Awards

Project HERCULES was awarded the prestigious EURORDIS Black Pearl Award for Patient Engagement in 2019, as recognition of the unique approach to collaboration that Duchenne UK and our partner organisations have adopted.  More information is available here.

Please watch the video played at the EURORDIS Black Pearl Award Ceremony 2019

Publications

Measuring carer quality of life in Duchenne muscular dystrophy: a systematic review of the reliability and validity of self-report instruments using COSMIN. Carlton J, Powell PA & Project HERCULES Carer Group Health and Quality of Life Outcomes (2022)

Deriving a preference-based measure for people with Duchenne muscular dystrophy from the DMD-QoL. Rowen D, Powell PA, Mukuria C, Carlton J, et al. Value in Health (2021)

Development of a new Quality of Life measure for Duchenne muscular dystropy using mixed methods – The DMD-QoL. Powell PA, Carlton J, Rowen D, Chandler F, et al. Neurology (2021)

Life expectancy in Duchenne muscular dystrophy: Reproduced individual patient data meta-analysis Broomfield J, Hill M, Guglieri M, Crowther M, Abrams KA Neurology (2021)

Measuring quality of life in Duchenne muscular dystrophy: a systematic review of the content and structural validity of commonly used instruments. Powell PA, Carlton J, Buckley Woods H & Mazzone P. Health and Quality of Life Outcomes (2020)

The Impact of Payer and Reimbursement Authorities Evidence Requirements on Healthcare Solution Design for Muscular Dystrophies. Lebmeier M, Chandler F, Godfrey J & Dando J (2020)

Producing a preference-based quality of life measure for people with Duchenne muscular dystrophy: a mixed-methods study protocol. Powell PA, Carlton J, Rowen D, et al. BMJ Open (2019).

Patient reported outcome measures of quality of life in Duchenne muscular dystrophy (DMD): a systematic review of content and structural validity using COSMIN. Powell P, Carlton J, Woods H & Mazzone P HEDS Discussion Paper Series (2019).

A review of quality of life themes in Duchenne muscular dystrophy for patients and carers. Uttley et al. Health and Quality of Life Outcomes (2018)

Collaboration – the missing piece in the access puzzle? Crossley E & Chandler F. PharmaTimes (March 2018).

Improving access to new treatments in rare diseases by Emily Crossley. Published in NICE Blog (June 2018).

Sharing is Caring: The Case for Company Level Collaboration, Hatswell A & Chandler F. Published in Pharmacoeconomics (2017)

Podcast: Decoding Duchenne – Project HERCULES. Listen to Emily Crossley, Fleur Chandler and Josie Godfrey speak about Project HERCULES.

Posters

Title Category
ISPOR 2021 Burden of steroid use in the treatment of DMD: Findings from a TLR and online survey. Tolley Health Economics Poster Download (0.361MB)
ECRD 2020 Project HERCULES: Overcoming the Health Technology Assessment hurdle through patient led collaboration. Poster Download (0.002MB)
ECRD 2020 Project HERCULES: The challenges of estimating multi-state model transitions in rare diseases. University of Leicester Poster Download (0.001MB)
ISPOR EU 2019 Project HERCULES: A new paradigm for collaboration in HTA Poster Download (0.002MB)
ISPOR EU 2019 A systematic review of content and structural validity of PROs used to assess quality of life in DMD. ScHARR, University of Sheffield Poster Download (0.001MB)
ISPOR EU 2019 Construction of a new preference based measure of quality of life for DMD. ScHARR, University of Sheffield Poster Download (0.001MB)
ISPOR EU 2019 A modified Delphi study to inform a burden-of-illness study in DMD. HCD Economics Poster Download (0.849MB)
ISPOR EU 2019: A case study in developing a multi-company, flexible cost-effectiveness model in a rare disease. Source HEOR Poster Download (0.611MB)
ISPOR EU 2019: A paradigm shift in the development of cost-effectiveness models in rare diseases. Source HEOR Papers Download (0.996MB)
ISPOR EU 2019 Estimating life-expectancy for patients with DMD using individual patient data extracted from the published literature. UoL Poster Download (0.001MB)
PROMs 2018 Measuring quality of life in people with DMD: developing a new patient reported outcome measure. ScHARR Poster Download (0MB)
ISPOR EU 2018 Measuring quality of life for economic evaluation in DMD from childhood to adulthood. ScHARR Poster Download (0MB)
ISPOR Asia Pacific 2018 - The burden of illness in Duchenne muscular dystrophy - how much evidence is available? Poster Download (0MB)
ECRD 2018 International collaboration in value assessment: the HERCULES experience Poster Download (0MB)
ISOQOL 2018 Quality of life in DMD: initial findings and protocol for a new preference-based measure. ScHARR Poster Download (0MB)
ISPOR EU 2018 The challenges of estimating multi-state model transitions in rare diseases - University of Leicester Poster Download (0MB)