RARE DISEASE WORKSHOP SERIES

Improving the Clinical Development Process

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


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