Policy and campaigning

Duchenne UK attend meeting for All-Party Parliamentary Group On Off-Patent Drugs

24th October 2018

Yesterday, representatives from Duchenne UK attended an All-Party Parliamentary Group (APPG) meeting on off-patent drugs in Westminster. The group, chaired by Nick Thomas-Symonds MP, was attended by representatives from patient organisations for different disease areas, including Parkinsons disease and Breast Cancer.

In December 2017, a report was published by the AMRC to look at the potential for using off-patent, repurposed medicines. The report was produced in response to a request from the Minister for Life Sciences, we are still awaiting a response from the minister about this report. 

The body of the meeting included an update from Rebecca Bloor, from the Royal Pharmaceutical Society, on the British National Formulary (BNF). The BNF is pharmaceutical reference book for the UK, which contains a wide spectrum of information and advice on prescribing medicine. The BNF is used by GP’s and pharmacists for guidance on how to treat common conditions. Having information about the use of off-patent drugs in the BNF could give doctors the confidence to prescribe these drugs.

In 2016, a project was initiated to improve the information about off-label prescribing in the BNF. 270-300 off-patent drugs were agreed with NICE to be researched and added into the BNF. The Royal Pharmaceutical Society looked at existing literature and examples of off-patent drugs being used to treat a certain condition. 80% of the 270-300 drugs determined in 2016 have been reviewed, the remaining 20% are either in progress or advice is being sought regarding issues such as dosing.

Rebecca Bloor highlighted that groups and patient organisations can write to the BNF to highlight other off-patent drugs which may be effective for another disease. This will trigger their usual process of researching and validating any claims made.

Duchenne UK is investigating the use of several off-patent drugs for the treatment of Duchenne muscular dystrophy, using a technique called repurposing. We will continue to support the great work achieved by this group so far, as part of our commitment to develop repurposed treatments for DMD. DMD is a rare disease with very few approved treatments, so doctors may prescribe patients with off-patent drugs if there is substantial evidence to show they are an effective treatment for DMD.



What is Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy is the most common fatal genetic disease diagnosed in childhood. 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. It almost exclusively affects boys. There is no cure. In the UK there are around 2,500 boys affected and around 300, 000 worldwide. It is classified as a rare disease. 

Who are 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. 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. 

Duchenne UK is entirely reliant on donations to fund and accelerate the search for treatments for DMDPlease help us and donate today:
  • Direct Debit – Duchenne Direct
  • Individual Donation – Donate
  • If you are a family or friend affected by DMD you can set up your own fund with Duchenne UK – Family and Friend Funds
  • Take part in one of our fundraising events – Events
  • Text DUCH10 £10 to 70070

Published on 24 October 2018

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