Challenges in demonstrating the value of disease-modifying therapies for Alzheimer’s disease

References

• Anderson G, Oderkirk J. Dementia research and care: can big data help? OECD Publishing, Paris. 2015.
   Google Scholar
• 2020 Alzheimer’s disease facts and figures. Alzheimer’s Dement [Internet]. 2020. 16:391–460. [cited 2020 May 21]. Available from: https://onlinelibrary.wiley.com/doi/abs/10.1002/alz.12068.
   Google Scholar
• Saha S, Gerdtham U-G, Toresson H, et al. Economic evaluation of interventions for screening of dementia. Work Pap. 2018;
   Google Scholar
• Xu J, Zhang Y, Qiu C, et al. Global and regional economic costs of dementia: a systematic review. Lancet. 2017;390:S47.
   Web of Science ®Google Scholar
• Wimo A, Handels R, Winblad B, et al. Quantifying and describing the natural history and costs of alzheimer’s disease and effects of hypothetical interventions. Michalowsky B, editor. J Alzheimer’s Dis. Internet]. 2020 ;1–12. [cited 2020 May 21]. Available from: https://www.medra.org/servlet/aliasResolver?alias=iospress&doi=10.3233/JAD-191055
   Web of Science ®Google Scholar
• Winblad B, Amouyel P, Andrieu S, et al. Defeating alzheimer’s disease and other dementias: A priority for European science and society. Lancet Neurol. 2016;15(5):455–532. .
   PubMed Web of Science ®Google Scholar
• Reed C, Happich M, Raskin J, et al. Utilization of observational data as a proxy cohort for comparison purposes with open-label study results: an example from alzheimer’s disease. J Prev Alzheimer’s Dis. 2019;6:90–99.
   PubMed Web of Science ®Google Scholar
• Green C, Shearer J, Ritchie CW, et al. Model-based economic evaluation in alzheimer’s disease: A review of the methods available to model alzheimer’s disease progression. Value Heal [Internet]. 2011;14:621–630. .
   Google Scholar
• Pouryamout L, Dams J, Wasem J, et al. Economic evaluation of treatment options in patients with alzheimers disease: A systematic review of cost-effectiveness analyses. Drugs. 2012;72(6):789–802. .
   PubMed Web of Science ®Google Scholar
• Hernandez L, Ozen A, DosSantos R, et al. Systematic review of model-based economic evaluations of treatments for alzheimer’s disease. Pharmacoeconomics. 2016;34(7):681–707. .
   PubMed Web of Science ®Google Scholar
• Nguyen KH, Comans TA, Green C. Where are we at with model-based economic evaluations of interventions for dementia? a systematic review and quality assessment. Int Psychogeriatrics. 2018;30(11):1593–1605.
   PubMed Web of Science ®Google Scholar
• Green C, Handels R, Gustavsson A, et al. Assessing cost-effectiveness of early intervention in alzheimer’s disease: an open-source modeling framework. Alzheimer’s Dement. 2019;15(10):1309–1321. .
   PubMed Web of Science ®Google Scholar
• Ebrahem AS, Oremus M. A pharmacoeconomic evaluation of cholinesterase inhibitors and memantine for the treatment of alzheimer’s disease. Expert Opin Pharmacother. 2018;19(11):1245–1259.
   PubMed Web of Science ®Google Scholar
• Aisen PS, Cummings J, Jack CR, et al. On the path to 2025: understanding the alzheimer’s disease continuum. Alzheimer’s Res Ther. 2017; 9(1). DOI: 10.1186/s13195-017-0283-5.
   PubMed Web of Science ®Google Scholar
• Vermunt L, Sikkes SAM, van den Hout A, et al. Duration of preclinical, prodromal, and dementia stages of alzheimer’s disease in relation to age, sex, and APOE genotype. Alzheimer’s Dement. 2019;15(7):888–898.
   PubMed Web of Science ®Google Scholar
• Samtani MN, Raghavan N, Novak G, et al. Disease progression model for clinical dementia rating–sum of boxes in mild cognitive impairment and alzheimer’s subjects from the alzheimer’s disease neuroimaging initiative. Neuropsychiatr Dis Treat;2014. 929. DOI: 10.2147/NDT.S62323
   PubMed Web of Science ®Google Scholar
• Balsis S, Benge JF, Lowe DA, et al. How do scores on the ADAS-Cog, MMSE, and CDR-SOB correspond? Clin Neuropsychol. 2015;29(7):1002–1009.
   PubMed Web of Science ®Google Scholar
• Aisen PS. Cognitive/clinical endpoints for pre-dementia AD trials. J Prev Alzheimer’s Dis. 2015;2(2):82–84.
   Google Scholar
• Cohen JT, Neumann PJ. Decision analytic models for alzheimer’s disease: state of the art and future directions. Alzheimer’s Dement. 2008;4(3):212–222.
   PubMed Web of Science ®Google Scholar
• FDA. Guidance for industry alzheimer’s disease: developing drugs for the treatment of early stage disease. 2013;1–7. Available from: http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm.
   Google Scholar
• O’Bryant SE, Lacritz LH, Hall J, et al. Validation of the new interpretive guidelines for the clinical dementia rating scale sum of boxes score in the National alzheimer’s coordinating center database. Arch Neurol. 2010;67(6):746–749.
   Google Scholar
• Andrews JS, Desai U, Kirson NY, et al. Disease severity and minimal clinically important differences in clinical outcome assessments for ALZHEIMER’S disease clinical trials. Alzheimer’s Dement Transl Res Clin Interv. 2019;5(1):354–363. .
   PubMedGoogle Scholar
• Escudero JMS, Beltrán J, Á P, et al. Neuropsychiatric symptoms as predictors of clinical course in neurodegeneration. A longitudinal study. Front Aging Neurosci. 2019;11:176.
   Web of Science ®Google Scholar
• Kansal AR, Tafazzoli A, Ishak KJ, et al. Alzheimer’s disease archimedes condition-event simulator: development and validation. Alzheimer’s Dement Transl Res Clin Interv. 2018;4(1):76–88. .
   PubMedGoogle Scholar
• Zhang L, Lim CY, Maiti T, et al. Analysis of conversion of alzheimer’s disease using a multi-state Markov model. Stat Methods Med Res. 2019;28(9):2801–2819. .
   Web of Science ®Google Scholar
• Cummings J. Lessons learned from alzheimer disease: clinical trials with negative outcomes. Clin. Transl. Sci. 2018;11(2):147–152.
   PubMed Web of Science ®Google Scholar
• Makin S. The amyloid hypothesis on trial. Nature. 2018;559(7715):S4-S7.
   PubMed Web of Science ®Google Scholar
• Selkoe DJ, Hardy J. The amyloid hypothesis of alzheimer’s disease at 25 years. EMBO Mol Med. 2016;8(6):595–608.
   PubMed Web of Science ®Google Scholar
• Lansdall CJ. An effective treatment for alzheimer’s disease must consider both amyloid and tau. Biosci. Horizons. 2014;7:hzu002-hzu002.
   Google Scholar
• Sperling RA, Jack CR, Aisen PS. Testing the right target and right drug at the right stage. Sci Transl Med. 2011;3(111): 111cm33.
   Web of Science ®Google Scholar
• Vos SJB, Verhey F, Frölich L, et al. Prevalence and prognosis of alzheimer’s disease at the mild cognitive impairment stage. Brain. 2015;138(5):1327–1338. .
   Web of Science ®Google Scholar
• Rizzuto D, Bellocco R, Kivipelto M, et al. Dementia after age 75: survival in different severity stages and years of life lost. Curr Alzheimer Res. 2012;9(7):795–800. .
   PubMed Web of Science ®Google Scholar
• Wilson RS, Aggarwal NT, Barnes LL, et al. Biracial population study of mortality in mild cognitive impairment and alzheimer disease. Arch Neurol. 2009;66(6). DOI:10.1001/archneurol.2009.80.
   Google Scholar
• Gasper MC, Ott BR, Lapane KL. Is donepezil therapy associated with reduced mortality in nursing home residents with dementia? Am J Geriatr Pharmacother. 2005;3(1):1–7.
   PubMedGoogle Scholar
• Guo S, Getsios D, Revankar N, et al. Evaluating disease-modifying agents: a simulation framework for alzheimer’s disease. Pharmacoeconomics. 2014;32(11):1129–1139. .
   Web of Science ®Google Scholar
• Skoldunger A, Johnell K, Winblad B, et al. Mortality and treatment costs have a great impact on the cost- effectiveness of disease modifying treatment in alzheimer’s disease - a simulation study. Curr Alzheimer Res. 2013;10(2):207–216. .
   PubMed Web of Science ®Google Scholar
• Xie J, Brayne C, Matthews FE. Survival times in people with dementia: analysis from population based cohort study with 14 year follow-up. BMJ. 2008;336(7638):258–262.
   PubMed Web of Science ®Google Scholar
• Helzner EP, Scarmeas N, Cosentino S, et al. Survival in alzheimer disease: A multiethnic, population-based study of incident cases. Neurology. 2008;71(19):1489–1495. .
   PubMed Web of Science ®Google Scholar
• Eldon Spackman D, Kadiyala S, Neumann P, et al. Measuring alzheimer disease progression with transition probabilities: estimates from NACC-UDS. Curr Alzheimer Res. 2012;9(9):1050–1058. .
   PubMed Web of Science ®Google Scholar
• Gustavsson A, Green C, Jones RW, et al. Current issues and future research priorities for health economic modelling across the full continuum of alzheimer’s disease. Alzheimer’s Dement. 2017;13(3):312–321. .
   PubMed Web of Science ®Google Scholar
• Janssen O, Vos SJB, García-Negredo G, et al., Real-world evidence in alzheimer’s disease: the ROADMAP data cube. Alzheimers Dement. 16(3): 461–471. 2020. .
   Web of Science ®Google Scholar
• Makady A, Ham RT, de Boer A, et al. Policies for use of real-world data in health technology assessment (HTA): a comparative study of six HTA agencies. Value Heal. 2017;
   Google Scholar
• Bouvy JC, Jonsson P, O’Rourke D, et al. Regulatory and health technology assessment considerations for disease-modifying drugs in alzheimer’s disease. CNS Drugs. 2018;32(12):1085–1090.
   PubMed Web of Science ®Google Scholar
• Gallacher J, De Vulpillieres FDR, Amzal B, et al. Challenges for optimizing real-world evidence in alzheimer’s disease: the ROADMAP project. J Alzheimer’s Dis. 2019;67(2):495–501. .
   PubMed Web of Science ®Google Scholar
• Wimo A, Guerchet M, Ali GC, et al., The worldwide costs of dementia 2015 and comparisons with 2010. Alzheimer’s Dement. 13(1): 1–7. 2017
   Web of Science ®Google Scholar
• Wimo A, Ballard C, Brayne C, et al. Health economic evaluation of treatments for alzheimer′s disease: impact of new diagnostic criteria. J Intern Med. 2014;275(3):304–316. .
   PubMed Web of Science ®Google Scholar
• Landeiro F, Wace H, Ghinai I, et al. Resource utilisation and costs in predementia and dementia: A systematic review protocol. BMJ Open. 2018;8(1):e019060. .
   Web of Science ®Google Scholar
• Deb A, Thornton JD, Sambamoorthi U, et al. Direct and indirect cost of managing alzheimer’s disease and related dementias in the United States. Expert Rev Pharmacoecon Outcomes Res. 2017;17(2):189–202. Taylor and Francis Ltd. .
   PubMed Web of Science ®Google Scholar
• Brüggenjürgen B, Andersohn F, Ezzat N, et al. Medical management, costs, and consequences of alzheimer’s disease in Germany: an analysis of health claims data. J Med Econ. 2015;18(6):466–473. .
   PubMed Web of Science ®Google Scholar
• El-Hayek YH, Wiley RE, Khoury CP, et al. Tip of the iceberg: assessing the global socioeconomic costs of alzheimer’s disease and related dementias and strategic implications for stakeholders. J Alzheimer’s Dis. 2019;70:321–339.
   Web of Science ®Google Scholar
• Krol M, Brouwer W. How to estimate productivity costs in economic evaluations. Pharmacoeconomics. 2014;32(4):335–344.
   PubMed Web of Science ®Google Scholar
• Aston L, Hilton A, Moutela T, et al. Exploring the evidence base for how people with dementia and their informal carers manage their medication in the community: A mixed studies review. BMC Geriatr. 2017;17(1). DOI: 10.1186/s12877-017-0638-6.
   PubMed Web of Science ®Google Scholar
• Afram B, Stephan A, Verbeek H, et al. Reasons for institutionalization of people with dementia: informal caregiver reports from 8 European countries. J Am Med Dir Assoc. 2014;15(2):108–116.
   PubMed Web of Science ®Google Scholar
• Dreier-Wolfgramm A, Michalowsky B, Austrom MG, et al. Dementia care management in primary care : current collaborative care models and the case for interprofessional education. Z Gerontol Geriatr. 2017;50(S2):68–77. .
   PubMed Web of Science ®Google Scholar
• Association A. Changing the trajectory of alzheimer’s disease: how a treatment by 2025 saves lives and dollars. Alzheimer’s Dement. 2013;9:208–245.
   PubMed Web of Science ®Google Scholar
• Babiarz P, Yilmazer T. The impact of adverse health events on consumption: understanding the mediating effect of income transfers, wealth, and health insurance. Heal Econ. 2017;26(12):1743–1758.
   PubMed Web of Science ®Google Scholar
• World Health Organization. Who guide to identifying the economic consequences of disease and injury. 2009;
   Google Scholar
• Kotsopoulos N, Connolly MP. Is the gap between micro- and macroeconomic assessments in health care well understood? The case of vaccination and potential remedies. J Mark Heal. 2014;2(1):23897.
   Google Scholar
• Attema AE, Brouwer WBF, Claxton K. Discounting in economic evaluations. Pharmacoeconomics. 2018;36(7):745–758.
   PubMed Web of Science ®Google Scholar