NICE reject two treatments for fatal, ultra-rare disease – hATTR

The NHS’ cost effectiveness body NICE has rejected two treatments for the rare inherited condition, hereditary transthyretin-related amyliodsis (hATTR), over concerns as to whether the drugs showed long term benefits.

While Akcea Therapeutics’ Tegsedi (inotersen) and Alnylam Pharmaceuticals’ Onpattro (patisiran) were found to offer benefits for people with hATTR in the short term by slowing disease progression and improving quality of life, it remained uncertain as to whether these benefits are maintained in the longer term.

As such, the cost effectiveness estimates for both drugs were much higher than the range that can be considered an appropriate use of NHS resources for highly specialised services.

There are currently no therapies available for the treatment of the ultra-rare condition hATTR, which is thought to affect around 150 people in the UK.

Thus treatments for the debilitating disease, which is often fatal, focus on symptom relief and supportive care.

Brendan Martin, general manager of Alnylam UK & Ireland commented: "Patisiran is breaking new ground, offering eligible people with hATTR amyloidosis a new treatment option with the potential to help improve debilitating neuropathy symptoms.

While we believe our current proposal demonstrates that patisiran represents real value, we welcome the recognition by NICE that this medicine provides ‘considerable clinical benefits’ and we look forward to continuing discussions about how we can make it available to patients in the UK.

He added: "Our focus now will be on addressing the immediate questions posed by the Committee and on working towards securing a positive, sustainable agreement that represents value for the NHS and that would help transform the lives of people living with this terrible disease.

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