PSC 4215 - Exam 1 - Drug discovery and Approval

Where can leads for new drugs come from? (6)

1. Competitor compounds

2. Endogenous ligands

3. Random screening/compound file

4. Natural products

5. Scientific literature

6. Combinatorial libraries/AI

Up to 5000 compounds screened to find _____ marketable drug

1

10,000 initial compounds → _____ make it to in vivo testing

10

Only _____ out of 12 development candidates becomes a marketed drug

1

Drug approval trends (1995-2004) show _____ recent trends

Upward

How long can drug discovery take?

1 to 5 years

What kind of information should be included in preclinical submissions?

• Everything from start to finish

  • Drug info (structure, metabolism, toxicity, contraindications, overdose treatments, etc)

  • Objective, methods, manufacturing, investigators, institutions

  • etc.

T/F: Preclinical studies involve in vitro human testing on healthy patients

F, preclinical studies do NOT involve humans.

T/F: A drug that is in preclinical studies is an investigational drug that needs FDA approval before in vitro studies begin

F, drugs in preclinical studies is NOT considered an investigational drug until the preclinical studies are DONE and the FDA has approved it

When is the first time an investigational drug is given to patients

Phase 2 (Phase 1 = healthy humans, to record baseline effect)

If phase 3 studies suggest that the drug is effective, what must be file?

New Drug Submission/Application

Assess mechanism of action (MOA), toxicity, metabolism, and drug properties in lab and animal models. Submit findings to the FDA for investigational drug approval.

Pre clinical studies which takes 1.5 years

First human trials in healthy volunteers to establish dose-response curves and pharmacokinetics.

Phase 1 which takes 0.5 to 2 years

Small-scale trials in patients to evaluate elimination, metabolism, therapeutic benefits, dose ranges, and efficacy.

Phase 2 which takes 1 to 3 years

Large, randomized, double-blind clinical trials to confirm safety and effectiveness. Results support a New Drug Application (NDA) submission for FDA approval.

Phase 3 which takes 3 to 5 years

The FDA evaluates all data before granting marketing approval.

NDA Review which takes 1–2 years

(Post-Marketing Surveillance) – The drug is marketed and monitored worldwide for long-term safety, rare side effects, and dosing adjustments.

Phase 4

Summarize all of the phases of the drug approval process

1. Preclinical Studies (1.5 years) – Assess mechanism of action (MOA), toxicity, metabolism, and drug properties in lab and animal models. Submit findings to the FDA for investigational drug approval.

2. Phase 1 (0.5–2 years) – First human trials in healthy volunteers to establish dose-response curves and pharmacokinetics.

3. Phase 2 (1–3 years) – Small-scale trials in patients to evaluate elimination, metabolism, therapeutic benefits, dose ranges, and efficacy.

4. Phase 3 (3–5 years) – Large, randomized, double-blind clinical trials to confirm safety and effectiveness. Results support a New Drug Application (NDA) submission for FDA approval.

5. NDA Review (1–2 years) – The FDA evaluates all data before granting marketing approval.

6. Phase 4 (Post-Marketing Surveillance) – The drug is marketed and monitored worldwide for long-term safety, rare side effects, and dosing adjustments.

The drug remains in new drug status until the FDA is confident in its safety and efficacy, at which point routine monitoring decreases.

T/F: While phase 1 is generally used to determine whether a drug is safe in humans, phase 2 is used to identify whether it works

T