A SYSTEMATIC LITERATURE REVIEW OF THE ECONOMIC AND HUMANISTIC BURDEN OF TRANSTHYRETIN AMYLOID CARDIOMYOPATHY (ATTR-CM)
Author(s)
Jaydeep Das, BSc, MSc1, Silvia Capucci, MSc2, Musku Kumaraswamy, M.Pharm3, Sunita Nair, PhD4.
1Novo Nordisk A/S, Bangalore, India, 2Novo Nordisk A/S, Søborg, Denmark, 3Clarivate, Bangalore, India, 4Clarivate, Mumbai, India.
1Novo Nordisk A/S, Bangalore, India, 2Novo Nordisk A/S, Søborg, Denmark, 3Clarivate, Bangalore, India, 4Clarivate, Mumbai, India.
OBJECTIVES: We conducted a systematic literature review to understand treatment costs/cost-effectiveness and drivers of economic and humanistic burden (patient-reported outcomes [PROs]) of transthyretin amyloid cardiomyopathy (ATTR-CM) by subtype, functional class, treatment status, and geography.
METHODS: Embase, MEDLINE®, the Cochrane Library, and EconLit were searched in February 2026, from database inception, and grey literature from 2023, to identify economic evaluations, and data on cost, resource use, and PROs in ATTR-CM.
RESULTS: Eighty-one studies were identified: 9 cost-effectiveness analyses (CEAs), 43 cost/resource-use studies, 22 reporting PROs, and 7 on both cost/resource use and PROs. The CEAs showed that, while disease-modifying therapies (e.g. tafamidis and transthyretin-silencing agents) improve survival (by 1.60-3.88 life-years) and quality of life (by 0.06-3.54 quality-adjusted life-years) versus symptomatic care, they were not cost-effective at accepted thresholds in Brazil (n=1), Italy (n=1), the USA (n=6), and multi-nationally (n=1). Cost-effectiveness was highly sensitive to drug acquisition costs (reported heterogeneously as monthly, annual, or over the treatment/trial period in different studies), as well as patient age and disease prevalence. Cost and resource use were primarily driven by the costs of disease-modifying therapy (two studies, ~245,000-287,000 USD/patient/year), hospitalisations (18,271-51,627 USD/cardiovascular-related admission), and disease progression, associated with complications and comorbidities. Early diagnosis and treatment were identified as a factor that could reduce the burden on healthcare systems. Kansas City Cardiomyopathy Questionnaire (KCCQ) overall summary scores showed a decline with increased symptom severity (~80 for New York Heart Association class [NYHA] I; ≥48 for NYHA class IV). Long-term treatment generally stabilised or improved KCCQ.
CONCLUSIONS: ATTR-CM imposes substantial economic and humanistic burden, driven by hospitalisation, drug costs, disease progression, and reduced quality of life. Current treatments are generally not cost-effective at accepted thresholds, underscoring the need for therapies that improve outcomes, lower resource use, and provide acceptable value.
METHODS: Embase, MEDLINE®, the Cochrane Library, and EconLit were searched in February 2026, from database inception, and grey literature from 2023, to identify economic evaluations, and data on cost, resource use, and PROs in ATTR-CM.
RESULTS: Eighty-one studies were identified: 9 cost-effectiveness analyses (CEAs), 43 cost/resource-use studies, 22 reporting PROs, and 7 on both cost/resource use and PROs. The CEAs showed that, while disease-modifying therapies (e.g. tafamidis and transthyretin-silencing agents) improve survival (by 1.60-3.88 life-years) and quality of life (by 0.06-3.54 quality-adjusted life-years) versus symptomatic care, they were not cost-effective at accepted thresholds in Brazil (n=1), Italy (n=1), the USA (n=6), and multi-nationally (n=1). Cost-effectiveness was highly sensitive to drug acquisition costs (reported heterogeneously as monthly, annual, or over the treatment/trial period in different studies), as well as patient age and disease prevalence. Cost and resource use were primarily driven by the costs of disease-modifying therapy (two studies, ~245,000-287,000 USD/patient/year), hospitalisations (18,271-51,627 USD/cardiovascular-related admission), and disease progression, associated with complications and comorbidities. Early diagnosis and treatment were identified as a factor that could reduce the burden on healthcare systems. Kansas City Cardiomyopathy Questionnaire (KCCQ) overall summary scores showed a decline with increased symptom severity (~80 for New York Heart Association class [NYHA] I; ≥48 for NYHA class IV). Long-term treatment generally stabilised or improved KCCQ.
CONCLUSIONS: ATTR-CM imposes substantial economic and humanistic burden, driven by hospitalisation, drug costs, disease progression, and reduced quality of life. Current treatments are generally not cost-effective at accepted thresholds, underscoring the need for therapies that improve outcomes, lower resource use, and provide acceptable value.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE13
Topic
Economic Evaluation, Patient-Centered Research
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Cardiovascular Disorders (including MI, Stroke, Circulatory)