UPDATE: CAMPAIGN WIN!!!
On 18 November NICE announced that they have reconsidered their decision, and have agreed to assess tofersen via the Highly Specialised Technology (HST) route.
This is what our petition was asking for, and it means that there’s a much greater chance of tofersen becoming available on the NHS.
More than 15,000 people signed our petition in just a few weeks, sending NICE a powerful message about how important tofersen is – a groundbreaking new treatment for the 2% of people with MND who have an alteration in the SOD1 gene. And NICE listened.
Thank you so, so much for signing the petition and being part of this amazing victory!
Why we're campaigning on Tofersen
There is huge excitement about tofersen in the MND community, and it’s easy to see why.
It’s the first proven, effective new treatment for MND since riluzole in the 1990s. And while riluzole only has a modest effect in extending life, tofersen has been shown to slow the progress of MND so much that it gives people the hope of many more years of life.
Tofersen is only effective for a small proportion of people with MND – those whose condition is caused by a specific change in the SOD1 gene. But for those people, this is the kind of treatment the MND community has been waiting for.
But there is no point in research leading to an exciting new treatment if the people who could benefit from it can’t then access it.
Sadly, there is no prospect of tofersen becoming available on the NHS any time soon. This raises the real prospect of people with SOD1 MND being able to access tofersen if they live in the United States or the European Union (where it has already been approved) but not if they live in the UK.
Why is tofersen not available on the NHS?
In one sense, the reasons for this are complicated. In attempting to explain them I fear I your eyes glazing over as I descend into the weeds of drug appraisal guidance.
But in another way, the reason is very simple: money. Specifically, tofersen is expensive, and the NHS doesn’t have the funds to pay for everything it wants to.
The National Institute for Health and Care Excellence (NICE, the body responsible for evaluating new treatments for NHS use, considering both cost and effectiveness) wants to appraise tofersen via its Standard Technology Appraisal (STA). But Biogen, the company that makes tofersen, wants NICE to use its Highly Specialised Technology (HST) route.
The difference between these two processes is that the HST route evaluates cost-benefit in a different way, so that expensive treatments have a higher chance of being approved. And following NICE’s decision not to use the HST route, Biogen thinks there is so little chance of success via the STA route that it has paused its application.
So we’re left with a standoff between NICE and Biogen, with people with SOD1 MND caught in the middle.
The one bit of good news is that Biogen is currently providing tofersen to patients under a compassionate access scheme, and has promised to continue for as long as those patients can benefit from it. But we have no idea if and when this scheme will close to new patients. We also fear that it not being available on the NHS means that not everyone who would benefit from it is actually receiving it.
Tofersen – a matter of life and death
This is literally a question of life and death, and that’s why we’ve launched our Prescribe Life Campaign.
We want both NICE and Biogen to be flexible. We believe that if both compromise, we can get to a point where tofersen is available in the UK. If other countries have done it, why can’t we?
But in particular, we think NICE should reconsider its decision not to assess tofersen via the HST.
NICE’s guidance says the HST should only be used for assessing drugs that make a big positive difference for people with a rare and life-limiting disease, and only when the number of people who could benefit from the drug is very small.
Tofersen comfortably ticks all but one of those boxes. Only a very small number of people (around 60) would benefit from it; without tofersen those 60 people would have a very poor prognosis; and tofersen is a game-changing drug that could potentially give them years of extra life.
The only box tofersen arguably doesn’t tick is the rarity one. While SOD1 MND is rare enough to qualify, MND as a whole is not. This is why NICE decided not to assess tofersen via the HST.
But NICE’s guidance says that it can make exceptions to that rule, if the drug strongly meets the other criteria.
We believe that in deciding not to make an exception, NICE has made the wrong decision. If a transformational drug for people with a disease that kills half of people within two years of diagnosis is not worth making this kind of exception for, what on earth could be?
In the absence of an answer to this, our Prescribe Life campaign is calling for NICE to make an exception and agree to appraise tofersen via the HST.
The MND community is crying out for tofersen
Even if NICE agreed to appraise tofersen via the HST, this wouldn’t be the end of the story. There would still need to be an appraisal process, and there is no guarantee what the result of that would be. But it would unblock the current logjam, bring Biogen back to the table, and give people with SOD1 MND hope for a future where tofersen is available on the NHS.
NICE’s own guidance allows for it. The MND community is crying out for it. And the more people who join our campaign, the better our chance of being heard.
Tofersen is an exceptional drug. MND is an exceptionally devastating condition. Please sign the petition and add your voice to all those calling for NICE to make an exception.
Richard Evans
Director of Engagement