While freelancing for Nature Biotechnology, I recently talked to US Food and Drug Administration (Rockville, MD) Commissioner Scott Gottlieb. The conversation ranged from pricing, to market competition to new therapeutic modalities like gene therapy coming down the pipeline. A more extended extract of our discussion is also available in the News Feature.
You’ve helped to insert FDA into the drug affordability debate in 2017, by emphasizing competition and, by extension, more approvals, as a means to reduced costs.
Scott Gottlieb: A lot of what FDA can do around competition comes down to what we can do on the generic drug side with respect to complex drugs that are hard to make generic because of scientific or regulatory obstacles. We also see companies sometimes taking advantage of certain regulations and policies to extend patents beyond the time Congress really intended. And built into the generic drug approval process is a sort of regulatory arbitrage, where a company can come in, pick off one of the 300 or so products that was typically a low-volume generic but didn’t face any competition and jack up the price. So we’ve been taking action to try to resolve what I think are regulatory policy obstacles to allowing more vigorous competition.
Focusing on complex drugs where the patents and exclusivities have lapsed but they don’t face any competition yet, companies maintain monopolies on these products. We will be putting out some analysis early in the year on what the total spend is on complex drugs that should be subject to competition but aren’t.
These are drugs like metered dose inhalers. Drugs that are hard to copy under the traditional framework of the generic drug approval process. When a drug can’t be easily measured in the blood or it acts locally on tissue because it’s a topical agent or it’s an eye drop, or it’s a metered dose inhaler, that framework doesn’t apply very well.
So we’ve committed to putting forward product-specific guidance two years ahead of the first potential patent expiry on any complex drug going forward. We’ve gone back and tried to revise guidances on existing complex drugs that aren’t subject to generic competition. We’ve revised general principles in various areas for demonstrating sameness when it comes to things like metered dose inhalers, or topical agents, or liposomal agents. We’ve also put forward changes in how we infer sameness in drug-device combinations when the generic device that delivers the drug might be slightly different than the branded device.
Would you have expected to see more biosimilar competition by now, given when that pathway was created?
SG: I think we’re going to. When we look at the pipeline we see a pretty robust pipeline of companies that have come in to us, starting to engage the agency on biosimilars they want to develop. We have to keep in mind, there’s a small subset of biologics that have come off of patent. And also the biosimilars that have been approved have been subject to litigation. I think that if you look back at the early days of Hatch/Waxman the experience that we’re having with the biosimilars isn’t that different. It took a while for firms to gain the sophistication to come through the regulatory process. It took a while for providers to gain confidence in adopting the generic drugs. And it also took a while for a lot of litigation to get settled. I think we’re basically in the early days of that. That said, the big initiative we’re going to announce next year on drug competition is a biosimilar policy initiative. It’s going to be a collection of policies that we undertake to try to loosen the framework for bringing biosimilars onto the market to try to instigate more competition. We’re also going to be spending money trying to help educate providers about biosimilars. We’ve done that on the generic drug side of the house where we do public service campaigns. We’re going to be undertaking and are in the throes of it right now a big public service campaign on adoption of biosimilars, trying to educate providers on using biosimilar drugs as well.
I think that we’re still in the early days for biosimilars. But I never had the expectation that this market would evolve in as robust a fashion in its early years as some of the initial policy estimates. There were estimates put forward in Washington that inferred and imputed enormous savings from biosimilars very early. I think it was always going to be the case that this is going to be a slower evolution. And I think we’re doing quite well. I feel pretty confident and I base that not on what’s been approved. I’m looking at what we see in terms of the action of companies coming in and engaging us.
How do you think about creating competition in areas like gene therapy?
SG: Right now we’ve validated a handful of tools and I think over time we’re going to validate more tools that are going to enable different ways to try to address the same disease through multiple modalities.
If you look at sickle cell disease, for example, there are people developing CRISPR/Cas9 approaches to it, people who are developing exogenous gene therapy, people doing in vivo gene therapy techniques, people who are using fetal hemoglobin, people trying to correct the underlying defect. There’s a lot of different approaches that will hopefully create some inherent competition in the market. Right now it’s early days because we’ve validated a handful of tools. –like antibodies I think the inflection point we’ve witnessed in gene therapy in the past couple of years is the advent of the AAV vector and more reliable vectors that don’t have any immunogenicity, and deliver the gene therapy products more reliably. And so I think we’re going to see other types of modalities come forward, just as we saw in the biologics space, where you saw multiple ways to humanize and develop fully human antibodies. I think the same thing will play out in gene therapy and you’ll see competition by virtue of that.