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HMX Pro Pharmacology
Drug Discovery and Development

Understand advances in drug discovery and development and how new therapeutics are developed.


Drug discovery and development has evolved dramatically over recent decades and the field continues to advance today. More sophisticated approaches and models are being developed, new technology is being incorporated into the entire pipeline of development, and exciting drug modalities continue to emerge. With all the advances in science and with closer collaboration between different scientific disciplines, a multifaceted approach is now being applied to drug discovery and development. Therefore, the likelihood of being able to discover a compound, transform this into a safe and effective therapeutic, and improve the lives of patients has increased. 

Learning about the process of drug discovery and development has important implications for anyone working in health care and related sectors. This advanced course offers a unique way for professionals to learn from leading Harvard Medical School faculty about how drugs are discovered and developed and about the advances happening in this field that are ultimately helping to improve the treatment and prevention of disease.

Course Topics

Overview of Drug Discovery and Development

  • Introduction to Drug Discovery and Development
  • The Promise of Drug Discovery and Development

Project Initiation and Selection

  • Introduction to Drug Research and Development
  • Understanding the Disease/Indication Being Targeted
  • Understanding the Drug being Developed
  • Understanding the Mechanism of Action of the Drug
  • Understanding the Effect of the Drug on the Disease/Indication
  • Having a Clear Plan for Drug Development
  • Genetics in Drug Discovery

Drug Discovery

  • Pharmacological Principles
  • Biochemical Models and Cell Models
  • Disease Models
  • Small Molecules
  • Protein Therapeutics
  • RNA Therapeutics
  • Clinical Linkage: Cystic Fibrosis

Nonclinical Drug Development

  • Goals of Nonclinical Drug Development
  • Animal Pharmacology
  • Good Laboratory Practice
  • Toxicology and Safety Pharmacology
  • Toxicology Testing
  • Genotoxicity, Carcinogenicity, and Reproductive Toxicity
  • Early Clinical Studies

Clinical Drug Development

  • Establishing a Causal Pathway
  • Chance and Bias
  • Confounding and Effect Modification
  • Basic Epidemiologic Considerations
  • Surrogate Endpoints
  • Fundamentals of Randomized Trial Design
  • Anatomy of a Randomized Trial
  • Clinical Linkage: PCSK9 Inhibitors

Wrap-up

  • The Future of Drug Discovery and Development

Download the Drug Discovery and Development course outline

Course Instructors

Kate McDonnell-Dowling, HMX Pharmacology Course Lead

Kate McDonnell-Dowling, PhD

Lecturer in Biological Chemistry and Molecular Pharmacology, Harvard Medical School
Associate Director of Curriculum, HMX


Mark Goldberg

Mark Goldberg, MD

Lecturer in Medicine, Harvard Medical School
Attending Physician in Hematology, Brigham and Women’s Hospital
Member of Board of Directors at Avacta Group PLC


Mark Namchuk

Mark Namchuk, PhD

Senior Lecturer in Biological Chemistry and Molecular Pharmacology, Harvard Medical School
Executive Director of Therapeutics Translation in Department of Biological Chemistry and Molecular Pharmacology, Harvard Medical School


William Clarke

William Clarke, MD

Professor of Anesthesia, Harvard Medical School
Pediatric Critical Care Physician, Boston Children’s Hospital


Paul Ridker

Paul Ridker, MD, MPH

Eugene Braunwald Professor of Medicine, Harvard Medical School
Senior Physician, Brigham and Women’s Hospital
Director of the Center for Cardiovascular Disease Prevention, Brigham and Women’s Hospital


More Information

Frequently asked questions

Who should take this course?

This course is designed for professionals in health care, life sciences, and related industries who want to have a better understanding of drug discovery and development for their work.

What do participants need to know to succeed in this course?

We recommend basic knowledge of chemistry, biology, and physics, as well as an understanding of key pharmacology concepts. If you need further background in pharmacology, we also offer an introductory course, HMX Pro Pharmacology – Essentials.

Not sure? Take this short quiz to gauge your knowledge of pharmacology concepts.

What is the time commitment?

Most people can expect to spend around 15–20 hours total, but this depends on your baseline knowledge, how carefully you take notes, and how seriously you take the assessments. New course material is released periodically, and remains available until the course period ends, so participants can work at their own pace. There is a final exam at the end of each course that can be taken any time during the final exam period.

Can participants earn certificates from Harvard Medical School?

There are two certification levels, based on participant scores:

  • Certificate of Achievement
  • Certificate of Completion