1.1 Patisiran is recommended, within its marketing authorisation, as an option for treating hereditary transthyretin amyloidosis in adults with stage 1 and stage 2 polyneuropathy. It is recommended only if the company provides patisiran according to the commercial arrangement.
Why the committee made these recommendations
Hereditary transthyretin amyloidosis is a rare condition that severely affects the health and quality of life of people with the condition, as well as the quality of life of their families and carers. At the time of the evaluation, there were no disease-modifying treatments in widespread use.
Clinical trial evidence shows that patisiran reduces disability and improves quality of life, by enabling patients to return to work, carry out daily activities, participate in a more active family and social life, and maintain their independence and dignity. There is also evidence suggesting that patisiran may provide long-term benefits by stopping the progression of amyloidosis and potentially reversing it.
Some assumptions in the economic modelling are uncertain, particularly around the utility values and the modelling of mortality. Also, 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.