Preclinical Safety: From In Vitro Assays to Human Dose Predictions

In this course about preclinical safety, students will learn methods for quantifying the safety of a potential drug. The three-week course starts with the origins of preclinical safety programs as well as preliminary safety testing for promising compounds in a drug program. The second week emphasizes in vivo safety studies in representative rodent and non-rodent preclinical animal species. In the final week, students will learn how the PK properties are used with efficacy data to predict the human efficacious dose and assist in drug formulation.

Students in the course should understand human anatomy. An understanding of both pharmacodynamics and pharmacokinetics will also be helpful. The course is designed in modules. Each module opens with a short video on a selected topic. The module continues with a short reading or exercise with an assessment activity. Each module will require approximately 1 hour to complete, and the course includes 15 modules. Two approaches to the course are recommended. One, a student might spend an hour per day on the course and complete all 15 modules in approximately three weeks. Two, a student with additional available time may be able to work through the entire course in just a single week or over an extended weekend.

Students signed up for the Audit Track have access to the instructional video materials. Students enrolled through the Verified Track have access to the instructional videos, the readings and activities, the assessments, as well as selected additional videos related to the primary instructional videos.

week 1

• pharmacology & toxicology

• preclinical safety timeline

• tragedies in drug safety

• in vitro safety assays

• risks from hERG channel inhibition

week 2

• on-target toxicity

• in vivo toxicology studies

• drug-drug interactions

• intra-family target selectivity

• drug purity and safety

week 3

• efficacy vs. safety risks

• maximum recommended starting dose

• safety in oncology drugs

• preclinical safety concurrent with clinical trials

• post-approval surveillance