Why Did the Pentagon Push the FDA to Approve a Powerful New Opioid?

https://portside.org/2020-01-11/why-did-pentagon-push-fda-approve-powerful-new-opioid
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Author: Kastalia Medrano
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Gizmodo
Editorial cartoon on addiction

In November 2018, the Food and Drug Administration approved America’s newest opioid, a formulation of sufentanil called Dsuvia. Sufentanil is the strongest prescription painkiller available—five to 10 times more potent than its parent drug fentanyl, which is in turn 30-50 times more potent than heroin. The outcry over Dsuvia—its potency, its potential for abuse—was immediate.

For a lot of people, the opioid overdose crisis was defined by prescription drugs like OxyContin until the prescription-writing frenzy finally leveled off around 2010, at which point many who’d become addicted to the medications coped by turning to heroin. The rate of fatal heroin overdoses exploded. A cheaper alternative for a similar high, heroin was pushed in by Mexican cartels and became the new face of the crisis before it was overtaken by what the Centers for Disease Control and Prevention identifies as the crisis’ third wave: synthetic opioids, namely fentanyl, which came on the scene in 2013 and surpassed heroin as the leading cause of overdose deaths by 2016. Since then, the line charting the rise in overdose deaths from synthetic opioids has become almost vertical.

Fueled by a combination of fentanyl panic and alarmed headlines, Dsuvia got a fair amount of negative attention in the weeks before and after its FDA approval. Significantly less discussed, however, was the drug’s relationship to a second government entity: the Department of Defense, which funded more than $22 million of Dsuvia’s development. The DoD pushed for the drug’s expedited approval, confidently endorses its benefits, and, according to my correspondence with the agency, appears to know very little about it.

Dsuvia is the first graduate of a new collaboration between the DoD and the FDA, formally announced the same day Dsuvia was approved. Per its terms, the two work together to identify new medical products and drugs that couldaddress “specific and life-threatening risk to the U.S. military” and fast-track them for approval. The DoD now oversees a Priority List of unapproved products it thinks could save lives in a military emergency. Dsuvia was on the list.

Over a period of several months, both the FDA and the DoD were either unwilling or unable to articulate to me how an opioid made it onto a list supposedly reserved for life-saving medical products, when acute pain is not a life-threatening condition. (Medications that manage chronic pain can indeed be life-saving for patients who might otherwise be driven to suicide over time, but Dsuvia’s whole thing is that it’s for very short-term use only; you’re not even supposed to take it home.) The DoD offered conflicting or off-topic answers or else punted to the FDA, which separately declined repeated requests for comment on how the terms of the collaboration applied to Dsuvia. A DoD representative variously emailed me that the Department had told the FDA the opioid was a priority item under the new collaboration, and that, “After checking, there was no formal request from DoD for expedited FDA action under that statute, but DoD strongly supports the FDA approval of Sufentinel [sic].”

In the last decade, the FDA has deposited more than a dozen new opioid formulations on the shores of commercial use without much notice, but Dsuvia was not one of them. Two weeks before the drug’s approval deadline, Dr. Raeford Brown, then-chair of the FDA advisory committee that evaluates analgesics, wrote a statement urging the FDA to reject it, arguing that IV sufentanil was “so potent that abusers of this intravenous formulation often die when they inject the first dose” and that abuse of Dsuvia would likely begin “within the early months of its availability on the market.”

From there, the headlines wrote themselves. Two weeks after the drug’s approval, Brown co-wrote an op-ed in the Washington Post saying that the FDA had made a grave mistake, that Dsuvia had “limited efficacy and no unique benefits,” and implying that it would have a similar-but-worse impact on the overdose crisis as fentanyl. The op-ed also stated that all but three members of the FDA’s drug safety advisory committee had been disinvited from the hearing ahead of Dsuvia’s approval. (Advisory committee meetings don’t dictate the FDA’s verdict, but the agency follows their lead about 87 percent of the time. Dsuvia’s ended in a 10-3 vote for approval.) FDA spokesperson Deborah Kotz told me certain members were absent due to scheduling conflicts; Dr. Pamela Palmer, co-founder and Chief Medical Officer of the company behind Dsuvia, AcelRx Pharmaceuticals, said they hadn’t been invited in the first place; Brown maintained they were disinvited. (An FDA law expert, speaking on the condition of anonymity, told me it’s not unusual for advisory committee members to not attend a safety hearing, but that it would be highly unusual to be disinvited. They also said that if the DoD wanted Dsuvia approved, it’s not surprising that it was approved.) 

The media panic covered the DoD sponsorship either as an afterthought or not at all. It also mostly failed to clarify that our overdose crisis is fueled by illicit fentanyl, not prescription fentanyl. According to the Substance Abuse and Mental Health Services Administration, the percentage of the population that misused prescription fentanyl in 2017 was 0.1 percent, compared to 1.4 percent for OxyContin and 4.1 percent for painkillers of any kind. Only 0.5 percent of people who misused prescription painkillers got them by stealing from a healthcare facility. Most get them from friends or relatives. Dsuvia isn’t a take-home drug for chronic pain—according to the limited research conducted on risks posed by sufentanil, the people most likely to abuse it are hospital workers like anesthesiologists. Brown agrees the concern lies “not so much in the general population.”

This doesn’t mean Dsuvia won’t be abused—just that it’s not likely to be abused more than any other opioid already out there. That’s enough to satisfy the FDA, which has the power, but not the obligation, to factor public health into its approval decisions.

“Everything gets abused,” Palmer said. “I am not at all trivializing the horrible, horrible outpatient disaster that is the overprescribing of opioids for outpatient pain conditions [but] sufentanil is one of the least-abused opioids because it’s always been only in medically supervised settings. That’s a fact, and as much as people want to get alarmist around it they cannot argue with the federal data.”

The FDA first approved IV sufentanil in 1984, 11 years before OxyContin came onto the scene and doctors began prescribing opioids with abandon. Sufentanil’s extraordinary potency kept it strictly confined to surgical settings, where it remained up until AcelRx rolled out Dsuvia. The drug’s official FDA indication is “for use in adults in a certified medically supervised setting, such as hospitals, surgical centers, and emergency departments, for the management of acute pain severe enough to require an opioid analgesic and for which alternative treatments are inadequate.” A battlefield might not look like a certified medically supervised setting, but it’s considered one when the DoD says it is. This is where Dsuvia is meant to shine.

“It was developed for battlefield use,” said Navy Capt. Mike Colston, director of Mental Health Programs under the DoD. “Decisions need to be made on a dime in the battlefield, and we want to increase the armamentarium of our folks in having an ability to treat pain acutely.”

That armamentum currently includes IV morphine, intramuscular (IM) morphine, and a fentanyl lozenge commonly known as “the lollipop,” which has been accessible to the DoD since 2004 but—unlike Dsuvia—is officially intended only for patients already tolerant to opioids. “That’s why they came to us,” Palmer said. “[Dsuvia] is an extremely unique product for them.” 

Dsuvia was approved on the basis of a single study. It started off, however, with four, at least two of which were funded by the DoD, including an Emergency Room study to justify Dsuvia’s battlefield application. Because that ER study was open-label—meaning that, unlike with more rigorous studies, there was no placebo group—a 2017 FDA clinical review found it insufficient to support Dsuvia’s efficacy, and it was removed from the body of evidence that would make the case for FDA approval. Additional reviews bounced the first of the two placebo studies from the safety and efficacy findings due to insufficient data; some stated that because the ER study wasn’t placebo-controlled, they hadn’t bothered to review it at all. By 2018, the journey to approval was supported by only the one remaining placebo trial, though the rejected data continues to be cited just the same, popping up in public statements and FDA memos in a way that puts you in mind of whack-a-mole.

“I was really intrigued by this product, but I think I’m disappointed with the data,” said one advisory committee member during the meeting. “We have no idea whether this drug works as well as, or better than, or worse than ibuprofen, acetaminophen, aspirin, or morphine … the peak action is in an hour. That’s pretty slow for a drug that’s supposed to be used for really acute pain.” 

IM morphine can take an hour to reach peak effectiveness. The fentanyl lollipops take around 30 minutes. IV morphine is much faster, but tricky to negotiate in the field. A big reason the DoD says Dsuvia is suitable for the battlefield is that it works quickly compared to existing options, and yet in Dsuvia’s pivotal study the average time it took patients to report “meaningful” pain relief was 54 minutes.

“Now, when you say to a patient, ‘hey, hit this stopwatch,’ which is the way that 54 minutes was measured,” Palmer told me, “they’re having blood levels drawn, there’s a lot of chaos going off, and they have to remember to hit this second stopwatch, and the average time that they remembered to hit was 54 minutes. So, to me, it’s like, pain is so subjective that it’s hard to sit there and say, ‘okay, well, based on this way it looks like you guys work in 15 minutes, but based on this particular second time a patient hit a stopwatch- I don’t even know what that means clinically, all I can look at is what the data shows us and, basically, based on plasma levels that we achieved and based on what their pain intensity levels dropped to, we work in 15 minutes.”

It’s true that pain is notoriously hard to quantify in medical settings. But in non-IV pain-management studies—including for the lollipops—15 minutes in is often just the first time patients are asked if they feel any difference. By that standard, any other approved pain medication could claim to work more quickly, too. “You’ll note that the applicant has emphasized different times to onset, but FDA considers time to meaningful pain relief as the most clinically relevant,” said a clinical reviewer during the advisory committee meeting.

Asked why the DoD pressed forward with Dsuvia even after the only study intended to justify battlefield use was found too shaky for the FDA to evaluate, Colston instead cited the surviving placebo study—which used patients recovering from abdominal surgery—as “a reasonable proxy for battlefield wounds.” He went on to claim that Dsuvia was “superior to placebo pretty early in—about 15 minutes or so.”

Whatever its shortcomings in execution, not all the ideas behind Dsuvia are bad. Morphine injections work poorly with blood loss or poor circulation, or on burn victims; pills can be difficult to swallow if someone’s in respiratory distress. Dsuvia is formulated as a flat, blue tablet, smaller than a Tic Tac, and dispensed sublingually, meaning it dissolves under the tongue. Each tablet comes pre-loaded into a transparent, single-use plastic applicator, which will be terrible for the environment but means there should be no leftovers to make off with. There is only one dose size—30 micrograms, i.e. 30 millionths of a gram—which should mitigate the dosing errors that can occur when doctors do quick calculations under pressure, a problem Palmer said she founded AcelRx to address. Because painkillers tend to leave patients groggy and sedated, the DoD requested AcelRx perform a cognitive assessment to make sure patients would be clear-headed at the one-hour mark. Dsuvia performed well, though it’s not clear whether Dsuvia would have also performed well on a test conducted by an independent third party, nor whether anyone at the DoD ever actually followed up about the results. (When I asked the DoD if they’d reviewed the assessment and formed an opinion on it, they said that was a question best directed to AcelRx.) Unlike the lollipops, Dsuvia was studied in patients who hadn’t already built up a tolerance to opioids. But the lollipops have also long since checked all the same boxes Dsuvia now purports to—including being deemed safe for the battlefield.

Five out of the nine total doctors who authored the Dsuvia studies (excluding those affiliated with AcelRx) accepted upwards of $40,000 per year from drugmakers at least once since 2014. In 2017, three averaged more than $70,000. The national average per year for contributions to doctors is a little over $3,300.

Dr. Jacob Hutchins—who led the second open-label study—averaged more than $166,000 between 2014 and 2016. Almost all of it came from the manufacturer of a painkiller with which he was injecting patients in a manner the FDA had rejected, as reported by the Star Tribune. He was one of at least two study authors AcelRx sent to testify at the advisory committee meeting, which is a routine practice. Palmer declined to comment on the doctors’ compensation histories, but stated that they had the experience necessary to conduct the Dsuvia research and that the lead author of the ER study was not among those found to have taken excessive payments. Hutchins declined to comment on his own compensation history, but told me that while he would be open to using Dsuvia on his patients when appropriate, his hospital does not stock it currently. The DoD told me that, after checking, it had not been aware of any of the doctors’ records of industry payments.

“I don’t think that they really understand anything about this drug, I really don’t,” Brown said. “I think that this was about, ‘we’re gonna do this because we can.’”

Around 2010, the U.S. military began pushing the FDA to approve French freeze-dried plasma (FDP), a dehydrated powder that promotes clotting and helps slow blood loss. Unlike frozen plasma, which has to thaw before use and then expires rapidly, FDP is ready to go in minutes and can sit on a shelf, unrefrigerated, for up to two years. Frustrated by what it perceived as the FDA dragging its feet, in late 2017 the Pentagon tried to amend the Federal Food, Drug and Cosmetic Act so it could authorize unapproved drugs itself. It was a stunning power grab, the first time any government entity had attempted to go over the FDA’s head. A toned-down version of the bill was ultimately passed as a compromise, and by January 2018 the FDA and the DoD were in the early stages of their new partnership. FDP was authorized by July.

Even if Dsuvia works the way AcelRx and the DoD describe it rather than the way the supporting evidence describes it, it still cannot possibly treat or prevent any “life-threatening risk”—unlike, say, something that slows rapid blood loss—because it is still a painkiller, and short-term pain is still not a life-threatening condition. And despite all the fuss over whether Dsuvia was safe, or whether it worked, or whether its approval was above-board, short-term pain isn’t really a battlefield priority to begin with.

“Pain management has always been secondary. Like, when we’re doing our training obviously they teach it to us, but when you’re actually in the field, training with other soldiers who have done the work, your main priority isn’t whether [patients are] in pain—it’s to get them stable and then get them out to the next level of care,” said Richard Brookshire, an army combat medic from 2009 to 2016 who served in Afghanistan in 2011. “There isn’t anything else I would say that would have made the job easier. Though in the past there were things like this powdered, kinda like blood-clotting powder that they had had; that was really useful. I think there was some complications with it.”

Drug patent experts, prescription drug abuse experts, and emergency medicine doctors alike told me that the medical community is actively trying to back away from non-IV opioids; we have enough of them. 

Where we have real unmet need, if we’re talking about management of acute pain, is in our non-opioid options. There is still no real evidence to date that opioids work any better than Advil and Tylenol—even for acute pain, even in the ER. But opioids are addictive, which makes the pursuit of new uses for them a lucrative one. You will not find Big Pharma nor the DoD sponsoring research of exciting new uses for Tylenol, though from a public-health perspective—rather than a financial one—they probably should be.

The collaboration that birthed Dsuvia is not so much a partnership between two entities as it is the DoD telling the FDA what it wants and leaving the FDA to figure out how to make it happen. The FDA retains power of approval, but whenever the DoD puts something on the Priority List the FDA is now required to fast-track whatever it is as if it had received breakthrough designation—a channel formerly reserved for new drugs that treat serious conditions and for which the evidence shows them to be so exceptional, compared to what’s already available, that the normal review procedures are waived. The FDA is further required to meet repeatedly with the company developing the Priority List product in question and advise how to get it approved, plus file regular accountability reports “to ensure the timely development and approval of products of importance to DoD.” That the very first approval borne of this collaboration had by definition no business being there is an ominous sign that nobody’s confining the DoD to the actual parameters of its own legislation—that the DoD is a lot closer to the unilateral control it wanted than the compromise it supposedly got, and that if it oversteps its bounds it’s not expecting anyone to notice or care. 

Commercial rollout of Dsuvia began earlier in 2019. To date, the DoD has not purchased any units itself, but told me it intends to do so “upon deployment,” at which time it will be able to procure the drug at wholesale pricing—an arrangement with AcelRx that will supply Dsuvia at a discount to not just the DoD, but also the Department of Veterans Affairs. In the meantime, the DoD has created a Medical Product Acceleration Committee to “informally” manage the Priority List. The US Army Medical Command’s webpage for the collaboration states that questions about the Priority List “are not questions for FDA. FDA will be unable to answer these questions.” Instead, future “private collaborators” interested in “ensuring your product solution is given due MPAC consideration for placement on the DoD Priority List” are encouraged to contact the DoD directly.

[Kastalia Medrano is a journalist based in New York. She can be reached via Twitter @kastaliamedrano.]


Source URL: https://portside.org/2020-01-11/why-did-pentagon-push-fda-approve-powerful-new-opioid