Patterns of Treatment Adherence and Administration Preferences for People With Transthyretin Amyloid Cardiomyopathy: A Real-World Survey in Europe, the United States, and Japan
Author(s)
Sanjana Chandrasekar, MPH1, Jack Wright, MSc2, Jade Garratt-Wheeldon, BSc2, Janice Chung, MPH1, Mónica Sofia Inês, MSc PhD3, Christine L. Baker, MBA MPH JD1, Ann Willet, MBA PhamrD1, Pedro Ferreira Branquinho, Postgrad3, Feng Dai, PhD1, Alisha Braid, BSc2, Brianne Kerr, BSc2.
1Pfizer, New York, NY, USA, 2Adelphi Real World, Bollington, United Kingdom, 3Pfizer, Porto Salvo, Portugal.
1Pfizer, New York, NY, USA, 2Adelphi Real World, Bollington, United Kingdom, 3Pfizer, Porto Salvo, Portugal.
OBJECTIVES: Describe treatment patterns among transthyretin amyloid cardiomyopathy (ATTR-CM) patients in a real-world setting.
METHODS: Secondary analyses using data from the Adelphi ATTR Disease Specific ProgrammeTM, a cross-sectional survey of physicians and their patients in Europe (France, Germany, Italy, Spain, and the UK), the US, and Japan, August 2024-February 2025. Disease modifying therapies (DMTs) included transthyretin stabilisers and silencers. Analyses were descriptive.
RESULTS: Overall, 226 physicians provided data for 1006 patients (n=630 Europe, n=256 US, n=120 Japan) and 279 patients self-reported data. Mean (SD) patient age was 70.4 (12.8) years, 70% were male, 80% were White, and 15% had a cardiomyopathy-neuropathy phenotype. At survey, 73% of patients were prescribed treatment, of which 93% were prescribed DMTs including tafamidis (85%), patisiran (6%), vutrisiran (2%) and diflunisal (2%). Overall, 24% had never received treatment, reasons included patient refusal (30%), waiting to start their first regimen (30%), or ineligibility (25%). Physicians reported that 83% of patients were ‘completely’ adherent with treatment. Reasons for lack of adherence included patients forgetting to take their treatment (69%), administration difficulties (12%), and side effects (10%). Overall, 65% of patients reported they were ‘completely’ comfortable with a once-daily oral medication, 18% were ‘somewhat’ comfortable. For twice-daily orals, 50% were ‘completely’ comfortable (18% ‘somewhat’). For injections, 15% were ‘completely’ comfortable (28% ‘somewhat’) with hospital/clinic administration,13% were ‘completely’ comfortable (32% ‘somewhat’) with at-home healthcare professional administration, and 14% were ‘completely’ comfortable (26% ‘somewhat’) with at-home self-administration. For intravenous infusions, 10% were ‘completely’ (15% ‘somewhat’) with hospital/clinic administration, 9% were ‘completely’ comfortable (16% ‘somewhat’) with at-home healthcare professional administration.
CONCLUSIONS: Tafamidis was the most frequently prescribed DMT, and adherence was high. Patients more frequently reported feeling comfortable with oral medications than injections/infusions, particularly once daily orals, underscoring the relevance of administration preferences among treatment landscapes. Further research is needed to explore outcomes with ATTR treatments.
METHODS: Secondary analyses using data from the Adelphi ATTR Disease Specific ProgrammeTM, a cross-sectional survey of physicians and their patients in Europe (France, Germany, Italy, Spain, and the UK), the US, and Japan, August 2024-February 2025. Disease modifying therapies (DMTs) included transthyretin stabilisers and silencers. Analyses were descriptive.
RESULTS: Overall, 226 physicians provided data for 1006 patients (n=630 Europe, n=256 US, n=120 Japan) and 279 patients self-reported data. Mean (SD) patient age was 70.4 (12.8) years, 70% were male, 80% were White, and 15% had a cardiomyopathy-neuropathy phenotype. At survey, 73% of patients were prescribed treatment, of which 93% were prescribed DMTs including tafamidis (85%), patisiran (6%), vutrisiran (2%) and diflunisal (2%). Overall, 24% had never received treatment, reasons included patient refusal (30%), waiting to start their first regimen (30%), or ineligibility (25%). Physicians reported that 83% of patients were ‘completely’ adherent with treatment. Reasons for lack of adherence included patients forgetting to take their treatment (69%), administration difficulties (12%), and side effects (10%). Overall, 65% of patients reported they were ‘completely’ comfortable with a once-daily oral medication, 18% were ‘somewhat’ comfortable. For twice-daily orals, 50% were ‘completely’ comfortable (18% ‘somewhat’). For injections, 15% were ‘completely’ comfortable (28% ‘somewhat’) with hospital/clinic administration,13% were ‘completely’ comfortable (32% ‘somewhat’) with at-home healthcare professional administration, and 14% were ‘completely’ comfortable (26% ‘somewhat’) with at-home self-administration. For intravenous infusions, 10% were ‘completely’ (15% ‘somewhat’) with hospital/clinic administration, 9% were ‘completely’ comfortable (16% ‘somewhat’) with at-home healthcare professional administration.
CONCLUSIONS: Tafamidis was the most frequently prescribed DMT, and adherence was high. Patients more frequently reported feeling comfortable with oral medications than injections/infusions, particularly once daily orals, underscoring the relevance of administration preferences among treatment landscapes. Further research is needed to explore outcomes with ATTR treatments.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HSD84
Topic
Health Service Delivery & Process of Care, Patient-Centered Research
Disease
Cardiovascular Disorders (including MI, Stroke, Circulatory), Rare & Orphan Diseases