OBJECTIVES: Project HERCULES is an international patient led multi stakeholder collaboration developing tools and evidence supporting HTA for new treatments for Duchenne Muscular Dystrophy (DMD) including a bespoke Quality of Life metric, a natural history model, a burden of illness study and a core economic model. The evidence reflects patient experience and patients and patient organisations can shape the framework for HTA in DMD. This has led to a new paradigm for developing evidence for HTA and an improved understanding of DMD impacting on HTA decisions.

METHODS:

Project HERCULES is led by Duchenne UK with a core team including two parents of boys with DMD, one of whom is also a health economist. This unique combination brings health economics and HTA expertise combined with the lived patient experience to deliver this ambitious health economics research programme. Patient organisation leadership enables access to data sources and expertise which may be inaccessible for individual researchers.

An iterative approach to the individual workstreams ensures each workstream has high levels of patient involvement, shaping outcomes to embody actual patient and family experience.

RESULTS:This patient led approach has led to each workstream of Project HERCULES better reflecting the true impact of DMD:

• Identification of a previously undefined disease stage between the traditional stages of late ambulatory and early non ambulatory
• Development of a bespoke Quality of Life metric
• An economic model encompassing the actual experience of patients and families
• A burden of illness study focussing on what is most impactful on patients and families.
• Identification of future research priorities reflecting what is important to patients.

CONCLUSIONS:Project HERCULES has demonstrated the potential impact of patient led evidence generation in a rare disease for use in HTA.