Accelerating access to treatments

We launched Project HERCULES in (HEalth Research Collaboration United in Leading Evidence Synthesis) in 2017: a groundbreaking, global project which brings pharmaceutical companies together to increase the chances of new DMD treatments being approved and made available to patients.

Pharmaceutical companies wanting to introduce new treatments for patients are asked to submit evidence of clinical and cost effectiveness to Health Technology Assessments (HTA) agencies and payers. We set up Project HERCULES in response to the challenges and delays in the HTA of the first licensed treatment for DMD.

The projects aims to improve the evidence base and tools available to pharmaceutical companies, so that their treatments can be better assessed.

Project HERCULES has already delivered: 

• A new natural history model (a model of how the disease progresses without treatment) for DMD and new mortality data analyses
• A new bespoke Quality of Life measure for DMD, the DMD QoL
• A burden of illness study to map the impact of DMD on patients, families and carers
• An economic model, which can be adapted by individual companies, to support the assessment and reimbursement of DMD treatments

Find out more on the Project HERCULES website

Patient organisations play a pivotal role in helping regulators and policy makers understand the realities of living with DMD. We need to engage with them early in the drug development process to ensure that when drugs do become approved for use, the NHS can afford to pay for them, and understands the true value of these potential treatments for patients and families living with DMD.

Duchenne UK works with regulators on a number of issues, from how to speed up the drug development process, to advising on the design of clinical trials and advising on what outcomes are meaningful for patients when drugs are being developed. We work at UK level with the Medicines and Healthcare Products Regulatory Agency (MHRA), and both of our founders are members of the MHRA’s Patient Consultative Forum.