Avid Bioservices Inc (CDMO)

[north40000]

http://www.raps.org/Regulatory-Focus/News/2017/09/20/28500/FDAs-Woodcock-The-Clinical-Trials-System-is-Broken/

...The clinical trials system is "broken" and there needs to be new ways to collect and utilize patient data, Janet Woodcock, director of FDA's Center for Drug Evaluation and Research, told a workshop on real world evidence (RWE) at the National Academies of Sciences, Engineering, and Medicine on Wednesday.

The comment came at the end of Woodcock's talk in which she also noted that use of master protocols (ie. protocols for trials that look at multiple therapies in a single disease or a single treatment in multiple diseases) and the development of new clinical trial networks "need to be the future."

Both the 21st Century Cures Act and the new user fee laws will expand FDA's use of RWE in certain circumstances, as FDA Commissioner Scott Gottlieb outlined Tuesday, though Woodcock noted there has been "very little historical use of real world experience in drug regulatory decisions about effectiveness."

The use of RWE in determining effectiveness is "obviously most important for incentives" for industry, she said, though she also cautioned that RWE works when there's a "big effect" but it's a lot more difficult in uncovering smaller effects because "so many biases are introduced."

As far as situations in which drug developers might be able to use RWE, Woodcock noted similarities with the medical device industry, and singled out the development of biomarkers, expanded indications (she offered the example of Vertex's cystic fibrosis drug Kalydeco (ivacaftor)) and possibly evaluating an investigational drug in a "hybrid model" that can use some RWE with randomization.

"Let's make generating this evidence a lot easier and randomizing within the care system as much as we can," Woodcock said in the Q&A portion of the workshop.