Thursday, July 28, 2016

FDA Issues Two Guidances on Real World Evidence; NEJM Article

Better use of pragmatic and real world evidence is critical for health care delivery.   While the gold standard for effectiveness is held to be a tightly controlled randomized trial, what really happens when patients are not so carefully screened, or when drug compliance is not monitored daily or weekly?   What would have happened to the data if the control arm was ordinary care with all its flaws and not an IRB-approved, carefully monitored standard of care at an academic center?

Reform efforts like 21st Century Cures legislation (drafted; not enacted) contain provisions about adaptive trials and real-world evidence.  FDA is taking strides in this area with two new guidances released in July 2016, one on "Adaptive Designs for Medical Device Clinical Studies" and one on "Use of Real World Evidence to Support Regulatory Decision Making for Medical Devices."

See the Federal Register announcements here (for Adaptive Designs) and here (for RWE).   See the FDA guidance documents here (for Adaptive Designs) and here (for RWE).  Comments through October 27.

Trade journal article here.  For publications on RWE at Pubmed, here.

For discussion of a June 2016 paper by the Center for Bipartisan Policy on RWE by Mark McClellan, Patrick Soon-Shiong, and others, here.

For an August 2016 NEJM article on Pragmatic Trials by Ford & Norrie, here.