Spinal Muscular Atrophy Treatments in France: What Are the Organizational Economic and Environmental Impacts of Oral and Intrathecal Administrations
Author(s)
Anne-Laure Kaminsky, Neurologist1, Marion Masingue, Neurologist2, Anne-Laurence Cazin, Pharmacist3, Charline Remer, PharmD4, Marine Sivignon, PharmD5, Lucile Gabrielle Bellier, PharmD6, Hortense Nanoux, PharmD7, KATELL LE LAY, MSc4, Gwladys Touvron, PharmD8.
1CHU de Saint-Etienne, Hôpital Nord, Saint-Etienne, France, 2Pitié-Salpêtrière, AP-HP, Paris, France, 3Pharmacie de la Croix de Monjous, Gradignan, France, 4Roche, Boulogne-Billancourt, France, 5Putnam PHMR, Lyon, France, 6Putnam PHMR, Paris, France, 7Roche, Boulogne, France, 8Roche, Boulogne billancourt, France.
1CHU de Saint-Etienne, Hôpital Nord, Saint-Etienne, France, 2Pitié-Salpêtrière, AP-HP, Paris, France, 3Pharmacie de la Croix de Monjous, Gradignan, France, 4Roche, Boulogne-Billancourt, France, 5Putnam PHMR, Lyon, France, 6Putnam PHMR, Paris, France, 7Roche, Boulogne, France, 8Roche, Boulogne billancourt, France.
OBJECTIVES: Spinal Muscular Atrophy (SMA) is a rare genetic neuromuscular disease. In France, two treatments are available for adults: a hospital-based intrathecal therapy and a daily oral option. This study compares the organizational, economic, and environmental impacts of these two modalities from health insurance, patient, caregiver, and HCP perspectives.
METHODS: The analysis models the first-year journey of 750 French adult SMA patients (national prevalence), on oral or intrathecal treatment. A simplified budget impact model was developed to quantify HCP, patient and caregiver time, public payer costs, and carbon emissions. Data sources included published literature, institutional reports, and expert interviews, with a priority given to French sources.
RESULTS: Intrathecal administration for 750 patients mobilizes 11,606 HCP hours for the first year of treatment, versus 6,500 hours for the oral form. Transferring care to pharmacy would free up 11,606 HCP hours in hospitals, allowing resource reallocation to other clinical needs. For patients and caregivers’ perspective, intrathecal administration mobilizes 66 and 6 hours respectively in the first year per patient, compared to one caregiver hour with oral therapy.
For patients and caregivers, the gain is €800 per patient, in time saved valued considering productivity losses. From the health insurance perspective, the cost of hospital dispensing amounts to €2.8 million for 750 patients in the first year, versus €133.000 in community pharmacies. This represents a potential saving of €2.6 million for the health insurance, explained by the decrease in hospitalizations and transportation.
From an environmental perspective, intrathecal administration generates 52 tons of CO₂/year, while community pharmacy administration generates 11 tons of CO₂/year, where transportation is the main source of emission.
CONCLUSIONS: The findings demonstrate to multiple stakeholders the advantages of shifting from intrathecal to oral administration in the management of SMA, promoting a more sustainable healthcare approach with optimization of resources, reducing caregiver burden and improving patients’ QoL.
METHODS: The analysis models the first-year journey of 750 French adult SMA patients (national prevalence), on oral or intrathecal treatment. A simplified budget impact model was developed to quantify HCP, patient and caregiver time, public payer costs, and carbon emissions. Data sources included published literature, institutional reports, and expert interviews, with a priority given to French sources.
RESULTS: Intrathecal administration for 750 patients mobilizes 11,606 HCP hours for the first year of treatment, versus 6,500 hours for the oral form. Transferring care to pharmacy would free up 11,606 HCP hours in hospitals, allowing resource reallocation to other clinical needs. For patients and caregivers’ perspective, intrathecal administration mobilizes 66 and 6 hours respectively in the first year per patient, compared to one caregiver hour with oral therapy.
For patients and caregivers, the gain is €800 per patient, in time saved valued considering productivity losses. From the health insurance perspective, the cost of hospital dispensing amounts to €2.8 million for 750 patients in the first year, versus €133.000 in community pharmacies. This represents a potential saving of €2.6 million for the health insurance, explained by the decrease in hospitalizations and transportation.
From an environmental perspective, intrathecal administration generates 52 tons of CO₂/year, while community pharmacy administration generates 11 tons of CO₂/year, where transportation is the main source of emission.
CONCLUSIONS: The findings demonstrate to multiple stakeholders the advantages of shifting from intrathecal to oral administration in the management of SMA, promoting a more sustainable healthcare approach with optimization of resources, reducing caregiver burden and improving patients’ QoL.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE670
Topic
Economic Evaluation, Health Service Delivery & Process of Care
Topic Subcategory
Novel & Social Elements of Value
Disease
Rare & Orphan Diseases