Clinical Forecasting: A Novel Bayesian Tool for Predicting Phase III Outcomes 

By Asher D. Schachter, MD 

TABLE OF CONTENTS

Section 1
Existing Predictive Tools for Pharmaceutical Forecasting
Biological Tools
    Biomarker and Target Discovery via High-Throughput Genomics and Proteomics
    Bioinformatics: High-Throughput Biomarker and Target Discovery
    In Silico Drug Discovery with the Connectivity Map
    Pharmacogenetics and Pharmacogenomics
    High-Throughput Screens and Animal Models
Clinical Tools
    Therapeutic Index
    Pharmacokinetics
    Population Pharmacokinetics
    Pharmacokinetic Models
    Microdosing
        Sidebar: Phase IV Postmarketing Surveillance
Bayesian Market Forecasting and Modeling of Cost-Effectiveness in Drug Development

Section 2
Description of a Bayesian Clinical Forecasting Model
Application of a Bayesian Network to Clinical Forecasting in Drug Development
    Prior Probability of NCE Success and Failure
    Conditional Probability Tables
    Training Dataset from Tufts CSDD Sources
    Independent Dataset Construction
    Model Evaluation Shows 78% Accurate Prediction of NCE Success on
    Independent Dataset
Existing Predictive Tools Empower Bayesian Clinical Forecasting
    Well-Designed Clinical Forecasting Models Can Boost Accuracy of Market
    Forecasts
    Biomarkers and Clinical Predictors Empower Bayesian Forecasting Tools

Section 3
Case Study: Recombinant Human Activated Protein C, Eli Lilly’s Xigris
Data Used For Forecast
Model Predicts Xigris Has Low Probabilities of Clinical Success, Safety and Efficacy

Section 4
Economic Impact of Bayesian Clinical Forecasting
Pharmacoeconomic Evaluation
    Monte Carlo Simulation to Determine Expenditures and Revenues for BN Model
    and for Pharmaceutical Industry
Model Reduced Median Expenditures, Increased Median Cumulative 7-Year Revenues
    Harnessing the Power of Late-Stage Failure Data and of Industrywide Data
    Sharing
    Data Storage Issues: Paper vs. Digital

Section 5
Societal Impact of Bayesian Clinical Forecasting
Impact on Children
Impact on the Elderly

Appendix A
Brief Overview of Bayesian Networks

Appendix B
Glossary