RARE Daily

Patient Organizations, Researchers Express Concerns about NICE Approach to ALS Drug

April 1, 2024

Rare Daily Staff

Patient advocates and researchers say a recent decision by the National Institute for Health and Care Excellence on how it will review Biogen’s ALS drug tofersen, likely dooms it to failure and could have broader consequences for the availability of new rare disease treatments.

NICE said it will review tofersen through the Single Technology Appraisal route rather than the Highly Specialized Technologies route.

Biogen developed tofersen as a targeted therapy to treat people living with ALS who have alterations in the SOD1 gene. This is approximately 2 percent of the ALS population, estimated at 60-100 people in the United Kingdom. Ordinarily products targeted to rare diseases that affect less than 1 in 50,000 people in England are appraised through the Highly Specialized Technologies route, but NICE has confirmed it will conduct a Single Technology Appraisal on the basis that it does not view SOD1 ALS as a clinically distinct disease.

“This decision goes against advice from patient representative organizations, including the MND Association, and leading MND clinical experts, which have made extensive representations to NICE throughout the process,” the Motor Neurone Disease Association, a UK-based patient advocacy organization, said in a statement released on its website earlier this month. “Unfortunately, we have been advised it is highly unlikely that tofersen will receive a positive decision if appraised through the standard route, meaning that it will not become accessible to patients through the NHS.”

In a letter to NICE leadership, members of the UK MND Research Institute’s Clinical Studies Group argued that if Biogen gave away tofersen for free, it would fail the NICE appraisal under the economic modeling it is applying. It also warned of potential ramifications from the decision beyond the ALS community.

“Of further concern is the chilling impact this will have on pharmaceutical companies to bring their future innovative science and clinical trials to the UK, given the perception it will create that there is no effective route to market for precision medicine or rare disease therapies,” the researchers wrote. “This is completely contrary to the UK Government’s stated ambition for the Life Sciences sector.”

The MND Association said it intends to pursue the issue with NICE and with the government.

Tofersen has not yet received marketing authorization in the United Kingdom and Biogen has not yet been applied to the Medicines and Healthcare products Regulatory Agency. The association said it is possible that the decision by NICE regarding the route of appraisal could affect whether an application is forthcoming.

People living with motor neuron disease across the UK took part in the clinical trials of tofersen and are part of its success story, but NICE’s decision means that this may not now translate to access for people in England and Wales. In Scotland, tofersen will be appraised by the Scottish Medicines Consortium, which has yet to release details of how it plans to conduct its appraisal.

“We believe the decision has further reaching consequences too,” the association said. “It goes against the government’s desire to be a leader in genomics and personalized medicine. It has serious implications for precision medicine – not just in MND but other diseases where there are proven treatments for small groups of patients.”

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