Home / Health / MND Drug Offers Hope, But Access Remains Unequal
MND Drug Offers Hope, But Access Remains Unequal
30 Jan
Summary
- A breakthrough drug, Tofersen, shows promise in halting motor neurone disease progression.
- Unequal access to Tofersen creates a 'postcode lottery' for patients.
- Despite being approved, some NHS trusts lack resources for essential drug administration.
Eleanor Dalley, diagnosed with motor neurone disease (MND) in 2019, has experienced a remarkable turnaround thanks to the drug Tofersen. This breakthrough treatment, the first effective therapy for MND symptoms, has halted the progression of her condition, allowing her to reach milestones she feared she wouldn't live to see.
Tofersen, developed by Biogen, is currently being offered to the NHS free of charge via an early access program. However, the drug requires regular spinal injections, and some NHS trusts report insufficient staff and resources for its administration. This has led to significant disparities in access, creating a 'postcode lottery' for patients.
This situation has caused immense distress for individuals like 19-year-old Lillia Jakeman, who was diagnosed with MND in August and has been unable to access Tofersen. Her family describes the experience as 'mental torture,' emphasizing the critical need for early treatment to preserve function.
The All Party Parliamentary Group on MND has highlighted instances of patients being unable to access Tofersen, with some even dying while awaiting treatment. The drug is specifically for those with the SOD1 inherited form of MND, which constitutes about 2% of cases.
Concerns are mounting over the accessibility of new treatments on the NHS, even as significant investment is made in the life sciences sector. While Tofersen received marketing authorization in July from the UK's Medicines and Healthcare Regulatory Agency, the National Institute for Health and Care Excellence (NICE) has only just begun its evaluation process for routine NHS funding, with an unclear timeline.
Experts, including Professor Ammar Al-Chalabi, call for a simpler system, questioning the purpose of developing new treatments if they cannot be made available to patients. A Department of Health and Social Care spokesperson stated their commitment to improving care for MND patients and ensuring timely access if NICE recommends Tofersen for routine funding.
