On 23 March 2017, the European Medicines Agency (EMA), together with the Heads of Medicines Agencies (HMA), established a new task force “to explore how medicines regulators in the EEA can use big data to support research, innovation and robust medicines development” in order to improve human and animal health. The task force is composed of experienced staff from several medicines agencies in the EEA and will be co-chaired by the Danish Medicines Agency and the EMA.

The decision to establish a joint task force was taken following an EMA workshop on big data in November 2016, which EMA’s Executive Director Guido Rasi concluded with the words “we can, and we must, seize the opportunities for public health coming from big data.” According to the EMA, big data – the collection of extremely large sets of information requiring specialised computational tools to enable their analysis and exploitation – and especially real word evidence (e.g. electronic health records, hospital records, health insurance data) have the potential to “significantly contribute to the way the benefits and risks of medicines are assessed over their entire lifecycle.”

While the opportunities of big data are significant (e.g., in drug discovery, promising compounds for screening can be selected in silico; in clinical trials, epidemiological information can inform trial designs in terms of defining outcome measures and site selection for optimal recruitment, in the post-marketing stage, real word data can be used continually assess the benefit-risk balance in the wider population), the EMA also recognises that there are “significant challenges in the exploitation of these data.” Big data is often unstructured, noisy and inaccessible, and there may be missing data, and known or unknown biases. It can be difficult to create trust in results and to establish causality and rule out coincidence, factors that are essential for evidence in medicines regulation. During the workshop, it was agreed that implementing privacy, security, ethics and transparency measures is essential to maintain the trust of patients and the public.

In the next 18 months, the EMA/HMA big data task force is expected to design a big data “roadmap” and issue recommendations on necessary changes to legislation, regulatory guidelines or data security provisions. To that end, the task force will map the relevant sources of big data, identify the potential applicability thereof, and explore existing regulatory expertise and competences. The task force plans to collaborate with the US Food and Drug Administration (FDA), Health Canada and other third country stakeholders to gather their insights on the topic as well.

At the European Commission level, several big data initiatives have been taken as well, including the eHealth action plan and Horizon 2020 funded research (four projects: MIDAS, Evotion, BigO, PULSE) as well as public-private projects (Big Data Value Association and IMI). In December 2016, the European Commission published the results of a study on big data in public health, telemedicine and healthcare. The final report concludes that one of the most important lessons of the study is that awareness raising regarding the added value of big data in health is needed quite urgently, and establishes ten recommendations (e.g. clarify and align existing legal and privacy regulation of big data in health) that aim to maximise the opportunities big data can bring to public health in the European Union.