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. 

In January 2017, at a meeting organised by Duchenne UK, several companies expressed an interest in working together with Duchenne UK to develop key tools to support HTA processes. Project HERCULES has since been established to take forward this work.  Project HERCULES will have two phases. The planned outcomes for each phase are described below. 


  • Data sharing platform to share the project outputs including a systematic literature review and burden of illness data.
  • Data analysis to support HTA and reimbursement decisions including a review of current data and mapping of trial endpoints to clinical outcomes and natural history data. 
  • Quality of Life metric – a review of current metrics and their suitability for use in DMD; the development of a de novo metric or add-ons to an existing metric; and the use of the metric to generate scores for the relevant health states.


  • A natural history disease model populated with data from global sources.
  • A core economic model, adaptable by individual companies, to support HTA and reimbursement decisions.
  • A burden of illness study to map the impact of DMD on patients, families and carers.
  • HTA support and advice, potentially including guidance for approaching managed entry agreements.  

Deliverables to be completed in 2020:

  • A critique of commonly used Quality of Life metrics – available here
  • A natural history model of DMD
  • A core economic model  
  • A bespoke Quality of Life metric
  • A guide to Health Technology Assessment
  • Burden of Illness study
  • A final project report


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.