All Times EDT
Keywords: RWE, RWD, effectiveness, regulatory submission
Real world data is increasingly playing an important role in regulatory decisions as technology to abstract meaningful clinical information from EHR develops. FDA has a long history of using RWD to evaluate safety in the post-marketing setting. However, the 21st Century Cures Act, passed in 2016, places additional focus on the use of these types of data to support regulatory decision making, including approval of new indications for approved drugs. Three RWE related guidance are expected this year and this might accelerate the acceptance of RWD in novel settings. Though there have been a lot of developments in addressing the technical and methodological challenges in collecting and analyzing RWD, there is a lower acceptability of RWD for efficacy/effectiveness outcomes. This is because of concerns around bias and confounding and randomized clinical trials (RCT) remain the best available standard for assessing efficacy. With the advent of advanced therapies like CAR T-cell therapy and personalized medication becoming a reality, there are an increasing number of products for which traditional drug development pathways do not work. Often for these advanced therapies or orphan products for conditions with significant unmet need, a traditional randomized clinical trial may be unfeasible or unethical. Regulatory acceptability of RWD/RWE not only depends on approaches to abstract data and methodology to draw reliable inference but also on technological aspects associated with the data, and require solutions addressing differences in data formats, quality, and content that exist across different RWD sources.
This round table will focus on: -Analytic techniques to generate RWE with focus on effectiveness outcomes to enhance clinical research and support regulatory decision in a variety of use cases. -Approaches to address technical challenges involved in generating regulatory grade evidence such as standardizing and validating data.