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New HTA Decisions in England
August 2019
Drug name
ONPATTRO® (patisiran)
Decision date
Therapeutic area
Genetic conditions
Therapeutic sub area
Genetic conditions: general and other
As an option for treating hereditary transthyretin amyloidosis in adults with stage 1 and stage 2 polyneuropathy.
Decision Detail
It is recommended only if the company provides patisiran according to the commercial arrangement.
Clinical evidence was available from APOLLO (a randomised controlled trial that assessed the efficacy and safety of patisiran compared with placebo over 18 months), OLE (a single-arm phase 2 open-label extension study that assessed the safety and tolerability of patisiran for up to 36 months) and Global OLE (an ongoing single-arm open-label study assessing the long-term efficacy and safety of patisiran for up to 48 months). Clinical trial evidence shows that patisiran reduces disability and improves quality of life. The committee therefore concluded that the evidence showed that patisiran offers considerable benefit for patients and that, in addition to stopping disease progression, patisiran has the potential to reverse it. There was no long-term clinical evidence available for patisiran, but future benefits could be greater than what was presented to the committee. The committee considered that the most plausible ICER for patisiran compared with BSC using the confidential commercial arrangement for patisiran was likely to be between £80,730 and £125,256 per QALY gained. The range of cost-effectiveness estimates presented is somewhat higher than what NICE usually considers acceptable for highly specialised technologies. However, taking additional factors into account, such as the uncaptured health-related benefits of stopping and potentially reversing the condition, the rarity and severity of the condition, the potential lifetime benefit for people with the condition and the innovative nature of the treatment, patisiran is recommended for use in the NHS.