Focus Group Interview of Stakeholders to Derive Checklists for Future Real-world Data and Real-world Evidence Use in Regulatory Decision Making for Drugs

Abstract

The limitations of randomized clinical trials (RCTs) have been highlighted in regulatory decision-making. Thus, the requirement for real-world data (RWD) and real-world evidence (RWE) is growing. However, the definition of RWD is ambiguous in domestic laws and regulations, and drug safety management systems that use RWD are insufficient. We conducted focus group interviews (FGIs) regarding the definition of RWD and the approaches national institutions or industries must adopt to introduce RWD/RWE in regulatory decisions for the entire drug life cycle. Experts from academia, industry, government, and medical fields who have experience with RWD/RWE research were requested to participate in FGIs by email. The participants were classified into three groups (academia, industry, and government and medical professionals). Online discussions were conducted for each group. A total of 16 stakeholders were recruited for the FGIs: five from academia, seven from industry, three from government agencies, and one from the medical field. Most participants agreed with the opinion that RWD has not been collected through traditional clinical trials and should be defined comprehensively. The experts also suggested that national institutions and industries should prepare guidelines and infrastructure regarding RWD collection, RWE generation, and RWD/RWE management. In conclusion, national institutions and industries should set short- and long-term goals to introduce RWD/RWE in regulatory decision-making.