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Session 1: Keynote – Patient Advocacy
Session Chair(s)
Zoran Antonijevic, MSc
Vice President, Statistical Consulting
Abond CRO Inc., United States
Daniel Millar, MBA
Senior Director, Strategic Business Transformation
Johnson & Johnson, United States
Families in the rare disease community are familiar with the complicated, and often frustrating, clinical trial process. How can we make clinical trials faster, less burdensome, and open to all comers, no matter the stage of disease? Would it be possible to understand the individual’s response to a compound or biologic (intervention) more quickly? Would it be possible to have less exposure to placebo? To initiate testing of combinations of promising therapeutics earlier? To get to a go/no-go more quickly? Could we learn from our recent experience with COVID-19?
One potential pathway to overcoming obstacles in clinical trials for rare disease therapeutics is to address trial protocols, and a master protocol is one innovative approach that may have advantages. Experiences from their use for COVID-19 and in the development of approaches for Duchenne Muscular Dystrophy can inform their uses for rare indications.
Learning Objective : At the conclusion of this activity, participants should be able to:
- Recognize the benefits of master protocols from a patient perspective
- Explain core patient-centric principles that can drive meaningful outcomes and uptake
- Evaluate existing opportunities to create further efficiency in clinical trials
Speaker(s)
Speaker
Patricia Furlong, BSN
Parent Project Muscular Dystrophy, United States
Founding President and Chief Executive Officer
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