DIA Europe 2024

Advanced cancers represent an area of high unmet medical need and active drug development. Given the pace of innovation, new cancer drug applications often pose a dilemma between the need to satisfy evidentiary requirements and speedy access to innovative agents. The current regulatory framework allows for early approval of products when comprehensive clinical information is not available subject to further confirmation. Currently, the experience with conditional approvals is limited, except in oncology where it has been used for 55 new drug approvals to March 2022. Regulatory decisions are complex due to different unmet medical needs, uncertainties affecting the evidence submitted, ranging from the design of trials, the choice of endpoint, and various limitations in terms of internal and external validity, and different risk attitudes. From a sponsor’s perspective, there is an important decision to be made about the timing of submission in relationship to the available data and the conduct of confirmatory studies. An added element is the amount of supportive data (indirect comparisons) and the nature of the confirmatory studies (randomized controlled trials or indirect comparison using real-world data). In this session, we present the results of a recent survey with European regulators and discuss factors that influence acceptability. Furthermore, we discuss possible optimal submission strategies to address the challenges of approval when comprehensive clinical evidence is lacking.