Clinical trials

Subjects/participants: Individual patients and healthy volunteers: People volunteer to take part in clinical trials either as a control or as a recipient to test medical interventions including gene and cell therapy such as advanced therapy investigational medicinal products, cells and tissues used for therapeutic purposes, but also other biological products, surgical procedures, radiological procedures, devices, behavioral treatments, or preventive care. 

Ethics Committees: Ethics Committees’ role is to verify that the research protocol of the clinical trials complies with all ethics principles. Ethics Committees are independent national or local bodies established by Member States in accordance with their national law and empowered to give opinions for the national competent authorities to authorise or not the start of clinical trials.  

It should be noted that it generally exists other Ethics Committees under national laws, such as national ethics committees in charge of advising the policy and law decision makers, or universities’ research ethics committees in charge of reviewing research protocols for other studies than clinical trials. 

Funders and Sponsors:  A clinical trials’ sponsor is the individual, the company, the institution or the organization which takes the responsibility for the initiation, the management, and the setting up of the financing of the clinical trial. In the European Economic Area, 61% of clinical trials are sponsored by the pharmaceutical industry and 39% by non-commercial sponsors (academia, fund raisers…). 

National Competent Authorities: National Competent Authorities, which are often national medicines agencies, are notably responsible for authorizing and oversighting clinical trials. 

European Medicines Agency: The role of the EMA regarding clinical trials was mainly limited to the review of clinical trials data in the context of marketing authorisation application. It is now being strengthened with the entry into force of the new European regulation on clinical trials as the EMA has been in charge of setting up and maintaining the Clinical Trials Information System (CTIS). More information here. 

Investigators: They are the individuals, researchers, who are responsible for the conduct of a clinical trial at a clinical trial site.  

Principal investigator: is an investigator who has the responsibility to lead the investigators team conducting a clinical trial at a clinical trial site.

Clinical study: “‘Clinical study’ means any investigation in relation to humans intended: 

1. to discover or verify the clinical, pharmacological or other pharmacodynamic effects of one or more medicinal products; 
2. to identify any adverse reactions to one or more medicinal products; or 
3. to study the absorption, distribution, metabolism and excretion of one or more medicinal products; 

with the objective of ascertaining the safety and/or efficacy of those medicinal products;” (article 2§2 (1), of European Regulation No 536/2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC) 

Clinical trial: “‘Clinical trial’ means a clinical study which fulfils any of the following conditions: 

1. the assignment of the subject to a particular therapeutic strategy is decided in advance and does not fall within normal clinical practice of the Member State concerned; 
2. the decision to prescribe the investigational medicinal products is taken together with the decision to include the subject in the clinical study; or 
3. diagnostic or monitoring procedures in addition to normal clinical practice are applied to the subjects.”  

(Article 2§2 (2), of European Regulation No 536/2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC) 

‘Low-intervention clinical trial’: “means a clinical trial which fulfils all of the following conditions: 

1. the investigational medicinal products, excluding placebos, are authorised; 
2. according to the protocol of the clinical trial, 
   • the investigational medicinal products are used in accordance with the terms of the marketing authorisation; or 
   • the use of the investigational medicinal products is evidence-based and supported by published scientific evidence on the safety and efficacy of those investigational medicinal products in any of the Member States concerned; and 
3. the additional diagnostic or monitoring procedures do not pose more than minimal additional risk or burden to the safety of the subjects compared to normal clinical practice in any Member State concerned” (Article 2§2 (3), of European Regulation No 536/2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC) 

Advanced therapy investigational medicinal product: “‘Advanced therapy investigational medicinal product’ means an investigational medicinal product which is an advanced therapy medicinal product as defined in point (a) of Article 2(1) of Regulation (EC) No 1394/2007 of the European Parliament and of the Council.” (Article 2§2 (7), of European Regulation No 536/2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC) 

Subject: “‘Subject’ means an individual who participates in a clinical trial, either as recipient of an investigational medicinal product or as a control;” (Article 2§2 (17), of European Regulation No 536/2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC). 

Ethics Committee: ‘Ethics committee’ means an independent body established in a Member State in accordance with the law of that Member State and empowered to give opinions for the purposes of this Regulation, taking into account the views of laypersons, in particular patients or patients' organizations; (Article 2§2 (11), of European Regulation No 536/2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC). 

Protocol: “‘Protocol’ means a document that describes the objectives, design, methodology, statistical considerations and organization of a clinical trial. The term ‘protocol’ encompasses successive versions of the protocol and protocol modifications;” (Article 2§2 (22), of European Regulation No 536/2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC). 

Good clinical practice: ‘Good clinical practice’ means a set of detailed ethical and scientific quality requirements for designing, conducting, performing, monitoring, auditing, recording, analysing and reporting clinical trials ensuring that the rights, safety and well-being of subjects are protected, and that the data generated in the clinical trial are reliable and robust; (Article 2§2 (30), of European Regulation No 536/2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC).