Vutrisiran for treating hereditary transthyretin-related amyloidosis

1.1 Vutrisiran is recommended, within its marketing authorisation, as an option for treating hereditary transthyretin-related amyloidosis in adults with stage 1 or stage 2 polyneuropathy. It is only recommended if the company provides vutrisiran according to the commercial arrangement.

1.2 If people with the condition and their clinicians consider vutrisiran to be 1 of a range of suitable treatments, discuss the advantages and disadvantages of the available treatments. After that discussion, if more than 1 treatment is suitable, choose the least expensive. Take account of administration costs, dosage, price per dose and commercial arrangements.

Why these recommendations were made

Hereditary transthyretin-related amyloidosis is usually treated with patisiran, which is already recommended in NICE's highly specialised technologies guidance on patisiran. Vutrisiran works in a similar way, but it is given as an injection under the skin instead of into a vein.

Evidence from a clinical trial and an indirect comparison shows that vutrisiran works as well as patisiran.

In the economic model, the company estimated costs for patisiran using pharmacy data showing the number of vials used per person. It provided a scenario using clinical trial evidence. The clinical experts suggested that more vials of patisiran would be used in clinical practice than in the clinical trial. If more vials of patisiran are used, vutrisiran is more likely to be cost saving.

The cost savings for vutrisiran also depend on how long it takes to administer patisiran and which type of healthcare professional administers it. The clinical experts agreed with the company's estimate of administration time. In the model, when the administration cost for patisiran increases, vutrisiran is more cost saving.

Taking the number of vials used per person in the pharmacy data and administration costs into account, a cost comparison suggests vutrisiran is cost saving compared with patisiran. So vutrisiran is recommended.

For all evidence see the committee papers. To see what NICE did for patisiran, see the committee discussion in NICE's highly specialised technologies guidance on patisiran.