Costs And Healthcare Resource Utilization In Patients With Transthyretin Amyloid Cardiomyopathy: A Systematic Literature Review
Author(s)
Sandesh Dev, MD1, Jose Nativi-Nicolau, MD2, Louis Lavoie, PhD, MSc3, Marissa Betts, MS4, Zaira Escamilla-Gonzalez, MPH3, Liana Hennum, MHA5, Jean-François Tamby, MD, MBA5, Heather Falvey, MSc6, Richard Wright, MD7, Ahmad Masri, MD8;
1Arizona State University, Scottsdale, AZ, USA, 2Mayo Clinic, Jacksonville, FL, USA, 3Evidera, Montréal, QC, Canada, 4Evidera, Waltham, MA, USA, 5BridgeBio Pharma, Inc., San Francisco, CA, USA, 6BridgeBio Corp, San Francisco, CA, USA, 7Pacific Heart Institute, Santa Monica, CA, USA, 8Oregon Health and Sciences University, Portland, OR, USA
1Arizona State University, Scottsdale, AZ, USA, 2Mayo Clinic, Jacksonville, FL, USA, 3Evidera, Montréal, QC, Canada, 4Evidera, Waltham, MA, USA, 5BridgeBio Pharma, Inc., San Francisco, CA, USA, 6BridgeBio Corp, San Francisco, CA, USA, 7Pacific Heart Institute, Santa Monica, CA, USA, 8Oregon Health and Sciences University, Portland, OR, USA
OBJECTIVES: Transthyretin amyloid cardiomyopathy (ATTR-CM), a progressive disease, can impose a considerable burden on healthcare systems. Systematic literature reviews (SLR) of costs and healthcare resource utilization (HCRU) related to disease severity are lacking.
METHODS: Embase, MEDLINE, CENTRAL, and EconLit were searched for peer-reviewed articles (2013-2023); conference abstracts from 2021-2023 were included. Clinical trials and observational studies reporting on costs and HCRU were included.
RESULTS: Thirty-five publications, including 1 clinical trial and 32 observational studies, were included. Evidence mainly described direct healthcare costs. Total direct costs (2022 United States [US] dollars) to public healthcare systems reported per patient per year (PPPY) were $1,365-$22,600 (depending on disease severity) in the United Kingdom (UK) and $25,371 in South Korea (KR). In the US, out-of-pocket costs for ATTR-CM were estimated at $16,488 PPPY in 2021-2022. Two studies showed that direct costs increased with New York Heart Association (NYHA) class disease severity. Hospitalization was the main driver of direct costs, accounting for 87% in patients from 1 UK hospital; 47% and 88% in patients with NYHA class II and IV ATTR-CM, respectively, from 2 clinics in Sweden; and 93% of costs in patients in KR. All-cause hospitalization rates were 1.5 (Brazil) to 4.6 (KR) PPPY and length of stay was 3.1 (US) to 5.7 (KR) days. The most used procedures across studies at baseline/with follow-up of 1.5-3.5 years were echocardiograms (100%), bone scintigraphy (47-99%), and magnetic resonance imaging (48-50%). The most used drugs at baseline/with follow-up of 7.8-39 months were diuretics (40-90%), anticoagulants (39-85%), and beta-blockers (44-71%).
CONCLUSIONS: This SLR suggests that hospitalization costs are a main driver of healthcare costs in the ATTR-CM population. The economic impact of ATTR-CM may be underestimated due to insufficient literature describing indirect costs. Early diagnosis and treatment may reduce hospitalizations and lower the burden on healthcare systems.
METHODS: Embase, MEDLINE, CENTRAL, and EconLit were searched for peer-reviewed articles (2013-2023); conference abstracts from 2021-2023 were included. Clinical trials and observational studies reporting on costs and HCRU were included.
RESULTS: Thirty-five publications, including 1 clinical trial and 32 observational studies, were included. Evidence mainly described direct healthcare costs. Total direct costs (2022 United States [US] dollars) to public healthcare systems reported per patient per year (PPPY) were $1,365-$22,600 (depending on disease severity) in the United Kingdom (UK) and $25,371 in South Korea (KR). In the US, out-of-pocket costs for ATTR-CM were estimated at $16,488 PPPY in 2021-2022. Two studies showed that direct costs increased with New York Heart Association (NYHA) class disease severity. Hospitalization was the main driver of direct costs, accounting for 87% in patients from 1 UK hospital; 47% and 88% in patients with NYHA class II and IV ATTR-CM, respectively, from 2 clinics in Sweden; and 93% of costs in patients in KR. All-cause hospitalization rates were 1.5 (Brazil) to 4.6 (KR) PPPY and length of stay was 3.1 (US) to 5.7 (KR) days. The most used procedures across studies at baseline/with follow-up of 1.5-3.5 years were echocardiograms (100%), bone scintigraphy (47-99%), and magnetic resonance imaging (48-50%). The most used drugs at baseline/with follow-up of 7.8-39 months were diuretics (40-90%), anticoagulants (39-85%), and beta-blockers (44-71%).
CONCLUSIONS: This SLR suggests that hospitalization costs are a main driver of healthcare costs in the ATTR-CM population. The economic impact of ATTR-CM may be underestimated due to insufficient literature describing indirect costs. Early diagnosis and treatment may reduce hospitalizations and lower the burden on healthcare systems.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
EE461
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
SDC: Cardiovascular Disorders (including MI, Stroke, Circulatory), SDC: Rare & Orphan Diseases