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Abstract:
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Traditional randomized controlled trials (RCTs), conducted under carefully controlled ideal conditions, have served as the main source of clinical evidence supporting market authorization decisions of new drugs. In recent years, pragmatic clinical trials (PCT) have received increasing attention in the regulatory and industry setting to using real world evidence (RWE) for new drug approval or label expansions. While tackling from a different angle on effectiveness of interventions, i.e., benefit under routine clinical practice maximizing generalizability, PCTs have encountered challenges in design, conduct and analyses. In this roundtable session, we will discuss some of the common issues from PCT in an oncology setting including 1) How to select and define real-world trial endpoints; 2) How to leverage accuracy and completeness of data captured from the electronic health record (EHR) with low level of interference under routine clinical practice; 3) Intent-to-treat vs. per-protocol analyses; 4) Measure adherence while drug accountability is not enforced; 5) Non-inferiority study in the PCT setting.
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