THE POTENTIAL BURDEN OF ATTR-CM IN PORTUGAL: A GLOBAL AND PER FUNCTIONAL CLASS COST-OF-ILLNESS ANALYSIS
Author(s)
Débora Figueira, PharmD1, Rita Meireles, PharmD1, Olga Azevedo, MD2, Patricia Rodrigues, MD3, Nádia Moreira, MD4, João Agostinho, MD5, Silvia Aguiar, MD6, Bruno Rocha, MD7, Cátia Mota, PharmD8, Margarida Borges, MD1.
1IQVIA Portugal, Porto Salvo - Oeiras, Portugal, 2ULS do Alto Ave, Guimarães, Portugal, 3ULS de Santo António, Porto, Portugal, 4ULS Coimbra, Coimbra, Portugal, 5ULS de Santa Maria, Lisbon, Portugal, 6ULS de São José, Lisbon, Portugal, 7ULS Lisboa Ocidental, Lisbon, Portugal, 8Pfizer, LISBON, Portugal.
1IQVIA Portugal, Porto Salvo - Oeiras, Portugal, 2ULS do Alto Ave, Guimarães, Portugal, 3ULS de Santo António, Porto, Portugal, 4ULS Coimbra, Coimbra, Portugal, 5ULS de Santa Maria, Lisbon, Portugal, 6ULS de São José, Lisbon, Portugal, 7ULS Lisboa Ocidental, Lisbon, Portugal, 8Pfizer, LISBON, Portugal.
OBJECTIVES: Transthyretin amyloid cardiomyopathy (ATTR-CM) is a rare, progressive and life-threatening disease associated with a substantial clinical burden and healthcare resource use. This study aimed to estimate the prevalence and economic burden of ATTR-CM in Portugal by New York Heart Association (NYHA) class.
METHODS: A cost-of-illness analysis was conducted from a societal perspective and a one-year time horizon. Prevalence was estimated using epidemiological assumptions validated by clinical experts. Inpatient costs were derived from the Portuguese Hospital Morbidity Database and valued at the prices defined by Ordinance No. 207/2007, in its last version. Outpatient costs including diagnosis, treatment, follow-up, and supportive care were estimated through an expert panel of six cardiologists experienced in ATTR-CM management. Unit costs were obtained from official national sources (IQVIA, INFARMED, and applicable legislation).
RESULTS: In Portugal is estimated that there are 1,826 diagnosed patients (1.78 cases per 10,000 inhabitants) with ATTR-CM. The mean diagnostic cost was €2,124.48 per patient. Among patients receiving disease-modifying therapies, mean annual treatment costs were €34,258.92 in NYHA I-II and €7,306.28 in NYHA III per patient, with follow-up costs ranging from €1,008.92 to €2,668.70, respectively. In patients not receiving disease-modifying therapies, treatment costs ranged from €1,875.55 in NYHA I-II to €2,698.22 in NYHA III per patient. Follow-up costs increased with disease severity, from €1,070.20 (NYHA I-II) to €3,897.27 (NYHA III). Supportive care costs across the overall patient population reached €2,752.02 and €5,270.79 per patient in NYHA III and IV, respectively. Considering the estimated number of diagnosed patients, the total annual economic burden was approximately €49,3 million.
CONCLUSIONS: ATTR-CM represents a significant clinical and economic burden in Portugal. Although disease-modifying therapies account for most costs, their use may prevent disease-related events, with greater impact in advanced disease (NYHA class III). Early diagnosis and timely treatment access may maximize health gains and optimize resource allocation.
Funding: Pfizer
METHODS: A cost-of-illness analysis was conducted from a societal perspective and a one-year time horizon. Prevalence was estimated using epidemiological assumptions validated by clinical experts. Inpatient costs were derived from the Portuguese Hospital Morbidity Database and valued at the prices defined by Ordinance No. 207/2007, in its last version. Outpatient costs including diagnosis, treatment, follow-up, and supportive care were estimated through an expert panel of six cardiologists experienced in ATTR-CM management. Unit costs were obtained from official national sources (IQVIA, INFARMED, and applicable legislation).
RESULTS: In Portugal is estimated that there are 1,826 diagnosed patients (1.78 cases per 10,000 inhabitants) with ATTR-CM. The mean diagnostic cost was €2,124.48 per patient. Among patients receiving disease-modifying therapies, mean annual treatment costs were €34,258.92 in NYHA I-II and €7,306.28 in NYHA III per patient, with follow-up costs ranging from €1,008.92 to €2,668.70, respectively. In patients not receiving disease-modifying therapies, treatment costs ranged from €1,875.55 in NYHA I-II to €2,698.22 in NYHA III per patient. Follow-up costs increased with disease severity, from €1,070.20 (NYHA I-II) to €3,897.27 (NYHA III). Supportive care costs across the overall patient population reached €2,752.02 and €5,270.79 per patient in NYHA III and IV, respectively. Considering the estimated number of diagnosed patients, the total annual economic burden was approximately €49,3 million.
CONCLUSIONS: ATTR-CM represents a significant clinical and economic burden in Portugal. Although disease-modifying therapies account for most costs, their use may prevent disease-related events, with greater impact in advanced disease (NYHA class III). Early diagnosis and timely treatment access may maximize health gains and optimize resource allocation.
Funding: Pfizer
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE617
Topic
Clinical Outcomes, Economic Evaluation, Epidemiology & Public Health
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Cardiovascular Disorders (including MI, Stroke, Circulatory), Geriatrics, No Additional Disease & Conditions/Specialized Treatment Areas