Perspectives on Value Frameworks in Alzheimer’s Disease
Eric Jutkowitz, PhD, Department of Health Services, Policy and Practice, Brown University School of Public Health, Providence Veterans Affairs Medical Center, Center of Innovation in Long Term Services and Supports, Providence, RI, USA; and Joseph Gaugler, PhD, Division of Health Policy and Management, School of Public Health, University of Minnesota, Minneapolis, MN, USA
Globally, 50 million people are living with Alzheimer’s disease and related dementias (ADRD) and by mid-century, an estimated 152 million people will be living with ADRD.1 ADRD disproportionately affects older people and women. In the United States, the prevalence of ADRD is nearly 2 times greater among Blacks and 1.5 times greater among Latinos compared to Whites.2 People living with ADRD receive most of their care from family and friend caregivers.3,4 Currently, there is no ADRD disease-modifying therapy on the market, but 80% of ADRD drugs in development are disease-modifying and 29 drug candidates are in phase III development.5 There are also a number of nondrug interventions that have demonstrated statistically and clinically meaningful benefits for people living with ADRD and their caregivers, including improving quality of life.6,7 Finally, there are a number of emerging diagnostic tests to identify ADRD earlier in the disease course.6 Despite scientific advances in improving ADRD care, reimbursement of ADRD interventions has been stymied by value assessment frameworks not equipped to evaluate the effects of these therapeutics on the complex epidemiology of ADRD. In this supplement of Value & Outcomes Spotlight, 3 contributors provide perspectives on modifying value frameworks to evaluate current and emerging ADRD interventions.
Decision makers use value assessment frameworks to allocate resources. Many high-income countries have formal value assessment frameworks to determine the funding of healthcare programs. The United States is a notable exception, with a fragmented payer system. Each payer makes its own funding decisions. In a fragmented system, payers may have conflicting objectives and are not always transparent about how value is defined. In the first article, Garrison et al propose that funders should evaluate the value of an ADRD intervention from a wide perspective and draw on the “value flower” as a framework to evaluate ADRD interventions.8 The value flower framework was developed as part of a 2018 ISPOR task force review of value frameworks.9 Within the value flower framework, Garrison et al note the importance of domains related to productivity and distributional or equity considerations as particularly relevant to ADRD interventions. The lost productivity of people living with ADRD and their caregiver(s) is one of the largest cost drivers of ADRD, yet value frameworks that adopt a healthcare system perspective typically ignore productivity losses. Ignoring intervention effects, even if small, on productivity greatly underestimates the value of a therapy. The equity considerations include giving weight to an intervention that helps people with ADRD since it is a disease that disproportionately impacts older individuals, women, low-resourced communities, and Black, indigenous, and persons of color.
"Without early detection efforts, we may not be able to identify people who would benefit most from emerging disease-modifying therapeutics."
Building on Garrison et al, Basu et al focus on the brain equity considerations within a value framework and suggest using an equity-efficacy impact plane to evaluate ADRD interventions.10 In an equity-efficacy framework, there is value in interventions that are less cost-effective treatments so long as they reduce health inequities. A challenge when considering the brain equity effects of an intervention is reconciling the multiple approaches to evaluate equity (eg, fair share approach, rights-based approach, moral rights). It is unclear how to weigh results from different but equally valid equity perspectives. Importantly, it is not just the fact that ADRD interventions can reduce inequalities, but there must be mechanisms in place to ensure all people living with ADRD can access treatment. For example, there are few neurologists in rural America, so this may impact the ability to diagnose and prescribe any new ADRD theraputic.11
Although reimbursement and implementation decisions are not based on a single data point, a key metric in traditional value frameworks is cost per quality-adjusted life years (QALYs). Cost per QALY is also a metric in the value flower framework. Garrison et al and Basu et al note the challenges with using a cost per QALY framework to evaluate ADRD interventions. First, QALYs can be used in a way that places a lower value of life on older people. Second, many ADRD-related interventions yield benefits to the person with ADRD and their caregiver(s). Yet, QALYs are often only evaluated for either the person with ADRD or caregiver, but not both. This poses a challenge in a fragmented payer system like that of the United States, where the person with ADRD and their caregiver(s) may be beneficiaries of different insurance schemes. Third, standard health-related quality of life instruments may not fully capture the benefits of ADRD interventions. Finally, cost per QALY is concerned with maximizing total benefit and ignores the equitable allocation of resources.
The reality is that healthcare payers have budget constraints. Payers must not only navigate funding ADRD care but also must pay for interventions that help people with other costly chronic diseases. Emerging value and subscription payment models hold promise as a way to reimburse for innovative ADRD interventions that may have long-term value. Most value frameworks focus specifically on the initial adoption/funding decision, and have limited mechanisms for evaluating value over time. ADRD is a neurodegenerative disease, and people can live many years with ADRD. Interventions may have long-term effects, but clinical trials of ADRD interventions are only conducted for relatively short time periods. A common theme in all the articles is the need to assess the long-term value of an ADRD intervention.
"There have been tremendous scientific advances in improving ADRD care. It is now time for value frameworks and payment systems to catch up."
Barbarino et al identify data gaps that must be addressed to evaluate the long-term value of ADRD interventions.12 First, many of the emerging ADRD therapeutic candidates will most likely be effective in the early stages of cognitive impairment; yet, payment and clinical policies do not support early screening efforts. Without early detection efforts, we may not be able to identify people who would benefit most from emerging disease-modifying therapeutics. Second, we must better understand the effect of interventions on outcomes (eg, quality of life, independence, productivity) that are meaningful to patients, caregivers, health systems, and society at large using validated measures. Meaningful ADRD-related measures are not systematically collected in administrative data or clinical practice, but they are what people care about and could be used to evaluate the long-term value of an ADRD intervention. Notable examples of incorporating meaningful ADRD outcomes include the application of goal attainment scaling in the context of multidisciplinary, collaborative dementia care models.13
Conclusions
Multiple ADRD therapeutics are in development and may come to market within the next decade. ADRD affects people living with the disease, family/friends, healthcare systems, communities, and society. The value of an intervention likely will vary across these entities. While the societal benefits of an ADRD intervention may be large, all stakeholders or payers may not obtain equal returns. Traditional value frameworks that rely on a cost per QALY approach and a narrow stakeholder perspective are not suited to the unique challenges of ADRD. Value frameworks, which incorporate multiple perspectives, health/brain equity considerations, and long-term evaluation are needed. Innovation in the systematic collection of meaningful ADRD outcomes will facilitate the adoption of new interventions. There have been tremendous scientific advances in improving ADRD care. It is now time for value frameworks and payment systems to catch up. •
Dr Jutkowitz is supported by grants from National Institute on Aging (1R21AG059623-01, 1R01AG060871, and RF1 AG069771).
References
1. O’Connor D. World Alzheimer Report 2019: Attitudes to Dementia. https://globalcoalitiononaging.com/wp-content/uploads/2019/09/WorldAlzheimerReport2019.pdf. Accessed December 1, 2020.
2. 2020 Alzheimer’s disease facts and figures. Alzheimers Dement. 2020 Mar 10 [Online ahead of print.].
3. Wolff JL, Spillman BC, Freedman VA, Kasper JD. A National profile of family and unpaid caregivers who assist older adults with health care activities. JAMA Intern Med. 2016;176(3):372-379.
4. Jutkowitz E, Kane RL, Gaugler JE, MacLehose RF, Dowd B, Kuntz KM. Societal and family lifetime cost of dementia: implications for policy. J Am Geriatr Soc. 2017;65(10):2169-2175.
5. Cummings J, Lee G, Ritter A, Sabbagh M, Zhong K. Alzheimer’s disease drug development pipeline: 2020. Alzheimers Dement Transl Res Clin Interv. 2020;6(1):e12050.
6. Livingston G, Huntley J, Sommerlad A, et al. Dementia prevention, intervention, and care: 2020 report of the Lancet Commission. Lancet. 2020 Aug 8;396(10248):413-446.
7. Nickel F, Barth J, Kolominsky-Rabas PL. Health economic evaluations of non-pharmacological interventions for persons with dementia and their informal caregivers: a systematic review. BMC Geriatr. 2018;18(1):69.
8. Garrison LP Jr, Baumgart M, El-Hayek YH, Holzapfel D, Leibman C. Defining elements of value in Alzheimer’s disease. Value & Outcomes Spotlight. 2021;7(1S):S7-S11.
9. Lakdawalla DN, Doshi JA, Garrison LP, Phelps CE, Basu A, Danzon PM. Defining elements of value in health care—a health economics approach: an ISPOR Special Task Force Report [3]. Value Health. 2018;21(2):131-139.
10. Basu A, Lynn N, Peschin S, Resendez J. Value assessment in Alzheimer’s disease: a focus on equity. Value & Outcomes Spotlight. 2021;7(1S):S12-S17.
11. Lin CC, Callaghan BC, Burke JF, et al. Geographic variation in neurologist density and neurologic care in the United States. Neurology. 2020 Dec 23;10.1212/WNL.0000000000011276.
12. Barbarino P, Gustavsson A, Neumann PJ. Long-term value demonstration in Alzheimer’s disease: evidence needs. Value & Outcomes Spotlight. 2021;7(1S):S18-S23.
13. Jennings LA, Ramirez KD, Hays RD, Wenger NS, Reuben DB. Personalized goal attainment in dementia care: measuring what persons with dementia and their caregivers want. J Am Geriatr Soc. 2018;66(11):2120-2127.
