DIA 2017 Annual Meeting

Agenda

Day 1 Jun 19, 2017

11:00 AM — 12:15 PM

FDA Rare Disease Town Hall

Day 2 Jun 20, 2017

10:30 AM — 11:45 AM

10:30 AM — 11:45 AM

Serving the Patient Who Needs Early Access to Treatments and Achieving Meaningful Outcomes

10:30 AM — 11:45 AM

Success in Early CNS Drug Development: Getting Proof-of-Principle by Stacking the Deck with Biomarkers

2:00 PM — 3:15 PM

4:00 PM — 5:15 PM

Challenges and Issues in Early-Phase Oncology Trials

4:00 PM — 5:15 PM

Integrating the Patient's Voice Across the Development Program of Rare Diseases: At the Table - Where to Sit?

Day 3 Jun 21, 2017

10:30 AM — 11:45 AM

Engagement, Education, Networks, Media, and Societies in Rare Diseases: The MUST Haves

10:30 AM — 11:45 AM

Is the Future Bright for Treatment of Neurodegenerative Diseases?

2:00 PM — 3:15 PM

Collaborative Efforts to Accelerate Rare Disease Research and Development

2:00 PM — 3:15 PM

Innovative Designs and Statistical Approaches for Small Trials: Rare Disease or Pediatric Indications

2:00 PM — 3:15 PM

Pregnancy and Lactation Labeling Rule: Unique Challenges to Meet Requirements

4:00 PM — 5:15 PM

Increasing Participation in Clinical Trials Among Under-Represented Populations

4:00 PM — 5:15 PM

Looking to the Future for EU Pediatric Investigation Plans

Day 4 Jun 22, 2017

10:45 AM — 12:00 PM

Exploration of Metabolic Diseases: Is the Liver the New Heart?

10:45 AM — 12:00 PM

Recruiting Rare Disease Patients: A Unique Set of Challenges

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