Assessing the Value of the Enzyme Replacement Therapy for Hypophosphatasia (HPP): A Spanish Subanalysis of a Pan-European Multidisciplinary Multicriteria Decision Analysis (MCDA)
Author(s)
Pedro Gómez, MSC1, María Isabel Maestre, N/A2, Gabriel Ángel Martos-Moreno, MD3, Andres Navarro Ruiz, PhD4, Fernando Tielve, N/A5, Carolina Tornero, MD6, Natalia Male, PhD7, Sandra Merino-Montero, PhD8, Tania Vico, PhD9.
1Ministry of Health, Madrid, Spain, 2Patient Advocate for Hypophosphatasia, INESCOP, Alicante, Spain, 3Hospital Infantil Universitario Niño Jesús, Universidad Autónoma de Madrid, Madrid, Spain, 4Hospital General Universitario de Elche, Alicante, Spain, 5Patient advocate for Hypophosphatasia, Madrid, Spain, 6La Paz University Hospital, Madrid, Spain, 7Alexion Pharmaceuticals, Barcelona, Spain, 8Alexion Pharma Spain S.L.U., Barcelona, Spain, 9Consultant, Alira Health S.L.U., Barcelona, Spain.
1Ministry of Health, Madrid, Spain, 2Patient Advocate for Hypophosphatasia, INESCOP, Alicante, Spain, 3Hospital Infantil Universitario Niño Jesús, Universidad Autónoma de Madrid, Madrid, Spain, 4Hospital General Universitario de Elche, Alicante, Spain, 5Patient advocate for Hypophosphatasia, Madrid, Spain, 6La Paz University Hospital, Madrid, Spain, 7Alexion Pharmaceuticals, Barcelona, Spain, 8Alexion Pharma Spain S.L.U., Barcelona, Spain, 9Consultant, Alira Health S.L.U., Barcelona, Spain.
OBJECTIVES: To identify the value provided by asfotase alfa to HPP patients from a Spanish multi-stakeholder perspective.
METHODS: This sub-analysis presents the Spanish clinical value perception from a pan-European MCDA study. Six Spanish experts participated (2 clinicians, 2 patient advocates and 2 evaluators), from a total panel of twelve experts, in weighting criteria, scoring the intervention, and estimating its added value. The study considered the standard of care in the absence of asfotase alfa (best supportive care) versus using asfotase alfa as a treatment option for HPP patients.
RESULTS: Disease severity and efficacy/effectiveness were consistently ranked among the highest‐priority decision criteria by the Spanish experts. HPP was perceived as a progressive, high-burden disease affecting individuals of all ages. There is an urgent need for treatments that address disease progression, ensure efficacy across diverse patient profiles, and maintain a favourable safety profile. In terms of comparative efficacy/effectiveness and patient-reported outcomes (PROs), asfotase alfa was considered to offer substantial value versus BSC, while its safety profile remained consistently favourable. Spanish experts, aligned with the pan-European panel perception, considered asfotase alfa to have demonstrated therapeutic benefits across all relevant patient subgroups, supported by robust and consistent clinical data. The global value estimate of asfotase alfa compared to BSC was 0.74.
CONCLUSIONS: In the Spanish context, experts concluded that asfotase alfa offers superior value compared to the BSC for all patient subgroups (0.74). This value contribution is even much higher than the reported in other recently financed drugs for rare diseases, ranging from 0.44 to 0.63. This reinforces its status as a potentially transformative therapy for HPP, and will inform future decisions regarding access, reimbursement and policy in Spain.
METHODS: This sub-analysis presents the Spanish clinical value perception from a pan-European MCDA study. Six Spanish experts participated (2 clinicians, 2 patient advocates and 2 evaluators), from a total panel of twelve experts, in weighting criteria, scoring the intervention, and estimating its added value. The study considered the standard of care in the absence of asfotase alfa (best supportive care) versus using asfotase alfa as a treatment option for HPP patients.
RESULTS: Disease severity and efficacy/effectiveness were consistently ranked among the highest‐priority decision criteria by the Spanish experts. HPP was perceived as a progressive, high-burden disease affecting individuals of all ages. There is an urgent need for treatments that address disease progression, ensure efficacy across diverse patient profiles, and maintain a favourable safety profile. In terms of comparative efficacy/effectiveness and patient-reported outcomes (PROs), asfotase alfa was considered to offer substantial value versus BSC, while its safety profile remained consistently favourable. Spanish experts, aligned with the pan-European panel perception, considered asfotase alfa to have demonstrated therapeutic benefits across all relevant patient subgroups, supported by robust and consistent clinical data. The global value estimate of asfotase alfa compared to BSC was 0.74.
CONCLUSIONS: In the Spanish context, experts concluded that asfotase alfa offers superior value compared to the BSC for all patient subgroups (0.74). This value contribution is even much higher than the reported in other recently financed drugs for rare diseases, ranging from 0.44 to 0.63. This reinforces its status as a potentially transformative therapy for HPP, and will inform future decisions regarding access, reimbursement and policy in Spain.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA46
Topic
Health Policy & Regulatory, Health Technology Assessment, Methodological & Statistical Research
Topic Subcategory
Decision & Deliberative Processes, Value Frameworks & Dossier Format
Disease
Diabetes/Endocrine/Metabolic Disorders (including obesity), Pediatrics, Rare & Orphan Diseases