1. The Art of Drug Discovery Flashcards
First-in-class drug
targets a protein that has not been targeted before by another drug
The FDA approved ____ novel drugs in ____, the fewest to pass regulatory scrutiny since 2016
37
2022
Takes about ___ years for molecule to be approved
15
_____ prescription drugs marketed in North America
1000
Discovery/ Preclinical Testing
- lab & animal studies
- Asses safety, biological activity and formulations
- 5000 compounds evaluated
- 6.5 years
Phase 1
- 20-100 healthy volunteers
- Determine safety and dosage
- 5 compounds
- 1.5 years
Phase 2
- 100-500 patient volunteers
- Evaluate effectiveness & look for side effects
- 5 compounds
- 2 years
Phase 3
- 1000-5000 patient volunteers
- Confirm effectiveness, monitor adverse reactions from long term use
- 5 compounds
- 3.5 years
FDA review and approval
- 1.5 years
- one compound approved
- review and approval process
Phase 4
(whole process 15 years total)
1. additional post marketing testing required by FDA
World Health Organization takes into account…
- disease prevalence
- impact on disability-adjusted life years, DALYs
- public health, community health
- Evidence of efficacy
- evidence that the drug is safe
Highest proportion of drugs improved in which therapeutic area?
- Oncology
- Infectious diseases
- Neurology
Who decides medical needs?
- FDA (US)
- Health Canada (Canada)
Drug target
a molecule (usually a protein) involved in the disease, that the drug binds to, often inactivation the molecule
What makes a protein “druggable”?
good protein structure
Phase 1 Clinical Trials Purpose
–Determine a safe drug dose
Pharmacokinetics
what happens to the drug in the body, how the drug moves in the body
Phase 1 Clinical Trials Design
Usually dose-escalation. Start low, increase dose (Cautious)
Phase 1 Clinical Trials Subjects
– Normal volunteers usually
– Sometimes patients with the disease (cancer)
– Must obtain informed consent from subjects
Placebos
Placebos look like real pills but contain no active drug ingredient (controls for the “placebo effect”) is the expected response to taking a drug, (real brain circuitry involved)
Phase 2 Clinical Trials Purpose
To obtain preliminary information on the efficacy of the drug
Phase 2 Clinical Trials Design
Compare treated individuals to control individuals
Phase 2 Clinical Trials Subjects
Patients with the disease
Efficacy
does the drug work to treat the medical condition?
Single-blind trials
subjects don’t know who is getting treatment and who is getting placebo
Double-blind trials
neither subjects nor investigators know (Computer records – we ultimately do know who received which treatment)- MOST COMMON
Phase 3 Clinical trials Purpose
To compare efficacy of the new treatment with the standard regimen
Phase 3 Clinical trials Design
Usually randomized control
Phase 3 Clinical trials Subjects
Patients with the disease
Informed Consent
the process of understanding what you are agreeing (consent) to
Generic drugs
same chemical, off-patent
ie: Plan-B & contingency one
Patent protection
20 years following filing (gives the manufacturer a monopoly for this amount of time- usually files for patent during discovery phase)
Cost and time of developing a drug
Costs about >2 billion dollars & takes about 15 years
