By Christina Poschen, Consultant
Biogen’s Spinraza, a treatment for the rare genetic disease spinal muscular atrophy (SMA) is currently making headlines for its high price and promising outcomes. Various European countries including Spain, Italy as well as The Netherlands and Belgium just made the product available to their patients or confirmed they will do so in the next few months.
At the same time, the English health technology assessment watchdog NICE published its draft guidance not recommending Spinraza for routine use in England due to uncertainties over the drug’s long-term effectiveness and its extremely high cost.
Interestingly, the drug was not appraised under the Highly Specialised Technology (HST) route. This is likely due to its broad label that includes four SMA subtypes, and also as SMA is not commissioned through a highly specialised service. The HST route has a threshold of up to a maximum of £300,000 per quality adjusted life year (QALY) gained. Instead, Spinraza went through Single Technology Appraisal (STA) which has a QALY threshold of £30,000.
The £30,000 threshold is in stark contrast to the cost-effectiveness estimates for Spinraza that ranged between £400,000 and £600,000 per QALY gained based on its list price. Even with a proposed confidential price reduction, the cost of Spinraza is too high for it to be considered a cost-effective use of NHS resources.
In a statement NICE confirmed it is open for discussions, however, finding common ground with an over 10-fold threshold difference seems rather challenging. In the meantime, patient organisations have called upon their members to submit their comments to NICE before the consultation closes in September.
Not only patient groups but also the biotech industry will be keenly awaiting the final recommendation from NICE. Considering the recent changes and additional thresholds in the English healthcare system (e.g. £20 million budget impact threshold over the first three years or the request to meet all criteria listed in the HST to be considered for the higher QALY threshold), it is becoming increasingly challenging to launch a specialized product in England.
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