Monday, March 12, 2012
12:00 pm Main Conference Registration
1:55 Organizer’s Welcome Remarks
Kate Skaare, Conference Producer, Cambridge Healthtech Institute
» 2:00 Keynote Presentation: Have We Lost the Plot in Phase III Development?
 Declan Doogan, M.D., CMO, Amarin
In 2010 twenty one NDA/NBAs were approved. The ratio of NME:NBE was 15:6. A decade earlier 29 entities were approved; NME:NBE ratio was 27:2. The rapid evolution of the science base has enabled access to new targets, but the failure rate has grown dramatically too. This can be traced back to poor decision making. The main reason is that candidates were inappropriately nominated at the end of Phase II or did not have the appropriate characterization during Phase II.
|
Sponsored by
3:00 Avoiding Failure and Delay: Simulation Modeling for Optimizing Design of Phase III Trials
Badri Rengarajan, M.D., Medical Director, Archimedes, Inc.
Mathematical simulation allows for testing trial designs before implementing them. It is used to estimate eligible population size, baseline event rates, and anticipate trial performance under different scenarios of inclusion criteria, therapeutics, protocols, and patient/physican behaviors. Simulation can improve powering, population choice, and endpoint selection.
3:15 Sponsored Presentation (Opportunity Available, please contact Carol Dinerstein: dinerstein@healthtech.com, 781-972-5471)
Chairperson: Holly H. Kimko, Ph.D., Research Fellow, Janssen Research & Development, L.L.C. of J&J; editor of “Clinical Trial Simulations”
3:30 Use of Modeling & Simulation to Design Phase 3 Trials
Holly H. Kimko, Ph.D., Research Fellow, Janssen Research & Development, L.L.C. of J&J; editor of “Clinical Trial Simulations”
Modeling & Simulation is frequently used in major pharmaceutical companies to facilitate decision of phase 3 study designs. There are many success stories published, and we will review two cases to learn how M&S was used in order to decide design factors of phase 3 studies. Holly Kimko, PhD is a Scientific Director (Research Fellow) at the Department of Advanced Modeling & Simulation in Janssen Research & Development, LLC, New Jersey, and Adjunct Professor of the Pharmacy School of Rutgers University, New Jersey. She was previously a faculty in the Center for Drug Development Science in Georgetown University Medical School, Washington DC. Trained in Biochemistry and Pharmacy, Dr. Kimko earned her Ph.D. degree in Pharmaceutical Science from the State University of New York, Buffalo. She has published key papers on indirect response modeling and applications of CTS, and co-edited two books, titled Simulation for Designing Clinical Trials and Clinical Trial Simulations.
4:00 Evaluating Probability of Success for Decision- Making in Early Drug Development – A Bayesian Multivariate Approach
Yue “Annie” Wang, Ph.D., Data and Statistical Sciences, Global Pharmaceutical R&D, Abbott Laboratories
In early drug development, learning stage studies (a proof-of-concept or a dose-ranging study) are expected to provide clear evidence of the drug candidate meeting desired target product profile, as decisions to continue or halt development of a compound must be made at the end of these studies. Relying solely on p-values for testing hypotheses of no treatment effect based on a single endpoint for making drug development milestone decisions is an inefficient approach, as these studies are generally powered with little or no information on the unknown treatment effect. It ignores the correlations between multiple endpoints included in a Phase II clinical trial. This presentation illustrates a Bayesian multivariate approach that exploits totality of accumulated data or knowledge for decision-making in early drug development.
4:30 Refreshment Break in the Exhibit Hall with Poster Viewing
5:00 Quantitative Decision-Making in Drug Development
Nidal Al-Huniti, Ph.D., Director, Pharmacometrics, AstraZeneca
5:30 Speaker Q&A
Moderated by: Holly H. Kimko, Ph.D., Research Fellow, Janssen Research & Development, L.L.C. of J&J; editor of “Clinical Trial Simulations”
5:45 Welcome Reception in the Exhibit Hall
6:45 Close of Day