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Fear of Losing Access to Life-Prolonging Medication Grips MND Patients in England and Wales

People affected by motor neurone disease are expressing their distress over the potential loss of access to a life-prolonging medication due to an unchanged cost threshold since 2004.

Tofersen, a drug that has demonstrated effectiveness in slowing the progression of the disease during trials, faces slim chances of approval for use in England and Wales.

The National Institute for Health and Care Excellence (Nice) enforces a cost-effectiveness threshold of up to £30,000 for a medication that provides a year of good-quality life, measured in Quality-Adjusted Life Years (QALYs).

Despite evidence suggesting that the threshold should be closer to £50,000 today if adjusted for inflation over the past two decades, the figure remains stagnant since 2004.

Manufactured by Biogen, Tofersen is a costly genetic therapy administered through monthly lumbar punctures, necessitating brief hospital stays.

This medication targets the 2% of individuals with motor neurone disease who exhibit alterations in the SOD1 gene, approximately 100 people in England and Wales.

Expectations were high for Tofersen to be evaluated as a treatment for an exceptionally rare condition with a £100,000 threshold. However, Nice is assessing it against the lower figure, citing that the SOD1 gene alterations are not clinically distinct from amyotrophic lateral sclerosis, the most prevalent form of motor neurone disease.

Mike Thomas, a 59-year-old from Bridgend diagnosed with MND four years ago, has been part of a Tofersen trial since June last year. He has experienced various improvements, from enhanced mobility to increased energy levels.

Expressing his concerns, Mike stated, “It has been proven to be effective, and I would be devastated if treatment ceased. Tofersen is a source of hope.”

Professor Ammar Al-Chalabi from King’s College London voiced apprehension that Biogen might halt efforts to obtain market authorization due to the challenging cost-effectiveness threshold.

Tanya Curry, CEO of the MND Association, urged an immediate review of the threshold, emphasizing the need to adapt to the rising costs of medications and eliminate barriers to innovative treatments.

While Nice emphasized that cost is not the sole determinant in its recommendations, the Department of Health confirmed that the threshold would not be revised until 2024 to maintain a balance between new medication expenses and existing healthcare services.