Glossaries

A predominantly First-in-Human (FIH) trial with a single protocol with an initial dose-escalation phase followed by three or more additional subject cohorts with cohort-specific objectives. NOTE: The objectives of these expansion cohorts can include assessment of antitumor activity in a disease-specific setting, assessment of a dose with acceptable safety in specific populations (e.g., pediatric or elderly subjects, subjects with organ impairment, subjects with specific tumor types), evaluation of alternative doses or schedules, establishment of dose and schedule for the investigational drug administered with another oncology drug, or evaluation of the predictive value of a potential biomarker. In general, comparison of activity between cohorts is not planned except when a prespecified randomization and analysis plan are part of the protocol design. [FDA Guidance: Expansion Cohorts: Use in First-in-Human Clinical Trials to Expedite Development of Oncology Drugs and Biologics Guidance for Industry. March 2022]

The act or process of eliminating waste products from the body. See also ADME.

List of characteristics in a protocol, any one of which may exclude a potential subject from participation in a study.

Planned protocol activities such as randomization and study completion, and occurrences, conditions, or incidents independent of planned study evaluations occurring during the trial (e.g., adverse events) or prior to the trial (e.g., medical history). [After SDTM, www.cdisc.org] See also general observation class, intervention, finding.

Pertains to data or subjects that meet Statistical Analysis Plan criteria for inclusion in efficacy/safety datasets.

The regulatory agency for the EU.

Denotes social groups with a shared history, sense of identity, geography, and cultural roots.

Group convened to protect research subjects. NOTE: Such bodies, depending on the country or region, are abbreviated as: CCI, CCPPRB, CHR, CPPHS, CRB, EAB, HEX, HSRC, LREC, MREC, NIRB, NRB, and REB. See also institutional review board, independent ethics committee.

A precise description of the treatment effect reflecting the clinical question posed by a given clinical trial objective. It summarizes at a population level what the outcomes would be in the same patients under different treatment conditions being compared. NOTE: The four characteristics of an estimand include the definition of the target study population, statement of the endpoint of interest, intercurrent event details, and the population level summary of the variable of interest. (ICH E9 R1 Addendum; After Estimand Framework: What it is and Why You Need it. Applied Clinical Trials. February 27, 2020]

The official name of a drug or pharmaceutical product, relevant in US regulations. [US FDA, 21 CFR 299.4] See also proprietary name, generic name, international nonproprietary name (INN), medicinal product name, compendial name.

Documents that individually and collectively permit evaluation of the conduct of a study and the quality of the data produced. [ICH E6 Glossary]

The human-readable rendering of an electronic record serving as an eSource document that is part of a case history. The eSRF supports capture, transmission, storage, editing and/ or display of eSource documents (original records and certified copies of original records of clinical findings, observations, or other activities in a clinical investigation) used for reconstructing and evaluating the investigation. NOTE: Intended use distinguishes eCRF and eSRF. The eCRF is for capture, review and editing of protocol data belonging to the sponsor; the eSRF is for the human-readable representation of the eSource document for review or to maintain the eSource document that is part of the case history under 21CFR312.62. See also eCRF, eSource document. [CDISC, relevant to FDA Final Guidance on eSource]

Electronic record containing source data for a clinical trial, used to aggregate a particular instance of eSource data items for capture, transmission, storage, and/ or display, and serving as a source document for a clinical investigation. NOTE: Electronic Source documents are recorded in electronic systems according to conventions (such as those for PDF documents) that ensure that all the fields of eSource data and associated contextual information (e.g. time of capture, time zone, authorship, origin, signatures, revisions, etc.) are linked to each other in a particular structure for presentation. The encoded specifications in the electronic record thus serve the same role as have the physical properties of paper (binding data items together). eSource documents are subject to regulations and guidance that apply to source documents. See also source documents. [relevant to FDA Final Guidance on eSource]

Source data captured initially into a permanent electronic record (eSource document) used for the reconstruction and evaluation of a clinical study or a source data item included in an eCRF when direct entry is made. NOTE: permanent in the context of these definitions implies that any changes made to the electronic data are recorded via an audit trail. See also eSource document, source data, permanent data, data originator. [From body of FDA Final Guidance on eSource]

Source record that is electronic. See also source, electronic record.