SOCIOECONOMIC BURDEN OF TYPE 1 SMA IN JAPAN: COMPARING THE IMPACT OF ONASEMNOGENE ABEPARVOVEC, NUSINERSEN AND RISDIPLAM ON PATIENTS AND CAREGIVERS
Author(s)
Michel Kroes, MSc1, Riku Ota, MBA, MSPH2, AI TSUYUKI, MSc2, Oliver Burn, MSc3, Kirsten Chalmers, MSc3, Curtis Wellington, BSc3, Kiera R. Lander, MSc3, Ataru Igarashi, PhD4.
1International HEOR, Novartis AG, Basel, Switzerland, 2HEOR, Novartis Pharma K.K., Tokyo, Japan, 3Source Health Economics, London, United Kingdom, 4Graduate School of Business Administration, Keio University, Tokyo, Japan.
1International HEOR, Novartis AG, Basel, Switzerland, 2HEOR, Novartis Pharma K.K., Tokyo, Japan, 3Source Health Economics, London, United Kingdom, 4Graduate School of Business Administration, Keio University, Tokyo, Japan.
OBJECTIVES: This analysis estimates the socioeconomic burden of Type 1 spinal muscular atrophy (SMA) and impact of treatment (intravenous onasemnogene abeparvovec (OAV), nusinersen, or risdiplam) on patients and caregivers in Japan.
METHODS: A lifetime Markov model with six health states defined by motor function and death was constructed. Clinical inputs were derived from pivotal trials for OAV, nusinersen, and risdiplam. The analysis adopted a societal perspective, capturing direct and indirect costs associated with absenteeism/presenteeism, lost unpaid work time, health visits, mortality and bereavement, and societal value of potential paid work. Total life years (LYs) and patient and caregiver quality-adjusted life years (QALYs) were assessed. Data linked to socioeconomic outcomes were obtained from the “Statistics Bureau of Japan” and published literature. All monetary inputs referred to 2024 prices. Deterministic and probabilistic sensitivity analyses were conducted.
RESULTS: OAV provided greater patient LYs, combined patient and carer QALYs, and work productivity versus nusinersen and risdiplam, driven by improved survival and motor milestones attainment. Total lifetime socioeconomic burden per patient was ¥1,114,889,344, ¥1,388,451,377 and ¥1,141,281,807 for OAV, nusinersen, and risdiplam, respectively, with corresponding combined QALYs of 16.02, 9.07 and 8.93. Increased potential combined work hours translated to 31% and 32% gains in total equivalent years of work gained vs nusinersen and risdiplam. OAV reduced total bereavement losses through improved survival (¥14,250,542 vs ¥17,531,006 and ¥17,869,908, respectively), and as a one-time treatment, reduced time and costs associated with healthcare visits (38.28 hours; ¥308,894) compared with nusinersen (85.66 hours; ¥694,778). Results were robust to sensitivity analyses.
CONCLUSIONS: Compared with nusinersen and risdiplam, the study suggests that OAV reduces the total burden of Type 1 SMA, improving patient and caregiver outcomes while reducing long-term socioeconomic impacts through a one-time treatment. These findings highlight the importance of incorporating the societal perspective into decision-making for life-threatening rare diseases.
METHODS: A lifetime Markov model with six health states defined by motor function and death was constructed. Clinical inputs were derived from pivotal trials for OAV, nusinersen, and risdiplam. The analysis adopted a societal perspective, capturing direct and indirect costs associated with absenteeism/presenteeism, lost unpaid work time, health visits, mortality and bereavement, and societal value of potential paid work. Total life years (LYs) and patient and caregiver quality-adjusted life years (QALYs) were assessed. Data linked to socioeconomic outcomes were obtained from the “Statistics Bureau of Japan” and published literature. All monetary inputs referred to 2024 prices. Deterministic and probabilistic sensitivity analyses were conducted.
RESULTS: OAV provided greater patient LYs, combined patient and carer QALYs, and work productivity versus nusinersen and risdiplam, driven by improved survival and motor milestones attainment. Total lifetime socioeconomic burden per patient was ¥1,114,889,344, ¥1,388,451,377 and ¥1,141,281,807 for OAV, nusinersen, and risdiplam, respectively, with corresponding combined QALYs of 16.02, 9.07 and 8.93. Increased potential combined work hours translated to 31% and 32% gains in total equivalent years of work gained vs nusinersen and risdiplam. OAV reduced total bereavement losses through improved survival (¥14,250,542 vs ¥17,531,006 and ¥17,869,908, respectively), and as a one-time treatment, reduced time and costs associated with healthcare visits (38.28 hours; ¥308,894) compared with nusinersen (85.66 hours; ¥694,778). Results were robust to sensitivity analyses.
CONCLUSIONS: Compared with nusinersen and risdiplam, the study suggests that OAV reduces the total burden of Type 1 SMA, improving patient and caregiver outcomes while reducing long-term socioeconomic impacts through a one-time treatment. These findings highlight the importance of incorporating the societal perspective into decision-making for life-threatening rare diseases.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE244
Topic
Economic Evaluation, Health Policy & Regulatory
Disease
Rare & Orphan Diseases