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 pharmacometrics 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 the 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 MIDD can address: 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
Week 1 Course Overview and Overview of Model-Informed Drug Development
Week 2 Introduction to Population PK-PD; The usage of monoclonal antibody models for the expansion of the Xolair (omalizumab) dosing table to support an unmet medical lead
Week 3 How technical pieces of MIDD are included in submissions for an IND/CTA/NDA/MAA, and best practices for including this information
Week 4 MIDD for New Drug Development
Week 5 TBD; MIDD to support regulatory concerns in pediatrics
Week 6 Streaming approval for generic drug inhalation
Week 7 MIDD Dose Optimization Oncology; MI Dosing Recommendation using PBPK and Translation to Prescribing Information
Midterm Exam
Week 8 Translational drug development using MIDD
Week 9 MIDD Approaches for Vaccine Development; Model-Based Approach to Select the Dose Regimen of GBT021601, a Next-Generation Sickle Hemoglobin Polymerization Inhibitor in Healthy Volunteers and Patients With Sickle Cell Disease
Week 10 Translation Modeling: How to predict FIH dose for clinical trials?; Using Clin. Pharm. and PMX to accelerate cell therapeutics development: A Patient’s CAR-T journey
Week 11 MIDD applications when organ impairment is a determinant of dose; Pharmacometric Approaches for change in Dosing Regimen for Anti-PD-1 monoclonal Antibodies
Week 12 TBD
Week 13 No classes – Thanksgiving Break
Week 14 Model-Based Meta-Analysis (MBMA) to Support Phase 3 Dose Selection of Apixaban; Decision-making in Pharma R&D with a focus on MIDD
Week 15 Optimizing efficacy and minimizing toxicity through data-driven drug dose and schedule selection; Course closure and overall discussion
Final Exam

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


Introductory knowledge of PK modeling and simulation.

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 Applications
    • Authors:. H. Derendorf, S. Schmidt
    • Wolters & Kluwer
    • 2020
    • ISBN number: 978-1496385048

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