Clinical Trial Reform Takes Center Stage in Califf’s Replies to HELP Committee

Robert Califf, President Barack Obama’s choice to head the FDA, is calling for clinical trial reform, but does not believe new regulations are needed.

Califf says a combination of small, focused trials for precision medicine and large retrospective trials using electronic health records can result in a “dramatically lower cost” for clinical trials.

Small precision medicine trials would cost less because of their lower sample size, while large, retrospective trials would take advantage of data already collected to include important populations.

Califf sent responses to inquiries from Senate HELP Committee Chairman Lamar Alexander (R-Tenn.) and Ranking Member Patty Murray (D-Wash.) during a confirmation hearing in November 2015.

This precision medicine approach has been “increasingly recognized as having significant applicability to the development of clinical trial protocols and is now included in an FDA guidance document on risk-based monitoring,” Califf writes. He says the agency is making substantial progress in developing a more efficient system, and he praises the FDA’s Sentinel project as an “excellent building block with claims data on [more than] 170 million Americans available to evaluate the safety of drugs and biologics.”

Moreover, he does not believe new FDA regulations are needed, but that the U.S. system needs to “organize around some key principles,” he says. He adds that the agency is making “substantial progress,” touting the Precision Medicine Initiative as playing a key role in the reformation.

Reacting to a question about a speech he gave about regulations being a barrier to innovation, Califf says he was talking about “disruptive innovation” as a way to enable innovators to iteratively improve a product until it becomes better and “supplants the old product.”

But the nominee adds, “I have never stated, implied, or argued that the barrier should be lowered or removed.”

Califf also frowns on the importation of drugs from Canada and other countries in his replies to Alexander, arguing that products that are not approved by the FDA don’t have assurances of safety, efficacy and quality. He notes there are documented incidences of these imported substances having contamination, being counterfeit or containing varying amounts of active ingredient.

The HELP Committee held a hearing Jan. 12 and unanimously voted to pass his nomination to the full Senate for a vote.

Read the replies to Alexander here:, and the replies to Murray here: — Michael Cipriano


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