Monday, December 23, 2019

Very Brief Bog: FDA New Guidance on Clinical Trial Quality and Weight of Evidence

On December 19, 2019, the FDA issued a new 21-page draft guidance document on what constitutes "substantial evidence" of clinical effectiveness, for the purpose of drug and biological trials.   The historical high benchmark has been two "adequate, well-controlld" randomized controlled trials.  However, as long ago as 1998 guidance (here) FDA considered the circumstances of particular drugs and diseases. 

  • RAPS trade journal article here.
  • FDA press release here.
    • Extensive quotes from Deputy Director Amy Abernethy.
  • FDA web page for the new draft guidance here.
  • Actual PDF here.
  • Federal Register announcement; submit comments til February 18, 2020; here.
  • For a negative viewpoint, that the guidance and other FDA actions makes FDA too loose and liberal, see Endpoints, here.
To my eye, there are other FDA guidances say very little that's new (e.g. you get the "I could have written this" feeling.)   This one is quite interesting.  While there are many books and articles on designing sound clinical trials, this is a pretty good 21-page birds-eye summary of some of the most important in's and out's in trial design and trial interpretation.   

While the context is guidance for drugs and biologicals, many of the considerations are similar for diagnostics and devices.


Contemporary with this guidance, see a December 23 WSJ article on data-mining for real world evidence to support drug trials; here.  

Not directly related, but also this month, flurry of news articles on "hidden medical device failure reports" in the MAUDE device database - e.g.  Kaiser Health News here.  See the FDA"s home page on its SENTINEL initiative here.  For recent publication by FDA's head of devices, Jeff Shuren, here.

See a Health Affairs blog on FDA communications with drug developers here, and a Health Affairs blog on coordinating FDA and CMS decisions to encourage innovation here.

FDA held a November 7 all-day workshop on clinical trial design (separate from but related to this draft guidance document on clinical trial design appearing in December).
  • See the website on the FDA's all-day November 7 meeting between FDA and stakeholders about how FDA could improve its communications: here.  
  • Webpage includes archived streaming video of the workshop and and 20 PDF presentations.  
    • I've put all the agenda, presentations, and unofficial transcript from November 7 in one 10mb cloud zip file here.