The opioid crisis. Right to Try legislation. The first human trials of CRISPR-Cas9 gene editing technology. These are just a few of the many topics at the feet of the FDA this year.
Speaking at the BIO CEO & Investor Conference in New York on Tuesday, principal deputy commissioner Rachel Sherman, the FDA’s second in command to commissioner Scott Gottlieb, fired through a variety of issues affecting the agency both now and in the future.
Here are just a few tidbits from the chat, moderated by Biotechnology Industry Organization president and CEO Jim Greenwood.
Right to Try
A Republican-led push is underway to give desperately ill patients greater access to experimental drugs without FDA oversight, a move President Trump endorsed during his State of the Union speech. But the potential passage of a “Right to Try” bill that might reduce or eliminate FDA oversight of compassionate use—the mechanism already in place to get patients experimental drugs outside of clinical trials—has met vehement opposition from medical organizations, patient advocacy groups, and more.
Sherman wouldn’t comment on any discussions about the current bill in Congress, but she defended existing FDA compassionate use programs.
“Our primary concern—really our only concern—in this bill is that patients be protected,” Sherman said. “In the initial iterations of this bill, there was no stipulation about manufacturing. You could be making [a drug] in your bathtub at home and you could be selling it. And if the first time patients that took it dropped dead, you’d have no obligation to tell anyone. We’ve shared the sentiment that we don’t think responsible companies will be terribly interested in that.”
Sherman said the agency, as it has historically done every few years, is undergoing an independent assessment of its compassionate use program.
Sherman was clear about one problem the FDA is facing right now. “We’re getting old,” she said.
Notwithstanding a short retirement a few years ago, Sherman has been with the FDA for almost three decades. Several longtime staffers in key positions, like top drug evaluator Janet Woodcock, have been at the agency for years as well. Greenwood said the biopharma industry is wary of the “frightening prospect” of FDA leaders retiring without experienced successors in place. Sherman didn’t allay those fears. “It’s not our strongest suit, to be perfectly honest,” she said.
Sherman said the FDA is good at hiring junior workers, but has a harder time—due to salary constraints and other issues—retaining them as they climb the ranks. “The first thing that always disappears when money is tight or time is tight is training and investing in our own employees. We have to do better,” she said. “The notion that the institutional memories could walk out the door—and will walk out the door—is scary to everyone.”
Publishing Drug Rejections…
Last month, FDA commissioner Gottlieb, in a speech published on the FDA’s website titled “Fostering Transparency to Improve Public Health,” wrote that publishing all complete response letters (CRLs), which the FDA sends to companies when it rejects their drugs, would be “administratively burdensome.” Instead, the FDA is evaluating whether to redact and release a “subset of letters…where there are especially important public health reasons.”
A biotech investor wanting much more transparency on CRLs asked Sherman for her thoughts on the matter. Sherman appeared to put the onus on drugmakers to do more. “It’s not our property to release [CRLs] unless there is a…public health issue. As a citizen I would argue that the result of human experimentation belong to the patients. … Most FDA leadership is very into transparency. Companies do have their shareholder interests, et cetera, and I can’t speak to that. There’s not an easy answer.”
…And Sharing Data
Sherman called out one particular thing that could help speed up the development of drugs, particularly for chronic conditions like heart disease and Alzheimer’s that affect millions of people: working together. Companies typically don’t work together on large “outcome” trials, like the massive, expensive studies that test whether cardiovascular drugs lead to fewer heart attacks and strokes, Sherman said. If they shared data and worked together to become “more efficient at collecting only the data we need,” the cost and time involved to test drugs might come down, and products could get to patients sooner, she said.
When to Contact the FDA? Early and Often
A company can wait months to get an hour-long meeting with the FDA. But when that meeting arrives and the clock starts ticking, Sherman says, drug companies too often are either afraid to ask something or don’t “carefully scrutinize” what they are asking before they do. Sometimes companies don’t know who their primary contact is at the FDA and communication isn’t steady. That can lead to delays, or more uncertainty as a drug advances through testing. Companies are much better better off engaging the agency “early and often,” according to Sherman.
“The mistake too many companies make is shy away, stay away from the FDA,” Sherman said. “Continuous probing will get you much further. If an answer makes you unhappy, that may or may not be ok. But if you’re burning through time, your product may never get developed, and that’s bad for everybody.”