Posted 11 September 2017
By Zachary Brennan
FDA commissioner Scott Gottlieb on Monday explained to attendees of RAPS’ Regulatory Convergence conference some steps FDA is taking to make the clinical end of drug development more efficient and effective.
Opening with a discussion of the ways in which the gap of time between the discovery of the science behind new treatments and the adoption of such treatments has been shrinking, Gottlieb outlined a few of the ways in which the agency is modernizing its approach to collecting and evaluating clinical information.
And on a day when the discussion of how much it costs to develop a new oncology drug is being hotly debated with the release of a new study, Gottlieb also discussed how the costs of drug development "are also high, and growing.
"There’s been criticism of the various estimates of how much it costs to develop a new drug," he said, according to the transcript of his speech. "Moreover, on a relative basis, in many cases the costs of early stage drug development has grown at a proportionally faster rate than the cost of late stage drug development. In other words, inflation in early stage drug trials is rising faster than inflation in late stage development.
"By front-loading the cost of drug discovery, the broader biomedical community is making it harder to advance new ideas. It’s economically harder to capitalize the cost of an early stage drug program, relative to funding a later stage project. So frontloading the costs are a recipe for reducing the amount of new ideas that can be advanced."
And while highlighting the use of adaptive clinical trial designs, which "allow scientists to enrich trials for patient characteristics that correlate with benefits, or that help predict which patients are least likely to suffer a certain side effect," he also touched on the more widespread use of master protocols "to enable more coordinated ways to use the same trial structure to evaluate treatments in more than one subtype of a disease or type of patient."
He also said that with the increased use of adaptive and seamless clinical trial approaches, "informed consent used in clinical trials must be updated as the trial progresses, not only to reflect new safety data, as is already standard practice, but also to incorporate data on the evolving view of efficacy."
The agency also needs to engage in more communication between sponsors, investigators, institutional review boards and other stakeholders involved in the development program, he added.
He also touched on how FDA is better using more advanced computing tools, and more sophisticated statistical and computational methodologies, as part of the drug development and the drug review process, as, "Almost 100 percent of all new drug applications for new molecular entities have components of modeling and simulation."
Gottlieb also announced that FDA will release "at least ten new disease-specific guidance documents over the next year," and in response to a question on commercial speech and FDA regulations, he made clear that although FDA has lost recent court cases related to the First Amendment, he said, "Our regulations cannot be in conflict with the courts," noting that the agency is currently "in a period of ambiguity."
Dr. Gottlieb's speech to the Regulatory Affairs Professsionals Society (RAPS) 2017 Regulatory Conference