NICE rejects Alnylam and Akcea’s amyloidosis drugs

NICE has rejected two pioneering drugs for the rare inherited condition, hereditary transthyretin-related amyloidosis (hATTR) in the first draft guidance. The cost-effectiveness body had been using an assessment system for highly specialized technology when judging whether Alnylam’s Onpattro (patisiran) and Akcea’s Tegsedi (internet) should receive funding from the NHS in England. Both drugs work by RNA interference – blocking expression of the gene that causes the abnormal transthyretin protein that is the root cause of the disease. NICE concluded that both treatments offer benefits for people with the condition in the short term by slowing the progression and improving quality of life.
But it is uncertain whether these benefits are maintained in the longer term, and there were further uncertainties in the manufacturers’ economic modeling for both treatments, NICE said. NICE said that the drugs exceeded a cost-effectiveness threshold of £100,000 per Quality Adjusted Life Year. In the guidance, NICE said that it could not add extra weighting allowing up to £300,000 per QALY in cases where drugs extended good quality life for 30 years or more. At full price, Onpattro costs almost £133,000 per year for each patient, while Tegsedi costs around £308,000, although manufacturers have agreed to offer them to the NHS at a confidential discount.