Letter from the editors

In December of 2018, the FDA released a new strategic framework to advance the use of real-world evidence (RWE) in regulatory decision making, as required under the 21st Century Cures Act. The framework serves as the roadmap for more fully incorporating RWE into drug development and regulatory life cycles to improve the efficiency of clinical research and provide answers to questions that were insufficiently answered throughout clinical trials. In our latest issue of FOCUS, we explore the capabilities of RWE and how its correct application in drug development can increase the value of care in today’s healthcare marketplace. Key articles include:

• The 21st Century FDA: Advancing Patient Care Through Real-World Evidence – The development of the FDA’s “Real World Evidence Program,” opens many avenues for using real-world data (RWD) to generate RWE for demonstrating both product safety and clinical and economical effectiveness to support regulatory decisions of new drug products. Dr. Eli Phillips Jr, Dr. Kevin Lord and Bela Bapat explore how the appropriate application of RWD and digital health technologies can provide value in the drug development lifecycle.
• Bringing Clinical Trial Rigor to Cancer Treatment Data Collected in the Real World – Drug developers and other industry stakeholders are still in the formative stages when it comes to developing reliable models for collecting RWD that is accurate, consistent and timely enough to be useful to the FDA’s review process. Dr. Bruce Feinberg and Dr. Andrew Klink produced a new, more reliable method for collecting RWD. We sit down with Dr. Klink to understand how this new method more closely aligns with the gold standard of patient response measurement used in clinical trials.
• Validity in Real-World Data – A Case Study in Chart Abstraction Research – Stakeholders believe that RWD can potentially support pharmaceutical product label approvals and label expansions, but its adoption is mired in methodological challenges requiring rigorous solutions. Dr. Jonathan Kish discusses two fundamental challenges, data quality and internal validity, and how Cardinal Health researchers are pursuing novel strategies to improve upon methods to achieve high levels of both.
• Challenges in Oncology PRO Research – Given the renewed importance that patients, providers, payers, and policymakers are placing on outcomes beyond survival, the study of patient-reported outcomes (PROs) in oncology continues to expand. Dr. Jonathan Kish discusses how to overcome institutional operational inertia in healthcare, as real-world PRO research in oncology poses unique challenges that require novel solutions.