OBJECTIVES : To compare HTA’s economic evaluation of nusinersen in France and UK.

METHODS : We compared CEESP (France) and NICE (UK) opinions published on the cost-effectiveness models of nusinersen in SMA type I (early-onset), type II/III for NICE and type II only for CEESP (later-onset).

RESULTS : For type I, model structures are based on relative health states according to HINE-2 scores (vs baseline). In nusinersen arm, patients can be reclassified in type II/III with absolute states (i.e. ability to sit/stand/walk w/o assistance). HAS type II model was similar with evolution of HMSE scores, while the NICE, after ERG’s request, took a model based on motor milestones. CEESP retained a 5-year time horizon for early-onset, and 60 years for later-onset, while NICE retained 60 and 80 years. CEESP evaluated only a cost-efficacy analysis for SMA type I while NICE appreciated both cost/LY (Life Year) and cost/QALY even if they noticed the difficulty to estimate robust utilities for such young children. Nusinersen stopping rules were scoliosis surgery and absence of improvement. After stopping treatment, patients were assumed to stay stable. Resources utilizations were determined through a survey in the NICE submission while they were mainly based on expert opinion in France. The base case ICER for early-onset SMA is around £370,000/LY and €930,000/LY and for later-onset SMA, it is £1,250,000/QALY and €2,660,500/QALY. However, ICER’s estimations are hardly comparable: CEESP assesses ICER based on list price claimed (before negotiation) while NICE takes into account the negotiated financial rebates.

CONCLUSIONS : Main comments are common between the two HTA agencies, with concerns about the extrapolation of motor function, the long-term survival and the utilities due to a lack of data in a context of rare disease. These opinions will have to be taken into account in the CE submissions for novel therapies in SMA.