Patisiran for treating hereditary transthyretin amyloidosis

Section 8(6) of the National Institute for Health and Care Excellence (Constitution and Functions) and the Health and Social Care Information Centre (Functions) Regulations 2013 requires clinical commissioning groups, NHS England and, with respect to their public health functions, local authorities to comply with the recommendations in this evaluation within 3 months of its date of publication. Because patisiran has been available through the early access to medicines scheme, NHS England and commissioning groups have agreed to provide funding to implement this guidance 30 days after publication.

The Welsh ministers have issued directions to the NHS in Wales on implementing NICE highly specialised technologies guidance. When a NICE highly specialised technologies guidance recommends the use of a drug or treatment, or other technology, the NHS in Wales must usually provide funding and resources for it within 2 months of the first publication of the final evaluation document.

When NICE recommends a treatment 'as an option', the NHS must make sure it is available within the period set out in the paragraph above. This means that, if a patient has hereditary transthyretin amyloidosis with stage 1 and stage 2 polyneuropathy and the doctor responsible for their care thinks that patisiran is the right treatment, it should be available for use, in line with NICE's recommendations.