RWD：不仅仅是另一个首字母缩略词

Report on the experience gainedwith regulator-led studies fromSeptember 2021 to February 2023 Executive Summary Overseen by theBig Data Steering Group(BDSG), EMAand the European Medicines Regulatory Network(EMRN) are working toestablish a sustainableframework to enable the use and establish the value ofreal-world evidence (RWE) across different regulatoryuse cases (Arlett et al., 2022). While the majority of RWE for regulatory use is generated by pharmaceuticalcompanies, the addedvalue ofregulator-led studies has beendemonstrated over thelast few yearsby the use ofreal-world data (RWD)tomonitorthe safety andeffectiveness of COVID-19 medicines. Nevertheless, further work is needed to betterintegrate RWD/RWE into regulatory decision making alongside the evidence comingfrom more established sources (notably randomised controlled trials)1. In 2021, EMA completed apilot with the Pharmacovigilance Risk AssessmentCommittee (PRAC)to test the Agency’s ability to conduct rapid analyses in a numberof databases containing electronic health records in order to address knowledge gapsarising in the context of PRAC scientific assessments. From September 2021 onwards,the pilot activities were expanded to explore opportunities for generating RWE in orderto also support the scientific evaluations of other committees and working parties,including the Paediatric Committee (PDCO), theCommittee for Orphan MedicinalProducts (COMP),theCommittee for Advanced Therapies (CAT), as well as theCommittee for Medicinal Products for Human Use (CHMP)and itsScientific AdviceWorking Party(SAWP). While these pilot activities are still ongoingin 2023, in line with the BDSG secondmultiannualwork plan (2022-2025) this report takes stock of the experience gained todate in conducting RWD studies2aiming at addressingneeds of EU regulators as wellas external stakeholders including health technology assessment (HTA)bodies andpayers’ organisations. It focuses on studies conducted in addition to thoseperformedin response to theCOVID-19 public health emergencyandthePharmacovigilanceimpact strategy.All of the three existing pathways available to EMA for RWEgeneration were considered: (i) studies conducted in-house by a team within EMA ofpharmacoepidemiologists and data scientists usingsix databases containingmainlyprimary caremedical recordsfrom different European countries; (ii) studies conductedvia aDARWIN EU®afederated network ofdata, expertise, and servicesinitiated inFebruary 2022 which has accessto a growing list of data partners; and (iii) studiescommissioned to one of eight research organisations and consortia via theAgency’sresearchframework contracts. The report covers the time period from the receipt of the first pilot study requests(September 2021) up to the end of year 1 of DARWIN EU® (7 February 2022). Duringthis period, a total of 61RWD research topics were identified (in addition to researchquestions relatedto theCOVID-19 public health emergencyand thePharmacovigilanceimpact strategy). This includes 44 (72%) study requests received from members ofEMA’s scientific Committees, SAWP, national competentauthorities (NCAs), or externalstakeholders or EMA functions. The remaining 17 studies (28%) were offered toRapporteurs and lead member states mostly as a result of proactive screening by EMAof certain new/upcoming regulatory procedures (e.g. signals and referrals). For more than half (36) of these topics, a study was considered feasible in terms of theavailability of relevant data and the timeframe, and, while six study offers from EMAwere refused by the Rapporteur/lead-member states as considered to be of limitedvalue, a total of 30studies proceeded to execution. Most of these studies weredescriptive cohort studies and some were comparative with more complex analysesperformed. The studies primarily addressed research needs of thePRAC,PDCO,COMP,and SAWP in the context of safety signals, periodic safety update report singleassessments, applications for paediatric investigation plans and waivers, (maintenanceof) orphan designations as well as scientific advice. There was a broad range of studytypes including safety studies,drug utilisationstudies, disease epidemiology studies,andstudies to inform thedesign and feasibility of clinical trials and clinicalmanagement. By the end of the reporting period, 27 studies had been completed and three wereongoing. Reflecting the status of the three pathways during the review period, most ofthese studies were conducted via the in-house pathway (24 completed, 1 ongoing) in arapid manner with the majority of study reports (19/24, 79%) being deliveredwithinless than 90 days from receipt of the initial request.Most of these studies used datafrom Germany, France and UK. Noting that the first DARWIN EU® data partners were onboarded only in the Autumnof 2022, an additional four studies were conducted via DARWIN EU® (in line with thepre-defined capacity for studies in year 1) and one study was commissioned to