FDA Seeks More Informative Clinical Research

There is a critical need to rethink standards of evidence and of the reliability of information used to make regulatory decisions, which involves placing greater reliance on data from sources outside traditional clinical studies, according to FDA officials and industry leaders. While the randomized, prospective placebo controlled trial remains at the “top of the pyramid” for reliability of evidence, new tools for collecting data and methodologies for evaluating different forms of information requires FDA to adapt to this new information matrix, said FDA commissioner Scott Gottlieb in keynote remarks at a recent workshop on the Impact of Real World Evidence on Medical Product Development at the National Academy of Sciences, Engineering, and Medicine (NASEM).

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Janet Woodcock, director of the Center for Drug Evaluation and Research (CDER), was more pointed in describing the nation’s clinical trial system as “broken” and in need of significant change to generate the evidence needed to support better medical decisions. Without new clinical trial platforms able to generate needed evidence efficiently and cost-effectively, she said at the NASEM workshop, the current system will “continue to be a barrier to innovation and to quality of care around the world.”

The current explosion in science is bolstering the drug development pipeline, added former FDA commissioner Robert Califf, but noted that sponsors are pulling projects off the shelf because they can’t afford to do the costly development work. Califf said that the traditional system for evidence generation has become “bloated and burdened” and is “not generating the evidence that is needed.” New methods for generating evidence from clinical practice does not exclude randomization, he pointed out, but it should focus on the most critical factors to study and what information really needs to be generated.

Reforming the current clinical research system also may involve re-writing the seminal guidance on good clinical practices (GCPs) developed by the International Council on Harmonisation (ICH). That organization is considering a “reflection paper” on revising GCPs, but workshop participants suggested that a whole new approach to regulating clinical research may be in order.

While this recent NASEM workshop focused on efforts to make greater use of real world evidence (RWE) from patient health care records to broaden the sources of data available to inform medical product development, FDA and sponsors also are exploring other approaches, such as pragmatic clinical trials, master protocols, and clinical trial networks able to assess multiple products on a range of patients. RWE won’t replace traditional clinical trial data in many cases, said Gottlieb. But he sees more opportunity in the pre- and post-market context to use this data to improve medical product development. FDA will collaborate with all stakeholders, he said, to achieve a “more appropriate adoption of RWE as part of the entire life cycle of medical products.”