Building trust in real world evidence (RWE): moving transparency in RWE towards the randomized controlled trial standard

Abstract

The increasing use of RWE in regulatory and reimbursement decision-making indicates the significant progress that has been made in building trust in RWE through greater transparency. This review of the published literature and key online sources was conducted to provide an update on progress towards greater transparency in RWE, based on four key barriers to trust identified in a 2016 paper and applying learnings from transparency initiatives established for RCTs, such as the US FDA Amendments Act (FDAAA) 2007 Final Rule. Multiple initiatives from the US FDA, EMA and organizations such as the ISPOR-ISPE Joint Task Force have yielded new guidance documents and tools that enable greater transparency in RWE study methodology (STaRT-RWE and HARPER templates), data source selection and quality (SPIFD2 framework, REQueST tool), strategy (the Center for Open Science RWE Study Registry), and will therefore improve transparency in RWE study reporting. Programs such as the REPEAT Initiative and RWE DUPLICATE are investigating reproducibility of RWE studies and improving understanding of the circumstances when valid inference on treatment effects can be obtained from RWE studies. Further work is needed to embed and to implement the new tools and guidance that are available, and thus raise standards for RWE transparency towards the levels expected for RCTs.