The current edition of the newsletter features real-world data (RWD) and real-world evidence (RWE) in addition to sections on FDA approvals, recent ODAC meetings, Biostat Bytes and WG updates. RWD is observational data and includes electronic health records (EHRs), medical claims data, data from product or disease registries, and data gathered from other sources (such as digital health technologies) that can inform on health status [1]. The popularity of RWD use for RWE has grown due to advancements in data sharing and cloud computing. These advancements have enabled the industry to leverage large, complex datasets more effectively and efficiently, leading to the emergence of new data strategies and methodologies. Dr. Sajan Khosla from AstraZeneca covers details of RWE in the pharmaceutical industry in the KOL message.

Since 2017, FDA stepped up the development of RWD/RWE guidance documents and released 10 guidance documents covering data sources, study design, and regulatory submissions. These guidance documents advise that the generation and utilization of RWD and RWE meet certain standards of reliability, validity, and relevance for informing regulatory decisions. An introduction to RWD data sources and a high-level non-exhaustive summary of recent regulatory filings using RWD are included in this newsletter. One ODAC meeting on October 4 is particularly interesting about eflornithine in high-risk neuroblastoma based on a study that used external control and obtained ODAC votes 14:6 in favor of eflornithine improving event-free survival and the drug was subsequently approved in December 2023. The Biostat Bytes panel explains several statistical terms used in RWD/RWE including immortal time bias, hybrid trial, and externally controlled trial. There are multifaceted purposes of RWD to improve the statistical efficiency in trial design [2-3], trial recruitment [4], health technology assessment (HTA) and reimbursement submissions [5-6] and in generalizing research findings in subgroup representation [7]. Furthermore RWD from multiple healthcare databases can be linked together for monitoring of drug safety as in FDA’s sentinel initiative [8] and the assessment of the real-world health outcomes in European health data and evidence network (EHDEN) project [9].

Detailed description of the content includes:

You can access and download the newsletter at https://doi.org/10.5281/zenodo.10909835.