REAL-WORLD CLINICAL, SAFETY AND ECONOMIC OUTCOMES OF ACORAMIDIS UNDER AN EARLY ACCESS PROGRAM: A PHARMACIST-LED EVALUATION FROM A PORTUGUESE NHS REFERENCE CENTRE
Author(s)
Tânia S. Laranjeira, PharmD1, Marina Alves, PharmD1, Carlos Aguiar, MD2, Bruno Rocha, MD2, Ana Mirco, PharmD1.
1Pharmaceutical Services, ULS Lisboa Ocidental, Lisboa, Portugal, 2Cardiomyopathy Clinic, Advanced Heart Failure and Heart Transplantation Department, ULS Lisboa Ocidental, Lisboa, Portugal.
1Pharmaceutical Services, ULS Lisboa Ocidental, Lisboa, Portugal, 2Cardiomyopathy Clinic, Advanced Heart Failure and Heart Transplantation Department, ULS Lisboa Ocidental, Lisboa, Portugal.
OBJECTIVES: The acoramidis Early Access Program (EAP) was approved in Portugal for the treatment of wild-type or variant transthyretin amyloid cardiomyopathy (ATTR-CM) in adult patients with disease progression despite tafamidis, or with intolerance or contraindication to this therapy. This study aimed to characterise the eligible population and access pathway at the highest-enrolling Portuguese centre and to evaluate real-world effectiveness, safety, cost avoidance and adherence.
METHODS: Retrospective, single-centre observational study including all patients initiating acoramidis under the national EAP between June 2025 and January 2026. Data were extracted from electronic health records and the outpatient pharmacy dispensing system. NYHA class, NT-proBNP and estimated Glomerular Filtration Rate (eGFR) were collected at baseline and most recent follow-up. Hospital pharmacists performed structured pharmacovigilance during dispensing visits. Adherence was measured using Proportion of Days Covered (PDC); persistence was defined as continuous treatment until progression, death, or transition to reimbursed access. Cost avoidance was estimated based on drug cost approved after reimbursement and duration of EAP.
RESULTS: Seven patients initiated acoramidis (5 male; median age 83 years), all with wild type ATTR-CM. Median follow-up was 6 months. Eligibility was driven by tafamidis contraindication due to severe eGFR reduction (6/7) and disease progression despite tafamidis (1/7). NYHA class improved in 6/7 and remained stable in 1/7. NT-proBNP changes were heterogeneous, likely reflecting renal decline and short follow-up. No cardiovascular hospitalisations or deaths occurred. One non-serious adverse event resolved without discontinuation. Median PDC was 100% (range 91-100%), with 100% persistence. NHS drug cost avoidance reached €188,446 (mean €26,921/patient).
CONCLUSIONS: In this EAP cohort, representing the largest national experience, acoramidis showed favourable functional outcomes and manageable safety profile in a predominantly renally impaired population otherwise ineligible for tafamidis. The EAP generated substantial cost avoidance while enabling timely access pre-reimbursement access, highlighting its dual role in access and early real-world evidence generation.
METHODS: Retrospective, single-centre observational study including all patients initiating acoramidis under the national EAP between June 2025 and January 2026. Data were extracted from electronic health records and the outpatient pharmacy dispensing system. NYHA class, NT-proBNP and estimated Glomerular Filtration Rate (eGFR) were collected at baseline and most recent follow-up. Hospital pharmacists performed structured pharmacovigilance during dispensing visits. Adherence was measured using Proportion of Days Covered (PDC); persistence was defined as continuous treatment until progression, death, or transition to reimbursed access. Cost avoidance was estimated based on drug cost approved after reimbursement and duration of EAP.
RESULTS: Seven patients initiated acoramidis (5 male; median age 83 years), all with wild type ATTR-CM. Median follow-up was 6 months. Eligibility was driven by tafamidis contraindication due to severe eGFR reduction (6/7) and disease progression despite tafamidis (1/7). NYHA class improved in 6/7 and remained stable in 1/7. NT-proBNP changes were heterogeneous, likely reflecting renal decline and short follow-up. No cardiovascular hospitalisations or deaths occurred. One non-serious adverse event resolved without discontinuation. Median PDC was 100% (range 91-100%), with 100% persistence. NHS drug cost avoidance reached €188,446 (mean €26,921/patient).
CONCLUSIONS: In this EAP cohort, representing the largest national experience, acoramidis showed favourable functional outcomes and manageable safety profile in a predominantly renally impaired population otherwise ineligible for tafamidis. The EAP generated substantial cost avoidance while enabling timely access pre-reimbursement access, highlighting its dual role in access and early real-world evidence generation.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE128
Topic
Clinical Outcomes, Economic Evaluation, Real World Data & Information Systems
Topic Subcategory
Budget Impact Analysis
Disease
Cardiovascular Disorders (including MI, Stroke, Circulatory), Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)