WASHINGTON — Competition to make new brand-name drugs in some of the newer drug categories is slowing, and that’s a problem, outgoing FDA Commissioner Scott Gottlieb, MD, said Friday.
“Part of [the] FDA’s mission is trying to enable product competition — not just with generic substitution, but also with novel drugs,” he said at a briefing sponsored by the Alliance for a Stronger FDA. “We’re going to be publishing data very soon showing that second-to-market innovation in novel drug categories is taking longer to come to market, and more of these novel drug categories are staying monopolies into perpetuity.”
“There’s reason why that’s happening,” Gottlieb, who is stepping down at the end of next week, told moderator and former FDA Commissioner Mark McClellan, MD, PhD. “When you’re trying to develop a therapy for an inherited disorder, once you’ve cured people with the disease, being second to market only to treat people who are newly diagnosed with the disease every year is splitting that market too small to justify the investment. So you quite literally see companies slowing down development programs if they think they’re going to be second to market, or pulling out entirely. My job was to create product competition … When you have product competition, you have price competition.”
Speaking of price competition, one area where it doesn’t happen is Medicare Part B, which covers drugs dispensed in the physician’s office. In Part B, “the government is largely a price taker and those drugs aren’t competitively bid; I think we need to think about that,” he said.
Gottlieb also discussed tactics used by brand-name drug companies to prevent generics from coming onto the market, including the use of Risk Evaluation and Management Strategies (REMS) as an excuse to deny generic drugmakers access to samples of brand-name drugs for clinical trials. “The underlying premise here is that branded companies make it hard for generic firms to access the physical doses that they need to do bioequivalence and bioavailability studies to file an application with the FDA,” he said. “I find this particularly pernicious and ruinsome to the whole system. We’re asking generic firms to do fairly straightforward studies to get on the market … and they can’t even get access to the doses they need to prove their drug is the same as the branded drug. That undermines the whole system.”
However, “behaviors and abuses around REMS aren’t as prevalent any more,” Gottlieb said. A new tactic instead is for companies to “distribute the drug through a specialty pharmacy company and make it hard in the supply chain for the generic firm to actually purchase the drug.”
“That’s why we’ve looked at, on the biosimilar side, allowing the biosimilar [company] to go to Europe and buy the product there,” he continued. “We know it’s the same product produced in the same facility.” But this raises a legal issue: “We know it’s the same product produced in the same facility, but does that information constitute CCI [confidential commercial information] that we can’t use for purposes of our own regulatory considerations? That’s what we’re working with the lawyers on right now.”
Another strategy the FDA is considering is “to make it easier for companies to file a global application for generic drugs, to allow for more of these small-market generic drugs that might be sold in one European country by a specialty pharmacy company to be more readily accessed in the U.S. in situations where we don’t have a lot of generic competition,” Gottlieb said. “There are a lot of places in the market where we pay too much for drugs … but there are also places where we pay too little, particularly when you’re dealing with the generic sterile parenteral drugs. That’s typically been where the shortages are.”
Combatting the Opioid Crisis
On the opioid front, “we’ve changed our view about our obligation and taken a much more affirmative role in trying to reduce the rate of new addiction and trying to rationalize prescribing, and we’re seeing that as a core responsibility of the agency,” said Gottlieb. “We’re trying to make sure that when prescriptions are written, they’re appropriate, and when they’re prescribed, it’s for a duration of use that works in the clinical circumstance. We’re doing things like expanding education to all providers and blister-packing immediate-release drugs pretty soon in 1- or 2-day packs to make it easier for doctors to dispense what we think is an appropriate duration for a lot of post-surgical uses, which is just 1 or 2 days.”
“The challenge now,” he added, “is that the nature of the crisis is changing dramatically, from one where the most new exposure was in the medical setting to one where more of the initial exposure is illicit drugs used recreationally. If you look at … the scope of decline in medical prescribing, which is good, it is being dwarfed by a rise in illicit [drug] flows.”
Gottlieb also discussed the agency’s work on nicotine use. “We are seeing anecdotal data suggesting that adult smokers are transitioning off of combustible tobacco to [e-cigarettes] … If you look at the survey data, a large portion of people using [e-cigarettes] are newly initiated on nicotine, but there is also a large portion migrating [away from tobacco] — either dual users or switching completely, which is a huge impact on public health if these trends continue.”
Gottlieb said he was “shocked” (a word he has used before) at results from the National Youth Tobacco Survey last August showing an epidemic rise of e-cigarette use among kids. “We made the announcement about a week later that we were going to have to step in to regulate access to flavored products at retail and online in order to close off the ‘on-ramp’ to kids, recognizing that this would at some level impede the ‘off-ramp’ for adults,” he said. “These are difficult tradeoffs.”
Changes in the Agency’s Scope
Gottlieb said the agency had changed greatly since his two earlier stints there. “The scope of the agency expanded so dramatically in a not-so-long period of time,” he said. “And it’s not just that we have whole new areas of regulation, like [the Center for Tobacco Products], but … we didn’t have cell-based regenerative medicine and gene therapy 10 years ago, and we didn’t regulate on the farm. These are dramatic expansions in the scope of the work we do that’s really changed the contours of the agency’s mission and made it far more diverse and vast.”
In addition, “there are new paradigms of regulating technology that are very novel — digital health, cell-based regenerative medicine, and gene therapy — [where] the existing statutory framework doesn’t apply very well, so we had to conceive of different ways to apply our current regulatory policies to some of these new technologies. That’s one of the things that will endure from this general time period: we are writing the modern rules on how to regulate new areas of technology.”
Asked what his plans were after he steps down, Gottlieb said he had “outsourced” to his three young daughters the destination choice for his family’s upcoming vacation. As a result, “we’re going to Disney World the week after I leave this job.”
And after that? “No idea,” he said.