Drug Hunting: The Science of Making New Medicines

Identify and validate the biological target at the heart of drug discovery, using genetics, omics, and functional assays to build a convincing case for clinical translation.

Explore high throughput screening, fragment based discovery, and AI to identify and optimize hit compounds from nature's own chemical library, turning starting scaffolds into potential medicines.

Explore hit-to-lead phase, a chemical speed dating process that triages hit series through resynthesis confirmation, orthogonal assays, SAR exploration, heat maps, and ADME safety checks to nominate a lead.

See how assay development drives drug discovery through a tiered cascade—from primary screens to in vivo models—using robust, validated reagents, z factor, and cellular assays for reliable decisions.

Explore lead optimization in drug discovery, balancing potency, selectivity, adme properties, and safety through the dmta cycle, using structure based design to forge a preclinical development candidate.

Explore admet, the pharmacokinetic journey from absorption through metabolism to excretion and toxicity, and how solubility, permeability, first-pass effects, and safety determine a drug's viability.

Fuse pharmacokinetics and pharmacodynamics to determine the right dose and schedule, using PK metrics like Cmax and AUC, and PD measures like Emax and MEC to define the therapeutic window.

Navigate the go/no-go decision at the candidate selection meeting. Rely on the TCP to set efficacy, PK and ADME, safety, and CMC criteria for a go, hold, or no-go verdict.

This lecture guides moving from discovery to preclinical development, detailing in vitro potency, mechanism of action, ex-vivo and in vivo testing, and the PK/PD efficacy relationship to support IND.

Explore how preclinical toxicology safeguards drug development through safety pharmacology, in vitro and in vivo studies. Assess genotoxicity, noael determinations, Herg assay, and establish safety margins for the human dose.

Explore how pharmaceutics transform a pure API into a real-world medicine by optimizing bioavailability, stability, dosing accuracy, and patient acceptability through pre-formulation, excipients, and advanced delivery systems.

Explain how the IND application secures human trials by compiling a five-module CTD dossier. Highlight module three quality control tests, module four nonclinical data, and module five the clinical protocol.

Trace the three-phase clinical trials—safety and pharmacokinetics in phase one, efficacy and dose in phase two, and definitive outcomes in phase three—bridging IND clearance to post-marketing surveillance.

Explore ethics and good clinical practice in clinical trials, balancing patient protection with data integrity through IRB review, informed consent, and ALCOA data standards.

Explore how endpoints and statistical analysis transform trial observations into credible evidence, using hypothesis testing, p value, power, and confidence intervals to gauge clinical and statistical significance.

Analyze high-profile clinical trial failures, from anti-amyloid therapies to NGF inhibitors, to uncover lessons in target validation and surrogate endpoints. Recognize risk-benefit dynamics and data from large trials.

Understand how the NDA/MAA compiles a comprehensive, evidence-based dossier to prove safety, efficacy, and scalable manufacturing. See how the common technical document organizes five modules to secure regulatory approval.

Learn how the FDA and EMA govern global drug reviews, including PDUFA timelines, NDA and MA submissions, CHMP opinions, and clock-stop reviews, and how parallel scientific advice accelerates development.

Explore how patents, pricing, and payers shape every stage of drug development from early discovery to reaching patients, driving strategic pivots and value oriented trial design for reimbursed medicines.

Bridge regulatory approval to patient access by scaling manufacturing, enforcing cGMP quality, and coordinating API production, formulation, branding, payer negotiations, medical affairs, and launch strategy across global markets.

Monitor post-marketing safety through phase four studies and pharmacovigilance, detecting signals, updating labels, and managing risks to safeguard patient safety in real-world use.

Explore health economics and outcomes research to prove a drug's value across clinical, economic, and humanistic outcomes; learn tools like CEA, ICER, BIA, and HTA.

Explore how patent expiration launches the drug into a second life via two paths—generics democratization and repurposing of existing medicines—under Hatch-Waxman with bioequivalence and new trials.

Analyze postmarketing safety signals from Vioxx and Zantac recalls, and learn why patient safety, long-term safety trials, data transparency, and independent trial registration reshape drug regulation.

Explore how pharmacogenomics drives personalized medicine by linking drug response to genetic variation in targets and metabolism, enabling biomarkers and companion diagnostics for targeted therapies.

Artificial intelligence and machine learning transform drug discovery by enabling in silico research across discovery to trials, from target identification and AlphaFold structure prediction to de novo design.

Explore RNA therapies, gene therapy, and cell therapy that rewrite disease at the genetic level, addressing undruggable conditions, delivery challenges, and the high costs shaping modern medicine.

Explore the global challenge of antimicrobial resistance and pandemic preparedness, detailing economic barriers, delinked incentives, and proactive platforms like mRNA and the 100 Days Mission to secure health security.