The National Institute for Health and Care Excellence (NICE) have published draft recommendation for Alnylam's medicine patisiran, brand name Onpattro▼, as an option for treating hereditary transthyretin amyloidosis (hATTR amyloidosis) in adults with stage 1 and stage 2 polyneuropathy.
It is recommended only if the company provides patisiran according to the commercial arrangement. Commercial arrangements are agreements between the manufacturer and the NHS, usually specifying a set of conditions under which reimbursement for the medicine will be made.
Why the NICE made these recommendations
Hereditary transthyretin amyloidosis is a rare condition that severely affects the health and quality of life of people with the condition, as well as the quality of life of their families and carers. At the time of the evaluation, there were no disease-modifying treatments in widespread use.
Clinical trial evidence shows that patisiran reduces disability and improves quality of life, by enabling patients to return to work, carry out daily activities, participate in a more active family and social life; and maintain their independence and dignity. There is also evidence suggesting that patisiran may provide long-term benefits by stopping the progression of amyloidosis and potentially reversing it.
Some assumptions in the economic modelling are uncertain, particularly around the utility values and the modelling of mortality. Also, the range of cost-effectiveness estimates presented is somewhat higher than what NICE usually considers acceptable for highly specialised technologies. However, taking additional factors into account, such as the uncaptured health-related benefits of stopping and potentially reversing the condition, the rarity and severity of the condition, the potential lifetime benefit for people with the condition and the innovative nature of the treatment, patisiran is recommended for use in the NHS.
About Hereditary transthyretin (hATTR) amyloidosis
Hereditary transthyretin (hATTR) amyloidosis is an ultra-rare condition caused by inherited mutations in the transthyretin (TTR) gene. This causes the liver to produce abnormal TTR protein, which accumulates as deposits in body tissues (amyloidosis). These deposits can disrupt the structure and damage the function of affected tissues.
Because hATTR amyloidosis can affect tissues throughout the body, people may have a range of symptoms relating to 1 or more systems. These can include the autonomic nervous system, peripheral nerves, heart, gastrointestinal system, eyes and central nervous system. The effects and complications of the condition can lead to death within 3 to 15 years of symptoms developing. At the time of the evidence submission, there were thought to be around 150 people with hATTR amyloidosis in the UK.
Onpattro was first made available in the EU in 2018. It is manufactured by Alnylam.
European Medicines Agency website page for Onpattro
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NICE information for Onpattro
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About Transthyretin amyloidosis