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FDA ‘open for business’ in considering real-world evidence, Sharpless says

PHILADELPHIA — The Food and Drug Administration is “open for business” in considering real-world evidence to clear expanded uses of new cancer drugs, acting agency head Ned Sharpless said Tuesday, but warned industry against relying too heavily on alternative sources of data.

Speaking at the BIO conference in Philadelphia, the former National Cancer Institute director acknowledged the rising cost and complexity of clinical trials, and indicated the FDA is aware of how difficult enrolling studies of rare disease subtypes can be.

“The FDA is always going to want to see really well designed beautiful trials with convincing endpoints,” he told conference attendees. “If you can do a large [randomized controlled trial] with overall survival as your outcome, there is nothing better.”

Still, “we realize there are circumstances where there is a need for flexibility,” he added.

Recently, for example, the FDA approved Pfizer’s Ibrance for male breast cancer, granting the pharma an expanded indication in April. In making its decision, the regulator used data from electronic health records and from real-world use of Ibrance, information gathered from Iqvia, Flatiron and Pfizer.

“Male breast cancer is a rare cancer and it’s not easy to do a clinical trial,” Sharpless noted. “Real-world evidence was supporting material — it wasn’t the sole factor.”

“The FDA is open for business to consider those kinds of data,” he said, while noting industry’s case can’t be made just by arguing it’s cheaper and faster than doing a clinical study.

The increasing segmentation of cancer into genetically defined subtypes gives drugmakers a better chance of proving efficacy with a targeted drug. But recruiting for such trials requires further steps, such as genetic testing, that can add time and expense.

“When I started my career as a cancer researcher we had one kind of breast cancer. Now we have 10 kinds of breast cancer,” Sharpless said. “That makes clinical trials much harder.”

The FDA’s willingness to consider alternate sources of data isn’t new. Drugs chief Janet Woodcock told a separate BIO session the agency has used real-world evidence for years in reviewing new treatments for rare diseases.

“We have vast experience with real-world evidence on the safety side,” she said.

Real-world evidence comes with concerns, both in quality and in interpretation. Still, for follow-on indications of already approved drugs, companies can make a plausible case.

“I think we have seen some of those particularly in oncology, where [drugmakers] are pulling some of that [data] from treatment use in rare cancers,” Woodcock said. “I think that is a very reasonable thing to do.”

Peter Marks, head of FDA’s biologics unit, echoed the theme, while sounding a note of caution.

“It is a really powerful technology to use these large databases” Marks said. “Unfortunately, the electronic medical record as it has grown up in the U.S. is highly fragmented and not correct a lot of the time.”

Marks encouraged industry to help ensure more accurate data in the records. “We have to get doctors to do a better job of what they put into the EMRs,” he said.