Project HERCULES (HEalth Research Collaboration United in Leading Evidence Synthesis) is a collaborative global project set up by Duchenne UK to increase the chances of patients with Duchenne Muscular Dystrophy (DMD) of accessing innovative treatments.

It brings together pharmaceutical companies to develop a better evidence base to help support their case for the pricing and reimbursement of new treatments for DMD.


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.

What Project HERCULES does

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 that identifies a new stage, the Transfer Stage, 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.

Project HERCULES team

Project HERCULES publications

Project HERCULES awards

Contact Us

If you would like to contact us or get involved in Project HERCULES then please email [email protected].

We are looking for people to respond to surveys and to review our findings so please do get in touch with us if you can help.