Access to treatments

Duchenne UK and Project HERCULES announce new patient centred quality of life tool for Duchenne muscular dystrophy

Duchenne UK and Project HERCULES announce the launch of a new patient centred, quality of life tool designed to support faster access to new treatments for people living with Duchenne muscular dystrophy

The tool which was uniquely developed through Project HERCULES, an initiative launched by Duchenne UK in collaboration with pharmaceutical companies, sets a new benchmark for rare disease drug development

As part of their ongoing work aimed at finding effective treatments and a cure for Duchenne muscular dystrophy (DMD), Duchenne UK and Project HERCULES have announced the launch of a new patient centred, quality of life tool which has been designed to support faster access to new treatments for boys and men living with DMD.

The quality of life (QoL) tool known as the DMD-QoL, developed through collaboration between pharmaceutical companies, The University of Sheffield’s School of health and related research (ScHARR) and Duchenne UK, is unique as it allows drug developers and regulators to assess new potential treatments against measures that have been found to be most meaningful to patients and their families. These measures go beyond the physical symptoms which have traditionally been the focus, to include the impact on mental health and ability to socialise. The measures were defined through a programme quantitative research conducted with people living with DMD and their families.

Quality of life is an important measure used by many drug regulators when deciding whether to reimburse a new treatment, and at what price. But historically the QoL measures used for DMD have inadequately captured the full lived reality of the disease. This could lead to drugs which are capable of making meaningful improvements to patients and their families not being reimbursed or having access delayed if they do not meet the traditional QoL measures.

Emily Crossley, CEO Duchenne UK said:

“Duchenne is a progressive and life-limiting disease, and the boys who are living with it today don’t have time to waste in gaining access to new treatments which could improve and lengthen their lives. We are incredibly proud of what we have been able to achieve through Project HERCULES and we look forward to seeing the DMD-QoL tool being used throughout the drug development and assessment pathway to accelerate access to new treatments”

Dr Philip Powell, Research Fellow ScHARR said:

“Developing the DMD-QoL has been a fantastic project for us to be involved in. The level of collaboration between members of the Duchenne community and other stakeholder organisations has been exceptional and has allowed us to develop a tool which we are confident will have a significant impact in the process of developing new therapies for DMD.”

Project HERCULES is now partnering with the Clinical Outcomes team at Oxford University Innovation in a ground-breaking collaboration to ensure that anyone undertaking research in DMD will be able to use the DMD-QoL. This includes companies undertaking clinical trials, charities, universities, and researchers. Companies who have developed new treatments for DMD will be able to use the DMD-QoL to measure QoL when they submit evidence to drug approval agencies, such as NICE in the UK.

Matt Perkins, CEO Oxford University Innovation said:

“It is wonderful to see this collaboration where expertise nurtured at OUI is now being deployed to support global research aimed at tackling Duchenne muscular dystrophy”

Licences to use the DMD-QoL in non-commercial academic research and publicly funded healthcare are provided free of charge as part of our commitment to supporting the DMD-QoL for the benefit of the Duchenne community.

Further information on the DMD-QoL can be found on the Clinical Outcomes website along with the facility to request use of the DMD-QoL and support materials, including access to the rapidly growing library of available translations.


Published on 7 September 2021

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Note for editors

What is Project HERCULES?

Project HERCULES (HEalth Research Collaboration United in Leading Evidence Synthesis) is a ground breaking multinational collaboration 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 patient organisations, clinicians, academics, leading pharmaceutical companies, Health Technology Assessment agencies and other advisers to build a better evidence base for DMD. 

Project HERCULES was awarded the prestigious EURORDIS Black Pearl Award for Patient Engagement in 2019, as recognition of the unique approach to collaboration that Duchenne UK and our partner organisations have adopted

What is Duchenne muscular dystrophy?

Duchenne muscular dystrophy (DMD) is a devastating muscle-wasting disease. It is the most common and severe form of muscular dystrophy. Typically diagnosed in early childhood, it mainly affects boys. Children born with DMD cannot produce the protein dystrophin, which is vital for muscle strength and function. Without it, their muscles gradually weaken, leading to a loss of mobility often by their teens. It eventually affects the heart and lungs, shortening their life expectancy. In the UK, there are around 2,500 people affected and around 300,000 worldwide. 

Who is Duchenne UK?

As the leading Duchenne muscular dystrophy (DMD) charity in the UK, we’re going further to find effective treatments for DMD to end its devastating impact. We’re doing it faster, too, by accelerating access to these treatments and therapies for this generation of patients. And we’re here to support every family affected and ensure that they receive the best possible care.  Together, we will end Duchenne.  

Our president is HRH The Duchess of Cornwall. Our patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell. 

Who is Clinical Outcomes at Oxford University Innovation?

Clinical Outcomes is a specialist team within Oxford University innovation (the Technology Transfer Company for the University of Oxford) providing access to, and support in the use of, some of the most prestigious patient centred outcomes tools available.