Access to treatments

Duchenne UK’s Project HERCULES Awarded Prestigious Rare Disease Award

15th November 2018

Duchenne UK’s Project HERCULES has been awarded the 2019 EURORDIS Black Pearl Award for Patient Engagement.

The EURORDIS Black Pearl awards celebrate the major achievements and outstanding commitment of patient advocates, patient organisations, clinicians, policy makers, scientists, industry and others who strive to make a difference in the rare disease community. Project HERCULES is the first of its kind to be awarded this respected title in recognition of the innovative approach to bringing stakeholders together. 

Project HERCULES is a unique international multi-stakeholder collaborative project set up by Duchenne UK to develop tools and evidence to support Health Technology Assessments and reimbursement decisions for new treatments for Duchenne Muscular Dystrophy (DMD). It brings together 7 leading pharmaceutical companies, academics, patient organisations and advisers to develop and build a better evidence base for DMD to aid the notoriously difficult pricing and reimbursement stage of drug development.

The annual awards ceremony, which will be held in February 2019, is part of a month of events to mark Rare Disease Day.  

Nominations for this prestigious award are submitted by those affected by rare diseases or working in this area. They are shortlisted by a committee of senior experts in this field. The winners are then selected by the EURORDIS Board of Directors, which is made up of senior members of the rare disease community.

Emily and Alex, Co-founders of Duchenne UK have said:

HERCULES is the first project of its kind in this space and we are delighted to have been acknowledged in this way with a Black Pearl Award.

The project is a truly multi-stakeholder collaboration that we hope will pave the way forward for more evidence generation to aid reimbursement decisions for Duchenne and other rare diseases.

We are grateful for the input into the project from people living with Duchenne and their families, patient organisations, healthcare professionals, HTA experts from around the world and for our industry partners. This would not have been possible without your willingness to collaborate for the greater good.

Josie Godfrey, Project Director for Project HERCULES added:

I am delighted that Project HERCULES has been recognised with a Black Pearl award. Bringing so many companies and experts together to build a better evidence base and the tools needed to better demonstrate the real value of treatments or DMD. This model of international collaboration between industry and patient organisations and other experts is a truly innovative model to approaching HTA that will not only bring real benefit to those living with DMD but could also benefit those with other rare conditions struggling to develop strong evidence of the value of new treatments.

Fleur Chandler, Chair of Project HERCULES Steering Group said:

I am honoured that Project HERCULES is to receive a Black Pearl award in recognition of the great strides that can be made when patient organisations, rare disease families, academia and the pharmaceutical industry unite. I am delighted that this award will also highlight the critical importance of the process of gaining access to medicines in rare diseases.

Duchenne UK and Project HERCULES would like to thank our industry partners Pfizer Inc, PTC Therapeutics International Ltd, Roche, Sarepta Therapeutics Inc, Solid Biosciences, Summit Therapeutics plc and Wave Life Sciences USA Inc.

Update 13th February 2019:

This film explaining the project was played at the awards ceremony:


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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 treatment or 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. 

How to donate?

Duchenne UK is entirely reliant on donations to fund research for treatments and a cure to DMD. This can be done via:

  • 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

For more information and interview requests

Visit www.duchenneuk.org

Molly Hunt – Communications Manger, Duchenne UK 

E: [email protected]


Published on 15 November 2018

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