Making sense a future perspective Making outcomes work SCOPE-DMD - PowerPoint PPT Presentation

Making sense – a future perspective “Making outcomes work” – SCOPE-DMD Workshop Annemieke Aartsma-Rus London, May 26 2016

COST Action BM1207 core group meeting 2 Department of Human Genetics Annemieke Aartsma-Rus

Meeting April 29 2016 http://exonskipping.eu/media/videos-workshop-2015/ 3 Department of Human Genetics Annemieke Aartsma-Rus

Paper will be online June 6 4 Department of Human Genetics Annemieke Aartsma-Rus

Take home messages • Need a collaborative and constructive dialogue between all stakeholders • Need to develop and implement care standards • Involve all stakeholders to develop functional and molecular outcome measures • Collect high-quality data for natural history and outcome measures • Trial design • Regulatory approval 5 Department of Human Genetics Annemieke Aartsma-Rus

Future directions (focus on OMs) • Define a core set of outcome measures for clinical trials • Develop outcome measures for non-ambulant patients • Involve regulators in development of outcomes measures • Publish date on natural history and outcome measures è This meeting 6 Department of Human Genetics Annemieke Aartsma-Rus

Wrap up – Molecular biomarkers • DNA and serum biomarkers • Standardization and collaboration • MRI • Correlation with function and strength • Many trials now include MRI • Regulatory requirements • Efficacy measurements • Data and quality important (collect, collaborate and share!) • Training important (standardization/replication) • Secondary outcomes from patients important (PROM & QoL) • Come early and discuss – regulators are willing to help • Choose appropriate marker(s) (therapy and stage) • Involve patients 7 Department of Human Genetics Annemieke Aartsma-Rus

Wrap up – Functional outcomes • Once scale cannot measure everything for all stages • Upper limb • Myotools, activity monitors, PUL, reachable workspace • Relation strength and function and quality of life • Decrease in upper limb function takes decades • Selection of outcome measures (stage related) • Collaboration with patients • Combined efforts: PUL and PROM (DMD-Upper PROM) • Collaboration with patients to develop scales and trial design (drug development starts and ends with patients) • Meaningful • Regulators need data confirming correlation to function that is important to patient 8 Department of Human Genetics Annemieke Aartsma-Rus

Thank you 9 Department of Human Genetics Annemieke Aartsma-Rus