DDD Flashcards
First drug created containing an active ingredient with established efficacy, safety, & quality and received approval for use
Innovator Drug
First retroviral inhibitor approved for the tx of AIDS
Zidovudine (Azidothymidine - AZT)
On average, drug discovery and development could take about ____ years.
12
The identification of a single marketed drug has a success rate of ____.
< 0.001%
DRUG DISCOVERY
The choice of disease and identification of biological target
Identify the step in drug discovery.
(1)
Target Discovery
DRUG DISCOVERY
Expected molecular target (gene, protein, nucleic acid of a small molecule) is certified
Identify the step in drug discovery.
(2)
Target Validation
DRUG DISCOVERY
Structurally related compounds with identified desired biologically activity are identified (HIT and LEAD) through biological screening of large numbers of compounds
Identify the step in drug discovery.
(3)
Lead Discovery
DRUG DISCOVERY
Lead compounds are modified and studied to identify potential candidate for drug development
Identify the step in drug discovery.
(4)
Lead Optimization
DRUG DISCOVERY
Maintain favorable properties and enhance deficiency on structure
Identify the step in drug discovery.
(4)
Lead Optimization
Goals
DRUG DISCOVERY
Early stages of pharmacological studies are helpful to characterize the MOA of the compound
Identify the step in drug discovery.
(5)
Product Characterization
DRUG DISCOVERY
A stage of drug development during which the physicochemical properties of APIS are characterized to produce a bioavailable, stable, and optimal dosage form for a specific administration route
Identify the step in drug discovery.
(6)
Pharmaceutical Formulation/ Formulational Development
DRUG DISCOVERY
Data mining using bioinformatics
Identify the step in drug discovery.
(1)
Target Discovery
Approaches
DRUG DISCOVERY
Genetic association
Identify the step in drug discovery.
(1)
Target Discovery
Approaches
DRUG DISCOVERY
Expression profile
Identify the step in drug discovery.
(1)
Target Discovery
Approaches
DRUG DISCOVERY
Pathway and phenotypic analysis
Identify the step in drug discovery.
(1)
Target Discovery
Approaches
DRUG DISCOVERY
Functional screening
Identify the step in drug discovery.
(1)
Target Discovery
Approaches
DRUG DISCOVERY
SAR of the analogs
Identify the step in drug discovery.
(2)
Target Validation
Strategies
DRUG DISCOVERY
Generation of drug-resistant mutant
Identify the step in drug discovery.
(2)
Target Validation
Strategies
DRUG DISCOVERY
Knockdown/over expression of target
Identify the step in drug discovery.
(2)
Target Validation
Strategies
DRUG DISCOVERY
Monitoring the known signaling systems downstream of the presumed target
Identify the step in drug discovery.
(2)
Target Validation
Strategies
DRUG DISCOVERY
Reproducibility
Identify the step in drug discovery.
(2)
Target Validation
Key Steps
DRUG DISCOVERY
Introduce variation to drug-target environment
Identify the step in drug discovery.
(2)
Target Validation
Key Steps
DRUG DISCOVERY
SAR identified
Identify the step in drug discovery.
(3)
Lead Discovery
Characteristics
DRUG DISCOVERY
Drug ability (preliminary toxicity)
Identify the step in drug discovery.
(3)
Lead Discovery
Characteristics
DRUG DISCOVERY
Synthetic feasibility
Identify the step in drug discovery.
(3)
Lead Discovery
Characteristics
DRUG DISCOVERY
Select mechanistic assays
Identify the step in drug discovery.
(3)
Lead Discovery
Characteristics
DRUG DISCOVERY
In vitro assessment of drug resistance and efflux potential
Identify the step in drug discovery.
(3)
Lead Discovery
Characteristics
DRUG DISCOVERY
PK/Toxicity of chemical class known based on preliminary toxicity or in silico studies
Identify the step in drug discovery.
(3)
Lead Discovery
Characteristics
DRUG DISCOVERY
Evidence of in vivo efficacy of chemical class
Identify the step in drug discovery.
(3)
Lead Discovery
Characteristics
DRUG DISCOVERY
Selectivity and binding mechanism as the final step in early-stage of drug discovery
Identify the step in drug discovery.
(4)
Lead Optimization
DRUG DISCOVERY
The final step in early-stage of drug discovery
Selectivity and binding mechanism during lead optimization
DRUG DEVELOPMENT
Decides whether a drug is ready for clinical trials and
involves extensive studies (animal models) that yield
preliminary information on efficacy, toxicity, pharmacokinetic, and safety
Identify the step in drug development.
(0)
Pre-Clinical Studies
DRUG DEVELOPMENT
General pharmacology
Identify the step in drug development.
(0)
Pre-Clinical Studies
Approaches
DRUG DEVELOPMENT
Toxicology
Identify the step in drug development.
(0)
Pre-Clinical Studies
Approaches
DRUG DEVELOPMENT
Requests permission to move a clinical candidate to human study
Identify the step in drug development.
(1)
INDA
Submission of an Investigational New Drug Application
DRUG DEVELOPMENT
Provides regulatory agencies with detailed preclinical data and detailed clinical protocols that describe how the compounds will be studies in human populations.
Identify the step in drug development.
(1)
INDA
Submission of an Investigational New Drug Application
DRUG DEVELOPMENT
Data collection and analysis
Identify the step in drug development.
(2)
Clinical Research
(Clinical Trials)
Purpose
DRUG DEVELOPMENT
Investigative FIH trials that follows USFDA guidelines
Identify the phase of clinical trial.
Phase 0
(2) Clinical Research (Clinical Trials)
DRUG DEVELOPMENT
Human micro dose studies with 10 to 15 healthy volunteers
Identify the phase of clinical trial.
Phase 0
(2) Clinical Research (Clinical Trials)
DRUG DEVELOPMENT
Safety and tolerability
Identify the phase of clinical trial.
Phase 1
(2) Clinical Research (Clinical Trials)
DRUG DEVELOPMENT
20-100 healthy individuals
Identify the phase of clinical trial.
Phase 1
(2) Clinical Research (Clinical Trials)
DRUG DEVELOPMENT
Safety and efficacy
Identify the phase of clinical trial.
Phase 2
(2) Clinical Research (Clinical Trials)
DRUG DEVELOPMENT
100-300 patients
Identify the phase of clinical trial.
Phase 2
(2) Clinical Research (Clinical Trials)
DRUG DEVELOPMENT
Efficacy of IND in larger population including comparison with standard therapies
Identify the phase of clinical trial.
Phase 3
(2) Clinical Research (Clinical Trials)
DRUG DEVELOPMENT
Comprehensive details of both animals and human studies including safety findings, manufacturing procedures, dosing methods, and storage conditions
Identify the step in drug development.
(3)
NDA submission to regulatory body
New Drug Application
DRUG DEVELOPMENT
FDA Review
Identify the step in drug development.
(3)
NDA submission to regulatory body
New Drug Application
DRUG DEVELOPMENT
Alterations to labeling based on safety results
Identify the step in drug development.
(4)
Phase 4 Clinical Trial
(Post-Marketing Surveillance)
Impacts
DRUG DEVELOPMENT
Detects rare and long term adverse effects across a much larger population of patients than could be supported in Phase III
Identify the step in drug development.
(4)
Phase 4 Clinical Trial
(Post-Marketing Surveillance)
DRUG DEVELOPMENT
Contraindication for use of the new drug in combination with other medications
Identify the step in drug development.
(4)
Phase 4 Clinical Trial
(Post-Marketing Surveillance)
DRUG DEVELOPMENT
Withdrawal of marketing approval if the findings are severe enough
Identify the step in drug development.
(4)
Phase 4 Clinical Trial
(Post-Marketing Surveillance)
DRUG DEVELOPMENT
Identify competitive advantages, new markets, and new indications for the products
Identify the step in drug development.
(4)
Phase 4 Clinical Trial
(Post-Marketing Surveillance)
