Achieving Meaningful Regulatory and Clinical Outcomes for Patients: Strategies in Rare Disease Therapy Development

A collaborative workshop designed to examine practical issues in the structuring of rare diseases therapeutic development programs.

Agenda

Day 1 Oct 06, 2015

1:00 PM — 4:30 PM

Tutorial: Study Endpoint Development in Rare Diseases

Day 2 Oct 07, 2015

7:00 AM — 8:00 AM

Registration and Continental Breakfast

8:00 AM — 8:15 AM

Welcome and Opening Remarks

8:15 AM — 8:30 AM

Case Study Presentation

8:30 AM — 9:30 AM

Session 1: Orphan Drug Development and Its Regulatory Challenges

9:30 AM — 10:00 AM

Refreshment Break

10:00 AM — 11:30 AM

Session 2: Clinical Development for Rare Diseases

11:30 AM — 12:30 PM

Luncheon

12:30 PM — 2:00 PM

Session 3: Impact of Early Patient Engagement

2:00 PM — 2:30 PM

Refreshment Break

2:30 PM — 4:00 PM

Session 4: Roundtable Discussion: Creating Solutions for Complex Challenges in Rare Disease Development

4:00 PM — 4:30 PM

Concluding Workshop Discussion

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