Advancing regulatory science, advancing regulatory practice

In an Editorial of Science in 2011, Margaret A. Hamburg, the then Commissioner of the US Food and Drug Administration highlighted that a strong field of regulatory science is required to develop new tools, standards and approaches that efficiently and consistently assess the safety, efficacy and performance of products, and that this field has long been underappreciated and underfunded.1 During the last decade, several international initiatives were taken to translate results of scientific research into everyday regulatory practice. The US Food and Drug Administration launched the Advancing Regulatory Science Initiative to transform the way medical products are developed, evaluated and manufactured.2 The Innovative Medicines Initiative (IMI) was launched in Europe as a public–private partnership aiming to improve health by speeding up the development of, and patient access to, innovative medicines, particularly in areas where there is an unmet medical or social need.3,4 Improving and strengthening the monitoring of the benefit–risk of medicines marketed in the European Union (EU) was one of the first topics adopted by IMI in 2008. Its main goal was to enable a more rapid detection and confirmation of new adverse drug reactions (ADRs) under ‘real world’ conditions and to develop new scientifically based and tested tools for the benefit–risk assessment of marketed drugs. The European Medicines Agency (EMA) considered it should play a role in this project and established the Pharmacoepidemiological Research on Outcomes of Therapeutics by a European ConsorTium (PROTECT) group to which the project was eventually awarded in association with a consortium of pharmaceutical companies coordinated by GlaxoSmithKline. Pharmacoepidemiological Research on Outcomes of Therapeutics by a European ConsorTium was conducted from 1 September 2009 to 28 February 2015 with 33 other organisations and pharmaceutical companies.5 In terms of research outputs, PROTECT was in no doubt successful, with more than 75 original publications in peer-reviewed journals and 100 presentations in various conferences and meetings.6 Projects from PROTECT were also the subject of 14 doctoral theses and three master theses carried out in universities across the EU. But in regulatory science, the number of reports, presentations, publications or databases should not be an ultimate marker of success: the project should generate outcomes in the form of strengthened regulatory systems that ensure patient safety, enhance public health and stimulate innovation.7 This article briefly reviews four examples of PROTECT results and discusses the different impact on regulatory practice and public health the results have already had or may have in the future.