Author(s)
Crossley E1, Chandler F2, Godfrey J3, Abrams K4, Hill M5, Mumby-Croft J6, Carlton J7, Powell PA7, O'Hara J8, Woodcock F9, Rose A8
1Duchenne UK, London, UK, 2Duchenne UK, Sunbury, LON, UK, 3JG Zebra Consulting, London, UK, 4University of York, Leicester, LEC, UK, 5University of Leicester, Leicester, LEC, UK, 6Source Health Economics, Oxford, OXF, UK, 7University of Sheffield, Sheffield, UK, 8HCD Economics, Daresbury, UK, 9Source Health Economics, Oxford, UK
OBJECTIVES: Project HERCULES is a collaborative global project set up by Duchenne UK, a patient organisation, to bring together pharmaceutical companies, academia, HTA bodies, clinicians, and patients to build tools and evidence at a disease level for use in HTA focused on Duchenne Muscular Dystrophy. METHODS: Duchenne UK leads a multistakeholder international collaboration developing a Quality of Life metric that better captures important aspects of Quality of Life for people living with DMD, a natural history model, a burden of illness study and an economic model. These workstreams are supported by leading academics and consultants who are working together to ensure that each RESULTS: Alongside the progress of individual workstreams, the poster will describe as the benefits and challenges of the Project HERCULES approach. This will include the challenges of having a diverse, dispersed, international evidence base, facing a lack of mortality data in a disease which is 100% fatal, the use of quality of life metrics in a paediatric progressive condition, creation of a disease level model suitable for different interventions and agreement on what is important in burden of illness. CONCLUSIONS: The benefits of this approach include presenting a credible, comprehensive, peer reviewed suite of evidence that best reflects patient and clinical experience. Working with world leaders in their respective fields ensures high quality outputs readily adaptable to the needs of HTA systems. This is a cost and time effective approach for all parties in a rare yet heavily researched disease population.
Conference/Value in Health Info
2019-11, ISPOR Europe 2019, Copenhagen, Denmark
Code
PND21
Topic
Epidemiology & Public Health, Patient-Centered Research
Topic Subcategory
Disease Classification & Coding, Patient Engagement, Patient-reported Outcomes & Quality of Life Outcomes
Disease
Musculoskeletal Disorders, Neurological Disorders, Pediatrics