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Challenges of Obtaining FDA Approval for Novel Agents


Barry Kreiswirth, PhD, founding director, Public Health Research Institute Tuberculosis Center, professor of medicine at Rutgers University, discusses challenges associated with the approval of novel antibiotics.

Interview Transcript (slightly modified for readability)

“The issue of drug discovery now has changed. Years ago we had a formula, we, being the pharmaceutical industry, had a formula that [we] followed. Because of resistance, now people are looking for novel approaches [and] treatment options that [they] probably wouldn’t [even] have thought of years ago, just because they were a little too 'out of the box.'

The problem with alternative treatment options [is that] they’re difficult to get approved; they’re difficult to do clinical trials. We don’t have paradigms that we can build on from our experience, [and] so, if you really had a novel agent that really worked differently, to get approval is going to be tricky because we don’t have precedent. Even though we’re all trying to think of novel approaches, different regimens, different ways to deliver drugs, different ways not to develop resistance, soon as you hit something that’s very novel, the challenges just change because, how do you show that it’s effective when we really don’t have a pathway for the FDA to go down that says, ‘Oh yeah, you do A, B, and C, and if it works, then we’ll approve the drug.’ Now, it’s [more] like, ‘Oh, I don’t know what A, B, and C is because this is a whole novel approach and it changes the game.’ It’s important to do, but it’s also very frustrating because there may actually be good options out there that we don’t know how to use yet.

Like I said, when we do come up with novel agents, the hardest thing is, you go to the FDA and they’re always looking for a paradigm to compare it to, which makes sense. They said, ‘Okay, we took this drug, you do A, B, and C; this drug was approved; look how well it’s doing.’ So, you take that same path, [but] now you say, ‘Oh, but this drug is a little different,’ so the FDA says, ‘Oh, okay. What do I compare it to?’ or ‘What path should this take?’

This is the problem with diagnostics as well, the FDA, or part of the FDA, has to approve rapid diagnostics. Again it’s always the idea of well, how do we know this is better or inferior or superior or equivalent to what’s out there? Sometimes, the novel agent or the novel diagnostic doesn’t have the perfect match to say, ‘I can really test it well and compare it,' because my comparator isn’t the same. That becomes a little different; it’s like if you developed the Tesla and now you want to compare it to a new electric car [and you realize], ‘Oh, there are not many electric cars to compare it to’ and that’s the good or bad of coming up with novel agents.

Academics have come up now, trying to get into the space with the idea that they’ll come up with a novel idea and hopefully [a] pharmaceutical company will pick it up, because no academic can develop a drug; [they] just don’t have the tools or the financial ability. A lot of academics are getting involved in the idea of trying to develop that first phase of discovery with the thought that maybe [someone in the] pharmaceutical industry or a biotech will be the ones, or even NIH, to take it to maybe the next step, which gets very expensive.”
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