Preliminary highly specialised technologies guidance on Akcea Therapeutics’ Tegsedi (inotersen) and Alnylam Pharmaceuticals’ Onpattro (patisiran) conclude that both therapies offer benefits for people with the condition in the short term by slowing progression of the disease and improving quality of life.
However, it is “uncertain” whether these benefits are maintained in the longer-term, and there were further in the economic modelling for both treatments, NICE said.
Consequently, cost-effectiveness estimates for both were “much higher” than the range that can be considered an appropriate use of NHS resources for highly specialised services, the Institute stressed.
hATTR is an ultra-rare (in England it is thought to affect around 150 people), inherited, progressively debilitating, and often fatal disease caused by mutations in the TTR gene, which cause abnormal amyloid proteins to accumulate and damage body organs and tissue, resulting in intractable peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy.
There are no therapies available to treat the underlying cause of hATTR, so current treatments are limited and mainly focus on symptom relief and supportive care, including pain management, nutritional and mobility support and mitigation of the effects of the disease on other organs.