IPS/CA2 Drug Discovery, Development and Delivery Systems

Drug

Articles recognized in official pharmacopoeias or formularies.

New Drug

Contains new active ingredients or combinations.

FDA Approval

Required for new drugs to ensure safety and efficacy.

Unmet Medical Need

Need for treatments for new or inadequately addressed diseases.

IND

Investigational New Drug application for clinical trials.

NDA

New Drug Application for marketing approval.

Preclinical Studies

Initial testing for safety and efficacy before clinical trials.

Clinical Studies

Testing drugs on human subjects for safety and effectiveness.

Sources of New Drugs

Includes plants, animals, synthetic, and biotechnological sources.

Total Synthesis

Complete chemical synthesis of a drug from basic chemicals.

Semi Synthesis

Modification of natural products to create drugs.

Biotechnology

Use of living systems to develop drugs.

Postmarketing Studies

Research conducted after drug approval to monitor effects.

Solid Dosage Forms

Includes tablets and capsules for drug delivery.

Semi-solid Dosage Forms

Includes creams and ointments for topical application.

Liquid Dosage Forms

Includes solutions and suspensions for oral or injectable use.

Parenteral Dosage Forms

Injectable forms bypassing the gastrointestinal tract.

Active Ingredient

Chemical responsible for drug's therapeutic effect.

Fixed Combination Drug

Two or more drugs combined in a single dosage form.

New Indications

New uses for existing drugs beyond original approval.

Dosage Strength

Concentration of active ingredient in a dosage form.

Health Costs

Economic burden of diseases and their treatments.

ADA deficiency

A genetic disorder affecting adenosine metabolism.

Goal Drug

Desired therapeutic effect of a drug.

Random screening

Unbiased selection of compounds for testing.

Nonrandom screening

Targeted selection based on specific criteria.

Bioassays

Tests measuring biological activity of compounds.

High throughput screening

Rapid testing of large compound libraries.

Molecular modification

Alteration of existing compounds to enhance properties.

Dichloroisoproterenol

Beta-blocker with partial sympathomimetic activity.

Pronethalol

Beta-blocker with side effects like nausea.

Propanolol

Beta-blocker with no sympathomimetic side effects.

Lead Compound

Initial compound with potential for drug development.

Mechanism-based drug design

Design targeting specific biological mechanisms.

Prodrugs

Inactive compounds converted to active drugs post-administration.

Ranitidine

Requires metabolic transformation for pharmacological activity.

Biologic Characterization

Study of drug effects on biological systems.

Pharmacodynamics

Study of drug effects and mechanisms of action.

Pharmacokinetics

Study of drug absorption, distribution, metabolism, and excretion.

Animal Testing

Evaluates drug effects in genetically modified animals.

Drug Metabolism

Biochemical transformation of drugs in the body.

First-pass effect

Metabolism of a drug before systemic circulation.

Toxicology

Study of harmful effects of substances on organisms.

Dose-response relationship

Correlation between drug dose and effect magnitude.

Dose Interval

2 weeks equals one dose administration.

Chronic Illness Duration

Chronic human illness lasts one year.

Initial Dose Calculation

Initial dose is one-tenth of highest non-toxic dose.

Subacute Studies

Studies lasting 90 days for toxicity assessment.

Carcinogenicity Studies

Long-term testing for cancer risk in animals.

Reproductive Studies

Evaluate fertility, mating, and offspring development.

Genotoxicity Testing

Assess DNA damage using various assays.

Ames Test

In vitro test using Salmonella typhimurium strains.

Micronucleus Test

In vivo test for chromosomal damage assessment.

Preformulation Studies

Evaluate drug's physical and chemical properties.

Partition Coefficient

Measure of drug lipophilicity in formulations.

Dissolution Rate

Speed at which a drug dissolves in solution.

Phase 1 Clinical Trial

Tests active ingredient for safety in humans.

Phase 2 Clinical Trial

Tests active ingredient with excipients for efficacy.

Phase 3 Clinical Trial

Final formulation tested for therapeutic effectiveness.

IND Application

Investigational New Drug application for clinical trials.

Clinical Study Protocol

Document detailing trial objectives and methodology.

IRB Approval

Institutional Review Board approval for ethical standards.

FDA Drug Classification

System categorizing drugs based on safety and efficacy.

Accelerated Approval

Fast-track process for drugs addressing serious conditions.

Surrogate Endpoint

Marker predicting clinical benefit, not direct measure.

Intermediate Clinical Endpoint

Predictive measure of therapeutic effect on health.

Phase 1

Initial testing for safety in humans.

Phase 2

Evaluates efficacy and side effects.

Phase 3

Confirms effectiveness and monitors adverse reactions.

Phase 4

Post-marketing surveillance for long-term effects.

Emergency Use IND

Allows use of experimental drugs in emergencies.

Treatment IND

For promising drugs in clinical testing.

Emergency Use Authorization

FDA authorization for urgent drug use.

Orphan Drug Act of 1983

Supports drugs for rare diseases.

NDA

New Drug Application for market approval.

Drug Product Labeling

Mandatory information on drug packaging.

Minimum Mandatory Information

Essential details required on drug labels.

Powders

Finely divided solids for use.

Granules

Agglomerated powdered materials for tablets.

Tablet

Solid dosage form for oral administration.

Capsule

Gelatin container for powdered drugs.

Solution

Homogeneous mixture of solute and solvent.

Suspension

Solid particles dispersed in liquid.

Ointment

Semi-solid preparation for external use.

Mesh Size Number

Indicates particle size in sieving.

Very Coarse

Particles pass No. 8 sieve,

Coarse

Particles pass No. 20 sieve,

Moderately Coarse

Particles pass No. 40 sieve,

Fine

Particles pass No. 60 sieve,

Very Fine

Particles pass No. 80 sieve completely.

Executive Order No. 121

Authorizes FDA for Emergency Use Authorization.

Emergency Use Authorization (EUA)

Allows rapid approval of COVID-19 drugs and vaccines.

Orphan Drug Act of 1983

Legislation to encourage drug development for rare diseases.

Orphan disease

Condition affecting fewer than 200,000 people in the U.S.

FDA Office of Orphan Products

Oversees orphan drug designations and incentives.

IND period

Timeframe to demonstrate drug safety and effectiveness.

Drug Product Labeling

Regulations for information on drug packaging.

A.O. 2016-0008

Regulations for generic drug labeling requirements.

DC No. 2021-0003

Compliance guidelines for maximum retail drug price.

Product Name

Official name of the drug product.