A Real-World Survey of Healthcare Resource Utilization Treatment Usage and Unmet Need for Hemophilia Patients in Spain
Author(s)
Sheena Thakkar, BS, MPH1, Carmen Peral, M2, Irene francisco, M3, Patricia Tarilonte, M4, Rabiyah Sahar, M5, Chris Blazos, M6, Sophie Lai, M6, Ella Morton, M6, Nathan Ball, M6.
1Director, Value & Evidence, Pfizer Inc., Cambridge, MA, USA, 2Pfizer, MADRID, Spain, 3Pfizer. Inc, Madrid, Spain, 4Pfizer Inc., Alcobendas - Madrid, Spain, 5Adelphi Real World, London, United Kingdom, 6Adelphi Real World, Bollington, United Kingdom.
1Director, Value & Evidence, Pfizer Inc., Cambridge, MA, USA, 2Pfizer, MADRID, Spain, 3Pfizer. Inc, Madrid, Spain, 4Pfizer Inc., Alcobendas - Madrid, Spain, 5Adelphi Real World, London, United Kingdom, 6Adelphi Real World, Bollington, United Kingdom.
OBJECTIVES: To describe healthcare resource utilisation (HCRU), treatment usage, and remaining unmet need among male haemophilia patients in Spain.
METHODS: Data were drawn from the Adelphi Real World Disease Specific Programme™, a cross-sectional survey with retrospective data collection of physicians and male haemophilia A (HA) and B (HB) patients in Spain (September 2023-April 2024). Physicians reported data on HCRU, use of standard half-life (SHL)/extended half-life (EHL)/non-factor therapies (NFT), and clinical unmet needs. Analyses were descriptive.
RESULTS: Overall, 36 physicians provided data for 183 patients (83% HA, 17% HB). Median (IQR) patient age was 23.0 (17.0-40.0), with 84% being ≥12 years old and 90% of patients were without inhibitors at time of survey. In the 12 months prior to survey, patients had a mean (SD) 14.4 (34.06) consultations with multiple physicians; with a mean (SD) 6.8 (2.95) test/assessments used to monitor.At the time of survey, 90% of patients received prophylactic therapy (HA: 38% SHL, 28% EHL, 26% NFT; HB: 56% EHL, 19% SHL); of which 79% were also prescribed on-demand therapy. Physicians reported that they weren’t ‘completely satisfied’ for 49% of patients’ prophylactic therapy.In the 12 months prior to survey, patients had a mean (SD) 0.7 (1.05) bleeds, with 40% experiencing ≥1. Overall, 42% of patients suffered from joint problems due to their haemophilia, of these 58% had target joints.
CONCLUSIONS: Physicians reported that nearly half of patients in Spain had bled in the 12 months preceding the survey, and/or had joint problems due to haemophilia. High numbers of consultations and test usage was reported, resulting in burden for patients. Future therapies should address the need for supportive medication and clinical unmet need which in turn could reduce HCRU.
METHODS: Data were drawn from the Adelphi Real World Disease Specific Programme™, a cross-sectional survey with retrospective data collection of physicians and male haemophilia A (HA) and B (HB) patients in Spain (September 2023-April 2024). Physicians reported data on HCRU, use of standard half-life (SHL)/extended half-life (EHL)/non-factor therapies (NFT), and clinical unmet needs. Analyses were descriptive.
RESULTS: Overall, 36 physicians provided data for 183 patients (83% HA, 17% HB). Median (IQR) patient age was 23.0 (17.0-40.0), with 84% being ≥12 years old and 90% of patients were without inhibitors at time of survey. In the 12 months prior to survey, patients had a mean (SD) 14.4 (34.06) consultations with multiple physicians; with a mean (SD) 6.8 (2.95) test/assessments used to monitor.At the time of survey, 90% of patients received prophylactic therapy (HA: 38% SHL, 28% EHL, 26% NFT; HB: 56% EHL, 19% SHL); of which 79% were also prescribed on-demand therapy. Physicians reported that they weren’t ‘completely satisfied’ for 49% of patients’ prophylactic therapy.In the 12 months prior to survey, patients had a mean (SD) 0.7 (1.05) bleeds, with 40% experiencing ≥1. Overall, 42% of patients suffered from joint problems due to their haemophilia, of these 58% had target joints.
CONCLUSIONS: Physicians reported that nearly half of patients in Spain had bled in the 12 months preceding the survey, and/or had joint problems due to haemophilia. High numbers of consultations and test usage was reported, resulting in burden for patients. Future therapies should address the need for supportive medication and clinical unmet need which in turn could reduce HCRU.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
RWD5
Topic
Epidemiology & Public Health, Health Technology Assessment, Real World Data & Information Systems
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)