Value contribution of olipudase alfa therapy for the management of the non-CNS manifestations of acid sphingomyelinase deficiency by multi-criteria decision analysis

Introduction: Olipudase alfa, an enzyme replacement therapy, is the first treatment approved for acid sphingomyelinase deficiency (ASMD), a rare lysosomal storage disease. Objective: To determine the value contribution of olipudase alfa to treat non-central nervous system (CNS) manifestations of ASMD using MCDA methodology. Methods: A literature review was conducted to populate an adapted MCDA framework for orphan-drug evaluation. A multidisciplinary panel of 8 experts were trained in MCDA methodology and scored the evidence matrix. Scoring consistency was explored through a test-retest reliability analysis. Results: ASMD was considered a severe disease (mean, 4.1 ± 0.6) with significant unmet needs (4.9 ± 0.4). Experts perceived that olipudase alfa may improve efficacy/effectiveness (4.4 ± 0.7), and has a favorable safety profile (3.0 ± 2.6). Additionally, its therapeutic benefit was considered relevant (4.3 ± 0.7) and patient-reported outcomes (PROs) (2.4 ± 1.8) were rated as moderate. Lower medical costs (2.6 ± 1.3) and indirect cost savings (2.9 ± 1.1) would be expected. Evidence presented was considered adequate (3.9 ± 1.0). The test-retest analysis indicated consistency in the scoring of almost all criteria. Lower correlation scores were observed in criteria with more dispersion as expert’s scoring perception was addressed during the discussion session. Conclusion: Olipudase alfa may provide a high-value intervention to treat non-CNS manifestations of ASMD.