Author + information
- S2213177916305959-28bcb45824ecad34309510724b56b83cJoseph G. Rogers, MD∗ ()
- Division of Cardiology and the Duke Clinical Research Institute, Duke University, Durham, North Carolina
- ↵∗Reprint request and correspondence:
Dr. Joseph G. Rogers, Division of Cardiology, Duke University, Box 3034, Duke University Medical Center (DUMC), 2400 Pratt Street, Durham, North Carolina 27710.
The medical press and lay press are filled with commentary about the cost of U.S. health care and the marginal “value” it brings to our population. The foundation of this concern is variably blamed on physicians’ practice patterns, delivery of non–evidence-based care, the cost of durable medical equipment, pharmaceuticals, end-of-life care, hospitalization costs, and medical innovation. Health care costs are anticipated to rise with the development of incrementally efficacious and life-prolonging treatments coupled with the aging populace. This issue of rising costs causes considerable angst among economists and health care strategists who remain convinced that the financial burden of U.S. health care is unsustainable and will consume an excessive portion of the overall economy. It is through this lens that medical innovation must be examined. In a constraint-focused system in which financial support for thorough investigation of novel diagnostics and therapeutics is increasingly limited, lingering questions remain about who will pay for medical advances, as well as the time horizon used to determine the ultimate “value” of such treatments.
The past decades have witnessed dramatic advances in cardiovascular care, many of which evolved over a protracted timeline. Consider that just a few decades ago myocardial infarction was treated with prolonged bed rest and analgesics, but it is now routine to open an occluded coronary artery mechanically within 90 min of symptom onset to avoid subsequent morbidity and death. Implantable defibrillators, originally conceived to treat patients with refractory ventricular arrhythmias, have evolved and now assume a critically important role in the prevention of sudden death in patients with ventricular dysfunction. Clinical trials of transcatheter aortic valve replacement began with patients at unacceptable risk for cardiac surgical procedures but are currently including low-risk patients, and transcatheter aortic valve replacement seems destined to play a pivotal role in the therapeutic armamentarium for the majority of patients with aortic stenosis. The field of advanced heart failure has similarly had its share of remarkable successes that have favorably altered quality of life and reduced mortality rates in this vulnerable population. The first heart transplant operation in 1967 by Christiaan Barnard in Cape Town, South Africa was followed by a worldwide series of clinical failures that in a contemporary era could have doomed the therapy. It was only an unwavering commitment to the procedure by a small number of clinician-scientists, coupled with the introduction of cyclosporine nearly 15 years later, that transformed a fledgling, ineffective, “low-value” treatment into an accepted treatment that has extended the life of more than 100,000 patients worldwide and provided new and exciting insights into the fields of immunology, ischemia-reperfusion injury, and novel pharmacotherapeutic agents.
Mechanically assisted circulation is on an accelerated trajectory compared with the early days of heart transplantation, although the parallels are difficult to ignore. Early-generation left ventricular assist devices (LVAD) were complex, large devices that provided short-term support, were prone to malfunction, and were associated with significant adverse events. Enthusiasm for LVAD therapy remained limited to a small number of centers and a few patients. However, newer-generation LVADs with simpler design principles, enhanced durability, and better hemocompatibility have improved clinical outcomes. As a result, there has been greater acceptance of VAD therapy as a viable option for patients with advanced heart failure. The device development timeline is important to acknowledge: the HeartMate II LVAD (St. Jude Medical, St. Paul, Minnesota) received approval from the U.S. Food and Drug Administration for bridging patients to heart transplantation in 2008 and as an alternative therapy in transplant-ineligible patients in 2010. Over the past decade more than 20,000 patients in the United States have been entered in INTERMACS (Interagency Registry for Mechanically Assisted Circulatory Support), a post-market data repository that has chronicled the development of LVAD therapy (1). Although the rapid dissemination of this therapy has primarily been driven by clinical need, engineers and clinicians alike recognize the need for continued improvements in device design and manufacturing, the role of adherence to established care principles, and the need for ongoing education to achieve the best outcomes.
From this background, several questions should be asked: 1) how long is a therapy “nascent”?; 2) when should we anticipate seeing value?; 3) who should bear the financial burden of advancing new and innovative treatment modalities?; and 4) at what point do we acknowledge that an efficacious therapy may be too expensive for its associated clinical value?
In this issue of JACC: Heart Failure, Shreibati et al. (2) present an economic analysis of patients treated with mechanically assisted circulation in 2009 and 2010. These investigators used a Medicare dataset to determine inpatient and outpatient care costs and stratified the group on the basis of severity of illness by using administrative claims data. Value was determined by evaluating the costs in the context of health status improvements (longevity and quality of life), and key clinical drivers of cost were identified.
Costs for device implantation were $175,000 in this period, with an additional cost of $3,000/month for outpatient management. LVADs did not reduce hospitalizations when compared with the 12-month period before device implantation. In fact, the duration and cost of LVAD-related hospital stays were higher. The projected life expectancy in this cohort of LVAD-treated patients was 6.28 years, at a total cost of $726,000. The cost-effectiveness of VADs in less sick LVAD recipients was unfavorable: LVADs increased survival by only 0.6 life-years or 1.74 quality-adjusted life-years (QALYs), at a cost of $364,000 or $209,000/QALY. LVADs had the greatest impact when they were implanted in high-risk patients, whose survival was increased by nearly 3 years (2.78 QALYs), at a cost of $475,500 or $171,000/QALY. Overall costs were most sensitive to hospitalizations and outpatient care. Further modeling of these data suggested that reducing readmission rates and outpatient costs by 50% dropped the incremental cost-effectiveness ratio (ICER) to $87,000. For reference, an ICER <$50,000 is considered cost-effective, whereas an ICER <$100,000 is acceptable.
Although the report by Shreibati et al. (2) highlights the potential value of LVAD therapy, several caveats should be considered in its interpretation. The field of assisted circulation is rapidly evolving, with new technologies and improved patient selection and management strategies. The data used in this analysis are 6 to 7 years old, from a time when these devices had just completed clinical trials. As a result, this information may not be representative of a contemporary state and does not account for changes in the incidence or cost of managing adverse events (3). Further, the analysis of outpatient costs is calculated on the basis of a small number of patients (n = 45) and may not reflect the larger population of LVAD recipients.
Although it is encouraging to see that the field of assisted circulation is making progress toward cost-effectiveness, benchmarks have not yet been achieved (4). Fortunately, Shreibati et al. (2) provide a roadmap to guide our efforts. On the basis of this analysis, multidisciplinary teams first should focus on reducing outpatient costs and repeat hospitalizations. The costs of managing outpatients supported with mechanical blood pumps require careful examination, particularly the costs of durable medical equipment and supplies. Future innovation that includes totally implantable systems may obviate the need for some of these supplies but will certainly be associated with higher initial costs as well as yet undefined maintenance costs. The second area of focus must be the reduction of repeat hospitalizations. As in the early days of heart transplantation, when nearly all physiological perturbations resulted in hospitalization, VAD-treated patients are hospitalized with impunity. Clinicians do not yet have the confidence or the tools to manage many VAD adverse events in the outpatient setting. Further, many of the complications, such as mucosal bleeding, stroke, and device malfunction, require inpatient care. Finally, patient selection also requires refinement because some of the adverse events may be predictable on the basis of pre-operative, candidate frailty or the severity of right-sided heart failure.
Beyond the primary data, the report by Shreibati et al. (2) highlights a critically important and deeper question: what are our clinical, temporal, and financial tolerances for innovation? Arguably there is a growing expectation that technology, biomedical research, and clinical trial infrastructure should expedite the translation of discovery to clinical application. It should not come as a surprise that maturation of many of our therapies will require careful analysis and refinement of these treatments by thoughtful clinician scientists if we hope ultimately to replicate the successful evolution of cardiac transplantation.
↵∗ Editorials published in JACC: Heart Failure reflect the views of the author and do not necessarily represent the views of JACC: Heart Failure or the American College of Cardiology.
Dr. Rogers has reported that he has no relationships relevant to the contents of this paper to disclose.
- American College of Cardiology Foundation
- ↵University of Alabama at Birmingham. Interagency Registry for Mechanically Assisted Circulatory Support. Available at: https://www.uab.edu/medicine/intermacs/about-us. Accessed November 16, 2016.
- Shreibati J.B.,
- Goldhaber-Fiebert J.D.,
- Banerjee D.,
- Owens D.K.,
- Hlatky M.A.
- Miller L.W.,
- Guglin M.,
- Rogers J.