Co-sponsored by:
  
8:30 am – 8:35 am Regency Ballroom C
Welcome Remarks
Joga Gobburu, Ph.D.
U.S. Food and Drug Administration
8:35 am – 8:45 am
Critical Path in Action
Janet Woodcock, M.D.
U.S. Food and Drug Administration
8:45 am – 11:00 am Regency Ballroom C
SESSION I: GENERAL BACKGROUND
The objective of this session is to provide an overall background of the perceived public health benefit of Parkinson’s disease modifying drugs, pathology of the disease, trial designs and outcomes in practice and summary of prior trial data.
Moderators:
Joga Gobburu, Ph.D.
U.S. Food and Drug Administration
Karl Kieburtz, M.D., M.P.H.
University of Rochester Medical Center
8:45 am – 9:00 am
Public Health Benefit of Modifying
Parkinson’s Disease
Jeff Martin, J.D.
9:00 am – 9:30 am
Pathology and Mechanisms to Modify Parkinson’s Disease
C. Warren Olanow, M.D., F.R.C.P.C.
Mount Sinai School of Medicine
9:30 am – 10:00 am
Parkinson’s Disease Modifications: Overview of Trial Designs and Outcomes
Karl Kieburtz, M.D., M.P.H.
University of Rochester Medical Center
10:00 am – 10:30 am Regency Ballroom Foyer
Coffee Break
10:30 am – 11:00 am
Learning from Prior Parkinson’s Trials Using Pharmacometric Approaches
Venkatesh Atul Bhattaram, Ph.D.
U.S. Food and Drug Administration
11:00 am – 12:30 pm Regency Ballroom C
SESSION II: EARLY DEVELOPMENT
The goal of this session is to provide clarity around two major questions: what information do we need to obtain in early clinical trials to ensure appropriate decision making about moving a potential disease-modifying therapy forward; and what are the best methods for obtaining this information.
Moderators:
Brian K. Fiske, Ph.D.
The Michael J. Fox Foundation for Parkinson's Research
Ramana S. Uppoor, Ph.D.
U.S. Food and Drug Administration
11:00 am – 11:30 am
Proof-of-Concept and Dose-finding Strategies for Parkinson’s Disease
Eric R. Siemers, M.D.
Eli Lilly and Company
11:30 am – 12:00 pm
Designing and Analyzing Early Clinical Trials to Support Development Decisions
Joga Gobburu, Ph.D.
U.S. Food and Drug Administration
12:00 pm – 12:30 pm
Panel Discussion
An overall discussion on what are the most critical and realistically obtainable parameters for early phase trials and the best methods for obtaining them.
Panel:
Joga Gobburu, Ph.D.
U.S. Food and Drug Administration
Bruce Levin, Ph.D.
Columbia University
Kenneth Marek, M.D.
Institute for Neurodegenerative Disorders
Eric R. Siemers, M.D.
Eli Lilly and Company
Vikram Sinha, Ph.D.
Eli Lilly and Company
12:30 pm – 1:30 pm Independence Center
Lunch
Complimentary to all attendees
1:30 pm – 5:00 pm Regency Ballroom C
SESSION III: REGISTRATION TRIALS
The topics in these sessions are focused on the trial design, endpoints and analysis options for registration trials which form the primary basis for disease-modifying claims. The different sessions in this part of the conference are separated based on the trial design.
Moderators:
Joga Gobburu, Ph.D.
U.S. Food and Drug Administration
Karl Kieburtz, M.D., M.P.H.
University of Rochester Medical Center
1:30 pm – 2:00 pm
Design and Analysis Options for Demonstrating Disease-modifying Effects: General Statistical Considerations
Michael P. McDermott, Ph.D.
University of Rochester
2:00 pm – 2:30 pm
Why Divergence of Slopes between Placebo and Active Treatment Alone Cannot Substantiate Disease-modifying Effects?
Russell Katz, M.D.
U.S. Food and Drug Administration
2:30 pm – 3:00 pm
Endpoints and Analysis to Discern Disease-modifying Effects for Delayed-start Design
Ohidul I. Siddiqui, Ph.D.
U.S. Food and Drug Administration
3:00 pm – 3:30 pm Regency Ballroom Foyer
Coffee Break
3:30 pm – 4:00 pm
Natural History Staggered Start Design to Demonstrate Disease Modification: Lessons from Alzheimer’s Trials
Suzanne B. Hendrix, Ph.D.
Pentara Corporation
4:00 pm – 5:00 pm
Panel Discussion
Panel:
Suzanne B. Hendrix, Ph.D.
Pentara Corporation
Russell Katz, M.D.
U.S. Food and Drug Administration
Michael P. McDermott, Ph.D.
University of Rochester
C. Warren Olanow, M.D., F.R.C.P.C.
Mount Sinai School of Medicine
Ohidul I. Siddiqui, Ph.D.
U.S. Food and Drug Administration
Eric R. Siemers, M.D.
Eli Lilly and Company
5:00 pm – 6:00 pm Independence Center
Reception
Tuesday, April 29
8:00 am – 12:00 pm Regency Ballroom Foyer
Registration
8:00 am – 10:00 am
SESSION IV: CONCURRENT BREAKOUT SESSIONS
Breakout Session 1: Biomarkers for Early Development Decisions Washington A Room
This breakout session will focus on the kinds of markers, both biologic and imaging, that can be used or developed to inform early clinical testing of potential neuroprotective/ disease-modifying agents.
Moderators:
Brian K. Fiske, Ph.D.
The Michael J. Fox Foundation for Parkinson's Research
Ramana S. Uppoor, Ph.D.
U.S. Food and Drug Administration
Use of Biomarkers in Early Trials
Kenneth Marek, M.D.
Institute for Neurodegenerative Disorders
Breakout Session II: Parkinson’s Disease Outcomes and Practical Aspects of Different Trial Designs Regency Ballroom C
This breakout session will focus on the different disease outcomes (such as total UPDRS versus Motor scores) relevant to demonstrate disease-modifying effects. Also, the practical aspects of the different study designs (for example, advantages of delayed-start versus withdrawal designs) will also be discussed.
Moderators:
Karl Kieburtz, M.D., M.P.H.
University of Rochester Medical Center
Stanley Fahn, M.D.
Columbia University
Functional Outcome Measures
Ira Shoulson, M.D.
University of Rochester
Delayed-start Design with Dopaminergic Therapies
Stanley Fahn, M.D.
Columbia University
Breakout Session III: Statistical Issues Specific to Disease Modification Endpoints Washington B Room
This breakout session will focus on power analysis and dealing with missing data for delayed-start, withdrawal and other alternative designs for the modification claim.
Moderators:
Ralph B. D’Agostino, Sr., Ph.D.
Boston University
Ohidul I. Siddiqui, Ph.D.
U.S. Food and Drug Administration
Selection Designs
Bruce Levin, Ph.D.
Columbia University
10:00 am – 10:30 am Regency Ballroom Foyer
Coffee Break
10:30 am – 11:00 am Regency Ballroom C
Panel Discussion and Audience Q&A
Group to reconvene and breakout session moderators will report on discussion.
Panel:
Breakout Session 1: Biomarkers for Early Development Decisions
Brian K. Fiske, Ph.D.
The Michael J. Fox Foundation for Parkinson's Research
Breakout Session II: Parkinson’s Disease Outcomes and Practical Aspects of Different Trial Designs
Stanley Fahn, M.D.
Columbia University
Breakout Session III: Statistical Issues Specific to Disease Modification Endpoints
Ralph B. D’Agostino, Sr., Ph.D.
Boston University
11:00 AM – 1:30 PM Regency Ballroom C
SESSION V: REGULATORY PERSPECTIVE
What are the key regulatory questions for discerning disease-modifying and symptomatic treatments? Is it more prudent to employ diverse trial designs to substantiate effectiveness? Should there be a clinically meaningful threshold for these claims?
Moderators:
Joga Gobburu, Ph.D.
U.S. Food and Drug Administration
Russell Katz, M.D.
U.S. Food and Drug Administration
11:00 am - 11:30 am
U.S. Food and Drug Administration Perspective
Russell Katz, M.D.
U.S. Food and Drug Administration
11:30 am – 12:00 pm
European Perspective
Cristina Sampaio, M.D., Ph.D.
Faculdade de Medicina de Lisboa
12:00 pm – 1:30 pm
Panel Discussion
Moderator:
Joga Gobburu, Ph.D.
U.S. Food and Drug Administration
Panel:
Ralph B. D’Agostino, Sr., Ph.D.
Boston University
Russell Katz, M.D.
U.S. Food and Drug Administration
Leonard P. Kapcala, M.D.
U.S. Food and Drug Administration
Karl Kieburtz, M.D., M.P.H.
University of Rochester Medical Center
Stanley Fahn, M.D.
Columbia University
Robert O’Neill, Ph.D.
U.S. Food and Drug Administration
Cristina Sampaio, M.D., Ph.D.
Faculdade de Medicina de Lisboa
Eric R. Siemers, M.D.
Eli Lilly and Company
1:30 pm
Adjournment
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