Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what recent assessment his Department has made of the availability of treatment for those with Friedreich’s Ataxia.

Under the UK Rare Diseases Framework, the Government is working to improve access to specialist care, treatments, and drugs across all rare conditions. In February, we published the fourth England action plan reporting on progress.

Following extensive consultation, NHS England has revised the national service specification for specialised neurology, which now includes an annex providing greater clarity for neurology sub-specialties. This includes the categories of both movement disorders and neurogenetics into which Friedreich’s Ataxia falls. Every specialised National Health Service neurology centre could and should see patients with Friedreich’s Ataxia.

Although Skyclarys (omaveloxolone) is now licensed for those aged 16 years old and over, following an update from the company, Biogen, the National Institute for Health and Care Excellence (NICE) has had to terminate its appraisal of this medicine as the company has withdrawn its evidence submission. NICE will review its decision if the company decides to make a new submission. Further information is available at the following link:

https://www.nice.org.uk/guidance/TA1061

To avoid the possibility of creating a way to circumvent the appraisal process, NHS England is unable to fund medicines where companies have not engaged with NICE.