TA868 · HST

Vutrisiran for treating hereditary transthyretin-related amyloidosis

RecommendedJanuary 2022

Recommended within its marketing authorisation, as an option for treating hereditary transthyretin-related amyloidosis in adults with stage 1 or stage 2 polyneuropathy, contingent on commercial arrangement (simple discount patient access scheme)

Source documents

Intervention

vutrisiran (Amvuttra)
· RNA interference (similar mechanism to patisiran) · subcutaneous injection

Condition

hereditary transthyretin-related amyloidosismetabolic_endocrine · stage 1 or stage 2 polyneuropathy

Comparators

NameType Established Committee preferred
patisiranactive drugYes

Economic model

cost comparison (company)

Methodological decisions (2)

cost assumption

Number of vials of patisiran used per person: company used pharmacy data showing vials used, but clinical experts suggested more vials would be used in clinical practice than in the clinical trial

Company: Estimated costs for patisiran using pharmacy data on vials used per person

Committee: Used pharmacy data showing higher vial usage; acknowledged clinical trial scenario where more vials used would make vutrisiran more cost saving

ICER impact: decreases

cost assumption

Administration time and costs for patisiran: the cost savings for vutrisiran depend on administration time and type of healthcare professional administering patisiran

Company: Estimated administration costs for patisiran

Committee: Clinical experts agreed with company's estimate of administration time; higher administration costs for patisiran increase vutrisiran's cost savings

ICER impact: decreases

Commercial arrangement

simple discount pas · confidential · critical for recommendation

Special considerations

Cross-references

comparator guidance — Patisiran is already recommended in NICE's highly specialised technologies guidance on patisiran