Vital Signs: Rare diseases

Briefing · Parliament

MPs and peers press ministers on rare disease screening, commissioning and access to medicines across multiple debates

1. 01The sharpest exchanges concerned newborn screening for spinal muscular atrophy. In a Commons petitions debate on 22 June 2026, Lewis Atkinson (Labour, Sunderland Central) and Liz Twist (Labour, Blaydon and Consett) pressed the Government to explain why only seven of thirteen laboratories would take part in the in-service evaluation starting in October 2026, leaving around 163,000 babies unscreened. Robbie Moore (Conservative, Keighley and Ilkley), Munira Wilson (Liberal Democrat, Twickenham) and Ruth Jones (Labour, Newport West and Islwyn) argued that for such a rare condition a wider trial would strengthen rather than weaken the evidence base. Minister Sharon Hodgson (Labour, Washington and Gateshead South) said the six excluded laboratories lacked the requisite equipment but that more could be included if that changed.
2. 02The infected blood compensation scheme dominated several sittings. In a Commons backbench debate on 18 June 2026, Clive Efford (Labour, Eltham and Chislehurst) and colleagues including Ian Lavery (Labour, Blyth and Ashington) and Jessica Morden (Labour, Newport East) raised concerns about the pace of payments, the burden placed on haemophilia centres, and the exclusion of carers from full recognition. Julian Lewis (Conservative, New Forest East) cited the Haemophilia Society's view that family carers were not being fairly compensated, and minister Nick Thomas-Symonds (Labour, Torfaen) said he was willing to look at the society's specific concerns. In a Lords debate on 22 July 2026, Baroness Brinton (Liberal Democrat) argued that scheme assessors lacked clinical expertise in the relevant conditions, causing distress to victims.
3. 03The Health Bill committee sittings of 23 and 25 June 2026 produced sustained questioning about what the proposed transfer of specialised commissioning to integrated care boards would mean for people with rare diseases. Caroline Johnson (Conservative, Sleaford and North Hykeham) warned that ICBs facing budget pressure would inevitably prioritise conditions affecting larger numbers of patients, and tabled an amendment requiring a published impact assessment before any transfer of commissioning responsibility. Liz Twist (Labour, Blaydon and Consett) asked the Government to explain how patient involvement and national clinical oversight would be maintained in a more dispersed system.
4. 04VAT charged on medicines supplied free through compassionate use and early access schemes drew questions in both chambers. In a Lords question session on 15 June 2026, Lord Kamall (Conservative) warned that companies might withdraw programmes providing innovative treatments to patients with rare conditions before routine NHS funding was available. In a Commons urgent question on 11 June 2026, Karin Smyth (Labour, Bristol South) said a range of options were being considered and pointed to the Government's decision to raise the NICE cost-effectiveness threshold, which she said had already enabled treatment of patients with Duchenne muscular dystrophy and a rare form of stomach cancer.
5. 05Motor neurone disease was raised in two separate Commons proceedings on 16 and 17 June 2026. Frank McNally (Labour, Coatbridge and Bellshill) presented a petition calling for a national MND strategy and asked the Prime Minister to arrange a meeting to discuss it; David Lammy (Labour, Tottenham), acting as Prime Minister, said the Minister for Care would be happy to meet. In a separate topical question on 9 June 2026, Mary Glindon (Labour, Newcastle upon Tyne East and Wallsend) raised the case of a six-year-old diagnosed with metachromatic leukodystrophy and asked whether newborn screening for the condition could be expedited; Sharon Hodgson (Labour, Washington and Gateshead South) said the Department was working with partners to explore a multi-condition evaluation.