Sharpless Begins to Learn the Ropes While FDA Center Directors Look to the Future

Regulatory NewsRegulatory News | 02 May 2019 |  By 

About a month into his tenure as acting FDA commissioner, Ned Sharpless was in the spotlight Thursday, speaking first as the keynote at the Food and Drug Law Institute’s (FDLI) annual conference in Washington, DC, and then again a few blocks away at the Reagan Udall Foundation’s annual public meeting at Pew Charitable Trusts.

While both appearances lacked any definitive signal as to what he’s seeking to accomplish at FDA, both talks also revealed Sharpless’s evenhandedness and interest in keeping the agency headed in the same direction that former FDA Commissioner Scott Gottlieb steered it.

At FDLI, Sharpless touched on highlights from 2018 and explained how he’s learning to juggle the various and wide-ranging FDA centers and the products they regulate, while keeping his focus on public health issues.
“We’re considering whether to mandate that certain oral forms of immediate-release opioid formulations be made available in small quantities in blister packaging. You’ll be hearing more about this in the near future,” he said.

Sharpless also discussed the current measles outbreak, calling it “disheartening,” especially as the disease was eliminated and is only making a comeback because of vaccine avoidance.

At the Reagan Udall meeting, meanwhile, he discussed a major and fundamental challenge for the agency in balancing how quickly new medicines should come to market and how much has changed in the oncology space.

He also touched on the wealth of data that FDA has, explaining the difficulties in sharing it: “The bad news is that it’s a lot harder than people realize …. the good news is that people want to do it, but layering in patient protections makes it harder,” he said. The FDA as a “black box” concept is gone, he added.

CDER, CBER and CDRH Directors

Also at the Reagan Udall meeting, FDA’s directors of the Center for Biologics Evaluation and Research (CBER), Center for Devices and Radiological Health (CDRH) and Center for Drug Evaluation and Research (CDER) discussed how they are working with different stakeholder groups and where the centers are headed in the future.

CBER Director Peter Marks discussed the expectation for sticker shock associated with the influx of incoming gene and cell therapies. He also said the prohibitive costs of gene and cell therapy manufacturing need to be addressed, as do the lack of standards. And he noted that even if those manufacturing costs come down, prices for the therapies may not follow suit.

CDER Director Janet Woodcock noted the “tremendous problem” with antimicrobial resistance, explaining how one of the biggest issues is the lack of rapid diagnostics. Without these tests, she predicted, there will not be much new drug development in this space.

She also discussed the difficulties that the agency is experiencing with the use of real-world evidence (RWE), noting the data often are not reliable or standardized, and that with any observational study there can be confounding bias.

But she said CDER is working on a comparative program where observational studies are being run in tandem with randomized clinical trials. And Woodcock predicted that drugs will become more like medical devices in their use of RWE, especially as there’s more of a mechanistic rationale.

She offered the example of a genetically targeted drug, like with cystic fibrosis, that is approved for common genotypes. Will CDER require a randomized clinical trial for every new genotype a company wants to add? No, impossible, she said. Instead, the company may tinker with the drug, such as with small base pair modifications, and target genetic variations of the same disease.

And CDRH Director Jeff Shuren touched on how with devices, there are many products that come to market for new indications because of RWE. He also explained how there has been a lot of effort around device registries to ensure the data is consistent across databases. And he mentioned that by 2020, there will be a full list of companies that have used RWE to win approvals.


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