Symposium: Best Practices in Clinical Study Design for Rare Diseases

Symposium: Best Practices in Clinical Study Design for Rare Diseases
April 29 - 30, 2013
George Washington University

The Clinical and Translational Science Institute at Children’s National (CTSI-CN) is hosting an event at the George Washington University Jack Morton Auditorium to talk about the challenges and potential solutions surrounding rare disease product development. The format of the two-day event will feature roundtable discussions with moderators and speakers like John McKew from the NIH, Mark McClellan from the Brookings Institute, and Gayatri Rao from the FDA, as well as for-profit companies, advocacy groups, and non-profit organizations. Topics will include:
  • Statistical and Study Design Challenges
  • Product Development such as the use of biomarkers
  • Considerations for Best Practices in Rare Disease Product Development
  • Sustainability of Rare Disease Product Development
  • Infrastructure for Rare Disease Product Development
Agenda:

Day 1: Monday, April 29, 2013
Setting the Stage for Discussion of Rare Disease Drug Development and Clinical Trials (.pdf) Co-Chair, Vincent A. Chiappinelli, PhD, George Washington University (GW)
Co-Chair, Edward M. Connor, MD, MBEChildren’s National
Rare Diseases: Landscape and Overview (.pdf) Katherine Needleman, MS, PhD, RACOffice of Orphan Products Development, FDA
Challenges and Solutions in Drug Development for Rare Diseases (.pdf) John McKew, PhDTRND, NIH
The Regulatory Pathway for Rare Diseases: Lessons Learned from Previous Examples of Clinical STudy Designs for Small Populations (.pdf) Robert Temple, MD, CDER, FDA
Regulatory Experience from Clinical Studies in Enzyme Deficiency Disease (.pdf) Anne Pariser, MDCDER, FDA
Potential Path for Regulatory Process in Rare Disease Space (.pdf) Marshall L. Summar, MDChildren's National
A Patient Advocate's View of Drug Development in Rare Diseases (.pdf) Peter L. Saltonstall, CEO, NORD
Clinical and Genetic Characterization of Duchenne Muscular Dystrophy Patients (.pdf) Paula R. Clemens, MDUniversity of Pittsburgh
Clinical and Genetic Characterizations of Cystic Fibrosis Patients (.pdf) Bruce C. Marshall, MDCystic Fibrosis Foundation
Roundtable 1: Statistical Challenges in Clinical Study Design in Rare Diseases  Moderator: Robert Temple, MD (.pdf), CDER, FDA
Panelists:
Edmund V. Capparelli, PharmD (.pdf), University of California San Diego
Avital Cnaan, PhD (.pdf), Children's National
Susan S. Ellenberg, PhD (.pdf), University of Pennsylvania
Edward M. Kaye, MD (.pdf), Sarepta Therapeutics
Michael A. Pacanowski, PharmD, MPH (.pdf), FDA
Prof. Martin Posch, PhD (.pdf), University of Vienna
Roundtable 2: Drug Development Tools in Rare Diseases  Moderator: Stephen C. Groft, PharmD (.pdf), NIH
Panelists:
Felix Ratjen, MD, PhD, FRCPC (.pdf), Hospital for Sick Children, University of Toronto
Barry J. Byrne, MD, PhD (.pdf), University of Florida
Talissa Altes, MD, University of Virginia
Marc K. Walton, MD, PhD (.pdf), FDA
Eric P. Hoffman, PhD (.pdf), Children's National
Anne Zajicek, MD, PharmD (.pdf), NICHD
Day 2: Tuesday, April 30, 2013
Roundtable 3: Considerations for Drug Development Programs in Rare Diseases (.pdf) Moderator: Anne Pariser, MDFDA
Panelists:
John N. van den Anker, MD, PhD, FCP, FAAP, Children's National
Cristina Csimma, PharmD, MPH, CEO, Cydan Development Inc.
Debra MillerCureDuchenne
Peter Mueller, PhDVertex
John D. Porter, PhD, NINDS
Adriana H. Tremoulet, MD, MASKawasaki Disease Research Center, University of California San Diego
Roundtable 4: The Economics of Rare Disease Drug Development  Moderator: Mark McClellan, MD, PhD (.pdf), The Brookings Institute
Panelists: 
Mark Trusheim, MS (.pdf), MIT
Bruce Quinn, MD, PhD (.pdf), Foley Hoag
Christopher-Paul Milne, DVM, MPH, JD (.pdf), Center for the Study of Drug Development, Tufts University Medical School
Amber Jessup, PhD (.pdf), Health and Human Services
Rare Disease Drug Development Infrastructure Mark Batshaw, MD (.pdf), Children's National
Barry J. Byrne, MD, PhD (.pdf), University of Florida
Stephen C. Groft, PharmD (.pdf), NIH
Mary Purucker, MD, PhD (.pdf), NCATS