Project HERCULES 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. 

Duchenne UK’s Project HERCULES Awarded Prestigious Rare Disease Award

Pharmaceutical companies wanting to introduce new treatments for patients in many countries are asked to submit dossiers providing evidence for clinical and cost effectiveness of the new technology to Health Technology Assessments (HTA) agencies and payers. For example, in the UK the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) are likely to review all new treatments for DMD. These assessments determine if a new treatment should be funded by the NHS and made available to patients. 

Developing evidence for HTA dossiers is particularly challenging in rare diseases such as DMD given the sparsity of available evidence to support the requirements of HTA agencies. Many pharmaceutical companies are developing potential treatments for DMD and are working individually to develop their approach to HTA. The variety of methods in use and the difficulties of generating data can lead to delays in introducing new treatments and inconsistent decision-making. 

Project HERCULES has already delivered: 

  • A new natural history model for DMD and new mortality data analyses.
  • DMD-QoL, a new bespoke Quality of Life measure for DMD, along with 26 translations/linguistic validations to ensure it can be widely applied in clinical trials and practice. 
  • A burden of illness study to map the impact of DMD on patients, families and carers.
  • A core economic model, adaptable by individual companies, to support HTA and reimbursement decisions.
  • A systematic review of Quality of Life measures for DMD carers and families.
  • A review of the burden of side effects from long-term corticosteroid treatment.
  • Analysis of patient data from Clinical Practice Research Datalink, to inform the natural history and burden of illness workstreams.

In 2022 we will produce: 

  • Further peer reviewed publications for project outputs.
  • A toolkit of resources to promote awareness and understanding of key findings among clinical and patient communities.


Why did Duchenne UK start Project HERCULES?

Duchenne UK started Project HERCULES in response to the challenges and delays in the Health Technology Assessment of the first licensed treatment for DMD. Fleur Chandler recognised that the process would have been much simpler if the evidence base and tools available to companies were improved and this led to the creation of Project HERCULES.

Is Project HERCULES just for DMD?

Project HERCULES is focused on DMD. The outputs from Project HERCULES could have real benefits for other disease areas, particularly life limiting paediatric or neurodegenerative conditions.