Drug Discovery and Development

At the end of the course, students should be able to 1. illustrate major approaches to drug discovery; 2. restate properties of drugs; 3. explain the techniques used to identify drug targets and lead compounds; 4. discuss methods for determining the pharmacological and toxicological properties of compounds; 5. describe the role of pharmacokinetic principles in the drug discovery process; and 6. discuss preclinical and clinical evaluation of new drugs.

Introduction: Medicines and discovery processes. Initial stages: Target discovery; applications of molecular biology, in silico methods and assays; lead discovery; lead optimization; biomarkers. Measurement of drug properties: assay techniques; agonists (full, partial, inverse and biased); competitive antagonists. In vivo models; pharmacokinetic profiles of compounds; pharmacogenetics and formulations. Pre-clinical safety assessment including acute toxicity evaluation, subacute and chronic toxicity studies, reproductive toxicity, carcinogenicity studies, mutagenicity and mechanistic toxicity studies. Limitations of preclinical studies. Problems associated with drug discovery and development. Later stages: Pharmaceutical development; phases of clinical evaluation; ethics (informed consent, good clinical practice, participation criteria, sponsorship) and concepts in clinical trial design. Pharmacovigilance. e-Learning Activity Links are provided to additional resources (further reading, videos, on-line activities) to support the lecture content. Online self-assessment modules on the course content can be provided for formative feedback. Practical: Experiments are designed to reflect the topics covered including spectrophotometry, chromatography, flow cytometry.