New hATTR Treatments Under Review for their ‘Value for Money’

Two vitally important reviews are underway for patisiran and inotersen, new treatments that have been trialed for hATTR patients.

One review is led by the National Institute for Health and Clinical Excellence (NICE) in the UK.

NICE is the organisation which decides whether new treatments should be available on the national (public) health system (the NHS).

If it approves the treatments they will be made available free of charge to eligible patients living in England and Wales.

To reach its decision NICE assesses the evidence on the treatments’ benefits, risks and costs. A new treatment has to be both ‘clinically and cost effective’ to be approved for routine use on the NHS. ARC is participating as a stakeholder in the review, providing comments and evidence from patients and carers about what it is like to live with hATTR and the value the new treatments may offer. NICE will complete its review in early 2019.

Are you an hATTR patient living in the UK?  If you’d like to be kept up to date on how the NICE review of Inotersen and patisiran is progressing or to get involved, send us an email with ‘NICE’ in the subject heading

At the same time, an organisation called the Institute for Clinical and Economic Research (ICER) is carrying out a similar review of patisiran and inotersen in the United States. A different and quicker process than NICE, ICER will conclude its review in the Fall. ICER does not ‘approve’ new treatments for reimbursement; it is up to public and private insurers to decide if and how to provide coverage. However, its conclusions about how much value for money new treatments offer and what price could be acceptable may influence what insurers and payers do. The ARC is working closely with ICER and providing comments and evidence to help inform the review. This week ICER published its ‘draft evidence report’. You can read the report here. ARC will be commenting on this report by mid-August. We want to make sure that all the right evidence and context is understood and that the subsequent recommendations help insurers and the companies find a practical solution that enables all hATTR patients to access these new treatments.

If you want to get involved in the ICER process or be kept up to date on the review, send us an email with ‘ICER’ in the subject heading. You can also send comments on the draft report directly to ICER. Please ask us for more details

ARC is determined to see important new amyloidosis treatments made available to patients. You can help us by signing up to get involved. Please send us an email using the above links to receive latest news and for information on how you can help us.

ARC will also be hosting a webinar shortly about access to hATTR treatments in the United States. Keep an eye out for information on our website.