Nearly a year after a major panel recommended it, the Food and Drug Administration has released new standards for how officials will evaluate new opioid drugs for approval to be sold in the United States. The proposed guidelines formally say that the FDA will consider opioids' effects on the wider public, not just whether they work if they're taken as intended, which is a first. But at least one advocacy group says it's not enough.
"If this is their view of what should go into a opioid framework, that is not acceptable," says Sidney Wolfe, a drug-safety expert with the consumer advocacy group Public Citizen, who previously served on an FDA advisory committee. Public Citizen has petitioned the FDA to stop approving any new opioid painkillers until it starts using stricter rules.
Typically, when deciding whether to approve a new drug, the FDA only looks at the risks and benefits to patients who take it as directed, not risks to everyone else. But in July of last year, a panel of scientists organized by the National Academies of Science, Engineering, and Medicine recommended the FDA create a special, stricter process just for opioids. The overprescribing of opioid painkillers played a critical role in creating the addiction and overdose epidemic in America today, the National Academies found, and so the FDA has an "important gatekeeping function" to play in keeping riskier new opioids from entering the market. The call for a tougher opioid approval process was underscored in November of last year, when the FDA OK'd an opioid called Dsuvia, generating intense controversy. Dsuvia is 10 times more powerful than fentanyl, an already-potent opioid that's the driver of overdose deaths in America. Groups, including Public Citizen, called the approval dangerous. The FDA argued that restrictions on Dsuvia's distribution would minimize its risk.
Among other things, the FDA now proposes that officials consider whether a new opioid works better than other painkillers—opioid and non-opioid—that are already on the market. Officials will analyze whether the new opioid has characteristics that might tempt people to misuse it, for example if it's very potent, or easy to crush, dissolve, and inject.
Richard Bonnie, a professor of health and drug law at the University of Virginia who chaired the National Academies panel that recommended a special opioid FDA process, says the panel hasn't had the chance to review the new proposal yet, so he couldn't comment on it in detail. "What I can say is that we are pleased that the agency has taken this first step in implementing our recommendations," he writes in an email. The panel plans to take a closer look in July.
Public Citizen agrees with the National Academies' recommendations and wants to see them implemented. In Wolfe's view, however, the FDA's guidance doesn't do that. For example, the guidance doesn't say the FDA definitely won't approve any opioid drug that doesn't work better than existing painkillers. The guidance also says the FDA will "consider" data on the unintended consequences of a new opioid drug, but doesn't require companies to submit that data to the agency. Indeed, as a guidance, not a formal regulation, it isn't legally binding. It's not uncommon for the FDA to issue guidances instead of regulations, says Patricia Zettler, an assistant professor at Ohio State University's Moritz College of Law, who previously worked as an associate chief counsel at the FDA. "Some have criticized FDA for over-using guidance," she writes in an email, but companies "often" follow them.*
Public Citizen would have no problem with the guidance if it had stronger language, Wolfe says.
When asked about Public Citizen's criticisms, Lyndsay Meyer, a spokesperson for the FDA, pointed to the agency's announcement that it will hold a public meeting in September to discuss whether the agency should require drug companies to prove a new opioid works better than those already on the market. The announcement suggests the agency can't legally do that now. Instead, it may need new powers, which Congress would have to grant.
The guidance is open to change. The public, including advocacy groups and drug companies, has until August 20th to comment on it.
*Update—June 26th, 2019: This post has been updated with Patricia Zettler's current position.