The aim of this project, which has been commissioned by NICE through the NIHR HTA Programme as part of the NICE’s Highly Specialised Technologies Appraisal process, is to review the submission of the pharmaceutical company (Ionis Phamaceuticals/Akcea Therapeutics) on the evidence for the clinical and cost-effectiveness of Inotersen (TegsediTM) for adults with  stage 1 or stage 2 hereditary transthyretin amyloidosis with polyneuropathy. 

The company’s clinical evidence consists mainly of one phase II/III RCT, the NEURO-TTR trial, which compares Inotersen with placebo. The co-primary endpoints are the change from baseline to week 66 in a composite neurological impairment score (the modified Neuropathy Impairment Score+7) and the participants’ perceived quality of life (the Norfolk Quality of Life-Diabetic Neuropathy questionnaire).

The Aberdeen HTA Group acts as the Evidence Review Group (ERG) for the project and provides an independent critique of the clinical and cost-effectiveness evidence submitted by the company. Based on the findings of the company submission and of the ERG’s critique.  NICE guidance was published on 22 May 2019.


Miriam Brazzelli;