VALUE CONTRIBUTION OF DONIDALORSEN FOR THE LONG-TERM PROPHYLAXIS OF HEREDITARY ANGIOEDEMA IN SPAIN: A MULTI-CRITERIA DECISION ANALYSIS (MCDA)
Author(s)
Mónica Climente Martí, Dr.1, Stefan Cimbollek, Dr.2, Mar Guilarte, Dr.3, Carlos González, Dr.4, Eva Bailles, Dr.3, Alicia Herrero, Dr.5, Jose Luis Trillo, Dr.6, Ana Calvo, Dr.7, Rafael Subiran, Dr.8.
1Hospital Universitario Dr. Peset, Valencia, Spain, 2Hospital Universitario Virgen del Rocío, Sevilla, Spain, 3Hospital Universitario Vall d’Hebron, Barcelona, Spain, 4Hospital Universitario de Bellvitge, Barcelona, Spain, 5Hospital Universitario La Paz, Madrid, Spain, 6Consejería de sanidad de Valencia, Valencia, Spain, 7Outcomes'10 (a ProductLife Group Company), Castellón, Spain, 8Otsuka Pharmaceutical, Barcelona, Spain.
1Hospital Universitario Dr. Peset, Valencia, Spain, 2Hospital Universitario Virgen del Rocío, Sevilla, Spain, 3Hospital Universitario Vall d’Hebron, Barcelona, Spain, 4Hospital Universitario de Bellvitge, Barcelona, Spain, 5Hospital Universitario La Paz, Madrid, Spain, 6Consejería de sanidad de Valencia, Valencia, Spain, 7Outcomes'10 (a ProductLife Group Company), Castellón, Spain, 8Otsuka Pharmaceutical, Barcelona, Spain.
OBJECTIVES: Hereditary angioedema (HAE) is a rare, chronic, potentially life-threatening disease associated with unpredictable attacks and quality-of-life impairment despite available long-term prophylaxis (LTP) options. This study assessed the value contribution of donidalorsen for prevention of HAE attacks in adults and adolescents aged ≥12 years in Spain using multicriteria decision analysis (MCDA).
METHODS: An MCDA framework was adapted from EVIDEM (Evidence and Value: Impact on DEcisionMaking) and following ISPOR good practice recommendations. A multidisciplinary scientific committee, including clinicians, hospital pharmacists, a nurse, a psychologist, and a healthcare decision-maker, selected and weighted evaluation criteria. Evidence from published sources and grey literature was organized in an evidence matrix. Quantitative criteria were scored using 0 to 5 scales for non-comparative criteria and −5 to +5 scales for comparative criteria. Donidalorsen was compared with pdC1INH i.v., pdC1INH s.c., lanadelumab, berotralstat, and garadacimab. Weighted scores were aggregated to estimate value contribution on a −1 to +1 scale.
RESULTS: The evaluation framework comprised 10 quantitative and 4 contextual criteria. Efficacy and safety received the highest weights. Comparative value contribution favored donidalorsen versus pdC1INH i.v. (0.31), berotralstat (0.29), pdC1INH s.c. (0.16), lanadelumab (0.06), and garadacimab (0.01). The overall value contribution scores were 0.66, 0.65, 0.52, 0.41, and 0.37, respectively. Donidalorsen offered advantages in efficacy, safety, patient-reported outcomes, and indirect costs compared to pdC1INH i.v., pdC1INH s.c. and berotralstat; and was broadly comparable to lanadelumab and garadacimab, providing added value in dosing flexibility. Main value drivers included sustained attack reduction, improved disease control, favorable patient-reported outcomes, high treatment satisfaction, and the potential to extend dosing to every 2 months in well-controlled patients.
CONCLUSIONS: Donidalorsen showed a positive value contribution for LTP of HAE in Spain. Its monthly or every-2-month subcutaneous administration, clinical profile, and alignment with healthcare priorities support its consideration as a valuable prophylactic option in clinical practice.
METHODS: An MCDA framework was adapted from EVIDEM (Evidence and Value: Impact on DEcisionMaking) and following ISPOR good practice recommendations. A multidisciplinary scientific committee, including clinicians, hospital pharmacists, a nurse, a psychologist, and a healthcare decision-maker, selected and weighted evaluation criteria. Evidence from published sources and grey literature was organized in an evidence matrix. Quantitative criteria were scored using 0 to 5 scales for non-comparative criteria and −5 to +5 scales for comparative criteria. Donidalorsen was compared with pdC1INH i.v., pdC1INH s.c., lanadelumab, berotralstat, and garadacimab. Weighted scores were aggregated to estimate value contribution on a −1 to +1 scale.
RESULTS: The evaluation framework comprised 10 quantitative and 4 contextual criteria. Efficacy and safety received the highest weights. Comparative value contribution favored donidalorsen versus pdC1INH i.v. (0.31), berotralstat (0.29), pdC1INH s.c. (0.16), lanadelumab (0.06), and garadacimab (0.01). The overall value contribution scores were 0.66, 0.65, 0.52, 0.41, and 0.37, respectively. Donidalorsen offered advantages in efficacy, safety, patient-reported outcomes, and indirect costs compared to pdC1INH i.v., pdC1INH s.c. and berotralstat; and was broadly comparable to lanadelumab and garadacimab, providing added value in dosing flexibility. Main value drivers included sustained attack reduction, improved disease control, favorable patient-reported outcomes, high treatment satisfaction, and the potential to extend dosing to every 2 months in well-controlled patients.
CONCLUSIONS: Donidalorsen showed a positive value contribution for LTP of HAE in Spain. Its monthly or every-2-month subcutaneous administration, clinical profile, and alignment with healthcare priorities support its consideration as a valuable prophylactic option in clinical practice.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HTA199
Topic
Health Technology Assessment, Methodological & Statistical Research, Study Approaches
Topic Subcategory
Decision & Deliberative Processes, Value Frameworks & Dossier Format
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases