Workshop: Real-world data: are we ready to take up the challenge?

Abstract Evidence provided by traditional clinical research often fails to answer patients’, physicians’ and healthcare decision-makers’ questions about real-world practice and outcomes; in fact, real-world effectiveness may considerably differ from efficacy assessed in clinical trial settings. The rise of interest in real-world data (RWD) - data routinely collected outside a controlled research environment - is driven by the increasing need for evidence in specific populations, such as comorbid or multi-treated people. RWD can also allow investigation of unanticipated, uncommon or long-term outcomes. In addition, RWD may represent the only source of information in some fields of special interest, e.g. rare diseases. Furthermore, current conditional reimbursement systems of drugs require dynamic evaluations of cost-effectiveness that have necessarily to take RWD into account. Indeed, RWD play a relevant role also in Health Technology Assessment (HTA) projects that should release the most up-to-date evidence and should be updated once new data are available. Nonetheless, common concerns about evidence derived from RWD include uncertainty about data quality, high possibility of bias, difficulties in managing a vast amount of data coming from different sources as well as legal and privacy requirements, in particular in the light of the new General Data Protection Regulation. However, HTA cannot exempt using RWD as it is entrusted to assess short- and long-term consequences of the application of health technologies, in terms of both health outcomes and costs. This workshop is an opportunity to discuss current and future challenges in the use of RWD in HTA and concerns preventing researchers from exploiting RWD full potential. Key messages Real-world evidence and randomized control trial data are considered mutually complementary in generating evidence and supporting decision-making. Real-world data have proved to be crucial as instrument to gather data in HTA and to support decision-making.