Glossary

Any untoward medical occurrence in a participant administered a pharmaceutical product, whether or not it has a causal relationship with the treatment.

An adverse event that results in death, is life-threatening, requires hospitalization, causes persistent disability, or is a congenital anomaly.

A pre-identified adverse event that requires close monitoring and rapid communication due to its scientific and medical significance.

An SAE assessed by the investigator or sponsor as related to the investigational product and not previously identified in the Investigator's Brochure.

A standardized grading system (Grade 1-5) used to classify the severity of adverse events in clinical trials.

An independent committee that reviews unblinded safety data during a trial to protect participant safety and recommend continuation, modification, or termination.

Data quality standard: Attributable, Legible, Contemporaneous, Original, Accurate — plus Complete, Consistent, Enduring, and Available.

A chronological record of all data entries, changes, and deletions with user identification, timestamps, and reasons for change. Required by 21 CFR Part 11.

A systematic process to investigate root causes of identified problems (corrective) and prevent their recurrence (preventive).

Also known as a monitor, the CRA visits clinical sites to verify data against source documents, ensure protocol compliance, and protect participant safety.

The individual responsible for the day-to-day operational execution of clinical trials at the site, including participant management, data entry, and regulatory compliance.

A document (paper or electronic) used to collect and report data from each participant in a clinical trial, as defined by the protocol.

An organization contracted by the sponsor to perform one or more sponsor-related clinical trial functions (e.g., monitoring, data management, biostatistics).

Software used to manage clinical trial operations: enrollment tracking, site management, milestone tracking, and budget management.

A trial conducted partially or fully outside traditional clinical sites using telemedicine, remote monitoring, home nursing, and wearable devices.

A side effect of a drug that is severe enough to prevent increasing the dose or continuing treatment. Key concept in Phase 1 dose-escalation studies.

A computerized system for collecting clinical trial data in electronic format, replacing paper CRFs. Examples: Medidata Rave, Veeva Vault EDC, REDCap.

Electronic informed consent process using multimedia presentations, interactive elements, and digital signatures to enhance participant understanding and convenience.

The European Union's regulatory authority for evaluating and supervising medicinal products across EU member states.

Electronic Patient-Reported Outcomes / Electronic Clinical Outcome Assessments — digital tools for patients to report symptoms and quality of life directly.

Electronic repository for all essential trial documents, organized according to the TMF Reference Model.

The United States federal agency responsible for regulating and supervising drugs, biologics, medical devices, and clinical trials.

International ethical and scientific quality standard for designing, conducting, recording, and reporting clinical trials involving human subjects (ICH E6).

European Union regulation on data protection and privacy, with strict requirements for processing personal health data in clinical research.

Quality standards for manufacturing investigational products, ensuring they are consistently produced and controlled according to quality standards.

U.S. law that requires authorization for the use of Protected Health Information (PHI) in research and establishes privacy and security standards.

A compilation of all known clinical and non-clinical data about the investigational product, provided by the sponsor to the investigator.

The document signed by the participant (or LAR) confirming voluntary agreement to participate in a clinical trial after understanding the study details.

An international body that creates harmonized pharmaceutical guidelines across regulatory regions, including the GCP guidelines (E6).

An independent body that reviews the ethical aspects of clinical research to protect the rights, safety, and well-being of participants. Also called IRB or REC.

An application submitted to the FDA before a new drug can be tested in humans, containing preclinical data, manufacturing information, and the clinical protocol.

Instituto Nacional de Vigilancia de Medicamentos y Alimentos — Colombia's regulatory authority for food, drug, and clinical trial oversight.

Comisión Federal para la Protección contra Riesgos Sanitarios — Mexico's federal commission for health risk protection and clinical trial regulation.

The pharmaceutical form of an active ingredient or placebo being tested or used as a reference in a clinical trial.

An independent committee that reviews and approves clinical research to protect the rights and welfare of human participants. U.S. term for ethics committee.

The collection of essential documents maintained at the clinical site, containing all regulatory, operational, and safety documents for the study.

An analysis population that includes all randomized participants, regardless of protocol adherence or completion, preserving the benefits of randomization.

Interactive Web Response System / Randomization and Trial Supply Management — systems for participant randomization and drug supply management.

An individual authorized under applicable law to consent to participation in a clinical trial on behalf of a prospective participant.

Application submitted to the EMA for approval to market a medicinal product in the European Union, based on clinical trial data.

A Phase 1 study design where participants receive multiple doses over days/weeks to evaluate steady-state pharmacokinetics and cumulative effects.

A standardized medical terminology used for coding adverse events and medical history in clinical trials and post-marketing safety reporting.

The highest dose of a drug that does not cause unacceptable side effects. Determined through Phase 1 dose-escalation studies.

Application submitted to the FDA requesting approval to market a new drug in the United States, primarily based on Phase 3 data.

The study of what the drug does to the body — its mechanism of action, therapeutic effects, and dose-response relationships.

The qualified individual at a clinical site who is responsible for the conduct of the trial and the care of participants.

The study of what the body does to the drug — absorption, distribution, metabolism, and excretion (ADME).

A detailed document describing the objectives, design, methodology, statistical considerations, and organization of a clinical trial.

Any change, divergence, or departure from the study design or procedures defined in the approved protocol.

A formal change to the approved protocol, requiring IRB/IEC review and approval before implementation.

A monitoring approach that focuses resources on the most critical data and processes, using centralized statistical monitoring and targeted site visits.

A study design where participants are randomly assigned to intervention or control groups, considered the gold standard for evaluating treatment effects.

A standardized set of criteria for measuring tumor response (complete response, partial response, stable disease, progressive disease) in oncology trials.

Evidence derived from real-world data (EHRs, claims databases, registries) about the usage and effects of medical products outside of controlled clinical trials.

A Phase 1 study design where small groups receive escalating single doses to evaluate initial safety and pharmacokinetics.

A detailed document describing the pre-specified statistical analyses to be performed, including primary, secondary, and sensitivity analyses.

An organization that provides clinical trial services at the site level, including CRC staffing, regulatory management, and operational support.

A detailed, written set of instructions for performing a specific task or process consistently across an organization.

Original documents, data, and records from which CRF data are collected. Examples: medical charts, lab reports, imaging results.

The individual, company, institution, or organization that initiates, manages, and/or finances a clinical trial.

The complete collection of essential documents that demonstrate the conduct and quality of a clinical trial. Must be maintained throughout and after the trial.

IATA-mandated packaging for biological specimens: primary receptacle (holds specimen), secondary packaging (watertight with absorbent), and outer packaging (rigid protection).

UN classification number for Biological Substance, Category B — the most common category for clinical research specimen shipments.

UN classification number for Category A infectious substances affecting humans.

UN classification number for dry ice (carbon dioxide, solid) — classified as a Class 9 Miscellaneous Dangerous Good.

IATA classification for infectious substances capable of causing permanent disability, life-threatening or fatal disease in otherwise healthy humans or animals.

IATA classification for biological substances that do not meet Category A criteria but still require regulated packaging and transport.

FDA regulation establishing criteria for electronic records and electronic signatures to be considered trustworthy and equivalent to paper records.

The World Medical Association's statement of ethical principles for medical research involving human subjects, first adopted in 1964.

A 1979 report establishing three core ethical principles for human research: Respect for Persons, Beneficence, and Justice.

A set of ethical principles published in 1947 following Nazi medical experiments, establishing voluntary consent as an absolute requirement.

One of the most widely used EDC platforms globally, offering data capture, validation, medical coding, and integration with the broader Medidata clinical cloud.

An integrated cloud platform offering EDC, eTMF, CTMS, and quality management modules with a unified user experience.

Research Electronic Data Capture — a free, web-based, open-source EDC tool developed by Vanderbilt University for academic research.

A digital platform for managing electronic Investigator Site Files (eISF) and study documents, enabling remote monitoring.

A clinical trial design that allows pre-planned modifications (e.g., sample size, dose, treatment arms) based on interim data analysis without compromising validity.

A measurable characteristic that is an indicator of normal biological processes, pathogenic processes, or responses to therapeutic intervention.

A procedure in which one or more parties are unaware of treatment assignments. Single-blind: participants unaware. Double-blind: participants and investigators unaware.

A study where participants receive multiple treatments in sequence, serving as their own controls, separated by washout periods.

A pre-defined outcome measure used to evaluate the effect of an intervention. Can be primary (main outcome), secondary, or exploratory.

The FDA's database for post-marketing safety reports, used to monitor adverse events reported after drug approval.

An inactive substance or procedure designed to look identical to the investigational product, used as a control in clinical trials.

The process of randomly assigning participants to different treatment groups to reduce bias and ensure comparable groups.

A period during which a participant discontinues a treatment before starting a new treatment or entering a study, to eliminate residual effects.

Documentation tracking the handling, transfer, and location of biological specimens from collection through analysis, ensuring integrity and traceability.