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New HTA Decisions in England
November 2019
Drug name
SPINRAZA® (nusinersen)
Biogen Idec
Decision date
Therapeutic area
Musculoskeletal conditions
Therapeutic sub area
Musculoskeletal conditions: general and other
Recommended with restrictions
Nusinersen as an option for treating 5q spinal muscular atrophy (SMA)
Decision Detail
Nusinersen is only recommended if: • people have pre-symptomatic SMA, or SMA types 1, 2 or 3 and • the conditions in the managed access agreement are followed.
The clinical evidence for nusinersen was provided by the randomised double-blind trials ENDEAR and CHERISH, demonstrating improvement in a number of outcomes that are important to people with early- (type 1) and later-onset (types 2 and 3) SMA. The committee agreed that ENDEAR showed a substantial benefit in survival for nusinersen compared with sham in people with early-onset SMA. Data from SHINE, an ongoing study which is a continuation of ENDEAR, CHERISH, CS12 and CS3A, appeared to show that patients having nurinersen in both ENDEAR and SHINE had improved outcomes in terms of time to death or permanent ventilation compared with patients who started nusinersen in SHINE. The committee agreed that nusinersen provides important health benefits for people with later‑onset SMA, but it was unclear how this affects survival because there were no deaths during the CHERISH trial. There is some evidence suggesting that nusinersen is effective for pre-symptomatic SMA, but the size of the benefit in this patient group has not been established compared with benefits seen in people with symptomatic SMA. Also, these results were based on ad-hoc subgroup analyses that may not have been powered sufficiently or on interim study results. However, there is no long-term evidence, so the long-term benefits are highly uncertain. The cost-effectiveness estimates presented are higher than what NICE usually considers a cost-effective use of NHS resources. However, these estimates are difficult to interpret because of the limited evidence base to substantiate longer-term benefits, the difficulty in clearly distinguishing between the SMA subtypes, and the difference in what can be achieved for these various patients without nusinersen. For example, the committee recalled that several of the parameters, when changed, resulted in an opposite impact on the ICER for the 2 populations (early-onset and late-onset SMA). Additionally, the committee noted that using higher resource costs increased the ICER for the early-onset model (because of increased management costs for longer survivors), but it reduced the ICER for the later-onset model (because more patients reached higher milestones that are associated with lower annual management costs).