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AUA ADVOCACY Implementing Cost-Effectiveness Into Guidelines to Lower Patient Cost of Care

By: Brian D. Cortese, MD, MBA, Perelman School of Medicine, University of Pennsylvania, Philadelphia; Vidit Sharma, MD, MS, Mayo Clinic, Rochester, Minnesota; Kevin M. Wymer, MD, Mayo Clinic, Rochester, Minnesota; Daniel D. Joyce, MD, MS, Vanderbilt University Medical Center, Nashville, Tennessee | Posted on: 18 Jun 2024

The drivers behind the meteoric rise in the cost of health care have been hotly debated since the 1960s.1 However, as Anderson et al,2 eloquently identified in the early 2000s, “it’s the prices, stupid.” It is thus generally accepted that rising prices, rather than increased health care utilization, are the primary driver of increased overall spending. Cost-effectiveness, which we will define as the costs of treatments divided by the quality of health outcomes, has become a point of national priority. Often, these health outcomes are defined by the quality-adjusted life-year (QALY), which incorporates both the quality and quantity of life when comparing 2 medical interventions. The standard metric used to compare the cost-effectiveness of a new intervention relative to a predefined standard is the incremental cost-effectiveness ratio, which reports the difference in cost divided by the difference in QALYs of the 2 options. As long as the incremental cost-effectiveness ratio is below the prespecified value or willingness-to-pay threshold of dollars per QALY, the intervention is considered cost-effective.

Although traditionally an area outside of the standard practice or knowledge of physicians, an understanding of cost and cost-effectiveness is increasingly needed as providers and patients continue to grapple with a complex and dynamic regulatory environment. This environment often puts the onus on providers to be stewards of high-value and cost-effective care. For instance, for providers subject to the Medicare Access and CHIP Reauthorization Act of 2015 who reach a threshold of care delivery for Medicare patients, enrollment in either the Merit-Based Incentive Payment System or the Alternative Payment Model Performance Plan is mandatory. Each of these payment schemes comes with its own reporting requirements and reimbursement structures that link payment to quality. Not only is physician compensation being increasingly tied to quality measures, but progressively expensive interventions have driven up cost-sharing for patients, leading to significant financial toxicity. For patients, financial toxicity can manifest as skipping doses or not filling prescriptions; increasing levels of anxiety, stress, and depression; and worsening oncologic outcomes and mortality.3

Further, cost-effective care and the evidence-based practice of medicine have become increasingly synonymous with one another. Through cost-effective care, physicians can improve clinical outcomes through the provision of evidence-based medicine, improve patient satisfaction through the reduction of financial toxicity, and improve resource stewardship, leading to more responsible health care dollars spent. For example, in a recently published comparative cost-effectiveness study examining the results of the Surgery in Early Metastatic Seminoma trial, we found that retroperitoneal lymph node dissection was the most cost-effective approach for stage IIA seminoma when compared to chemotherapy or radiotherapy.4 Specifically, the combination of high costs and accrued disutility secondary to chronic toxicities from radiotherapy and chemotherapy made retroperitoneal lymph node dissection the most cost-effective choice.4 In another cost-effectiveness study that examined the impact of adjuvant pembrolizumab after nephrectomy for high-risk renal cell carcinoma patients, we found that adjuvant pembrolizumab was only cost-effective for patients with at least a 60% risk of 5-year progression after nephrectomy.5 Such insights into trial data are more readily apparent when viewed via these health care economics perspectives and are critical to help guide implementation of high-value care.

Currently, the inclusion of cost-effectiveness in clinical practice guidelines within urology and urologic oncology is inconsistent. For example, although evidence-based practice guidelines from the AUA do not explicitly incorporate cost-effectiveness, other practice guidelines such as those from the National Comprehensive Cancer Network (NCCN) have made an intentional effort to do so. However, to date, these efforts rely on expert opinion and do not incorporate high-quality cost-effectiveness data into the value assessments. And in the case of the NCCN “evidence blocks,” very few physicians read and understand what they indicate. As a result, urologists are not given guidance on what set of recommendations are low value and what set are high value.

While incorporating cost-effectiveness into clinical practice guidelines is paved with good intentions, admittedly there are real drawbacks on how the findings of cost-effectiveness analyses are implemented. For example, the use of cost-effective measures can be potentially discriminatory toward certain demographics. Older patients or patients with disabilities can experience less benefit when modeling QALYs because they have a shorter life expectancy and their baseline quality of life may be less than younger, healthier patients.6,7 But these problems are easy to address with modifications in how QALYs are weighted and calculated for the given patient. Additionally, the perverse incentives surrounding fee-for-service care drive up procedure volumes, making it difficult to incorporate cost-effectiveness of those procedures for individual patient decisions. In fact, by statute, the Centers for Medicare and Medicaid Services are unable to use cost-effectiveness when deciding on what interventions receive coverage.8 In response to these barriers, the introduction of alternative payment and pay-for-performance models and a greater shift toward managed care (eg, Medicare Advantage, Medicaid Managed Care, accountable care organizations) represent the proverbial “baby steps” toward integrating cost and quality metrics into US health care spending.

Despite these obvious challenges and barriers, weaving cost-effectiveness into urologic clinical practice guidelines should be a top priority. At the federal level, only the CDC’s Advisory Committee on Immunization Practices robustly uses cost-effectiveness in their national recommendations.9 However, several professional medical societies and institutions routinely use value assessment frameworks in their recommendations, including the American College of Cardiology, American Heart Association, American Society of Clinical Oncology, Institute for Clinical and Economic Review, and as mentioned previously, the NCCN.10

Importantly, in the United States, clinicians essentially act as the gatekeepers of providing high-value, cost-effective care for our patient populations. As physicians routinely deliver guideline-directed and evidence-based medicine to their patients, incorporating indicators of cost-effectiveness alongside each guideline recommendation into clinical guidelines will make a difference. Therefore, we urge the AUA to strongly consider analyzing and incorporating the cost-effectiveness of individual clinical practice guideline statements to ensure the members of our specialty are the ones who define what the delivery of high-value care looks like.

  1. Somers HM. Financing of medical care in the United States. N Engl J Med. 1966;275(13):702-709. doi:10.1056/NEJM196609292751306
  2. Anderson GF, Reinhardt UE, Hussey PS, Petrosyan V. It’s the prices, stupid: why the United States is so different from other countries. Health Aff. 2003;22(3):89-105.
  3. Hussaini SMQ, Gupta A, Dusetzina SB. Financial toxicity of cancer treatment. JAMA Oncol. 2022;8(5):788. doi:10.1001/jamaoncol.2021.7987
  4. Joyce DD, Sharma V, Wymer KM, et al. Comparative cost-effectiveness of contemporary treatment strategies for stage IIA seminoma. J Natl Cancer Inst. 2024;116(3):468-475. doi: 10.1093/jnci/djad211
  5. Sharma V, Wymer KM, Joyce DD, et al. Cost-effectiveness of adjuvant pembrolizumab after nephrectomy for high-risk renal cell carcinoma: insights for patient selection from a Markov model. J Urol. 2023;209(1):89-98. doi:10.1097/JU.0000000000002953
  6. Basu A, Carlson J, Veenstra D. Health years in total: a new health objective function for cost-effectiveness analysis. Value Health. 2020;23(1):96-103. doi:10.1016/j.jval.2019.10.014
  7. Kim DD, Basu A. How does cost-effectiveness analysis inform health care decisions?. AMA J Ethics. 2021;23(8):E639-647. doi:10.1001/amajethics.2021.639
  8. Jacqueline F. Medicare should, but cannot, consider cost: legal impediments to sound policy. Buff L Rev. 2005;53:577-633.
  9. Leidner AJ, Chesson HW, Meltzer MI, Messonnier ML, Lee GM, Prosser LA. Guidance for health economics studies presented to the Advisory Committee on Immunization Practices (ACIP), 2019 update. Centers for Disease Control and Prevention. 2019. Accessed April 1, 2024. https://www.cdc.gov/vaccines/acip/committee/downloads/Economics-Guidance-for-ACIP-2019.pdf
  10. Neumann PJ, Willke RJ, Garrison LP Jr. A health economics approach to US value assessment frameworks-introduction: an ISPOR special task force report [1]. Value Health. 2018;21(2):119-123. doi:10.1016/j.jval.2017.12.012

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