麻豆传媒

With all the news about drug shortages, there's another type of possible shortage that the public should be concerned with, an FDA official said Wednesday.

"We need help from Congress on our authorities to prevent device shortages," Jeffrey Shuren, MD, JD, director of the FDA's Center for Devices and Radiological Health, said at a on FDA regulation of drugs, biologics, and devices. "They're happening every day, and we don't have all the tools we need to prevent that."

Device shortages was one of a number of topics that came up during a wide-ranging hearing featuring FDA officials that lasted for several hours. One topic that had interest from members on both sides of the aisle was the FDA's recent issuance of a final rule regulating laboratory-developed tests (LDTs) -- in vitro diagnostic products designed, manufactured, and used within a single clinical laboratory. LDTs can be used to measure or detect markers like proteins, glucose, cholesterol, or DNA to help provide information about a patient's health, including diagnosing, monitoring, or determining treatments.

"I've heard concerns that FDA regulation of LDTs could slow the approval process for all drugs and devices in addition to LDTs," said Rep. Raul Ruiz (D-Calif.). "While FDA will not collect user fees for LDTs for years, there's significant work that must begin now, including issuing guidance documents related to the rule." He asked Shuren whether FDA had enough resources to carry out the new rule.

"So we do have the resources to begin implementation of that rule, and we're already moving forward," Shuren said. "We're also looking at other opportunities for reducing costs, both to test makers and to the FDA," including ways to streamline premarket review.

Rep. John Sarbanes (D-Md.) asked what the agency was doing to encourage more generic drugs to come onto the market. "This is an area that is critical for us," said Patrizia Cavazzoni, MD, director of the FDA's Center for Drug Evaluation and Research. "Of course, the entire generic drug program is pointed towards making drugs more accessible to patients by creating competition and therefore lowering prices. This is a program that has historically had a lot of high volume; we are increasingly focusing on what we see is coming up in the pipeline for generics."

There are things Congress could do to help things along, she added. "For instance, one of the problems that we encounter is what brand-name drug manufacturers use to stifle competition -- constraints around what we can disclose to generic manufacturers when it comes to all of the ingredients in a drug. We have put forward some proposals to help remove some of those barriers," said Cavazzoni.

Rep. Richard Hudson (R-N.C.) was concerned about the FDA's failure to meet some of its own deadlines for deciding whether to approve a particular drug. Peter Marks, MD, PhD, director of the FDA's Center for Biologics Evaluation and Research, agreed that this was a problem. "We have a group that actually tracks these quite closely and ... I agree with you. We can sometimes do better here," he said. "We try to do, essentially, 'after action' [reports] to understand why these events occur, so we can help prevent them ... Bureaucratic reasons for this are unacceptable. There has to be a good scientific reason why we miss things."

Cavazzoni said it was "very rare" for her division to miss those deadlines, noting that if a drug sponsor wants to submit additional data while their application is being processed, "we extend the goal date to allow them to submit the data and allow it to be reviewed."

On a related note, Rep. Robin Kelly (D-Ill.) asked about getting more diverse populations included in clinical trials. "It is very important that we enroll, in clinical trials, a population that is representative of the population that will ultimately be taking the drug once it's marketed," said Cavazzoni. "We're paying a lot of attention to clinical trial diversity," including by issuing guidance about who should be included.

On the other hand, "We don't want the inclusion or exclusion criteria for trials to be so restrictive that it really impacts the generalizability of the data," she said. "And lastly, it is very important that we find a way to actually have recruitment for clinical trials in the communities that have historically been excluded from clinical trials."

Communication with the agency was another issue of concern. "One thing that has come to my attention in meeting with some really interesting innovators in the healthcare space is the lack of communication with FDA," said Rep. Dan Crenshaw (R-Texas). "They often feel like it's sort of a black hole of communication. They don't know what they're doing wrong -- they're just told to do it again, to try something differently." He asked how the agency is supporting "smaller companies that don't have legions of lawyers" to help them with the application process.

"We recognize that many of the sponsors working in regenerative medicine, even some of the small gene therapy companies -- they do not have the experience dealing with [FDA] and need someone to listen to their concerns and respond," said Marks, adding that the agency has several programs to help such companies.

For one program, "it may not be a [specific] product but a set of products they're intending to develop, and they can have a very informal meeting with some of my colleagues about manufacturing or clinical development," he said. Another program allows sponsors to "come in with just a couple of pages" about what they're trying to develop, and get advice on how to do it. "It's a 'no fault' dialogue -- we don't hold the sponsor to taking our advice, but it allows them to get feedback."

Comment