75 terms covering drug development, regulatory pathways, clinical trial design, pharmacology, safety, and intellectual property.
Regulatory
NDA (New Drug Application) — New Drug Application — the FDA submission required to market a new small-molecule drug in the US.
BLA (Biologics License Application) — Biologics License Application — the FDA submission required for biologics (proteins, antibodies, gene therapies).
PDUFA date (PDUFA) — The FDA target action date for an NDA or BLA, set under the Prescription Drug User Fee Act.
Priority review — FDA designation that shortens NDA/BLA review to 6 months for drugs offering significant improvement.
Breakthrough Therapy — FDA designation that fast-tracks review of drugs with substantial improvement on clinically significant endpoints.
Accelerated approval — FDA pathway allowing approval based on a surrogate endpoint, requiring confirmatory post-approval trials.
Orphan drug — US designation for drugs treating rare diseases (fewer than 200,000 US patients).
NICE (National Institute for Health and Care Excellence) — The UK body that decides whether the NHS will reimburse a drug, based on cost-effectiveness.
IND (Investigational New Drug) — The FDA authorisation required before testing an experimental drug in humans.
EMA (European Medicines Agency) — The EU regulatory agency that authorises and supervises medicines for use across member states.
CHMP (Committee for Medicinal Products for Human Use) — The EMA committee that issues opinions on whether a medicine should be authorised in the EU.
EPAR (European Public Assessment Report) — The publicly available EMA assessment of a centrally-authorised medicine.
PDUFA (Prescription Drug User Fee Act) — The US law that authorises FDA user fees from sponsors in exchange for performance commitments.
CRL (Complete Response Letter) — The FDA notification that an NDA or BLA cannot be approved in its present form.
AdCom (Advisory Committee, FDA AdCom) — An external expert panel that votes on whether the FDA should approve a contested drug.
IND (Investigational New Drug) — The FDA authorisation that lets a sponsor start human trials in the US.
CTA (Clinical Trial Application) — The EU equivalent of the US IND — required to begin clinical trials in member states.
GMP (Good Manufacturing Practice) — The quality-control standards drug manufacturers must follow.
ICH (International Council for Harmonisation) — The global body that harmonises drug regulation across the US, EU, Japan, and other markets.
PMDA (Pharmaceuticals and Medical Devices Agency) — Japan's medicines regulator.
ANDA (Abbreviated New Drug Application) — The FDA pathway for approving a generic copy of a previously-approved small-molecule drug.
NCE (New Chemical Entity, NME) — A drug containing an active ingredient never previously approved by the FDA.
Indication — The specific use of a drug as authorised by a regulator.
Clinical trials
Phase 1 trial (Phase I) — First-in-human study, primarily testing safety and pharmacokinetics in 20–100 participants.
Phase 2 trial (Phase II) — Mid-stage trial testing efficacy and dose-response in 100–500 patients with the target disease.
Phase 3 trial (Phase III, pivotal trial) — Large randomised registration trial (300–3000+ patients) used to support regulatory approval.
Primary endpoint — The pre-specified outcome measure used to determine whether a trial succeeded.
Surrogate endpoint — A measurable biomarker (e.g. tumour shrinkage, LDL) used as a proxy for a clinical benefit.
CDx (companion diagnostic) — A diagnostic test required to identify patients who will benefit from a specific drug.
OS (Overall Survival) — Time from treatment start (or randomisation) until death from any cause.
PFS (Progression-Free Survival) — Time from treatment start until disease progression or death.
ORR (Objective Response Rate) — The proportion of patients with a complete or partial tumour response.
HR (Hazard Ratio) — The relative risk of an event (progression, death) in the treatment vs. control arm.
p-value — The probability of seeing the observed (or more extreme) result if the null hypothesis were true.
ITT (Intention-to-Treat) — Analysing every randomised patient in the arm they were assigned, regardless of treatment received.
NNT (Number Needed to Treat) — The number of patients to treat to prevent one adverse outcome.
RECIST (Response Evaluation Criteria In Solid Tumours) — The standardised criteria for measuring tumour response on imaging in solid-tumour oncology trials.
NCT (ClinicalTrials.gov identifier) — The unique identifier (NCT########) assigned to every trial registered on ClinicalTrials.gov.
Pharmacology
Mechanism of action (MoA, MOA) — The specific biochemical interaction by which a drug produces its pharmacologic effect.
Pharmacokinetics (PK) — How the body processes a drug — absorption, distribution, metabolism, excretion (ADME).
Pharmacodynamics (PD) — How a drug affects the body — its biological effects, receptor occupancy, and dose-response.
PROTAC (Proteolysis-Targeting Chimera) — A bifunctional molecule that recruits the cell's protein-degradation machinery to destroy a target protein.
ADC (Antibody-Drug Conjugate) — An antibody covalently linked to a cytotoxic payload, delivered selectively to target cells.
CAR-T (Chimeric Antigen Receptor T-cell therapy) — A personalised cell therapy where a patient's T cells are engineered to attack their cancer.
mAb (Monoclonal Antibody) — A laboratory-produced antibody designed to bind a single epitope of a target protein.
mRNA (messenger RNA therapy) — Drugs that deliver mRNA to cells so they produce a therapeutic protein.
MOA (Mechanism of Action) — Synonym for "mechanism of action."
Safety
Drug interaction (DDI, drug-drug interaction) — A change in a drug's effect or safety caused by co-administration with another drug.
Adverse event (AE) — Any untoward medical occurrence in a patient receiving a drug, regardless of causality.
Boxed warning (black box warning) — The strongest FDA warning, displayed in a black border at the top of a drug label.
REMS (Risk Evaluation and Mitigation Strategy) — An FDA-required safety plan for drugs with significant risks (e.g. teratogenicity, addiction).
FAERS (FDA Adverse Event Reporting System) — The FDA database of post-marketing adverse event and medication error reports.
PV (Pharmacovigilance) — The science of detecting and managing adverse drug effects after marketing.
Patents & exclusivity
Patent cliff — The steep drop in revenue when a drug's patents expire and generic competition launches.
Paragraph IV — A generic manufacturer's certification that a brand drug's patents are invalid or not infringed.
Orange Book — The FDA-maintained list of approved drug products with patent and exclusivity information.
BPCIA (Biologics Price Competition and Innovation Act) — The 2010 US law that created the regulatory + patent pathway for biosimilars.
Hatch-Waxman — The 1984 US law that created the modern generic-drug approval pathway and 30-month patent stay.
Commercial & access
Biosimilar — A biologic product highly similar to an already-approved reference biologic.
Generic drug — A bioequivalent copy of a small-molecule drug after its patent and exclusivity expire.
QALY (Quality-Adjusted Life Year) — A measure combining length and quality of life used in cost-effectiveness analysis.
NICE TA (Technology Appraisal) — The NICE process that decides whether the NHS will fund a new medicine.
ICER (cost) (Incremental Cost-Effectiveness Ratio) — The cost-per-extra-QALY a treatment delivers compared to the standard of care.
WAC (Wholesale Acquisition Cost) — The US list price of a drug — manufacturer to wholesaler, before any discounts or rebates.
NADAC (National Average Drug Acquisition Cost) — The CMS-published actual acquisition cost from a national survey of US pharmacies.
ASP (Average Sales Price) — The weighted average net selling price of a drug to all US purchasers.
340B — The US programme that requires manufacturers to sell drugs at deep discount to safety-net providers.
IRA (Inflation Reduction Act) — The 2022 US law that lets Medicare negotiate prices for high-spend drugs.
CRO (Contract Research Organisation) — A company that runs clinical trials on behalf of pharmaceutical sponsors.
CMO (Contract Manufacturing Organisation, CDMO) — A company that manufactures drug substance or drug product on behalf of pharmaceutical sponsors.
OTC (Over-the-Counter) — A drug sold without a prescription.
PBAC (Pharmaceutical Benefits Advisory Committee) — The Australian committee that decides which medicines are listed on the publicly-funded PBS.
HTA (Health Technology Assessment) — The systematic evaluation of a medicine's clinical and cost-effectiveness for reimbursement decisions.
Off-label — Prescribing a drug for a use not specifically approved by the FDA.