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two pioneering drugs for the rare inherited condition, hereditary transthyretin-related amyloidosis (hATTR) in first draft guidance.

The cost-effectiveness body had been using an assessment system for highly specialised technology when judging whether Alnylam’s Onpattro (patisiran) and Akcea’s Tegsedi (inotersen) 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 modelling 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 both drugs cost around £300,000 per year  per patient, although manufacturers have agreed to offer them to the NHS at a confidential discount.

NICE said cost-effectiveness estimates for both treatments were much higher than the threshold.

hATTR is ultra-rare, progressive condition thought to affect only 150 people in England, that causes the liver to produce abnormal transthyretin protein. Accumulations of transthyretin form deposits in the tissues of the body (amyloidosis) which can disrupt the structure and damage the function of the affected tissues.

Over time, these deposits can cause symptoms of polyneuropathy (such as pain, loss of sensation and weakness in the hands, arms, legs or feet) and cardiomyopathy (such as chest pain, shortness of breath and fluid overload).

In some cases, the autonomic nervous system, which controls involuntary body functions such as blood pressure, heart rate, and digestion, may also be affected by amyloidosis.

There are no treatments 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 disease’s effects on other organs.

NICE is consulting on the draft guidance until 9 January, giving the manufacturers scope to drop their prices or submit further supportive data.


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