Background Real-world data (RWD) and real-world evidence (RWE) has long been used in drug development programs: to help generate hypotheses and assess the feasibility of clinical trials, or for post-marketing surveillance purposes. There is a wealth of RWD sources; data can be gathered from medical records, registries and wearables, among others. This data is then analysed to obtain RWE. Today, RWD/RWE is also increasingly being used to support regulatory decisions, especially during market authorisation approval for new drugs or new indications. A number of immuno-oncology drug products have owed their approval to the use of RWD/RWE.
Methods The objective of this experiment is to identify and trace the regulatory history of selected immuno-oncology products to understand how RWD/RWE has been applied in their drug development programs to market, and the perspective of the regulators at the time of review. Using Cedience, we identify the RWD/RWE-generating trials used in an immuno-oncology drug’s submission. Cedience is a next-generation regulatory intelligence platform that allows a user to answer regulatory precedent questions, monitor topics of interest, and analyse past development strategies by using a database of documents compiled from various regulatory agencies. We have previously used Cedience to identify drug products associated with RWD/RWE and reconstruct a timeline of said drug products by collecting correspondence letters from the FDA.
Results Using Cedience, case studies were built that show the timeline of a drug’s regulatory history; a picture of the drug’s journey through development. This timeline focuses on RWD/RWE-generating trials used to examine the immuno-oncology drug, as well as the evidence available prior to said trials. In the context of these regulatory submissions, RWD/RWE functions to support randomised clinical trials by providing a second source of data that is more generaliseable to the wider population.
Conclusions From this data, an analysis of how RWD/RWE was used in the real world could be seen: what percentage of FDA approvals are RWD/RWE-backed, the clinical evidence that can be generated from RWD/RWE, and what type of study designs are appropriate for the generation of RWD/RWE. The regulator’s perspective could be seen as well: how they evaluate RWD/RWE, and whether any issues or concerns are raised.
Statistics from Altmetric.com
If you wish to reuse any or all of this article please use the link below which will take you to the Copyright Clearance Center’s RightsLink service. You will be able to get a quick price and instant permission to reuse the content in many different ways.