Morning Session

7:00 - 8:00

Breakfast, Registration, and Poster Set-up

8:00 - 8:10

Welcome and Introduction

Review agenda, introductions

Stephen Groft, PhD

8:10 - 8:30

“Challenges and solutions to conducting clinical research in rare diseases”

Outline of major issues with additional intent of setting up the day

Peter Merkel, MD, MPH

8:30 - 9:15

“Clinical trial design issues and options for study of rare diseases”

Studies of small sizes; trial design (open-label, multicenter, N-of1), etc CASE STUDIES

Jeffrey Krischer, PhD

9:15 - 9:45

“Ethical issues, conflicts of interest, and privacy issues unique to rare diseases research”

Outline unique ethical and privacy issues surrounding conducting research in rare diseases

Ezekiel Emmanuel, MD, PhD

9:45 - 10:00

Break

10:00 - 11:00

“From the lab to labeling: clinical drug to development for rare diseases.  Investigator, industry, and regulatory Perspectives”

Based on a single case study of a new drug successfully developed, studied, and achieving FDA labeling.

Bonnie Ramsey, MD
Eric Olson, PhD

11:00 - 11:30

“Energizing and engaging patient advocate groups to partner in rare diseases research”

Investigator and CPAG partner

12:30 - 2:00

Poster session and lunch

Afternoon Session

2:00 -3:15

Breakout session 1

• Career development and mentoring OR
• Logistics of conducting rare disease research

All breakouts will be repeated in session 2

3:30 - 4:45

Breakout session 2

• Career development and mentoring OR
• Logistics of conducting rare disease research

5:00 - 5:30

Critical evaluation of day’s program

Written (anonymous) evaluation form with comments; open discussion

Attendees and Organizing Committee

Evening Session

5:30 - 7:00

Wine and cheese receptions
Trainee Networking

Informal session