People with ALS in England and Wales fear losing access to life-extending medicines

People with motor neurone disease have spoken of their devastation at the likely loss of access to a life-extending drug due to an NHS cost threshold that has not been increased since 2004.

Tofersen has slowed the progression of the disease in trials, but the chances of the drug being recommended for use in England and Wales would be slim.

The National Institute for Health and Care Excellence (Nice) has a ‘cost-effectiveness threshold’ of up to £30,000 for a drug that gives a patient a year of quality life, known as a Qaly.

The figure was set in 2004 and has not changed, despite studies showing that the threshold today would be closer to £50,000 if it had risen in line with inflation over the past twenty years.

Tofersen, made by the pharmaceutical company Biogen, is expensive because it is a genetic therapy that is administered via monthly lumbar puncture and requires short periods in the hospital.

The drug is said to be aimed at helping the 2% of people with ALS who have changes in the SOD1 gene – the equivalent of 100 people in England and Wales.

MND patients had hoped Tofersen would be reviewed as a treatment for an extremely rare condition that has a £100,000 threshold.

Nice compares this to the lower figure because they do not consider the changes in the SOD1 gene to be clinically different from amyotrophic lateral sclerosis, the most common form of MND. The NHS is legally obliged to provide Nice-recommended medicines.

Mike Thomas, 59, from Bridgend, was diagnosed with ALS four years ago. He has been taking Tofersen since June last year as part of a trial and the improvements in his condition range from an increased ability to walk to more energy.

He said: “It has been proven to be effective and I would be devastated if the treatment ended. Tofersen is a source of hope. At the conclusion of the trial, I saw all the publicity about the promising results.

“I try to be realistic and keep my expectations in check, but deep in my heart I hope not only for an end to the disease progression, but also for an improvement in symptoms.

“Tofersen could be snatched up because Biogen said they would continue to provide the drug until Nice made a decision. This decision could eliminate our only chance for recovery and long-term survival.”

Prof Ammar Al-Chalabi, a researcher in neurology and complex disease genetics at King’s College London, said he was concerned that Biogen would stop applying for market approval.

He said: “There’s a risk that the company might not want to bother bringing it to market and then no one will have it because they’ll say, ‘Well, there’s no way it will with that Qaly are approved. level from £20,000 to 30,000.”

Al-Chalabi said it was “insane” that the lower cost-effectiveness threshold had not been raised over the past 20 years as the purchasing power of the pound has declined.

“It should (also) be higher for this specific treatment because it is a rare condition,” he said. “It requires precision medicine. Precision medicines will become expensive because they are more difficult to develop.

“And if Britain wants to be at the forefront of biotechnology in the world, as it currently is, you need to be attractive to pharmaceutical companies for exactly these types of therapies.

“We are not in some kind of blunderbuss approach. We are in a targeted medical approach. So if you want to push companies to continue doing that, and that’s the only way we’re going to cure some of these diseases.”

Tanya Curry, the chief executive of the MND Association, called for an urgent review of the threshold.

She said: “It is devastating that people with a certain genetic subtype of motor neurone disease are being faced with what could be a life-changing treatment because of the NHS’s outdated systems for approving new medicines.

“I would like to see an urgent review of the cost threshold that Nice uses to assess whether new medicines should be approved for the NHS. It has been stuck in the same place for 20 years, indifferent to drug price increases, and has become an increasingly harmful barrier to getting innovative treatments to patients.”

Nice said cost was not the only basis for its recommendations.

A spokesperson said: “Since 2000, 84% of technology assessments in Nice have resulted in a positive decision – either recommended, an optimized recommendation, or referral to the Cancer Medicines Fund or ‘investigational use’.

“The figures for cancer medicines have risen sharply since the introduction of the cancer medicine fund in 2016. Only a small number of medicines receive negative advice.

“Our approach allows us to focus on the topics that have the greatest impact on improving health and care outcomes and allows us to provide flexibility in the assessment of medicines that treat serious conditions. It is important to note that considering the cost-effectiveness of a technology is a necessary, but not the only, basis for decision making.”

A Ministry of Health spokesperson said the threshold would remain the same in 2024.

She said: “It is vital that we have this system in place to ensure that spending on new medicines does not crowd out spending on other, more effective services and treatments.”