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Open Fund NHS access to Omaveloxolone for patients with Friedreich's Ataxia

Created by MRS ANN MCGREGOR
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We call on the Government to fund NHS access to Omaveloxolone in the UK. We believe the drug should also be considered under the HST programme, which we feel is the only appraisal route which reflects the realities of Friedreich's Ataxia. This is a degenerative disease & time is of the essence.

Friedreich's Ataxia patients need access to the drug Omaveloxolone, the only drug available for this disease. Without treatment, this degenerative disease can get worse. Patients & families may have to see their loved ones deteriorate when they are aware there is a drug available to stop the progression of this disease & in many cases, to improve the symptoms. The drug is available in EU countries & America. It passed the safety test in April 2025 and we believe it should be available on the NHS!

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  • This petition will be considered for debate in Parliament

    This petition got more than 100,000 signatures meaning the Petitions Committee will consider it for debate in Parliament.

  • Government responded to this petition

    The Government remains committed to improving access to effective and innovative medicines for patients. NICE cannot evaluate omaveloxolone unless the company re-engages with its appraisal.

    Read the response in full

    The Government recognises the concern and urgency felt by people affected by Friedreich’s ataxia and their families, particularly given the progressive nature of the condition.

    The National Institute for Health and Care Excellence (NICE) is the independent body responsible for developing evidence-based recommendations for the NHS on whether new, licensed medicines represent a clinically and cost-effective use of NHS resources. The NHS in England is legally required to fund medicines recommended by NICE.

    NICE initiated the appraisal of omaveloxolone for the treatment of Friedreich’s ataxia for people aged 16 and over in anticipation of a licence being granted by the Medicines and Healthcare products Regulatory Agency (MHRA). The MHRA is the independent body responsible for licensing medicines for use in the UK, based on an assessment of their safety, quality and efficacy.

    NICE’s appraisal process requires the manufacturer of the drug to make an evidence submission. NICE has unfortunately been unable to develop guidance for the NHS on the use of omaveloxolone for treating Friedreich's ataxia in people 16 years and over because the company, Biogen, withdrew its evidence submission. NICE will reopen the appraisal if the company decides to make a new evidence submission.

    The Government understands that Biogen has not approached NHS England or NICE to suggest a resubmission or seek a commercial surgery. Before taking any further action, Biogen must confirm to NHS England that it is willing to reengage in the NICE appraisal process and commercial discussions.

    While the Government understands the desire for urgent access, to avoid the possibility of creating a way to circumvent the appraisal process, NHS England does not fund medicines in circumstances where companies have not engaged with NICE. Funding a medicine before a positive recommendation by NICE also risks creating temporary access to a treatment that could be withdrawn at a later date, if not recommended.

    Decisions on whether medicines should be evaluated through NICE’s highly specialised technologies (HST) programmes are taken by NICE itself against a set of published criteria that have been developed through public and stakeholder engagement. Omaveloxolone satisfied three out of four of the routing criteria to be considered for evaluation through NICE’s HST programme. It did not satisfy criterion 2 – “Normally no more than 300 people in England are eligible for the technology in its licensed indication and no more than 500 across all its indications” – and was therefore considered through NICE’s mainstream technology appraisal programme.

    The vast majority of medicines are evaluated through NICE’s technology appraisal programme. NICE has a strong track record of recommending medicines for the treatment of rare diseases, and its standard technology appraisal process has been proven to be suitable for the evaluation of these medicines, where companies engage constructively in the appraisal and commercial discussions with NHS England. Since March 2024, NICE has recommended 89 per cent of the rare disease medicines, including advanced therapies, that it has appraised for some or all of the patients who would be eligible for the treatment under the marketing authorisation.

    The Government has recently announced several pilots and projects, following the US/UK partnership on pharmaceuticals, that will shape a commercial environment that actively encourages innovation and improves patient access. As part of that work, NICE will look at the approach to valuing rare disease medicines through its Health Technology Assessment Innovation Laboratory. The research will assess a range of both radical and incremental options to improve the current framework.

    The Government remains committed to improving access to effective and innovative medicines for patients, including those living with rare diseases, through a process that is fair, evidence-based and sustainable for the NHS.

    Department of Health and Social Care

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    If this petition gets 100,000 signatures, it will be considered for debate in Parliament.

    This petition will stay open until 24 September 2026.