FDA’s IT Systems Are ’20 Years Behind’ Industry Standard, Official Says

WASHINGTON — The FDA is “20 years behind the industry standard” in its information technology (IT) operations, said Peter Marks, MD, PhD, director of the agency’s Center for Biologics Evaluation and Research (CBER), at a briefing sponsored by the Alliance for a Stronger FDA.

“We really do want to modernize our business processes, which will require new IT systems … [and] move to a next level of safety surveillance systems,” Marks said at the Tuesday event. “And that really will require a fair amount of investment in IT partnerships and contracts to make that happen.”

Marks, who became CBER’s director in 2016, outlined his top eight priorities for the agency:

Responding to the COVID-19 outbreak. “We still have a very important role in COVID-19 vaccine authorizations and approvals,” Marks said. “That includes dealing with additional vaccine submissions for emergency use authorizations for expanding populations — into, for instance, the youngest pediatric [groups] — and for dealing with booster vaccinations, and then transitioning these emergency use authorizations over into traditional approvals, which will likely have to also occur during the next year.”

Eliminating the application backlog. “We’ve done a pretty good job of not missing major PDUFA dates [deadlines for making decisions on biologics applications], but we are lagging behind in getting people feedback on their applications in some cases,” said Marks. “And we are running behind where we would normally like to be in getting through our application load. So we want to try to do our best to catch up on that backlog of applications by year’s end.”

Expediting work on gene therapies. “There’s been a lot said about whether the center is taking a more cautious attitude or a more liberal attitude towards gene therapy,” he said. “We view this as an incredibly exciting field, which obviously will require a lot of nurturing as any nascent field does when it’s growing. And so we will continue to work diligently with sponsors and with stakeholders to try to move this forward.”

CBER also will “probably start to explore what would be potentially intermediate pathways for certain cellular therapy products,” he added. “We’re aware that there are certain cell therapy products that do not fall clearly into our tissue rules as not requiring premarket authorization, and yet also are probably not regulated ideally by submission of full biologics license applications. So we’re going to explore whether there is some intermediate pathway that might be appropriate there. That’s an exploration that I think is important to do, inasmuch as we are trying to lean into those things that we regulate to find the best methods to bring them forward to the benefit of patients.”

Producing more timely reviews and feedback. Marks called this a “major priority” for his agency.

Advancing blood donation policies. “This year is a critical one for completing a blood donation study looking at our blood donor deferral criteria and how we could potentially move past our current set of deferral criteria — for instance, for HIV to what would be perceived as a gender-neutral deferral criteria,” Marks said.

Taking regulatory science to the next level. “That includes doing additional work in the focused applied science of manufacturing and product characterization,” he continued. “Sometimes people don’t realize this, but we do have about 80 principal investigators that work at CBER … These are researcher reviewers and they make a big difference because they tend to keep up with the leading edge of the science, which helps them better review the products that come before them.”

Improving CBER’s business processes. “Obviously we need to modernize our business processes and our information technology systems to better serve public health,” said Marks.

Increasing diversity in the workforce. “We have to recruit, retain, and train a diverse professional workforce that’s capable of keeping up with the wealth of novel products that comes before us,” he said. Hiring for the agency will be a little easier now that working remotely is becoming more acceptable, Marks said. “We now have realized that we can have at least a reasonable fraction of our workforce … at remote locations, so hopefully that will allow us to draw on a larger talent pool to get a diverse workforce with the experience that we’re hoping to have.”

Marks also addressed the issue of what he would do if he had more money to spend than is currently in President Biden’s budget for the next fiscal year. “First of all, we’d hire more vaccine and gene therapy reviewers because I think we are clearly in a position that we are not giving the kind of feedback that I’m comfortable with in real time,” he said. “We are hiring up now, but we would even go further.”

Marks said he also would use the money to beef up safety surveillance, including the use of artificial intelligence and natural language processing to help CBER process “the literally thousands upon thousands — now almost bordering on millions — of adverse event reports that come in, in a given year.”

Safety surveillance work related to COVID vaccines is made more difficult because patients saw a lot less of their primary care providers during the pandemic, and they received free vaccines which weren’t billed to insurance companies and therefore don’t have claims attached to them, Marks said. “It becomes very challenging to do safety surveillance, because the data on immunizations are kept by immunization information systems, and there are 60 of those scattered around the United States and its territories.”

“We have had to essentially claw our way through agreements with each of those 60 in order to get our data partners to be able to get access to the data necessary to do the type of safety surveillance work that we would like to be able to do,” he added. “So this is something that we would love to fix moving forward, and that will require both funding as well as probably even legislative help.”