Executive Director Bayr Pharmaceuticals Inc Whippany, New Jersey, United States
Background: Real-World Evidence (RWE) and Real-World Data (RWD) have become increasingly important in the drug development of oncology early-stage products (ESP). RWD refers to data collected outside of traditional clinical trials, including electronic health records, insurance claims data, patient registries, digital health solutions and patient-reported outcomes. The drug development community has shown interest in the use of RWE and RWD for ESP, particularly in pharmacovigilance.
Objectives: To review the scientific literature and summarize the existing evidence on the use of RWE and RWD in pharmacovigilance for the development of oncology ESP.
Methods: A literature search was conducted from Jan 1, 2014, to Dec 31, 2024, to identify scientific publications related RWE/RWD and pharmacovigilance data for ESP. PubMed, Embase, Scopus, and Web of Science were used using the following key words: pharmacovigilance, safety, surveillance, patient safety, adverse events, benefit risk balance AND clinical trial, drug life cycle, drug safety monitoring, drug development, early development, phase I-IIb CTs, FinH studies AND use of RWE, RWD, big data AND oncology, cancer. Publications using RWE/RWD for safety of oncology ESP were included. Non-English publications and Product-specific publications, case reports, case series, post-marketing or other studies focused on specific adverse events were excluded. The initial list of references was complemented with manual review of references of relevant publications.
Results: The search identified 23 publications, 8 were excluded: 7 because were not related to oncology, and 1 focused on radiotherapy. Of the 15 remaining publications,7 articles were focused on signal detection, 4 on characterization of risks including 1 on risk factors, 1 on benefit/risk assessment, 2 on AE management and 1 compared the safety profile across same class products. For signal detection, FAERS and Vigibase were the most frequently used adverse event reporting systems. An analysis of risk factors identified that male, 65 years of age or older and skin cancer are more likely associated with bullous pemphigoid among immune checkpoint inhibitor users. Immune-related gastrointestinal toxicities were identified using RWD and the treatment courses and management were evaluated with significant impact of early gastroenterology consultation and early CT/endoscopy diagnosis on clinical outcomes.
Conclusions: RWE/RWD can complement CT data by providing insights into safety signal identification and assessment, characterization and management of risks. It can contribute to a more comprehensive understanding of a drug's performance in real-world settings, hence supporting better decision-making throughout the drug development lifecycle.
