Prime Minister Sir Keir Starmer was challenged in the House of Commons over the case of Jake McGregor-How, a 16-year-old from Aigburth, Liverpool, who has to crowdfund for life-saving treatment not available on the NHS. Jake's grandmother, Ann McGregor, said she was overcome with emotion hearing the Prime Minister address her grandson's battle.
Diagnosis and condition
Jake started noticing minor issues with his balance around five years ago, initially thought to be foot problems. His father, Paul, 48, said insoles seemed to fix the issue. However, Jake then began feeling tired and was diagnosed as anaemic and coeliac before the true cause was found. He was diagnosed with Friedreich's ataxia, a rare genetic neurodegenerative disease that often appears in adolescence. The condition progressively impacts the heart, mobility, balance, and coordination, and can cause sensory, sight, and hearing loss.
Drug not available in UK
A new drug, Omaveloxolone, has been medically proven to slow, arrest, and in some cases reverse the effects of Friedreich's ataxia. However, it is not available in the UK. Jake's family is crowdfunding to pay for treatment in Germany. The case was raised in Parliament by Paula Barker, Labour MP for Liverpool Wavertree. She said: "Despite the NHS’s founding principle that care should be based on clinical need, not ability to pay, Jake’s father is crowdfunding to pay for treatment in Germany with a new drug, Omaveloxolone, which has received clinical approval here but is not available through the NHS."
Barker criticised the National Institute for Health and Care Excellence (NICE) for stalling assessment. The condition is not classed as ultra-rare, yet rare enough that any drug will struggle to meet NICE's cost-effectiveness criteria. She added: "Families should never have to crowdfund for treatment abroad for a life-threatening disease. Will the Prime Minister arrange for me and other colleagues who have constituents with this cruel condition to meet the Health Secretary to discuss a way forward?"
Grandmother's advocacy
Ann McGregor was in Parliament last week advocating for government funding of Omaveloxolone for all UK patients. She said: "I am asking the government to direct NICE to drop the present assessment criteria, which is doomed for failure and they know it. They are using a standard assessment, used for every medicine including paracetamol. They are not taking into account the rarity and severity of the disease." She called for a highly specialised approach used for very rare diseases, stating: "My belief is that it is not just a numbers game but a moral issue. People with Friedreich’s Ataxia do not have the time to wait while those in power delay making life-changing decisions."
Prime Minister's response
Responding in the Commons, Sir Keir Starmer said: "May I start by sending my best wishes to Jake and his family? The change we have made to the way that NICE evaluates medicines is already increasing access to innovative new medicines. I will make sure that my hon. Friend gets the chance to meet a Health Minister to discuss Jake’s case and the related issues." Ann McGregor said: "It was quite emotional hearing the Prime Minister discussing my lovely grandson and referring to him by name."
Fundraising and community support
Last month Jake flew to Germany for his vital treatment. The family is fundraising with more than £133,000 raised towards a goal of £300,000 for a year of medicines. Jake's story has inspired Billy Cartawick, from Brentwood in Essex, who has never met him, to take on the Three Peaks Challenge, Basildon Half Marathon, and Everest Stairmaster Challenge to support the family. Symptoms of Friedreich's ataxia usually worsen gradually over years; life expectancy is often into the 30s, but some live into their 60s or beyond.
