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Scientists Not Paying Enough Attention to Regulatory Issues, Pep Talk Presenter Warns

SAN DIEGO — Researchers developing protein therapeutics need to pay better attention to regulatory issues, especially to changes in the way the US Food and Drug Administration reviews new product applications, according to a presentation at Cambridge Healthtech Institute’s Pep Talk meeting, held here this week.
While most of the sessions focused on the science of protein research, one session homed in on regulatory issues surrounding the development of new protein therapies.
According to David Lin, a senior consultant at Biologics Consulting Group in Alexandria, Va., too often biotechnology researchers developing new therapies are oblivious to the regulatory process and to the fact that what they may find interesting is not viewed similarly FDA reviewers.
“My point of view is that the FDA reviewer is a scientist, and they need to be able to understand your product,” Lin said. “They need to be able to say, ‘OK, I find that all the information provided to me allows me to make an assessment that it’s safe to proceed with investigations in humans.’”
Otherwise, if a researcher just provides raw data without explanations, the reviewer may see a product application as an opportunity to conduct an experiment of his own and come up with different results, he said.
In particular, he said, smaller firms funded by venture capitalists and with little experience dealing with regulatory agencies can be naïve about what government assessors are looking for. 
“What’s important to [the researchers] is proof of concept,” Lin said. “So they don’t feel it’s necessary to spend the time to understand the process. They figure, ‘Show proof of concept, get some funding, maybe licensing, [but] let somebody else deal with it.’”
Reorganization within the FDA during the past three years, especially how the agency regulates biologics, has compounded the challenge. In 2003, overview of some therapeutic biologics was transferred from the FDA’s Center for Biologics Evaluation and Research to its Center for Drug Evaluation and Research.
At the time, the agency said, “this consolidation initiative provides the opportunity to further develop and coordinate scientific and regulatory activities between CBER and CDER. FDA believes that as more drug and biological products are developed for a broader range of illnesses, such interaction is necessary for both efficient and consistent agency action.”
Within CDER, the Office of New Drug Quality Assessment reviews therapeutic hormone proteins, while other therapeutic proteins and enzymes are reviewed by the Office of Biotechnology Products.
The result of the changes, Lin said, has been greater consistency in review expectations, communications, scheduling of meetings, and review timelines.  But differences remain in compliance inspections that can affect outcomes, he said.

“What’s important to [the researchers] is proof of concept, so they don’t feel it’s necessary to spend the time to understand the process. They figure, ‘Show proof of concept, get some funding, maybe licensing, let somebody else deal with it.’”

For example, while OBP uses a team approach in its product reviews, ensuring a greater chance that a reviewer with biologics expertise will see the product, ONDQA uses what Lin called a small-molecule field inspection approach. Such an approach increases the possibility that someone without biologics expertise may review a product.
According to Lin, researchers must put themselves in the shoes of FDA reviewers. “Sometimes, what’s interesting [to a scientist] is not what’s important” to an FDA reviewer. “Sometimes, people want to investigate the mechanism of action to the 10th degree. But that’s not what’s important. … Sometimes, the more you look into it, the more you understand that when it comes to proteins, you don’t know what this protein’s going to trigger.”
To be sure, the changes that have occurred have streamlined the regulatory process, said Lin. But it is still up to the scientists to provide the right information and ask the right questions.
“The FDA is not a consulting company, so they’re not going to answer questions for you that you don’t ask,” he said. “When it comes down to it, they have certain things that they need to satisfy when they make that assessment, but they’re not going to look at it and say, ‘Oh, I’m going to offer it to you.’ It just doesn’t happen.”

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