Clinical trials in human medicines

The European Medicines Agency (EMA) relies on the results of clinical trials carried out by pharmaceutical companies to reach its opinions on the authorisation of medicines. Although clinical trials are authorised at national level in the European Union (EU), EMA plays a key role in further developing the EU as a competitive centre for innovative clinical trials, and in maintaining IT systems for the coordination of clinical trials.

Management of clinical trials impacted by the war in Ukraine

Sponsors can adjust the way they run clinical trials that have been affected by the war in Ukraine using the experience gained during the COVID-19 pandemic. They can also apply the approaches and flexibilities agreed in the context of the pandemic.

The European Commission, EMA and the Heads of Medicines Agencies (HMA) issued this initial advice for sponsors on 30 March 2022:

Additional recommendations for sponsors are available from the Clinical Trials Coordination Group (an HMA group uniting clinical trials experts):

Update: Guidance is also available from EMA's Biostatistics Working Party on actions sponsors of affected clinical trials can take to help ensure the integrity of their studies and the interpretation of the study results while safeguarding the safety of trial participants as a first priority:

EMA strongly encourages sponsors to capture data affected and unaffected by the war, and to use the 'estimand framework' described in the ICH E9 (R1) guideline for dealing with events impacting the trial:

Guidance on clinical trial management during the COVID-19 pandemic is available below.

EMA advises sponsors to also check any guidance available at national level.

Sponsors with specific questions relating to EU guidance can contact EMA using the Send a question form. Questions relating to national guidance should be addressed to the relevant national authority.

Sponsors can also contact either EMA or the national competent authorities for scientific advice on:

  • methodological aspects relating to their trials;
  • the use of affected clinical trial results for regulatory purposes.

Clinical trials are studies intended to discover or verify the effects of one or more investigational medicines.

The regulation of clinical trials aims to ensure that the rights, safety and well-being of trial participants are protected and the results of clinical trials are credible.

Regardless of where they are conducted, all clinical trials included in applications for marketing authorisation for human medicines in the European Union (EU) / European Economic Area (EEA) must have been carried out in accordance with the requirements set out in Annex 1 of Directive 2001/83/EC. This means that:

In the EU / EEA, approximately 4,000 clinical trials are authorised each year.

Approximately 60% of clinical trials are sponsored by the pharmaceutical industry and 40% by non-commercial sponsors, mainly academia.

For more information, see Clinical Trials Regulation.

EMA's role

EMA's Committee for Medicinal Products for Human Use (CHMP) is responsible for conducting the assessment of a human medicine for which an EU-wide marketing authorisation is sought. As part of its scientific evaluation work, the CHMP reviews the clinical trial data included in the application.

Clinical trial data is included in clinical-study reports that form a large part of the application dossiers submitted by applicants such as pharmaceutical companies and small and medium enterprises applying for a marketing authorisation via the Agency.

Assessments are based on purely scientific criteria and determine whether or not the medicines concerned meet the necessary quality, safety and efficacy requirements in accordance with EU legislation.

Furthermore, the Agency manages two clinical trial databases, EudraCT and the Clinical Trial Information System. EMA also co-leads the business change programme Accelerating Clinical Trials in the EU (ACT EU), together with the Heads of Medicines Agencies (HMA) and the European Commission.

For more information, see Clinical Trials Regulation.

Clinical trial management during the COVID-19 pandemic

Guidance is available for clinical-trial sponsors on how they should adjust the management of clinical trials and participants during the COVID-19 pandemic:

This covers how to deal with the extraordinary situations that the pandemic presents and specific advice on clinical trials for potential COVID-19 treatments.

Guidance is also available on the actions that sponsors of affected clinical trials should take to help ensure the integrity of their studies and the interpretation of the study results while safeguarding the safety of trial participants as a first priority:

In line with this guidance, EMA will be flexible and pragmatic during the assessment of affected clinical trial data submitted to the Agency as part of marketing authorisation applications.

Accelerating Clinical Trials in the EU (ACT EU)

The Accelerating Clinical Trials in the EU (ACT EU) initiative aims to develop the EU further as a competitive centre for innovative clinical research. 

The initiative builds on the Clinical Trials Regulation and Clinical Trials Information System's launch on 31 January 2022. It supports the European medicines agencies network strategy and the European Commission’s Pharmaceutical strategy for Europe in the field of clinical trial innovation.

An ACT EU strategy paper lists ten priority actions for 2022 and 2023 including:

  • enabling innovative trial methods;
  • developing and publishing guidance on key methodologies;
  • supporting the modernisation of good clinical practice;
  • establishing a multi-stakeholder platform. 

The European Commission, EMA and Heads of Medicines Agencies (HMA) launched ACT EU in January 2022 and run the initiative together, establishing a steering group in March 2022. 

Update: Under ACT EU, the European medicines regulatory network publishes statistics on the authorisation of clinical trials in the EU and EEA every month. This information provides an insight into how the Clinical Trials Regulation is transforming the clinical-trial environment in the EU / EEA. For more information, see:

Guidance on complex clinical trials (new)

New: Guidance is available for clinical trial sponsors, clinicians engaged in clinical trials and marketing authorisation applicants on planning and conducting complex clinical trials

The guidance covers the following topics: 

  • Planning and conducting complex clinical trials
  • Designing and conducting master protocols
  • Methods for clinical trial analysis (e.g. Bayesian approaches)
  • Biomarkers and their assays
  • Reporting trial information to regulatory authorities
  • Transparency

EMA, the European Commission and the Heads of Medicines Agencies (HMA) published this guidance under the ACT EU initiative. 

It aims to strengthen the link between scientific advice offered by regulators and innovative clinical research.

They published the guidance in May 2022.

For more information, see: 

Clinical Trials Regulation and the Clinical Trials Information System

The Clinical Trials Regulation (Regulation (EU) No 536/2014) entered into application on 31 January 2022, repealing the Clinical Trials Directive (EC) No. 2001/20/EC

The Regulation is part of a broad initiative to transform the EU / EEA clinical trials environment in support of large clinical trials in multiple European countries, to the benefit of medical innovation and patients.

For more information, see Clinical Trials Regulation.

During a transition period until 31 January 2023, sponsors can choose whether to apply to start a clinical trial under the Clinical Trials Regulation or the Clinical Trials Directive. 

For more information, see Transition period for clinical trial sponsors.

Clinical Trials Information System

The Clinical Trials Information System (CTIS) supports interactions between clinical trial sponsors (researchers or companies that run clinical trials and collect and analyse the data) and regulatory authorities in the EU Member States and EEA countries, throughout the lifecycle of a clinical trial.

CTIS went live with a searchable public website on 31 January 2022.

It serves to implement EU pharmaceutical law in the Clinical Trials Regulation - Regulation (EU) No 536/2014.

For more information, see: 

EudraCT database and the EU Clinical Trials Register

EMA is responsible for the development, maintenance and coordination of the European Union Drug Regulating Authorities Clinical Trials (EudraCT) database.

The EudraCT databse includes data on the following clinical trials:

A subset of this data is available through the European Union Clinical Trials Register, which EMA manages on behalf of EU Member States. Users are able to view protocol and result information regarding:

  • phase-II to phase-IV adult clinical trials where the investigator sites are in the EEA;
  • any clinical trials in children with investigator sites in the EU and any trials that form part of a PIP including those where the investigator sites are outside the EU.

Information about the results of clinical trials is shown in the European Union Clinical Trials Register - if published by the relevant sponsor on the EudraCT database. 

In line with European Commission guideline 2012/C 302/03, sponsors are required to post results of all clinical trials in EudraCT that are completed globally.

In July 2019, EMA published a letter co-signed by the European Commission and the Heads of Medicines Agencies (HMA) reminding all sponsors of their obligation to post on EudraCT the results of completed trials:

From October 2020, sponsors can also post results of clinical trials that ended prematurely, whether they were approved but never started or started but terminated early. Sponsors should provide the reasons for the premature end and any partial results, if available.

For more information, see the EudraCT database website. 

The Agency has a separate policy on the publication of clinical reports submitted by pharmaceutical companies to support their regulatory applications for human medicines.

For more information, see Clinical data publication.

Clinical trials conducted in countries outside the EU

Clinical trials conducted outside the EU but submitted in an application for marketing authorisation in the EU have to follow the principles which are equivalent to the provisions of the relevant EU legislation. 

For more information, see Clinical Trials Regulation.

In addition, sponsors are required to submit information on all clinical trials in EudraCT if they are:

  • conducted outside of the EU / EEA and part of an agreed paediatric investigation plan (PIP);
  • in scope of Article 46 of the Paediatric Regulation (Regulation (EC) No 1901/2006).

For more information, see EudraCT database and EU Clinical Trial Register.

In 2012, the Agency published the final version of this paper:

This paper aims to strengthen existing processes to provide assurance that clinical trials meet the required ethical and GCP standards, no matter where in the world they have been conducted.

The number of clinical trials and clinical trial subjects outside Western Europe and North America has been increasing for a number of years. More information is available in this document:

Clinical Trials Coordination Group

The Clinical Trials Coordination Group (CTCG) is a working group of the Heads of Medicines Agencies.

The CTCG aims to help make the EU / EEA more attractive for clinical trials activities.

It is responsible for harmonising and optimising the regulatory environment while assuring the protection of rights, safety and wellbeing of clinical trial participants and ensuring the generation of robust data.

The group is composed of experts in the classification, assessment and oversight of clinical trials from national agencies.

The Clinical Trials Coordination Group replaced the Clinical Trials Facilitation and Coordination Group. 

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