FDA's Woodcock: The Clinical Trials System is 'Broken'

Regulatory NewsRegulatory News | 20 September 2017 |  By 

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.

Draft guidance on RWE and a new framework on its use are both expected to be released by FDA before 2021, though Woodcock said she could not speculate on when exactly the guidance would be ready, noting there's a direct correlation between delays in a guidance's release and interest in a guidance.

Rory Collins, head of the Nuffield Department of Population Health at the University of Oxford, also told participants that just reforming the International Council on Harmonisation's (ICH) guidance on good clinical practices (GCP) (ICH released a reflection paper on GCP last January) might not be enough, as the document might need to be re-written completely or just abandoned.

However, Woodcock countered that at its heart, regulation is often just aimed at the bottom 1% of companies, which is a problem, though the ICH GCP guidance is necessary.

"You wouldn't believe what some will do – there are what I call bottom feeders out there that will astonish anyone. We have to have some kind of structure to guard against that," she added.


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