Now that would be a nice problem to have, or would it?

With the best will in the world, we cannot expect our dedicated researchers to work alone and carry the full burden of the challenge to defeat this disease.

Researchers are working the science. Biotechnology and pharmaceutical companies are investing in treatment development and charities support care, advocacy and research funding. 

But there is a vital important missing piece that, as new treatments are developed, increasingly has the potential to become a major choke point. This is the need for UK regulatory and NHS access reform.

Many might say

 ‘That would be nice problem to have! We need to get the treatments developed first, we should only be focusing on that!’ 

The truth, however, is that unless comprehensive reform discussions begin now, the outcome is sadly very predictable. More on this at the end of this post.

What is the challenge that necessitates our regulators to reform?

Our researchers, globally, work in earnest on treatment candidates. They aim to discover/create disease modifying treatments. This can take many years in itself. Then, and only after a successful trials(s), they have to present the evidence to our regulators (MHRA and NICE) for scientific scrutiny and price negotiations. 

Imagine, for one moment, a treatment is found to be effective through trials.

Actually we don’t have to imagine! One was trial proven and safe some 3 years ago, tofersen for SOD1 MND/ALS.

Tofersen demonstrated true clinical benefit and received FDA approval for eligible MND patients in the USA way back in 2023.

It is now 2026 and tofersen is still not fully approved for routine use in the UK!

During that time, patients have faced extraordinary stress, uncertainty, inconsistent information and ongoing barriers to access.

During this delay, some patients will already have suffered irreversible and life-limiting loss of function, including loss of speech, swallowing, mobility and independence, while regulatory and funding systems continued to debate process, timelines and cost.

Fortunately, Biogen, the dedicated Pharma behind tofersen, have deep pockets and are providing the treatment free, until NICE agree full authorisation, under an early access program. But as per our earlier post described this year, access remains problematic.

We almost dare, therefore, not to think of a future viable, trial proven, treatment developed by a small biotech, perhaps for a far wider eligible group of patients. A small biotech which perhaps doesn’t have the funds to operate an early access plan? The current bureaucracy of the MHRA/NICE and unfathomable rules surrounding government funding would be potentially killing patients.

This is not only about tofersen. Tofersen is, however, the proverbial canary in the coal mine. Let’s heed the warning?

The UK is just simply not structurally prepared for future inherited or sporadic MND treatments, including precision, biomarker-led, RNA-based, gene-altering and disease-modifying therapies. It took, for eg, over 18 months for the UK MHRA to just agree the most appropriate currently available pathway for tofersen! We can’t see this as anything other than a broken system.

It most certainly is not a nice problem to have!

Brutally put, and we in United2EndMND hate to say this, unless this challenge is tackled right now, when the next treatment is taken to the UK regulators, it will take 3 or more years before it is widely available. Given the average survival post diagnosis is 3 years, the math, as they say, says it all.

Our disease desperately needs this reform along with integrated and clear schemes to provide early access funding. It’s not only MND (a fast progressive disease) that is crying out for reform, other diseases with a similar prognosis will also need changes. Each disease, however, might need specific criteria, for example disease measures to be agreed with regulators.

Watch out for further posts on this subject, as we believe, like us, the whole community will innately care about this issue that is boiling under the surface.

We never forget, that there are things patients, campaigners and advocacy groups can say publicly that researchers and charities often cannot. This includes challenging Government, regulators and NHS systems directly when delays are causing avoidable harm. 

United2EndMND