The Artificial Heart Prototypes, Policies, and Patients (1991) / Chapter Skim
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10. Conclusions and Recommendations
10. Conclusions and Recommendations
Pages 173-200
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From page 173... ...
To succeed in improving patient care, the goals of the NHLBI artificial heart program and the outputs of its R&D efforts must be compatible with the U.S. health care system, particularly because: This discussion and the report as a whole do not consider devices that are intended only for temporary cardiac support, as they were not a focus of the study.
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From page 174... ...
Most of the devices implanted during the first decade of MCSS use will be VADs. As many as 10,000 to 20,000 of each year's primary group of patients have impairment of both of the heart's ventricles, however, and will be candidates to receive a total artificial heart (TAM)
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From page 175... ...
The level of past funding of MCSS contracts has been constraining for MCSS developers; the annual total may need to be slightly greater if NHLBI accepts the committee's recommendations to continue R&D with TAHs, as well as with more than one model of VAD, for an interim period. Not including the costs of the Novacor VAD trial, for which funds are already committed, approximately $2 million per year will be needed for additional VAD development for at least the next two fiscal years, and thereafter a possible additional amount to support further clinical trials.
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From page 176... ...
The NHLBI artificial heart program is unusual among health care R&D in the extent of its government support, in that the government has funded virtually all work to date, including the device development and testing costs usually borne by industry for similar technologies. As discussed later in this chapter and in Chapter 9, existing circumstances warrant continued NHLBI funding of both VAD and TAH research for at least a two- to three-year period.
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From page 177... ...
Among the relevant activities that can begin immediately are the development of provisional clinical practice guidelines (indications for use) 2 and of guidelines for the institutional resources and staff expertise necessary for appropriate MCSS use, as well as the initiation of selective coverage programs (e.g., third-party payers approving coverage for MCSS use only when the device is implanted at designated institutions)
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From page 178... ...
· Recognizing the cost of this technology, adequate federal and private research funds should be devoted to clinical studies and technology assessments of MCSS use. · Staff of the NHLBI artificial heart program should participate in these appropriateness-enhancing activities and work with AHCPR to develop support for the needed studies.
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From page 179... ...
Clinically Limited Access At least in the early stages of MCSS diffusion, it will be imperative to limit access on clinical grounds, much as the number of heart transplantation candidates is limited by stringent approval criteria for placement on the transplant waiting list. If provisional indications for long-term MCSS use
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From page 180... ...
Limited access to heart transplantation is warranted by the small number of available donors; with a manufactured device, no such curbs will occur on the supply side. Furthermore, those developing any restrictive indications for use and the clinicians who apply them to individual patients must take great care that they do not favor a particular demographic group (e.g., middle-class white males)
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From page 181... ...
Clinical Effectiveness It is premature to judge precisely when a clinically effective long-term VAD will be approved for general marketing, but such an approval appears likely to occur before the end of the century. A fully implantable TAH will likely follow about 10 years later, depending on future NHLBI decisions about funding of TAH development.
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From page 182... ...
~ ~ meets to support comprehensive assessments of health-related quality of life in patients receiving MCSSs during clinical trials and even thereafter. A core set of quality-of-life domains, similar to those used for this study's utility measures, should be assessed in all MCSS clinical trials.
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From page 183... ...
The committee's CEA examined the projected cost-effectiveness of TAH use and heart transplantation, comparing both with conventional medical treatment for end-stage heart disease as of 2010, the earliest that TAHs are likely to be in routine use. This required making a number of estimates of such aspects of TAH use as device failure rates and the probability of various TAH-related clinical complications, as well as lengths of hospital stays and outcomes of such events.
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From page 184... ...
These actions by FDA, the Medicare program, and numerous other third-party payers are the regulatory counterparts to the development of clinical practice guidelines and other measures that help to improve the appropriateness of technology use.
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From page 185... ...
Relatively few third-party payers have explicit criteria or standards for their coverage decisions, particularly ones that can be applied to an emerging technology for which data are limited. Clinical practice guidelines may eventually be able to guide third parties in their actions concerning MCSSs, both in developing coverage and payment policies and in acting on pretreatment authorization requests.
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From page 186... ...
Considerations favoring the continuation of this support include: · the substantial progress achieved over the artificial heart program's 27 years; · the number of end-stage heart disease patients probably at least 10,000 annually for whom no other form of treatment appears able to prevent disability and death; and · the likelihood, as explained elsewhere, that this development effort will not move forward without government support. Offsetting these arguments is the current great uncertainty about virtually every aspect of this technology's likely status early in the next century.
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From page 187... ...
NHLBI and other organizations regularly commit funds to R&D ventures with varying certainties of outcome; no reason exists to demand guaranteed success from the artificial heart program. Uncertainty admittedly exists about the ultimate TAH C/E ratio and its consequences, but moving ahead cautiously with additional TAH development during 1993-1995 offers the possibility of substantial benefits to patients who might otherwise die if TAH development is delayed by a suspension of funding.
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From page 188... ...
If some such entity were to decide that even $60,000 or $70,000 per QALY is too great a cost, then it would be reasonable to suspend both TAH development and R&D on new approaches to VADs until the current VAD development efforts yield MCSS clinical effectiveness data that are sufficiently improved to reduce the C/E ratio below $60,000. The committee thus concludes that continuing NHLBI support of TAH development, for a limited period of two to three years beyond the current contracts' expiration in September 1993, is in the public interest because of its possible future benefit to patients and the additional inflation to be developed during that time.
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From page 189... ...
A much broader range of sensitivity analyses than this study's resources allowed will also be helpful in testing the impact of the estimates developed for NHLBI's next decision. The committee concludes that NHLBI should continue to support scientifically meritorious TAH development for a two- to three-year period until preliminary results of the Novacor VAD trial (and perhaps other trials)
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From page 190... ...
OTHER RECOMMENDATIONS The preceding recommendations and discussions fulfill the committee's principal obligation to NHLBI, responding to the nine questions set forth in the 1989 report of the IOM planning committee and making recommendations concerning future TAH development. The committee believes that several additional conclusions and recommendations about aspects of MCSS development are also appropriate.
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From page 191... ...
FDA review of a developer's investigational device exemption application is theoretically sufficient to ensure that all aspects of a trial are properly designed and conducted, including periodic postimplantation assessments of patients' physiologic status and health-related quality of life. In practice, however, the committee recalls the criticisms of past artificial heart trials at Salt Lake City, Louisville, Phoenix, and other locations as having important shortcomings; by continuing its R&D involvement NHLBI can exert strong influence over all aspects of VAD trials.
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From page 192... ...
It is important that MCSS trials, whether supported by public or private funds, make provision for each patient to provide a fully informed consent, an advance directive concerning termination of treatment, and legal designation of a proxy; see the review of these ethical safeguards in Chapter 8. The committee thus recommends that all NHLBI-supported and private MCSS clinical trials include adequate funding for the
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From page 193... ...
Posttrial Follow-up The two-year follow-up period that is typical of clinical trials of implanted devices is not long enough to reveal all problems that may occur with MCSS use. In addition to any registry that is established, an adequate sample of patients receiving MCSSs during clinical trials should be followed periodically after the trial period ends, to ascertain and study details of posttrial patient management and outcomes.
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From page 194... ...
The policies and mechanisms by which the artificial heart program operates were originally developed to review, approve, and manage traditional investigator-initiated, nontargeted research by basic scientists, not the development of complex devices and support of their extensive technical evaluation, redesign, preclinical testing, and clinical trials. Additionally, although traditional scientific peer review is as important to MCSS research as it is to any other field, considering artificial heart program proposals in the existing peer-review committees puts this truly interdisciplinary research involving biomedical engineers, clinicians, and others at a serious competitive disadvantage, as discussed in Chapter 9.
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From page 195... ...
The committee therefore concludes that NHLBI should not use the development of MCSSs as a reason to reduce its level of funding of basic heart disease research. Instead, the committee recommends that NHLBI continue to support, at an adequate level, scientifically meritorious research into the mechanisms of heart failure and approaches to the prevention and medical treatment of end-stage heart disease.
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From page 196... ...
Better basic data will make future projections of MCSS use more accurate. The committee therefore applauds NHLBI for supporting the longitudinal studies recently begun; further, the committee recommends that, in addition to current longitudinal studies of heart disease in persons over age 65, NHLBI undertake as a high priority, and possibly with the involvement of the National Center for Health Statistics, the development of additional epidemiological data about the natural history of end-stage heart disease in patients of all ages, with special attention to the inclusion of women and minority populations.
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From page 197... ...
Most important, any one of the comprehensive studies of the artificial heart that have been performed could serve as a model of the type of study that should become an integral part of the U.S. health care system.
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From page 199... ...
Appendixes
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Key Terms
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