Duchenne UK responds to a new system to allow NHS patients faster access to ‘breakthrough’ medicines

The Government has today, Friday 3 November, announced a new scheme that will offer drug companies a fast-track route to get ‘breakthrough’ medicines approved for use by the NHS. The Accelerated Access Pathway initiative could help to make drugs available to patients up to four years sooner.

The initiative will launch in April next year and will offer a ‘breakthrough’ designation to up to five promising new drugs each year. These products will receive public sector support to speed up commercial deals with the NHS. 

The Accelerated Access Pathway is part of the Government’s response to the long-standing issue that patients often receive a raw deal when accessing the latest treatments through the NHS. A frustration that is further compounded when many innovative treatments have been made possible by UK science.

Responding to the news, Emily Crossley, CEO and Co-founder at Duchenne UK, said: “With the pace of innovation in drug development quickly outstripping the time it takes to approve them for use by NHS patients, the Accelerated Access Pathway is a welcome move. However, it doesn’t solve the problem entirely – the number of treatments that get designated as ‘breakthrough’ may be quite small.

“With potentially life-altering treatments for Duchenne muscular dystrophy (DMD) tantalisingly close, we need to be doing everything we can to promote greater efficiency in the approvals and pricing process. This is why Duchenne UK will be launching a program in the coming weeks to help companies collaborate to develop evidence for a better engagement with reimbursement bodies.”



About Duchenne Muscular Dystrophy (DMD)


  • There are around 2,500 boys affected by DMD in the UK and around 300,000 worldwide. It is classified as a rare disease
  • Duchenne Muscular Dystrophy is the most common fatal genetic disease diagnosed in childhood, almost exclusively affecting boys
  • Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. There is no treatment or cure


About Duchenne UK


  • Duchenne UK is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy (DMD). Duchenne UK has raised more than £5.5million in five years, and spent or committed £5million to DMD research projects – more than any other organization
  • Duchenne UK is leading the drive towards the efficient repurposing of existing medicines to bring new treatments for DMD
  • Duchenne UK believes that every child with Duchenne should be given the opportunity to access a clinical trial. Duchenne UK created the DMD Hub in 2016 to expand and develop the infrastructure for DMD trials in the UK and accelerate access to clinical studies for those who are interested in participating
  • The charity has been formed by the coming together of Joining Jack and Duchenne Children's Trust, the two biggest funders of research in the UK in the last three years. Its president is HRH The Duchess of Cornwall. Its patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell



Visit duchenne.org for more information or follow @DuchenneUK on Twitter.


Contact or more information and interview requests: Molly Hunt, Communications Manager: [email protected]


Published on 11 March 2017

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