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POLITICO – Big money questions remain on CARA

POLITICO – Big money questions remain on CARA

CALIFF’S VIEWS: REAL-WORLD DATA IN DRUG APPROVALS — The FDA head said he sees openings for “real-world” evidence in drug development and approvals. Drug makers and some members of Congress have been pushing the topic, as the industry hopes to capitalize on all that data from electronic health records to speed up and reduce the cost of drug approvals. Califf at the Friends of Cancer Research meeting last week described himself as a pioneer in using data from every-day physician practices to do research back in the 1970s. But he cautioned that even though the data can be important, not every use of real world evidence is appropriate for drug approval. Nor should the real world data be used simply because it’s a short-cut. “The mistake we want to avoid is to say that we can infer causality when the methods really don’t support it and we are really using it to say we don’t have the discipline to do the right study to answer the critical question.”

… On where he’d be more comfortable with real-world data: “Many of us at FDA believe the most useful source of knowledge will come from randomization in the context of clinical practice.” He noted that A-B comparisons is one of the first things now taught in business school. “It’s beyond me why we don’t employ this routinely in the practice of medicine where there is so much more at stake in getting the right answer.”

What he might find concerning: The agency is much more cautious of retrospective analyses of data from medical practice. This is less likely to be useful for making causal inferences about the risk and benefits of medical products, Califf said. FDA drug center director Janet Woodcock said the agency will put out guidance on using evidence from non-traditional clinical trials for regulatory purposes, but added that a lot of “grunt work” has to be done to make data from electronic health records usable for FDA’s purposes. For starters, it will need to be validated. Right now, it’s not easy to even confirm that a diagnosis in an academic medical center’s clinical records is correct.

MOON SHOT UPDATES COMING — A June 29 Cancer Moon Shot Summit in DC and in 100 locations throughout the country will provide updates on getting the Vice President’s initiative up and running, including proposals that specific government agencies are putting forth. Califf said he’s impressed with how fast things are moving, telling the Friends of Cancer Research meeting he’s “already seen some things happen that I didn’t think were possible.”…