Budget Impact Analysis of Risdiplam for the Treatment of Spinal Muscular Atrophy in Colombia

Speaker(s)

Vásquez E1, Lasalvia P2, Suárez F3, Quitian D4
1Neuroeconomix, Bogotá, Colombia, 2NeuroEconomix, BOGOTA, CUN, Colombia, 3Pontificia universidad Javeriana, Bogotá, Colombia, 4Productos Roche S.A., Bogotá, Colombia

Presentation Documents

INTRODUCTION: Spinal muscular atrophy (SMA) is a rare disease that affects motor neurons in the spinal cord. Risdiplam is the only disease modifying oral therapy approved to treat SMA.

OBJECTIVES: To estimate the budgetary impact of risdiplam for SMA treatment from the Colombian health system perspective.

METHODS: A global budget impact model was adapted to the Colombian perspective. Incident and prevalent patients with type 1-3 SMA were estimated using Colombian public databases and a published literature review. Two scenarios were assessed over a 5-year time horizon: i) the market was constituted by nusinersen (12% to 24% of the share) and the best supportive care (BSC) throughout the time horizon; ii) risdisplam increased its market share until reaching 20% at year 5. Drug acquisition, administration, follow-up and adverse events costs were considered. Cost-generating events were identified and validated with clinical experts. Costs were estimated using public rate manuals for the 2021 period and were expressed in USD (1 USD = 4.011 COP). Sensitivity analysis was performed to assess the impact of uncertainty on the results.

RESULTS: Nusinersen was associated with the highest treatment cost per patient during the loading year compared with risdiplam. The adoption of risdisplam resulted in a budget impact of USD $439.862 and $969.311 at years 1–5, respectively. The average annual budget impact was 6,45% (min: 5,07% - max: 8,85%). Cost overrun were mainly related to acquisition´s cost of risdiplam and nusinersen. There were savings related to the cost of drug administration, concomitant medications and general disease management with risdiplam use. Price of risdiplam is the variable that most impact the results of the model.

CONCLUSIONS: Risdiplam offers a stable budget impact from the Colombian health system perspective, providing an alternative treatment option for Colombian SMA patients.

Code

EE397

Topic

Economic Evaluation

Topic Subcategory

Budget Impact Analysis

Disease

STA: Drugs