Improving access to new treatments in rare diseases

Emily tells us how a ground-breaking HERCULES project will help NICE to get the evidence we need faster.

Emily Crossley, co-founder and co-chief executive of Duchenne UK

I was working as a journalist when my son, Eli, was diagnosed with Duchenne Muscular Dystrophy (DMD). In that moment, I went from covering other people’s tragedies to being at the centre of my own.   

We started looking into treatment options, only to find there were effectively none. There are many promising treatments in development, but small patient populations and limited resources mean gathering evidence is a significant challenge.

And without a robust evidence base, health technology assessments (HTA) risk being delayed, which then means patients like Eli miss out on potentially life-changing treatments.

At Duchenne UK, we are committed to accelerating new treatments for DMD. We couldn’t accept that a new treatment might be delayed because the right kind of evidence wasn’t being presented to HTA bodies like NICE.

In late 2017 we launched HERCULES (HEalth Research Collaboration United in Leading Evidence Synthesis) where seven pharmaceutical company partners committed to working with Duchenne UK and the Universities of Sheffield and Leicester, to generate, align and share data for HTAs.  

An ambitious series of activities is already underway, including mapping clinical trial progress, developing a common economic model and a new quality of life metric. This information will be available to all partners, ensuring a consistent, common approach that we hope will save money, effort and – most importantly – time.

This is a completely ground-breaking and unique collaboration. And, while HERCULES focuses on DMD, the model has the potential to better demonstrate the value of medicines for other rare and orphan diseases too.

DMD is the most common fatal genetic disease diagnosed in childhood. It almost always affects boys. Around 2,500 children and young people have DMD in the UK - they will be paralysed by the end of their teens, and are unlikely to live beyond their 20s.

For Eli and the hundreds of other boys like him, time is running out. There are treatments on the brink of a breakthrough, but can we be sure that they will available as quickly as possible? HERCULES is at the heart of a quiet revolution that could transform the lives of rare disease patients for many years to come.

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