Webinar: Impact of Engaging Patients

Sanofi and BioMarin share their initiatives on the significant progress they’re making in clinical outcomes by engaging patients through their clinical research experience. Q&A with the presenters will immediately follow the presentations.

Part 1: Sanofi’s New Initiatives Firmly Embedding Patient Experience, Perspectives and Priorities from Discovery Through Patient Access

Sanofi discusses how they created and implemented an end-to-end fully integrated patient informed R&D organization that works directly with patients as advisors in 100% of development staged programs from discovery and supporting research pipeline prioritization to registration. Key topics include:

• Aligning with the patient community’s most pressing needs
• Gaining insights that established clinical programs and studies and reshaped clinical trial designs and logistics
• Prioritizing early diversity & inclusion planning
• Generating patient relevant data that supports improved patient experience

Part 2: Educating Regulatory on What Matters Most to Patients

Biomarin shares initiatives they implemented to carve a path for FDA and EMA to openly acknowledge the ways in which patient voice matters to inform regulatory decision-making, especially when moving medical development forward. Key topics include:

• Patient community insights on what type of PED best characterizes the patient experience beyond Clinical Outcome Assessments to support regulatory decision-making
• How the patient community can partner on PED generation and how this can be incorporated into the regulatory review process throughout the medical product lifecycle
• Pragmatic approaches (e.g., through publicly available examples) that help patient advocacy groups and stakeholders tackle common challenges to applying regulatory guidance on PED generation (e.g., in rare disease and pediatric populations)