The announcement that the National Institute for Health and Care Excellence (NICE), the body that decides which treatments should be available on the NHS, has changed its mind on how it will assess tofersen is a huge campaigning breakthrough for the motor neurone disease community.
The language of the announcement – about NICE using its Highly Specialised Technology (HST) route rather than its Standard Technology Assessment (STA) route – might feel impenetrable to all but the wonkiest of policy wonks. But dig past the jargon, and it’s really important news.
What is tofersen and why is it important?
Tofersen is the first effective treatment for MND identified in decades. It is only effective for the one in 50 people with MND who have a particular type of the disease called SOD1 MND. But for those people, tofersen offers the hope of years more life, and it’s vital it becomes available to them on the NHS.
Until now, there was no prospect of that happening. NICE’s original decision to use the STA rather than the HST (a special evaluation route for drugs for rare diseases) meant there would be a higher bar to tofersen being approved, and a lower ceiling on the price NICE could agree to pay for it.
Biogen, the company that developed the drug, believed the chances of tofersen being approved at a price it could accept via the STA were so low it was not worth progressing its NICE application.
This meant the process had reached deadlock.
This was potentially a tragedy for the MND community. Biogen is giving tofersen to people with SOD1 MND free of charge at the moment, but there is no guarantee how long this scheme will stay open to new patients. This raised the very real possibility of people with MND in the UK dying for want of a drug that would be readily available in the United States and the European Union (where tofersen has been approved).
This is why we launched our Prescribe Life campaign.
Asking NICE to make an exception for tofersen
We believed NICE was wrong in its decision not to use the HST. It argued MND was not rare enough for tofersen to qualify, and SOD1 MND (which is rare enough) is not a clinically distinct condition in its own right.
But we made the case that SOD1 MND is clinically distinct from other types of MND. And we said that even if NICE disagreed, its own guidance on HST allows it to make an exception to the rule on rareness if the treatment will only be used for a small number of people AND will make a big positive difference for people with a very serious conditions.
If people with SOD1 MND aren’t worth making an exception for, we asked, then who on earth would be?
We were confident we had a strong argument, but we were just one voice. So a key part of the campaign was a petition to show the strength of feeling in the MND community.
The response was amazing! Incredibly, within a month of the campaign launching more than 15,000 people had already signed the petition, asking NICE to change its mind and use the HST to assess tofersen.
We’ve now had the brilliant news that NICE is going to do just that. We should give it credit for being open to changing its mind.
This has unblocked the logjam, meaning we now have real hope of seeing tofersen becoming available on the NHS before too long.
A long way still to go
This is an amazing campaigning win. But it’s far from the end of the story.
Yes, NICE’s decision has breathed new life into an application process that had ground to a halt. But while the decision to use the HST has tilted the odds in tofersen’s favour, the evaluation process will still take time and there is still no guarantee it will result in the outcome people with SOD1 MND need.
So, in the months to come, we will continue to do everything we can to help the process, whether that’s submitting scientific evidence or making sure the voices of people with SOD1 MND are heard. We’ll also keep you up-to-date.
NICE’s decision to use the HST route isn’t the long-term win we’re all working towards – that will only come when tofersen is available on the NHS. But it does mean the ball is back in play.
Richard Evans
Director of Engagement