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Bloomberg BNA – FDA Mulling Ways to Break Silos, Review Products by Clinical Areas

Bloomberg BNA – FDA Mulling Ways to Break Silos, Review Products by Clinical Areas

The FDA is considering ways to review more medical treatments by clinical area, following in the steps of the cancer center established this summer.


“Historically, as almost everyone knows, FDA has been remarkably siloed,” Food and Drug Administration Commissioner Robert M. Califf said Nov. 4. “But what you hear loud and clear from patients is they’d like a have a way to be in contact with the FDA that’s not by product type but by what’s relevant to their disease. And we obviously heard that loud and clear from the cancer patients.”


Califf delivered the keynote address at a biopharmaceutical conference organized in part by the Friends of Cancer Research—the group that successfully lobbied for the FDA Center for Excellence the agency established in June. An effort to streamline approval of cancer products by bringing together reviewers from all the different FDA device, drug and biologic centers, the center is part of the agency’s effort to support the White House cancer moonshot initiative.


This new approach to review also has implications to advance precision medicine—another Obama administration priority—since a critical component of these targeted treatments is the use of a diagnostic tool to determine if a patient will benefit from a treatment.

From Silos to Matrix

For a whole host of reasons, Califf said, the FDA didn’t follow academia and industry as they restructured from a siloed approach to a more integrated, or “matrix” approach 15 to 20 years ago. The Oncology Center for Excellence was the first step in responding to the call to look at medical products by clinical relevance and not by their classification as a drug, device or biologic.


“Exactly how far the matrix goes is a matter of discussion,” Califf said. “And it may be quite different for different fields.”


While there may be reason to have cohesion around the clinical portion of experimental treatments, different products have fundamental qualities that still may warrant review from the different centers.


“Devices are very iterative. They’re engineering. They’re fundamentally very different from drugs,” Califf said. “And I’ve learned that bioloigcs are somewhere in the middle. There’s a lot of tweaking that can and does go on with biologics.”

Two Priorities

In the final days of the Obama administration, Califf identified two of his overarching priorities, in addition to specific policy topics such as opioid addiction. The first is revamping the human resources system to enable the FDA to hire the staff it needs to keep pace with what he described as “an amazing explosion of biotechnology.”


The second priority is what he described as an “evidence generation” by harnessing available data to help inform the agency’s regulatory decisions. Califf cited the FDA’s Sentinel Initiative, which pulls information from electronic health records (EHR), insurance claims data and registries and other data sources to develop postmarket risk identification and analysis systems.


Califf added that most of the decisions about what’s not working are made “well before anyone would think about putting an application together.”


“Given that 92 percent of drugs that get to phase I don’t make it to market, this is really critical,” he said.


Phase I refers to the first time an investigational product gets tested on humans, usually for safety and dosage purposes, not to determine if the drug actually works.

New Drug Approvals Drop by Half

In a separate session, John Jenkins, director of the FDA Office of New Drugs, said the agency has approved 19 novel drugs. This number is less than half the new drugs the agency has approved in previous years. In 2015, the FDA approved 45 new drugs and in 2014 it approved 41.


Jenkins said the drop-off is due to fewer applications coming in to the FDA combined with fewer approvals after the first review cycle.


Rejections often arise from problems meeting manufacturing standards instead of a lack of substantial evidence that the investigational drug didn’t work. “It’s not a lack of effort on our part. It’s more deficiencies that just didn’t meet the standards,” Jenkins said.