A groundbreaking study reveals that onasemnogene abeparvovec, a one-time gene therapy, offers a cost-effective treatment alternative for presymptomatic infants with spinal muscular atrophy (SMA) in Italy. The research highlights significant healthcare savings and improved patient outcomes compared to existing therapies and standard care.
Comprehensive Economic Evaluation Conducted
Researchers employed a robust Markov model to assess the long-term costs and benefits of onasemnogene abeparvovec (OA) against nusinersen, risdiplam, and best supportive care (BSC). The study encompassed a cohort of 1,000 children diagnosed with SMA through genetic testing, focusing on those with two or three copies of the SMN2 gene. By simulating a lifetime perspective up to 100 years of age, the analysis provided a thorough understanding of the economic implications from the viewpoint of the Italian National Health Service.
Significant Cost-Effectiveness Demonstrated
The findings indicate that OA not only reduces overall treatment costs but also enhances effectiveness when compared to nusinersen and risdiplam, presenting negative incremental cost-effectiveness ratios (ICERs) of -€4,562,815 and -€718,640 respectively. Additionally, OA proved to be a cost-effective option compared to BSC, with an ICER of €65,894. These results were consistent across different SMN2 gene copy scenarios, showcasing OA’s versatility and economic advantage in diverse patient groups.
• OA offers substantial cost savings over long-term therapies like nusinersen and risdiplam.
• The one-time treatment approach of OA reduces the financial burden on the healthcare system.
• Patients receiving OA demonstrate comparable or superior health outcomes versus those on continuous treatments.
• The robustness of the model was confirmed through rigorous sensitivity analyses, ensuring reliable results.
The probabilistic sensitivity analysis further affirmed the model’s stability, reinforcing the validity of the deterministic results and underscoring OA’s position as a financially viable treatment option within the Italian healthcare framework.
Adopting onasemnogene abeparvovec could revolutionize the management of SMA in Italy by providing an effective, long-term solution that alleviates the economic strain associated with chronic treatments. Healthcare providers and policymakers are encouraged to consider gene therapy as a sustainable and impactful strategy to enhance patient care and optimize resource allocation.
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