Weiss Smith, Sharfstein: Door is Open at FDA for Drug Safety and Review

University of Maryland BioPark tenants get a primer on “Inside the FDA” and how best to work with the huge regulator.

By Steve Berberich
September 23, 2011

Too often, people think of the U.S. Food and Drug Administration (FDA) as a huge wall of regulations and don’t consider there’s an open door in that wall, drug safety scientist Sheila Weiss Smith, PhD, said Sept. 16 at “Inside the FDA,” a panel critique of one of America’s most pervasive and often controversial regulating agencies.

Weiss Smith, a professor of pharmaceutical health services research at the University of Maryland School of Pharmacy, encouraged faculty and company executives in attendance to prepare themselves well in advance of FDA officials reviewing their product for approval by engaging in constructive dialogues with the review division.

Weiss Smith and Joshua Sharfstein, MD, Maryland’s Secretary of Health and Mental Hygiene, were featured speakers at the Greater Baltimore Committee panel, which was held at the University of Maryland BioPark. Weiss Smith is often consulted by FDA regulators and the pharmaceutical industry on issues of drug safety. Sharfstein previously served as principal deputy commissioner at the FDA.

Along with moderator Steven Grossman, JD, author of the blog FDA Matters, the two speakers offered advice on working with a “new FDA” they said has become far more transparent in recent years.

“FDA is people, too,” said Grossman, who is president of Health Policy Solutions Group, LLC. “FDA is a lot more committed to working with you than you might expect. They are people who want to help you with your own development plan. That has been strengthened in recent years,” he said. He is a former deputy assistant secretary for health in the U.S. Department of Health and Human Services.

“Inside the FDA” was Sharfstein’s first public discussion of the FDA since he left the agency earlier this year to become the chief Maryland health official. He said, “The industry is very interested in getting guidance on technical matters, but the FDA doesn’t have the time to consider all of those.” Companies need to come to the FDA with “some general plan” at least, he said. The company may not have the wherewithal to check into all the questions of competitive drugs out there. And the FDA may not have the funding for all the questions, he advised. Sharfstein, however, added, “I was very impressed when I was with the agency of the broad expertise there.”

Weiss Smith broadened the point, saying, “Yes, there are published guidance documents on many topics, but it is good to know going in what the current thoughts are within the review division [at the FDA] that might apply to your product, so you can anticipate key concerns and questions.”

Grossman said the agency has been criticized at different times as being both too slow and too fast for drug approval. “It’s a tremendous responsibility to regulate food and drugs. When you stop and think about it, that’s one quarter of every consumer dollar spent.”

“In the past, I had described FDA as too lax in certain areas of drug safety. But now I think they are a lot more savvy,” said Weiss Smith. In a widely quoted editorial in the New England Journal of Medicine in 2007, Weiss Smith denounced the FDA’s response to a September 2006 report by the Institute of Medicine (IOM), “The Future of Drug Safety,” writing that the agency’s response demonstrated “a lack of understanding of the magnitude of the changes required to create a culture of safety.” The IOM report had identified weaknesses in the laws, regulations, resources, and practice of ensuring drug safety.

She advocates that whether one believes the FDA is emphasizing approvals or safety, the agency needs to balance both to make wise decisions. “There are a lot of different connotations to drug safety,” she explained to the BioPark audience. “They include making sure the drug is the right chemical and not counterfeit. Making sure it is not contaminated. Does it dissolve right [in the body]? Plus, you need to know and clearly articulate the risks of a drug, because once it is on the market it becomes a public health issue, particularly for drugs which are widely used.”

Weiss Smith is currently working with the National Cancer Institute to study safety issues for medicines used in cancer therapy and the potential cancer risk among some commonly used prescription medications.

Sharfstein and Weiss Smith also advocated more funding for little appreciated “regulatory sciences,” Sharfstein said, “Sometimes we need more research on the tools of regulation.” The two said such tools are the “how tos” of the many connotations of drug safety. Weiss-Smith said that ranges from “designing tests to rapidly identify counterfeit drugs to developing and testing new statistical techniques to study rare adverse events.”

Sharfstein praised the University of Maryland School of Pharmacy as a leader in regulatory sciences training. “Having a partner in the academic world is very important. There is an entire enterprise out there for many companies,” he said.