FDA Commissioner Scott Gottlieb pinned his hopes on several key initiatives, including a new robust knowledge management system, in an appearance before the US House Subcommittee on Agriculture and Rural Development on April 17.

The President’s 2019 budget request for the FDA totals $5.8 billion – an increase of $663 million from the FY18 resolution. Gottlieb delivered a statement to the subcommittee outlining the FDA’s plans, assuming the budget is secured.

Gottlieb began his statement by highlighting an impressive accomplishment from 2017: the FDA approved a record number of generic drugs, novel drugs and medical devices. Gottlieb credited advancements in science and technology as the catalyst for this milestone.

Moving into his vision for the future, Gottlieb outlined new efforts aimed at bringing a more team-based approach to medical product review, facilitating easier information sharing across scientific disciplines, and bringing more consistency to decision making across different disease and product areas.

The increased spending plan would allow the agency to work on several priorities, including:

  • Investments in advanced manufacturing that can bring more production back to the U.S. while improving our ability to respond to public health emergencies.
  • New efforts to help more compounding pharmacies expand and grow their businesses.
  • Programs to modernize generic drug review to increase competition and address high drug costs.
  • New approaches to support developing treatments for rare pediatric diseases.

Following these priorities, Gottlieb noted one major area of investment: building a knowledge management system that could not only increase transparency, but also make the drug development process more efficient.

Investing in a Knowledge Management System

To modernize medical product review programs and establish scientific precedents, the proposed knowledge management system would:

  • Store and manage medical review staff’s experiences
  • Identify how decisions are made across different functions
  • Make note of how scientific precedents are established
  • Capture the knowledge that’s developed through the process

Gottlieb said these changes have the potential to make the FDA’s review process more efficient, modern and scientifically rigorous.

“Right now, if you asked me how we made a particular review decision in the past, I’d begin by asking our review staff if they’ve confronted a similar clinical circumstance, how it was decided and why,” Gottlieb said. “We have limited options to query review decisions to extract how we reached certain conclusions. We can’t store and interrogate the scientific precedent we establish every day.”

Gottlieb went on to provide an anecdote of how the knowledge management system could play a crucial role in advancing medicine.

“In the late 1990s and early 2000s, several drugs were discovered after-approval to carry a risk of sudden cardiac death leading to their withdrawal. To identify drugs with this “pro-arrhythmic” potential, a new clinical study requirement was introduced as a pre-approval obligation for all new drugs.

These study requirements looked at something called QT prolongation, where the heart’s beat becomes elongated.

But doing these QT studies is very costly, and the results are imperfect. The studies can flag drugs as having a risk when none really exists, and can miss drugs that carry the danger.
Recently, we’ve improved on this approach by developing cell-based assays that can better discriminate the medicines that are likely to have this side effect.

We’ve done this using a “data warehouse” that we’ve built by collecting knowledge and information over many years and across many different drug reviews, and evaluating the differences between drugs that do and don’t have this risk.

Through a collaborative group of researchers, led by FDA’s Division of Cardiovascular and Renal Products, we’ve developed a faster, less expensive and more accurate method to screen drugs for this safety issue. This new cellular assay is being introduced across all drug programs, across all FDA divisions, and we plan to have it replace the old QT study requirements.”

“This is precisely the sort of innovation in how we assess risk and benefit that we can make more routine with a better capability for curating knowledge gleaned across drug reviews,” he said.

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Author: Ryan Clark

Ryan Clark is a strategic client director working on partnerships between CCC and commercial clients. Ryan has been with CCC over five years and has worked in rightsholder relations and business development prior to joining the strategic client group. Ryan enjoys spending time with his family, and following Bruins hockey and Ireland rugby in his time away from work. 
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