Biopharmaceutics Foundations and Drug Approval Process (Week 1 pt 2)

Comprehensive vocabulary flashcards covering the drug approval process phases, regulatory milestones, clinical trial requirements, and pharmaceutical equivalence terminology.

IND

The Investigational New Drug application; the formal regulatory submission and gateway between laboratory research and human testing.

NDA

New Drug Application; the extensive submission (usually 1-2 years) that packages clinical trial data to build the case for market approval.

Preclinical Testing

A 2-5 year stage where potential drug candidates are identified, screened in laboratories, and tested in animal models to evaluate safety and biological mechanisms.

Phase 1 Clinical Trials

The initial human testing phase using dose escalation in small volunteer groups to establish a safe dosage range and identify the Maximum Tolerated Dose (MTD).

Phase 2 Clinical Trials

The stage involving "dose-ranging" studies in real patients to look at how effectiveness and safety shift across different doses to refine the optimal therapeutic dose.

Phase 3 Clinical Trials

Pivotal trials directy supporting the NDA; the formulation used must match the final market version in dosage form, strength, and manufacturing process.

Maximum Tolerated Dose (MTD)

The highest dose of a drug that does not cause unacceptable side effects, identified during Phase 1 dose escalation.

Formulation Lock

The requirement that the drug formulation used in Phase 3 must be the final version that patients and doctors will actually use in the market.

Pre-Approval Inspection

An on-site inspection of the manufacturing facility by regulators before granting approval to verify cGMP compliance and data matching.

Current Good Manufacturing Practices (cGMP)

Regulated standards that a manufacturing facility must follow to ensure the production process is reproducible at full commercial scale.

Expedited Pathways

Regulatory mechanisms such as priority review that can shorten the timeline for drugs treating serious or life-threatening conditions.

Bioavailability

The extent and rate of absorption from a dosage form as reflected by a time-concentration curve of the drug in the systemic circulation.

Biological equivalent (Bioequivalent)

Pharmaceutical equivalent or alternative products that result in comparable bioavailability when administered to the same individuals in the same dosage regimen.

Pharmaceutical equivalent

Drug products containing the same active ingredients in identical amounts, dosage forms, and routes of administration, though they may differ in inert ingredients like color or flavor.

Therapeutically equivalent

Pharmaceutical equivalents that provide the same therapeutic effect as measured by the control of a disease or symptom.

Pharmaceutical Alternate

Drug products containing the same therapeutic moiety but as different salts, esters, complexes, dosage forms, or strengths.

Therapeutic moiety

The active portion or molecule of a drug common to pharmaceutical alternatives, even if the salt or complex form differs.

Semaglutide

The active ingredient found in both Ozempic and Wegovy, administered via weekly injections.

Ozempic

An FDA-approved drug (2017) for Type 2 diabetes and reducing cardiovascular risk, supplied in multi-use pens (1 per month).

Wegovy

An FDA-approved drug (2021) for weight loss and reducing cardiovascular risk, supplied in single-use pens (4 per month).

Commercial Reasons

The leading cause of discontinuation in clinical development, accounting for $40\,\%$ of cases.

Lack of Efficacy

A significant reason for the discontinuation of clinical drug development, accounting for $30\,\%$ of cases.

Adverse Effects in Man

A reason for terminating drug development after human trials begin, representing $10\,\%$ of discontinuations.

Pharmacokinetics (Discontinuation)

Issues regarding how the drug moves through the body that lead to the discontinuation of clinical development in $10\,\%$ of cases.

Animal Toxicity

Findings in preclinical models that lead to the discontinuation of development in $5\,\%$ of drug candidates.

Novo Nordisk

The manufacturing company responsible for the production of Ozempic and Wegovy.

Dose-escalation

The Phase 1 protocol of starting with a very low dose and gradually increasing it to identify the relationship between dose and side effects.

Multi-use pen

The delivery system for Ozempic where one pen is used for several injections over the course of a month.

Single-use pen

The delivery system for Wegovy where each pen is used for only one injection, requiring four pens per month.

Bridging Studies

Additional bioequivalence studies triggered if a drug formulation is changed after Phase 3 to prove it still behaves the same in the body.