Introduction to Model-Informed Drug Development
PHA 6418 Introduction to Model-Informed Drug Development


This is a 3-credit course.


Introduction to Model-Informed Drug Development (MIDD) is a 3-credit course that serves as a broad overview of applying modeling and simulation strategies to improve drug development decision-making and trial designs. Examples of applications include translational approaches from preclinical to clinical research, translation from biomarker to clinical endpoint, dose finding during Phase 1 and 2, clinical confirmation during Phase 3, avoiding the need for certain clinical trials, characterizing sources of variability in drug response, regulatory interactions and post-marketing assessment. Upon completion of the course, students will have a basic understanding of how model-based drug development can be applied to help bring new drugs to market more efficiently.

Upon completion of this course, the student will be able to:

  1. Discuss and appreciate the role of model-informed drug development during the following stages: discovery, preclinical, clinical, regulatory review and post-approval.
  2. Identify specific drug development questions or issues that can be addressed using MIDD.
  3. Select and apply pharmacometric method(s) used in MIDD: population PK or PK-PD, clinical trial simulation, meta-analysis, systems pharmacology, PBPK, etc.
  4. Contrast MIDD applications to support regulatory agency decisions versus drug development decisions within pharmaceutical industry.
  5. Understand the development strategies for various therapeutic areas and identify the question that needed to be answered using MIDD
  6. Describe MIDD approaches used to answer the above question: e.g. modeling, simulation, results, key assumptions and uncertainties.
  7. Explain the impact of MIDD on specific drug development examples and regulatory decisions or interactions.
  8. Apply MIDD concepts to all Phase(s) of drug development: preclinical, Phase 1, 2, 3, regulatory agency interactions, and post-regulatory approval.
  9. Explain the critical aspects of conducting clinical trials including human subject protections, Phase 1, 2, and 3 trials, regulatory requirements, and clinical data management.
  10. Answer specific drug development questions or issues that can be addressed by MIDD: dose selection, trial design for Phase 1, 2, or 3, go/no go, labeling, development strategy, avoiding the need to conduct a clinical trial, selecting between compounds in development, etc.
  11. Apply MIDD to various therapeutic areas: cardiovascular, neuroscience, metabolic diseases, oncology, rare diseases, etc.


Week Topic
Module 1
Week 1 Overview of Model-Informed Drug Development; Fundamental Concepts and Basic Pharmacokinetic and Pharmacodynamic Parameters
Week 2 Introduction to PK/PD; Introduction to Population PK/PD
Module 2
Week 3 Introduction to PBPK; Drug Interactions
Week 4 Preclinical to Clinical Scaling of PK and PD; Model-Based Meta-Analysis (MBMA) to Support Phase 3 Dose selection of Apixaban
Module 3
Week 5 PBPK of Perampanel (Role in Approval); PBPK of Perampanel (Support of Monotherapy)
Week 6 Model informed Drug Development to Optimize Dosing in Oncology; Model Informed Dosing Recommendation using PBPK and Translation to Prescribing Information
Module 4
Week 7 MIDD in Drug Development of the anti-PD1 Mono-clonal Antibody Nivolumab; Use of Pharmacometric Approaches to Support a change in Dosing Regimen for Anti-PD-1 monoclonal Antibodies
Week 8 MIDD in Oncology: Rational Dose Selection; Model-Based Meta-Analysis to Replace a Head-to-Head Clinical Trial in Type 2 Diabetes Mellitus
Module 5
Week 9 MIDD in Phase 2 Development; MID3 Strategies and Antibacterial Drug Development
Week 10 First Exam; Quantitative DDI Analysis for Regulatory Interactions: Case Examples
Module 6
Week 11 Model-Informed Drug Development for Dose Selection and DDI; Modeling and Simulation Approaches to Support Clinical Drug Development
Week 12 Introduction to IVIVC; Practical Decision Analysis Tools to Enable Key Drug Development Decisions
Module 7
Week 13 Application of Model Informed Drug Development to RNAi Therapeutics; Pediatric Drug Development
Week 14 Using Nonclinical Data and Physiologically-Based Pharmacokinetic Modelling to Inform on Risk of Victim Drug-Drug Interactions in Discovery’; Role of Biomarkers in Developing CNS Drugs
Week 15 Final Exam

Note: For a further breakdown of modules and course topics please see the full syllabus.


There are no prerequisites for this course.

Required Materials

Recommended Textbooks:

  • Title: Applied Pharmacometrics
    • Authors: S. Schmidt, H. Derendorf
    • AAPS Press/Springer
    • 2014
    • ISBN number: 978-1493913039
  • Title: Rowland and Tozer’s Clinical Pharmacokinetics and Pharmacodynamics: Concepts and Application
    • Authors:. H. Derendorf, S. Schmidt
    • Wolters & Kluwer
    • 2019
    • ISBN number: 978-1496385048

Additional reading assignments will be made available on the Canvas course site.