What is the new drug for motor neurone disease (MND)?

The new drug is called Tofersen, and it is the first effective treatment for MND symptoms, showing promise in halting the disease's progression.

Why is there a 'postcode lottery' for Tofersen access on the NHS?

A 'postcode lottery' exists because while Tofersen is free, some NHS trusts lack the necessary staff and resources to administer the required regular spinal injections.

When will Tofersen be evaluated for routine funding by NICE?

The National Institute for Health and Care Excellence (NICE) began its evaluation process for Tofersen in March, but the timeline for a decision on routine NHS funding is not yet clear.

Home / Health / MND Drug Offers Hope, But Access Remains Unequal

MND Drug Offers Hope, But Access Remains Unequal

30 Jan

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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.

MND Drug Offers Hope, But Access Remains Unequal

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.