OBJECTIVES: To determine the value of olipudase alfa compared with placebo in the treatment of non-central nervous system (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD) using MCDA.

METHODS: EVIDEM (v 4.0) MCDA Framework adapted to the context of orphan drugs and rare diseases was selected and adjusted for this study. A targeted literature review was conducted to populate the framework, comprised of 9 quantitative and 3 contextual criteria. The weighting used was performed by 98 evaluators and decision makers. The value contribution was obtained from the scoring of the criteria by a multidisciplinary panel including 5 hospital pharmacists and 2 clinicians with experience in ASMD, and a patient representative (n=8).

RESULTS: ASMD was considered a severe disease (mean±SD: 4.1±-0.6), with significant unmet needs given that current treatment is purely symptomatic (4.9±0.4). Experts perceived that olipudase alfa might provide a substantial improvement in efficacy/effectiveness (4.4±0.7) and has a favourable safety profile (3.0±2.6), whereas reduced differences were found in patient-reported outcomes (PROs) (2.4±1.8). Additionally, the therapeutic benefit generated by olipudase alfa was considered high (4.3±0.7). Experts anticipated an average of lower medical costs (2.6±1.3) and indirect cost savings (2.9±1.1). Evidence presented for olipudase alfa was considered high in the context of orphan drugs (3.9±1.0). In contextual criteria, most experts perceived a positive impact on population priorities and access (88%), common goal and specific interests (88%), and system capacity and appropriate use (100%). The result of the value contribution of olipudase alfa was 0.73 (in a -1 to +1 scale).

CONCLUSIONS: ASMD is a rare disease with significant unmet needs and a high impact on morbidity, mortality, and quality of life. Olipudase alfa provides very high added value to the treatment of non-CNS manifestations of ASMD using MCDA, showing a significant improvement in all severity parameters studied and potentially modifying its clinical course.