12 December 2018 - Room for manoeuvre after initial rejection.
NICE has issued draft guidance not recommending two new rival treatments for the rare inherited condition hereditary transthyretin-related amyloidosis (hATTR) – Alnylam’s Onpattro and Tegsedi from Akcea (a subsidiary of Ionis).
The draft highly specialised technologies guidance on Tegsedi (inotersen) and Onpattro (patisiran) concluded that both treatments slows progression of the disease and improve quality of life, but says uncertainty around these benefits being maintained in the longer-term, as well as uncertainties around economic modelling for both treatments, means its first ruling is a ‘no’.