Ways to measure clinical effectiveness in the investigation of medicinal products for the treatment of DMD/BMD

DMD workshop 2013

Session 1 - What is clinically meaningful and how to measure this?

Chaired by Jan Verschuuren

Jan Verschuuren
Video showing natural history of the disease demonstrating functions and milestones

Natural History of DMD/BMD
Craig McDonald
Summary of the decision framework for inclusion of clinical outcome measures in clinical trials and studies across the natural history of DMD

What patients see as being clinically meaningful
Pat Furlong, Elizabeth Vroom,
and Phillippa Farrant
What are the major milestones of the disease and how it is important to measure and quantify these in the context of a clinical study?

Outcome measures in young children
Eugenio Mercuri
Selection criteria for outcome measures

Outcome measures in ambulatory patients
Craig McDonald

Outcome measures in non-ambulant patients
Anna Mayhew

Session Discussion
Natural history and major milestones of the disease
Selecting outcome measures for clinical trials

Session 2 – Methods of efficacy measurement – strength versus function

Chaired by Francesco Muntoni


Francesco Muntoni

Natural history studies showing relationship in the lower extremities
Craig McDonald

Natural history studies showing relationship in the upper extremities
Thomas Voit

Session Discussion and round-up of morning presentations
Strength and Function as co-primary endpoints – is this the best choice in trial design?

Session 3 – Animal Models and Biochemical Outcome Measures

Chaired by Annemieke Aartsma-Rus
How reliable are Animal Models?
Annamaria de Luca and Nic Wells

Biochemical Outcome Measures and Biomarkers to improve patient selection and homogeneity for clinical studies

Prosensa and Annemieke Aartsma-Rus

Session summary


Annemieke Aartsma-Rus

Session 4 – Strategy & Design of Clinical Studies
- Extrapolating to other stages of the disease

Chaired by Thomas Voit

Accelerated design
Use of natural history data and historical controls
Extrapolating from ambulant to non-ambulant patient studies

Clinical perspective
Thomas Voit and Nathalie Goemans

Video showing quality of life in non-ambulant boys and men
Patient perspective
Elizabeth Vroom and Filippo Buccella

Final comments and round-up by the Session Chairs

Including on-going work and other issues to be addressed
12 Apr 2017