29 terms that appear in clinical planning, review, and readiness workflows.
A basket trial is a master-protocol design that enrolls patients into one study by shared molecular target rather than by disease, testing a single therapy across multiple histologies as separate, independently analyzed cohorts.
Bioequivalence is the regulatory standard requiring a test product to deliver the same rate and extent of active-ingredient exposure as its comparator, unlike pharmaceutical equivalence, which compares only composition, strength, and dosage form.
Blinding is the bias-control measure in a clinical trial that conceals treatment assignment from subjects, investigators, or analysts, distinct from randomisation, which determines how subjects are assigned rather than who knows the assignment.
A bridging study is a clinical study run in the region where approval is sought that generates the pharmacodynamic, pharmacokinetic, or clinical data needed to extrapolate foreign pivotal trial results to that region's population.
A clinical hold is an FDA order under an IND that delays a proposed clinical investigation or suspends an ongoing one, halting dosing until the sponsor corrects the deficiencies FDA cited in writing.
A Contract Research Organization is an independent contractor that assumes, under written transfer from a sponsor, one or more of that sponsor's regulatory obligations for a clinical investigation and must itself comply with the regulations governing them.
A Data Monitoring Committee is an independent group of clinicians and statisticians that reviews unblinded accumulating data during a trial and recommends to the sponsor whether to continue, modify, or terminate it.
Decentralized Clinical Trials are clinical investigations in which some or all participant activities, including consent, dosing, assessments and safety monitoring, happen at the participant's home or a local provider instead of the investigator's research site.
IND-enabling studies are the nonclinical pharmacology, toxicology and ADME work a sponsor runs to support the conclusion that human dosing is reasonably safe, distinguishing them from exploratory discovery studies never submitted to FDA.
Informed Consent is the legally effective authorization a research subject gives before any study procedure, obtained under conditions that allow unhurried consideration, minimize coercion, and use language the subject actually understands.
The Investigator's Brochure is the sponsor-maintained compilation of clinical and nonclinical data on an investigational product, functioning as its pre-approval labeling: the reference investigators use to judge risk-benefit and comply with the protocol.
An IRB is the committee a research institution formally designates to approve, require modification of, or disapprove human-subject research before it begins, and to re-review it at least annually under FDA rules.
Master Protocol is a single overarching clinical trial protocol under which multiple sub-studies run simultaneously, evaluating several investigational products, several populations, or both within one shared infrastructure, control group, and statistical framework.
Minimum Anticipated Biological Effect Level (MABEL) is the human exposure predicted to produce the lowest biologically observable effect, calculated from pharmacology rather than toxicology, and used by EMA as one of three permitted anchors for a first-in-human starting dose.
A patient narrative is the case-level prose account of one trial subject's death, serious adverse event, or other significant adverse event, written for the clinical study report rather than for expedited safety reporting.
Patient-reported outcome (PRO) is an outcome measure captured directly from the patient about their own symptoms, function, or health status, with no interpretation, filtering, or amendment by a clinician, caregiver, or investigator.
Pharmacodynamics is the branch of clinical pharmacology that quantifies what a drug does to the body: target engagement, biochemical effect, and clinical response, measured against dose and blood concentration rather than the body's handling of the drug.
Pharmacokinetics is the quantitative description of what the body does to a drug over time: absorption, distribution, metabolism, and excretion, summarized as exposure parameters such as Cmax, AUC, clearance, and half-life that set dose and dosing interval.
Phase 1 clinical trials are the first human studies of an investigational drug, designed to characterize metabolism, pharmacologic actions, and dose-related side effects rather than to prove effectiveness, typically enrolling 20 to 80 subjects.
Phase 2 clinical trials are the controlled studies that evaluate whether a drug is effective in patients with the target disease and determine the dose, regimen, and endpoints that Phase 3 will confirm.
Phase 3 clinical trials are expanded controlled studies run after preliminary evidence of effectiveness, sized at several hundred to several thousand subjects to establish the benefit-risk relationship and supply the substantial evidence supporting approval and physician labeling.
The primary endpoint is the pre-specified outcome measure a clinical trial is powered and statistically tested against, carrying the trial's efficacy claim; secondary and exploratory endpoints add supporting evidence but cannot substitute for it.
A protocol amendment is the IND submission that adds a new clinical protocol, changes an existing protocol, or adds an investigator; protocol changes must be submitted to FDA and approved by the IRB before implementation.