Better safety for patients through EU-funded research

4 April 2016

Findings from PROTECT are improving benefit-risk monitoring of medicines

The results of the Pharmacoepidemiological Research on Outcomes of Therapeutics by a European Consortium (PROTECT) project, that were published in a series of different medical journals, are making a difference in the assessment of benefits and risks of medicines available for patients in Europe. The goal of this project was to develop innovative methods to improve and strengthen the monitoring of the benefits and risks of medicines marketed in the European Union. PROTECT concentrated on improving early and proactive signal detection, establishing a framework for pharmacoepidemiology studies, developing methods for continuous benefit-risk monitoring of medicines and enhancing data collection directly from patients.

Some of the key findings of PROTECT have now been published in medical journals.

"PROTECT has made a significant contribution to the body of scientific knowledge of benefit-risk monitoring methodologies", said Peter Arlett, Head of the European Medicines Agency's (EMA) Pharmacovigilance department. "Results are being implemented into routine pharmacovigilance and regulatory practice. They have already started to improve day-to-day medicines monitoring operations of regulators and pharmaceutical companies, for better safety of European patients".

The main results and recommendations include:

• A guidance for observational studies on medicines in several databases and several countries with common protocols; this guidance will support the use of real world evidence for regulatory purposes by increasing consistency in findings from safety studies and revealing causes of differential drug effects, and will lead to updates to the methods guide of the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP); details of this research can be found in a series of 16 articles published in a special issue of the journal Pharmacoepidemiology and Drug Safety;
• A comprehensive review of good detection practices has identified significant improvements tosignal detection methods applied by national and international regulatory agencies and in pharmaceutical companies; this guidance was used to update methods for signal detection from EudraVigilance and will be integrated in revised regulatory guidance on signal management in 2016. Details about this review are published in an article in the journal Drug Safety;
• Recommendations for benefit-risk assessment methodologies and visual representations based on real-world case examples to facilitate clear and transparent decision-making; this has already led to initiatives that explore practical application of harmonised methods and the involvement of patients and the wider public in the assessment of benefits and risks of medicines. More details are available in an article in the journal Pharmacoepidemiology and Drug Safety;
• Exploring new methods to collect data directly from patients, including via the internet; this research included the collection of information from pregnant women via the web to better understand the safety of medicines during pregnancy. This project is described in an article in the journal JMIR Public Health and Surveillance.

A PROTECT: key results and recommendations of these publications is provided.

PROTECT has generated a significant amount of scientific research across the European Union. The project is behind a total of 72 original articles in peer-reviewed scientific journals, of which 26 were co-authored by EMA staff.In addition, projects from PROTECT were the subject of 14 doctoral theses and 3 master theses carried out in universities across the EU. Reports, publications, presentations and databases generated by PROTECT are available on the PROTECT website and a specific PROTECT benefit-risk website on case studies and recommendations for benefit-risk assessment. Further publications are anticipated including on overview of the impact of the PROTECT project on patient protection and life-cycle management of medicines.

PROTECT was funded by the Innovative Medicines Initiative joint undertaking. The 34-strong multinational consortium of academics, regulators and pharmaceutical companies was coordinated by EMA and GlaxoSmithKline from September 2009 until June 2015.