measures for secondary endpoints , patient subpopulations of interest . So , in a nutshell , RWE is useful before , during and after RCTs are implemented .
RWE for regulatory purposes From a regulatory perspective , the use of RWE has also expanded from its traditional pharmacovigilance role . Patients and health practitioners of therapeutic areas with high unmet needs are understandably demanding fast access to new , innovative drugs that could potentially be a life-saving opportunity for them . 2 This has increased the challenge faced by regulators who must weigh up the risk-benefit of a new drug while also facing these external pressures being exerted by patients and health practitioners . To this end , RWE has acted as an accelerator to providing faster access to innovative new therapies . 2 Regulatory agencies have created facilitated regulatory pathways such as adaptive licencing , accelerated approval and breakthrough designation status . 2 , 5 These pathways have allowed approval of new products in areas of high unmet need where a higher degree of uncertainty is accepted in terms of risk at the time of approval . This approval is given in exchange for collection of extensive post-authorisation data on product safety – and in some cases even on efficacy – to validate their early access / approval decision . Capturing evidence from real-world populations via electronic health records ( EHRs ) as well as patient registries has made such accelerated regulatory pathways possible and will undoubtedly become more common as further innovative products are developed . used are determined by the protocol , whereas in the real life , such decisions are freely made and can vary a lot . It is more relevant to conduct real-world observational studies to capture realistic data on economic outcomes . Another , more traditional question which is properly addressed by RWE is the burden of illness : what are the various healthcare costs and non-healthcare costs borne by patients , caregivers and employers ( for example , workdays lost , productivity losses )? This knowledge helps understanding of where a new intervention has impacts . These cost of illness or burden of illness studies are RWE . With such studies , RWE can allow us to learn a lot on how a disease is managed and what its diverse consequences are on various stakeholders . This may be very useful when designing the clinical studies plan for a new pharmaceutical , because it can inform aspects such as patient characteristics , relevant outcomes
RWE quality considerations RWE can be generated from RWD of a broad range of sources , but for data to be effectively used in RWE , it must be of appropriate quality . 6 It must be ensured the RWD is appropriate to address the objectives of the RWE study while also ensuring it is available and accessible for the timeframe of the study . Strengths and limitations are associated with different data sources and an awareness of such limitations is important for anyone wanting to utilise RWD to generate RWE . Patient registries are seen by many as one of the most reliable sources for RWD for pharmaceuticals post commercialisation . 7 However , various limitations may still exist such as a lack of standardised data collection methods across hospitals / regions and the lack of a comparator for product specific registries . 7 One initiative to improve the quality of RWD sources is the cross-border PAtient REgistries iNiTiative ( PARENT ) – a joint EU and Member State project aimed at improving quality , reliability and cross-border usability of EU-patient registry data . 7 Its objective is to harmonise practice and issue methodological guidelines to improve interoperability . With RWE becoming more and more important , it is likely that we will see many similar initiatives in the future .
How regulatory and HTA agencies are adapting to RWE Regulatory Both the European Medicines Agency ( EMA ) and the US Food and Drug Administration ( FDA ) have made considerable efforts to incorporate RWE into their regulatory decisions . As already outlined , new regulatory pathways utilising RWE have been implemented to allow faster access to new medicines . In Europe , the EMA has introduced both EMA adaptive pathways and EMA conditional
hospitalpharmacyeurope . com | 2021 | 5