Workshop 1
Optimizing the Choice of Statistical Analysis
and Study Design for Ultra Rare Diseases
November 8, 2010 8:00 a.m. – 3:00 p.m.
Bethesda Marriott, 5151 Pooks Hill Road, Bethesda, MD
Presentation slides can be accessed by clicking on the links within the agenda.
Agenda: |
8:00 am | Welcome
Emil Kakkis, M.D., Ph.D.
President & Founder, Kakkis EveryLife Foundation |
8:15 am | Keynote Presentation
Thomas Fleming
Professor of Biostatistics, University of Washington |
Ultra rare disease case study presentations: |
Section 1: Single Variable Methods |
9:00 am | Pompe Late Onset Treatment Trial
L.J. Wei, Ph.D.
Professor, Harvard University |
9:20 am | Post Marketing Fabry Trial
P.K. Tandon, Ph.D.
Senior Vice President Global Biomedical Data Sciences & Informatics, Genzyme Corporation |
9:40 am | Naglazyme Phase III
Emil Kakkis, M.D., Ph.D.
Former CMO, BioMarin Pharmaceutical Inc. |
10:00 am | Discussion of Single Variable Methods |
Section 2: Multivariate or Multiple Domain Methods |
10:30 am | Aldurazyme Responder Index
Gerry Cox, M.D., Ph.D.
Vice President, Clinical Research, Genzyme Corporation |
10:50 am | Multivariate Methods and Characterization of Clinical Effect of Complex Diseases
Brent A. Blumenstein, Ph.D.
Lead Statistician, Trial Architecture Consulting |
11:10 am | Hunter Phase III O'Brien analysis, issues with Elaprase
David A. Amato, Ph.D.
Director of Biostatistics, Shire Human Genetic Therapies |
11:30 am | Discussion of Multivariate or Multiple Domain Methods |
Strategies on Improving the Treatment Development Process |
11:45 am | FDA Perspective
Yuqun Luo, Ph.D., Mathematical Statistician, FDA\CBER
Estelle Russek-Cohen, Ph.D., Deputy Director, Division of Biostatistics, FDA\CBER |
12:05 am | Critical Path Institute Strategies
Marc Cantillon, M.D.
Director of Coalition Against Major Disease (CAMD), Critical Path Institute |
Planned statistical analysis on endpoints for approved ultra rare drugs |
1:10 pm | Overview on Analysis of Ultra Rare Drugs Data
Emil Kakkis, M.D., Ph.D.
Introduction to the Clinical Studies
Brigitta Miyamoto
Intern, Kakkis EveryLife Foundation |
1:20 pm | Explanation of Analysis on Endpoints for Approved Treatments & Findings
Mei Sheng Duh, MPH, ScD, Managing Principal, Analysis Group, Inc.
James Signorovitch, Manager, Analysis Group
Discussion on Additional Analyses and Modification of Analysis |
- Methods to control variation
- Multivariate methods
- Alternative study designs for rare disease studies
- Proposals for rare disease double-blind, placebo-controlled data sets to obtain for analysis
3:00 pm Workshop Summary
3:15 pm Workshop Adjourns
Approved Treatments Analyses