8.H. Workshop: Policy and practice: the role Real-World Data (RWD) in Health Technology Assessment

Abstract   The European Medicines Agency (EMA) has developed multiple schemes to improve access to medicines. Among them, the Conditional Marketing Authorisation (CMA), namely an approval based on less complete data followed by post-authorisation obligations, can be granted to medicines addressing unmet medical needs, debilitating or life-threatening diseases, rare diseases, or public health threats. In the post-authorisation obligations, real world data (RWD) routinely collected from different sources, such as electronic health records, administrative claims, or product and disease registries, are used for Real World Evidence (RWE) generation. In the context of the COVID-19 pandemic, this effort took the form of establishing an infrastructure and planning for the generation and synthesis of said data. Over the past few years, frameworks have emerged, and the European Union's new Pharmaceutical Strategy, adopted on November 2020, highlighted the need to systematically examine and transform current policies and practices to allow for the use of RWD/RWE to support the development, authorisation and use of medicines. Indeed, regulatory and Health Technology Assessment (HTA) bodies are opening the doors to the use of RWD/RWE in their evaluation frameworks. Nevertheless, currently there is a lack of established infrastructures to facilitate robust EU-wide data collection and transfer. Furthermore, there is a fragmented EU-wide landscape of heterogeneous policies on the acceptance and the appraisal of RWD/RWE. Given the COVID-19 pandemic, we also need to consider comparative effectiveness research and population information. This critically necessitates collaboration and mechanisms of information exchange supported by well-established research infrastructures. Additionally, several challenges need to be overcome, from methodological ones (quality and transferability of data) to governance issues and legislative challenges, including GDRP considerations in terms of data processing and transfer. During the 2019 HTA international (HTAi) Global Policy Forum, a call for actions and guidance to manage RWD/RWE to inform decision-making across the whole lifecycle of health technologies was launched. In fact, there is an urgent need for regulators and payers to make the best use of RWD/RWE also to allow a timely access to innovative health technologies and to reassess them across the lifecycle. This workshop is intended to carry forward the discussion on policies and practices on the use of RWD/RWE by regulatory and HTA bodies. Key messages Real world data (RWD) routinely collected from different sources and within different countries can be employed for rapid policy relevant research responses. HTA/Payer should agree on how collect and use data in the assessment and reassessment process of the value of health technologies.