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Glossary

Our glossary provides you with precise definitions of key terms from clinical research, clinical trials and the work of contract research organizations (CROs). Learn more about essential concepts, regulatory requirements and technical terms that play a role in drug and medical device development.

A

A study type that allows modifications to the trial procedures or protocols based on interim results, providing flexibility for changes during the trial to enhance efficiency and effectiveness.

Gene therapy, tissue-engineered products, and somatic cell therapy products regulated by the EMA in the EU and the FDA in the US, representing a specialized subset of biologics.

Any undesirable experience timely associated with the use of an investigational medicinal product or procedure in a clinical trial participant.

The German law that took effect in 2011, whose provisions form the basis for price negotiations in Germany.

B

German National Authority/ Regulatory agency, (Federal Institute for Drugs and Medical Devices, Bundesinstitut für Arzneimittel und Medizinprodukte).

Medical products derived from living organisms, including vaccines, blood products, and therapeutic proteins made using biotechnology.

Blinding is a methodological tool in clinical studies where information about which treatment a participant receives is concealed from one or more parties.

C

A global, non-profit organization that develops data standards to streamline clinical research and improve the quality, consistency, and interoperability of clinical data.

A systematic investigation involving one or more human subjects and conducted for the purpose of evaluating the safety or performance of a medical device.

A research study related to (a) medicinal product(s) entailing specific interventions where human subjects are assigned to one or more interventions to evaluate the effects of medicinal products on health-related biomedical or behavioral outcomes.

A regulatory submission required before initiating a clinical trial, containing comprehensive information about the trial protocol, investigational product, and safety measures.

An EU regulatory framework (No 536/2014) aiming to streamline the approval process and harmonize regulatory requirements for clinical trials across member states.

A centralized platform developed by the EMA to streamline the submission, assessment, and monitoring of clinical trials in the EU and EEA, part of the Clinical Trial Regulation (CTR).

A company that provides outsourced clinical research services, managing clinical trials on behalf of sponsors.

A group in clinical studies that does not receive the experimental treatment, serving as a baseline for comparison to determine the treatment’s efficacy and safety.

D

Trials utilizing digital technologies and decentralized methods, allowing for remote data collection, virtual visits, and home-based participation.

“Digital Health Applications,” a regulatory framework integrating digital health technologies into the German healthcare system, introduced by the Digital Healthcare Act.

The relationship between the dose of a drug and its effects on the body.

E

A digital tool used in clinical research to collect and manage data from clinical trial participants.

A digital method for collecting self-reported health information directly from trial participants, typically through electronic devices such as smartphones.

A group that reviews and monitors biomedical research involving human subjects, ensuring the study’s ethical standards and participant safety.

Contract Research Organization located in Europe that conducts clinical studies for sponsor companies.

A regulatory agency responsible for evaluating and supervising medicinal products in the EU and EEA.

G

Treatments that involve altering genetic material within a patient’s cells to treat or prevent diseases.

A comprehensive EU regulation aimed at protecting personal data and regulating its processing by organizations, applicable within the EU and EEA.

An international ethical and scientific quality standard for designing, conducting, and reporting clinical trials involving human participants.

I

Guideline on “Good Clinical Practice”, an international ethical and scientific quality standard for planning, initiating, performing, recording, oversight, evaluation, analysis and reporting clinical trials involving human participants.

The process by which individuals voluntarily agree to participate in a clinical trial after being informed of the trial’s purpose, procedures, risks, and benefits.

A global initiative to harmonize guidelines for the pharmaceutical industry, including those related to clinical development.

A new (biological) drug used in clinical investigations but not yet approved for marketing by the FDA.

The individual responsible for conducting a clinical trial at a trial site, often leading a research team.

International guidelines for conducting clinical investigations of medical devices in humans, ensuring ethical and scientific integrity.

L

A local sponsor representative (entitiy) in the EU/EEA, which a non-EU/EEA sponsor company must appoint to carry out a clinical study there when lacking an own subsidiary in the desired study country.

M

Market access for a new drug relates to swift and continuous access to new medical therapies for eligible patients at an acceptable cost.

An EU regulation (2017/745) governing the marketing, distribution, and use of medical devices within the EU/EEA, replacing the previous Medical Device Directive.

The UK agency responsible for regulating medicines, medical devices, and blood components for transfusion.

Each EU or EEA country where a clinical trial is intended to be conducted.

The act of overseeing the progress of a clinical trial to ensure compliance with protocols, SOPs, GCP, and regulatory requirements.

O

A status granted in Europe for medicinal products intended to treat rare diseases, providing incentives to stimulate research and development for unmet medical needs.

A term for medicinal products that are intended for the treatment of rare diseases.

P

German National Authority/ Regulatory agency, Paul-Ehrlich-Institute (Federal Institute for Vaccines and Biomedicines).

The study of how drugs affect the body, including mechanisms of action and biological responses.

The study of how the body absorbs, distributes, metabolizes, and excretes a drug.

The science and activities related to detecting, assessing, and preventing adverse effects or drug-related problems after a drug has been marketed.

A phase of a clinical trial refers to the stage of development that a potential new drug therapy is in. A clinical trial can be a Phase I, Phase II, Phase III or Phase IV.

Clinical trials that are conducted in the intended target population with the intend to gather safety and efficacy data for regulatory approval.

Integration of pharmacokinetics and pharmacodynamics to understand the relationship between drug concentration and effects, optimizing dosing regimens and therapeutic efficacy.

The ongoing monitoring of the safety and effectiveness of pharmaceutical products after they are granted marketing authorization.

A document detailing the objectives, design, and methodology of a clinical trial.

R

Assigning clinical trial subjects to treatment or control groups using an element of chance to reduce bias and support the validity of study conclusions.

A medical condition that affects no more than 1 in 2,000 persons living in any WHO-defined region in the world.

An approach to clinical trial monitoring that focuses resources on high-risk areas for patient safety and data integrity.

S

An untoward medical occurrence that results in death, life-threatening conditions, hospitalization, disability, or birth defects.

Regulatory benefits for SMEs conducting clinical trials in the EU, as defined by the European Commission.

Medical treatments that use living or non-living cells to treat various diseases and conditions, such as stem cell therapy.

An individual or organization responsible for initiating, managing, and financing a clinical trial.

Detailed instructions to achieve uniformity in the performance of specific functions.

The primary or secondary outcome used to judge the effectiveness of a treatment in a clinical trial.

An individual participating in a clinical trial, receiving the investigational product or serving as a control.

T

Products developed from tissues or organs for implantation or grafting into patients.

A collection of essential documents that support the conduct, management, and compliance of a clinical trial, serving as the central repository for all trial-related documentation.

The locations where clinical trial activities are conducted.