(C03) Contemporary pharmacoepidemiology practice for real world data source identification and feasibility assessment – Results from an ISPE-sponsored online survey
Director PEPI Consultancy Limited Eastleigh, United Kingdom
Background: There has been rapid growth in the variety and number of RWD sources, and new or draft regulatory documents that provide frameworks for assessing the suitability of RWD sources. This current research forms part of an ISPE-funded initiative to publish practical guidance for RWD identification and feasibility assessment with a focus on regulatory purposes among specific geographic regions.
Objectives: 1. Identify key criteria and map across existing RWD guidance documents; and 2. assess variability in current practices for identifying and assessing feasibility of RWD sources for pharmacoepidemiology studies among the ISPE community.
Methods: An expert multi-stakeholder working group, covering multiple research settings and regions, outlined knowledge gaps and key criteria for RWD feasibility assessment. Desk research was carried out to appraise relevant regulatory guidance documents across the US, EU, and APAC regions. Content from each document was mapped to criteria identified using expert input for consensus. Subsequently, an online survey was designed to describe background of RWD researchers, current and common practices, and potential strengths and limitations of RWD identification sources and existing guidance documents. Invitations to complete the online survey were sent to all ISPE members.
Results: In total, 11 RWD guidance documents were identified and mapped against 14 RWD assessment criteria within 3 broader domains (design features, data quality, data accessibility) to demonstrate variability in the existing guidance to assess RWD suitability. Between December 2022 and January 2023, 37 respondents with Biopharma (15, 41%), Service Provider (10, 27%), and/or Academic affiliations (6, 16%) participated in the survey. Majority had used RWD within the EU (29, 78%) and/or US (26, 70%). Most used RWD for post-marketing commitments (34, 92%) and/or background epidemiology (28, 76%). RWD were identified through literature (33,89%), data landscaping (26, 70%), data networks (21, 57%), and/or Expert consultation (20, 54%). For data source feasibility assessment, guidance documents referenced included: FDA (20, 54%), ENCEPP v10 (17, 46%), EMA (16, 43%), SPIFD [Gatto et al.] (11, 30%) and/or NICE (8, 22%). Strengths of RWD identification sources included broad coverage of sources, while limitations focused on the lack of specificity and not being up-to-date. Challenges for conducting feasibility assessments included RWD accessibility, ability to complete validation, and custodian responsiveness.
Conclusions: Current practices show guidelines are used extensively, but key criteria were not reflected consistently. Challenges remain in RWD identification and feasibility assessment, validating the broader aim of this initiative.
