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10 - Health Outcomes

A Comprehensive Approach

from Part II - Rethinking Research

Published online by Cambridge University Press:  13 March 2025

Karen B. Schmaling
Affiliation:
Washington State University
Robert M. Kaplan
Affiliation:
Stanford University
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Summary

We use healthcare in an effort to live longer or feel better. Yet many evaluations do not consider these outcomes, which are of high importance to patients. Instead, they concentrate on variables that are considered surrogates for what treatment is attempting to achieve. Prevention of heart disease, for example, might be estimated from changes in LDL cholesterol levels. These surrogate markers are often poorly correlated with the outcomes of most importance to patients. Understanding the basic biological mechanisms is valuable, but sometimes irrelevant. The chapter reviews patient-reported outcomes that are becoming more commonly used to evaluate health care. These measures are used to create indexes that combine how long people live with the quality of life during the years that precede death. The measures are generic and can be used to compare the value of investing in interventions that have different specific objectives. Cost-effectiveness analysis can directly compare health gain associated with treatments as different as exercise training versus organ transplantation. The public policy implications associated with these metrics are discussed.

Type
Chapter
Information
Rethinking Clinical Research
Methodology and Ethics
, pp. 201 - 216
Publisher: Cambridge University Press
Print publication year: 2025

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References

Ubel, PA. Medical facts versus value judgments – Toward preference-sensitive guidelines. N Engl J Med. 2015; 372(26):24752477. doi:10.1056/NEJMp1504245.CrossRefGoogle ScholarPubMed
Ubel, PA, Comerford, DA, Johnson, E. Healthcare.gov 3.0 – behavioral economics and insurance exchanges. N Engl J Med. 2015; 372(8):695698. doi:10.1056/NEJMp1414771.CrossRefGoogle ScholarPubMed
Roland, M, Dudley, RA. How financial and reputational incentives can be used to improve medical care. Health Serv Res. 2015. doi:10.1111/1475-6773.12419CrossRefGoogle ScholarPubMed
Kaplan, RM. The Ziggy theorem: Toward an outcomes-focused health psychology. Health Psychol. 1994; 13(6):451460.CrossRefGoogle ScholarPubMed
Singer, P, McKie, J, Kuhse, H, Richardson, J. Double jeopardy and the use of QALYs in health care allocation. J Med Ethics. 1995; 21(3):144150. doi:10.1136/jme.21.3.144CrossRefGoogle ScholarPubMed
Neumann, PJ, Weinstein, MC. Legislating against use of cost-effectiveness information. N Engl J Med. 2010; 363(16):14951497. doi:10.1056/NEJMp1007168.CrossRefGoogle ScholarPubMed
Kaplan, RM. The Hippocratic Predicament: Affordability, Access, and Accountability in American Medicine. Academic Press; 1993.Google Scholar
Kaplan, RM. Value judgment in the Oregon Medicaid experiment. Med Care. 1994; 32(10):975988.CrossRefGoogle ScholarPubMed
Kaplan, RM, Anderson, JP. A general health policy model: Update and applications. Health Serv Res. 1988; 23(2):203.Google ScholarPubMed
Neumann, PJ, Kim, DD, Trikalinos, TA, et al. Future directions for cost-effectiveness analyses in health and medicine. Med Decis Making. 2018; 38(7):767777.CrossRefGoogle ScholarPubMed
Neumann, PJ, Sanders, GD, Russell, LB, Siegel, JE, Ganiats, TG. Cost-effectiveness in Health and Medicine. Oxford University Press; 2016.CrossRefGoogle Scholar
Woolf, SH, Aron, LY, National Academies (U.S.). Panel on Understanding Cross-National Health Differences Among High-Income Countries, Institute of Medicine (U.S.). Board on Population Health and Public Health Practice. U.S. health in international perspective: Shorter lives, poorer health. The National Academies Press; 2013:xxv, 394 pages.Google Scholar
Kirsch, I. The emperor’s new drugs: Exploding the antidepressant myth. ReadHowYouWant.com; 2010.Google Scholar
Checkland, P. Systems theory and management thinking. Am Behav Sci. 1994; 38(1):7591.CrossRefGoogle Scholar
Action to Control Cardiovascular Risk in Diabetes Study G, Gerstein, HC, Miller, ME, et al. Effects of intensive glucose lowering in type 2 diabetes. N Engl J Med. 2008; 358(24):25452559. doi:10.1056/NEJMoa0802743.Google ScholarPubMed
Singh, AK, Szczech, L, Tang, KL, et al. Correction of anemia with epoetin alfa in chronic kidney disease. N Engl J Med. 2006; 355(20):20852098. doi:10.1056/NEJMoa065485.CrossRefGoogle ScholarPubMed
Drueke, TB, Locatelli, F, Clyne, N, et al. Normalization of hemoglobin level in patients with chronic kidney disease and anemia. N Engl J Med. 2006; 355(20):20712084. doi:10.1056/NEJMoa062276.CrossRefGoogle ScholarPubMed
Gharajedaghi, JAR. Mechanisms, organisms, and social systems. Strategic Manage J. 1984; 5:289300.CrossRefGoogle Scholar
Kaplan, RM, Ries, AL. Quality of life: Concept and definition. Copd. 2007; 4(3):263271. doi:10.1080/15412550701480356.CrossRefGoogle Scholar
Fiscella, K, Ransom, S, Jean-Pierre, P, et al. Patient-reported outcome measures suitable to assessment of patient navigation. Rev Cancer. 2011; 117(15 Suppl):36033617. doi:10.1002/cncr.26260.Google ScholarPubMed
Pandol, S, Gukovskaya, A, Edderkoui, M, Dawson, D, Eibl, G, Lugea, A. Epidemiology, risk factors, and the promotion of pancreatic cancer: Role of the stellate cell. J Gastroenterol Hepatol. 2012; 27(s2):127134.CrossRefGoogle ScholarPubMed
Cohn, JN. Introduction to surrogate markers. Circulation. 2004; 109(25 Suppl 1):IV20–IV21. doi:10.1161/01.CIR.0000133441.05780.1d.CrossRefGoogle ScholarPubMed
Maulden, A. Ignoring the experts: Implications of the FDA’s Aduhelm approval. Am J Law Med. 2022; 48(1):108133.CrossRefGoogle ScholarPubMed
Karlawish, J, Grill, JD. The approval of Aduhelm risks eroding public trust in Alzheimer research and the FDA. Nat Rev Neurol. 2021; 17(9):523524.CrossRefGoogle ScholarPubMed
Tanzi, RE. FDA Approval of Aduhelm Paves a New Path for Alzheimer’s Disease. ACS Publications; 2021. pp. 27142715.Google Scholar
Gusmano, MK. Aduhelm and the politics of drug approval in the United States. Public Policy Aging Rep. 2022; 32(2):6671.CrossRefGoogle Scholar
Vellas, BJ. The Geriatrician, the Primary Care Physician, Aducanumab and the FDA Decision: From Frustration to New Hope. Springer; 2021. pp. 821823.Google ScholarPubMed
Qiao, Y, Chi, Y, Zhang, Q, Ma, Y. Safety and efficacy of lecanemab for Alzheimer’s disease: A systematic review and meta-analysis of randomized clinical trials. Front Aging Neurosci. 2023; 15:1169499.CrossRefGoogle ScholarPubMed
Kurkinen, M. Lecanemab (Leqembi) is not the right drug for patients with Alzheimer’s disease. Adv Clin Exp Med. 2023; 32(9):943947.CrossRefGoogle ScholarPubMed
Kaplan, RM. Behavior as the central outcome in health care. Am Psychol. Nov 1990; 45(11):12111220.CrossRefGoogle Scholar
O’Fee, K, Deych, E, Ciani, O, Brown, DL. Assessment of nonfatal myocardial infarction as a surrogate for all-cause and cardiovascular mortality in treatment or prevention of coronary artery disease: A meta-analysis of randomized clinical trials. JAMA Intern Med. 2021; 181(12):15751587.CrossRefGoogle ScholarPubMed
Kaplan, RM. Health outcome models for policy analysis. Health Psychol. 1989; 8(6):723735.CrossRefGoogle ScholarPubMed
Kaplan, RM. More than Medicine: The Broken Promise of American Health. Harvard University Press; 2019.CrossRefGoogle Scholar
Guirguis-Blake, J, Evans, CV, Perdue, LA, Bean, SI, Senger, CA. Aspirin use to prevent cardiovascular disease and colorectal cancer: An evidence update for the U.S. preventive services task force. 2022.Google ScholarPubMed
Calderone, D, Greco, A, Ingala, S, et al. Efficacy and safety of aspirin for primary cardiovascular risk prevention in younger and older age: An updated systematic review and meta-analysis of 173,810 subjects from 21 randomized studies. Thromb Haemost. 2022; 122(03):445455.Google ScholarPubMed
Berger, JS. Aspirin for primary prevention – Time to rethink our approach. JAMA Netw Open. 2022; 5(4):e2210144–e2210144.CrossRefGoogle ScholarPubMed
Bolt, L, Speierer, A, Bétrisey, S, et al. Is there a shift from cardiovascular to cancer death in lipid-lowering trials? A systematic review and meta-analysis. Plos One. 2024; 19(2):e0297852.CrossRefGoogle Scholar
Abrams, DB. Applying transdisciplinary research strategies to understanding and eliminating health disparities. Health Educ Behav. 2006; 33(4):515531.CrossRefGoogle ScholarPubMed
Kaplan, RM. The future of outcomes measurement in rheumatology. Am J Manag Care. 2007; 13(Suppl 9):S252–S255.Google ScholarPubMed
Kaplan, RM, Frosch, DL. Decision making in medicine and health care. Ann Rev Clin Psychol. 2005; 1:525556.CrossRefGoogle ScholarPubMed
Group, AS. Long-term effects of intensive glucose lowering on cardiovascular outcomes. N Engl J Med. 2011; 364(9):818828.CrossRefGoogle Scholar
Han, BH, Sutin, D, Williamson, JD, et al. Effect of statin treatment vs usual care on primary cardiovascular prevention among older adults: The ALLHAT-LLT randomized clinical trial. JAMA Intern Med. 2017; 177(7):955965.CrossRefGoogle ScholarPubMed
Neale, RE, Baxter, C, Romero, BD, et al. The D-Health Trial: A randomised controlled trial of the effect of vitamin D on mortality. Lancet Diabetes Endocrinol. 2022; 10(2):120128. doi:10.1016/S2213-8587(21)00345-4.CrossRefGoogle Scholar

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