended, and direct and indirect consequences of the use of technology. A technology assessment usually first defines the technology to be studied, identifies the alternative technologies with which it competes, describes the patients using the technology, and considers the goals of decision makers for whom the technology assessment is intended; then it evaluates the process and outcomes of using the technology for these patients.
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. Alternative Technologies Similar information as described above will need to be obtained for alternative competing technologies used for the same functional requirements.
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Clinical Safety Technology assessments usually need information as to clinical hazards, toxicity, adverse effects, etc. EVALUATIVE INFORMATION FOR INDIRECT, UNINTENDED EFFECTS An indirect or unintended consequence of the use of a technology may be very extensive, and usually the decision makers for whom the assessment is being prepared have special interests which will determine the specific information needed.
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Such a minimum data set could contain essential and necessary data for many technology assessments. Of course, this could not provide sufficient comprehensive data for every assessment, and special data subsets could be developed for the different technology types and for those that require analytic methods.
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Budgets for clinical trials at the National Institutes of Health (NIH) are under constant surveillance, and vigilant members of Congress will want to know that resources have been well spent.
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Obviously this necessity for breadth multiplies the research effort enormously. AN ANALYTIC FRAMEWORK FOR ASSESSING COST-EFFECTIVENESS We suggest a general conceptual model for evaluating the cost-effectiveness of medical evaluations, and illustrate its applicability to two particular clinical trials.
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of a medical procedure? Moreover, since resources for providing such information (e.g., for clinical trials)
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The study, following more than 20,000 physicians over a 5-year period, is now in progress. Mild Hypertension Calculation of the potential benefits and costs of a mild hypertension trial was made at the time such a trial was being considered by the Veterans Administration and the National Institutes of Health (Laird et al., 1979~.
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From page 510... ...
It reported a significant and important treatment effect, especially in the mildly hypertensive ASSESSING MEDICAL TECHNOLOGY group. The controversy continues as to whether this community-based study was really measuring the effects of antihypertensive medication or whether other differences between the treatments could have accounted for the difference in mortality.
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APPENDIX B.: SELECTED PAPERS Does the second, or third, RCT seem to make more of a difference than the first? Perhaps, as Cochrane (1972)
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(1977) took a small step in this direction by reviewing randomized clinical trials in surgery over a 10-year period.
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3. Clinical trials can help, however; and we need to learn what their value is and how to increase it.
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1976. HyperASSESSING MEDICAL TECHNOLOGY tension: A Policy Perspective.
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The TA process, in general, follows that recommended by the Office of Technology Assessment TOTAL, and has been applied in prepaid group practice (POP) to a variety of medical technologies, including procedures, equipment, or systems that are used for diagnostic, therapeutic, or coordinating patient care services.
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From page 516... ...
Such a TA will be more comprehensive, and the document becomes one in the public domain through publication in the scientific literature. When a technology has been selected for assessment, it is necessary first to define the technology precisely and determine its objecASSESSING MEDICAL TECHNOLOGY fives for its specific applications, then to identify alternative technologies also used for the same applications.
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From page 517... ...
Economic analysis sometimes includes the impact of different payment modes for medical services, such as prepayment versus Medicare cost-reimbursement, purchase versus lease financing for capitalized equipment, etc. Cost-effectiveness analysis provides comparative costs of employing alternative technologies to achieve similar, specified levels of desired effectiveness.
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From page 518... ...
Pediatric geneticists requested a TA on alpha-fetoprotein screening because of impending legislation requiring it for pregnant women as a screening test for fetal neural tube defects. Technology procedures suitable for TA also have been identified by monitoring organizational gross expenditures; e.g., two-view chest x rays were the most frequently ordered diagnostic radiology procedure, so a TA was conducted to assess the consequences of alternative criteria for utilization of this technology.
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From page 519... ...
A TA is being conducted that compares cohort members randomly assigned upon joining the KPMCP to either the new team or to traditional primary care services. This is a followup study of a new approach to ambulatory care involving the entry of patients through a paramedically staffed health evaluation service.6 7 This study is supported in part by a grant from the H
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From page 520... ...
Areas that have been addressed include open heart surgery, CT scanning, radiation therapy, neurosurgery, hemodialysis, skilled nursing facilities, acute rehabilitation services, neonatal intensive care units, and chemical dependency rehabilitation. In summary, the KPMCP southern region's efforts include not only the issues of hard technology that are usually considered as technology assessment, but also the assessment of human and management factors that have an impact on the ultimate value of the use of technology.
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From page 521... ...
Studies have also been conducted on clinical trials, such as prophylactic use of phenobarbital to prevent febrile seizures in children. TA in Group Health Cooperative of Puget Sound Group Health Cooperative of Puget Sound (GHC)
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The professional staff has formed and chairs a medical services committee, with representation from management and the board. Through this committee the professional staff fills what it sees as its responsibility to assess predominantly new but also old medical technologies and makes appropriate recommendations to the board.
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The question of whether the increasing utilization was medically appropriate was referred back to the professional staff committee. Ultrasound for Diagnostic Assessment of Pelvic and Intrauterine Structures This technology is currently provided and covered as medically appropriate.
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Appendix C in Medical Technology and the Health Care System. A Study of the Diffusion ASSESSING MEDICAL TECHNOLOGY of Equipment-Embodied Technology.
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It was found that control enrollees were quite knowledgeable about insurance coverage and income tax deductions for medical care and most maintained detailed records for tax purposes. From these records and surveys, control ambulatory services utilization was estimated and compared to study group data.
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There were other major contributors to increased MCG ambulatory service rates than office visits per se, especially diagnostic ASSESSING MEDICAL TECHNOLOGY x-ray and laboratory services. The total rate for ambulatory services in MCG was severalfold that for controls (p ~ 0.01~.
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From page 527... ...
Suffice it to say here that no differences were found in the types of conditions for which surgery was done in the MCG enrollee group compared with controls, nor were there differences in the proportion of different operations, including those often thought of as elective or unnecessary. These studies provided sound evidence for the often-stated belief that hospital utilization can be decreased and ambulatory services utilization can be increased by a system which combines prepayment for comprehensive medical care services with an organized medical care delivery system.
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From page 528... ...
ASSESSING MEDICAL TECHNOLOGY SUMMARY Organization of medical care into an effective group with prepayment did lead to reduced hospital and increased ambulatory services utilization, but in and of itself did not lead to reduced medical care costs. Several other aspects of prepaid group practice were identified and may lead to cost control, especially reduced cost of physicians' services.
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From page 529... ...
Duncan Neuhausert Randomized clinical trials are said to be expensive, hard to organize, and fraught with ethical difficulties. The demand for more such trials persists, however, because of the need to definitively evaluate the large number of medical interventions of debatable or undemonstrated efficacy.
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Subspecialty consultants come to the ASSESSING MEDICAL TECHNOLOGY TABLE B-3 Volume of Services and Staffing in the Four Metro Firms Number F lrms Inpatients admitted per firm per month New inpatients admitted per firm per month Outpatient visits per firm per month Inpatient beds in each firm Number of physicians in each firm 4 80 55 240 28 18 firms on request.
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Costs Although a determination of the real costs involved in conducting clinical trials is a complicated issue, the extra costs, requiring outside funding for the first three trials, totalled less than $100,000. Suffice it to say, these trials are not expensive in relation to many randomized clinical trails.
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In the Metro firm trials the firms are randomly selected to serve as experimental or ASSESSING MEDICAL TECHNOLOGY control groups, and physicians and patients are randomly assigned to firms. Thus, there are four potential levels of analysis: the experimental versus control firms, each firm as a separate entity, the physician within each firm, or the individual patients.
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A randomized clinical trial with unexpected findings.
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1981. Composite randomization designs for clinical trials.
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From page 535... ...
outcome states for both treated and untreated patients are well documented. The Framingham study on cardiovascular diseases and the Veterans Administration (VA)
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How do these styles influence the choice a patient might make between or among alternative therapies? Data from cognitive psychology have recently suggested that the way data are presented can have a major impact on treatment choice.9 One pilot study illustrates this effect in medicines Treatment for operable lung cancer can involve either radiation therapy (RT)
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Perhaps the reader will think of other analogous examples. Screening for Disease One example of this category relates to the screening of pregnant women for neural tube defects, using the alpha-fetoprotein (AFP)
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, the data in Tables B-7 and B-8 emerged. Specifically, the 5-year survival rate is equal for the no-test and the ASSESSING MEDICAL TECHNOLOGY test strategies if a perfect test (sensitivity and specificity both equal to 100 percent)
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From page 539... ...
~9 On a scale of TABLE B-9 Influence of the Measure of Therapeutic Efficacy on the Choice of Therapy when Excellent Surgical Results Prevail Measure of Therapeutic Efficacy Percent Who Should Receive Radiation Therapy Rather than Operation with Operative Mortality Rates of 5~0 10% 15~o 20% At age 60: 5-year survival Expected utility 7 At age 70: O O 21 5-year survival 0 . 0 Expected utility 14 43 O O 43 64 O O 50 71 SOURCE: N
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From these two perspectives and from the above prototypical examples, it should be clear that additional work is needed in two areas: (1) refinement of the methodology for assessing values and ASSESSING MEDICAL TECHNOLOGY preferences, and (2)
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From page 541... ...
1982. The effect of private attitudes on public policy: Prenatal screening for neural tube defects as a prototype.
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The block grants proposed for FY 1982 called for reduced federal funding and a minimum of federal strings, eliminating or reducing requirements for state matching funds, planning, reporting, maintenance of effort, and the like. In the health field, the Reagan administration proposed two block grants, a health services block and a prevention block, funded at approximately 75 percent of FY 1981 program levels (Feder et al., 1982~.
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From page 543... ...
Renewed federal interest may take the form of research support for the Prospective Payment Assessment Commission, a more narrowly conceived successor to the National Center for Health Care Technology. The evolution of the DRG system, and associated federal support for technology assessment, will likely be as important as health block grants, Medicaid financing and reimbursement reform, and the success or failure of competitive incentive schemes in influencing future state support for technology assessment.
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Evidence of such action, however, is scanty ASSESSING MEDICAL TECHNOLOGY and is largely anecdotal. As third-party payers, some states limit payment for experimental procedures under Medicaid.
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The professional standards review and national health planning laws enacted in 1972 and 1974, respectively—were intended by Congress to foster increased professional regulation and centralized resource allocation across the 50 states. National legislation provided political and financial incentives for previously inactive states and private groups to establish certificate-of-need agencies and peer review organizations where they had not before existed.
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State Resources and Capacity If states were motivated to expand technology assessment efforts, would they have the resources and technical capacity to do so? At a minimum, states would need the financial resources to adequately fund technology assessments.
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To the extent that neither the federal government nor the states currently have a large capacity to conduct technology assessments, future capacity can be built at either level, if funds are available. To the extent that well-conducted technology assessments require access to large data bases, the conduct of large-scale clinical trials including diverse populations over long pe547 riods of time, and the development of complex methodologies, states' capacity to complete successful studies may be limited.
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Is technology assessment one of those functions that should be decentralized via new federalism, or is it primarily a responsibility of the federal government? The literature on federalism offers several economic and political criteria for ASSESSING MEDICAL TECHNOLOGY allocating responsibilities between federal and state governments.
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From page 549... ...
On balance, we believe that the arguments supporting a primary federal role in financing, technical assistance, standard setting, and data gathering if not in directly conducting technology assessments are stronger than those supporting increased state responsibility. Technology assessment as such would probably benefit more from nationally accepted guarantees of scientific integrity and access to nationwide data sets than from innovation, diversity, or grass roots participation at the state level.
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From page 550... ...
Alternatively, if Medicaid financing reforms took the form of federal caps or block grants to the states, states' financial stake in cost control would be maintained insofar as they were at risk for expenditures exceeding federal contributions. Under current matchASSESSING MEDICAL TECHNOLOGY ing arrangements, states pay between 25 and 50 percent of total Medicaid costs.
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On the whole, however, it 551 seems unlikely that health policy analysts and other technology assessment advocates will be as influential in the individual states as they have been at the national level. Third, block grants as enacted under the Reagan administration have increased fiscal constraints on currently funded programs and services.
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From page 552... ...
In contrast, a major new federal ASSESSING MEDICAL TECHNOLOGY initiative to conduct technology assessments, resulting, for example, from the need to administer the DRG payment system; the federalization of Medicaid; state adoption of glob-al budgeting systems; the withering of block grants, and/or continued fluctuations in state fiscal conditions would probably undermine the modicum of existing state support for technology assessment. NOTES ~ The health services block grant would have consolidated 15 categorical programs, including community health centers; alcohol, drug abuse, and mental health programs; maternal and child health; and migrant health programs.
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1981. Block grants and state discretion: A study of the implementation of the Partnership for Health Act in three states.
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A brief overview of the largest governmental programs that provide medical services and benefits is followed by a more comprehensive examination of the Medicare program. THE MEDICARE AND MEDICAID PROGRAMS The Health Care Financing Administration (HCFA)
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Although it is not a health insurance program, CHAMPUS is similar in many respects to health insurance and especially to the Medicare program. Authorized medical services and supplies are cost shared by the government from money appropriated by the Congress to the Department of Defense for this purpose.
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HEALTH CARE FINANCING ADMINISTRATION HCFA was established in 1977 to combine health financing and quality assurance programs into a single agency. It is responsible for the Medicare program, federal participation in the Medicaid program, and a variety of other health care quality assurance programs.
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Intermediaries act as the link between the provider and the Health Care Financing Administration, which is responsible for the administration of the Medicare program. The major role of the intermediaries is to review and pay claims for the costs of providing care to beneficiaries.
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Under the Medicaid program, states may enter into ASSESSING MEDICAL TECHNOLOGY an agreement with the secretary of DHHS to finance health care services for certain categories of low-income individuals, primarily those eligible to receive cash payment under the Aid to Families with Dependent Children (AFDC) program and the Supplemental Security Income (SSI)
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From page 559... ...
The Medicare program pays for some or all of the cost of certain medical services furnished to eligible beneficiaries. In this regard, the Medicare program is similar to the insurance programs of other thirdparty payers such as Blue Cross and Blue Shield and commercial insurance companies.
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From page 560... ...
These policy statements are continuously undergoing change as medical services and procedures evolve and new research findings emerge. Decisions on Individual Claims The claim review process for the Medicare program is designed to identify services which may not be covered or about which there may be a question of medical necessity or reasonableness.
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From page 561... ...
When the panel confirms the need for further expert medical opinion and evaluation, HCFA refers the question with the background information to PHS. USE OF EVALUATIVE DATA IN COVERAGE DECISIONS The criteria currently used to determine if an item or service is reasonable and necessary in terms of the Medicare program are as unspecific as safe and effective.
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From page 562... ...
In such cases, economic considerations or issues of distribution of services and access to services may be an implicit factor in the decision to refer the issue to HCFA for evaluation or to initiate a thorough evaluation. Such considerations may implicitly affect the coverage decision if for no other reason than that a ASSESSING MEDICAL TECHNOLOGY greater burden of proof may be required before a decision is made to cover the service as a Medicare benefit.
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From page 563... ...
Fur563 thermore, there are very significant differences in delivery of medical care services in different areas of the country. A physician evaluating alternative approaches and selecting those services that best serve the needs of an individual patient will draw upon very different information and values than will a policy analyst evaluating information to determine if a service qualifies as reasonable and necessary and, therefore, will be paid for by the Medicare program.
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From page 564... ...
An opposite view could require that payment be made only for those services that have been evaluated with evidence as to safety and efficacy. In practice, the Medicare program looks to the judgment, experience, and opinion of physicians and to sound scientific evidence.
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