Pharm & Society Final Exam

IND (Investigational New Drug)

Request submitted to regulatory agencies (e.g., Health Canada) to begin human clinical trials; ensures safety and proper study design; includes preclinical data, manufacturing info, and protocols; requirements vary by agency (FDA, EMA, etc.)

NDA (New Drug Application)

Comprehensive submission requesting approval to market a drug; includes preclinical data, clinical trial results, manufacturing details, and labeling; approval is required for legal sale

CTA (Clinical Trial Application)

Regulatory submission used outside the U.S. to approve clinical trials; similar to IND; must follow region-specific regulations (e.g., EU guidelines)

Phase I Trial

First human testing phase assessing safety, dosage, and pharmacokinetics; usually involves small numbers of healthy volunteers; often open-label

Phase II Trial

Tests efficacy and side effects in patients with the target disease; helps determine therapeutic effectiveness

Phase III Trial

Large-scale trials confirming efficacy and safety; compares drug to standard treatments; typically randomized and blinded

GMP (Good Manufacturing Practice)

Regulations ensuring drugs are consistently produced with quality, purity, and safety; covers manufacturing, testing, and quality control

GLP (Good Laboratory Practice)

Guidelines ensuring reliability and integrity of preclinical studies; includes protocols, data management, and quality assurance

Regulatory Affairs

Field ensuring compliance with drug laws; involves preparing submissions (IND, NDA, BLA), interacting with regulators, advising on compliance, and monitoring regulatory changes

Post-Marketing Surveillance

Monitoring drug safety and effectiveness after approval; detects rare or long-term adverse effects using reporting systems and data analysis

Adverse Event Reporting

Process of reporting negative drug reactions; done by healthcare providers, patients, and companies; identifies safety signals

BLA (Biologics License Application)

Approval submission for biologic drugs; requires additional data due to complexity; differs from NDA in product type and requirements

Orphan Drug Designation

Status for drugs treating rare diseases; provides incentives such as tax credits, market exclusivity, and reduced fees

Fast Track Designation

Speeds development/review of drugs for serious conditions with unmet needs; allows frequent communication with regulators

Breakthrough Therapy Designation

Given when early clinical evidence shows major improvement over existing treatments; accelerates development and review

Priority Review

Shortens regulatory review time for drugs with significant clinical benefit; faster access to market

Labeling

Official drug information including indications, dosage, contraindications, warnings, and adverse effects; must meet regulatory standards

Rational Drug Design

Designing drugs based on understanding biological targets and receptor structures; uses computational tools and chemical modification to create effective molecules

Pharmacogenomics

Study of how genetic variations affect drug response, metabolism, efficacy, and toxicity

Personalized Medicine

Tailoring treatments based on individual genetic and biological characteristics

Biologics (Large Molecule Drugs)

Complex drugs derived from living systems (e.g., monoclonal antibodies); offer targeted treatment but have manufacturing challenges

Dose-Response Relationship

Relationship between drug dose and magnitude of effect; used to determine effective and safe dosing

Pharmacokinetics (ADME)

Study of how drugs are absorbed, distributed, metabolized, and excreted; determines drug exposure and bioavailability

Pharmacodynamics

Study of drug effects on the body and mechanisms of action; explains how drugs produce physiological responses

Acute Toxicity

Adverse effects occurring within 24 hours after a single high dose

Subacute/Subchronic Toxicity

Toxic effects from repeated exposure over 28–90 days

Chronic Toxicity

Long-term toxic effects from prolonged exposure (months to years)

No Effect Dose

Highest dose where no toxic effects are observed

Minimum Lethal Dose

Smallest dose that causes death in test animals

LD50

Dose that kills 50% of the test population; used to assess toxicity

NOAEL

Highest dose with no observed adverse effects

LOAEL

Lowest dose where adverse effects are observed

Genotoxicity

Ability of a substance to damage DNA

Carcinogenicity

Ability to cause cancer

Reproductive Toxicity

Harmful effects on fertility or reproductive function

Immunotoxicity

Damage to the immune system

Hepatotoxicity

Toxic effects on the liver

Nephrotoxicity

Toxic effects on the kidneys

Cumulative Toxicity

Toxic effects that build up with repeated dosing

Reversibility

Ability of toxic effects to disappear after stopping exposure

Therapeutic Index (TI)

Ratio of toxic dose to effective dose; indicates drug safety margin (higher = safer)

Safety Margin (Certain Safety Factor)

Ratio comparing lethal/toxic dose for 1% (LD1/TD1) to effective dose for 99% (ED99); indicates extreme safety limits

Narrow Therapeutic Index (NTI)

Drug with small difference between effective and toxic doses; requires close monitoring

FIH (First-in-Human)

First time a drug is tested in humans after animal studies; highest risk stage for unexpected effects

Perinatal Depression

Depression occurring during pregnancy or within one year postpartum

PPD (Postpartum Depression)

Mood disorder affecting mothers after childbirth; common and impacts both mother and child

DSM-5 MDD with Peripartum Onset

Diagnostic criteria for major depression occurring during pregnancy or shortly after birth

ACE (Adverse Childhood Experience)

Early-life stressors (e.g., maternal depression) that affect long-term development and health

EPDS (Edinburgh Postnatal Depression Scale)

Screening tool used to identify postpartum depression

CBT (Cognitive Behavioral Therapy)

Therapy focusing on changing negative thoughts and behaviors

IPT (Interpersonal Therapy)

Therapy focusing on improving relationships and social functioning

Geriatric Pharmacology

Study of drug effects in older adults, considering age-related physiological changes

GFR (Glomerular Filtration Rate)

Measure of kidney function; decreases with age, affecting drug clearance

Bioavailability

Fraction of drug reaching systemic circulation

Volume of Distribution (Vd)

Extent drug distributes into tissues; increased for lipophilic drugs in elderly

Cockcroft-Gault Formula

Equation used to estimate creatinine clearance for dosing adjustments

Polypharmacy

Use of multiple medications; increases risk of adverse events

ADE (Adverse Drug Event)

Any harm caused by medication use

ADR (Adverse Drug Reaction)

Harmful response to a drug at normal doses

Beers Criteria

Guidelines for healthcare professionals to identify potentially inappropriate medications (PIMs) for adults aged 65 and older, aiming to reduce adverse drug events. Widely used in Canada, the criteria—updated by the American Geriatrics Society (AGS)—list medications to avoid or use with caution due to high risks in seniors.

Drug-Drug Interaction

One drug altering another’s effect

Hyperkalemia

Elevated potassium levels in blood

Anticholinergic Agents

Drugs that block acetylcholine; cause stronger side effects in elderly

Phase I Metabolism

Drug modification (e.g., oxidation); often reduced in elderly

Phase II Metabolism

Drug conjugation; less affected by aging

NSAIDs

Anti-inflammatory drugs; can cause kidney damage, fluid retention, and GI issues

Benzodiazepines

Sedative drugs; increased risk of sedation, falls, and cognitive impairment in elderly

Creatinine Clearance

Estimate of kidney function for drug dosing

Lipophilic Drugs

Fat-soluble drugs with increased distribution and prolonged action in elderly

Drug-Disease Interaction

Drug worsening an existing condition

Reduced Plasma Protein Binding

Leads to higher free drug levels and stronger effects

Individualized Drug Therapy

Tailoring treatment based on patient-specific factors

Adherence Challenges

Difficulty following medication regimens due to cognitive or physical issues

Clinical Monitoring

Ongoing assessment of drug safety and effectiveness

Deprescribing

Systematic process of stopping medications when risks outweigh benefits

Withdrawal Syndrome

Symptoms that occur after stopping a drug

Rebound Effect

Return of symptoms, often worse, after discontinuation of medication

GLP-1 Receptor Agonist

Drug that mimics incretin hormone to increase insulin, decrease glucagon, and slow gastric emptying

Half-life

Time required for drug concentration to decrease by half (~7 days for semaglutide/ozempic)

Proteolysis & Beta-Oxidation

Metabolic pathways used instead of liver CYP450 system

MACE (Major Adverse Cardiovascular Events)

Includes heart attack, stroke, and cardiovascular death

Dose Titration

Gradually increasing dose to reduce side effects

Contraindications

Conditions where drug should not be used (e.g., history of certain cancers)

Medullary Thyroid Carcinoma (MTC)

Thyroid cancer contraindication for GLP-1 drugs

MEN-2

Genetic condition increasing risk of endocrine tumors

Prevention Paradox

Situation where population-level benefit exists but access limits individual benefit

Cytochrome P450 (CYP450)

A family of liver enzymes responsible for Phase I drug metabolism (mainly oxidation); convert drugs into more water-soluble forms for elimination; activity can vary due to genetics, age, and environmental factors; major source of drug-drug interactions because some drugs inhibit or induce CYP450 enzymes, altering drug levels and effects

Therapeutic Drug Monitoring (TDM)

The measurement of drug concentrations in blood to ensure levels remain within the therapeutic range; especially important for drugs with a narrow therapeutic index to avoid toxicity or subtherapeutic dosing

Teratogenicity

The ability of a drug or substance to cause abnormalities in fetal development; assessed in preclinical studies using animal models; critical for evaluating drug safety during pregnancy

Cardiotoxicity

Toxic effects of a drug on the heart, often assessed using ECGs and cardiac biomarkers; can include arrhythmias or impaired cardiac function; important in both preclinical and clinical safety evaluation

Prescribing Cascade