Main roads
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Confidence in the US Food and Drug Administration is under attack after it rejects a new skin cancer treatment
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The resignation of FDA Commissioner Dr. Marty Macari also caused concern
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Executives in industries regulated by the FDA are frustrated by the agency’s guidance
SATURDAY, May 16, 2026 (NewsDay News) – US Food and Drug Administrationthe last decisionThe refusal to approve a new skin cancer treatment came down to doctors treating melanoma and patients who found the drug extended the lives of a third of clinical trial participants.
“It was devastating news,” he said Dr. Trisha Wise-Drapera University of Cincinnati dermatologist who enrolled patients in the trial.
“It’s probably life or death for 2,000 patients,” he added Dr. Eric Whitmanmedical director of oncology services at Atlantic Health System.
A Wall Street Journal editorialdecried the ruling, noting that it would “have a chilling effect on drug development”.
Despite the benefits to some patients, oncologists and pharmaceutical industry analysts say there are legitimate concerns about the treatment, called RP1, which may have the FDA reject it anyway. They noted that the company has ignored repeated suggestions from the FDA to change the trial design used to seek approval for the drug.
The FDA’s decision raised a few eyebrows before the current administration took over. But Dr. Marty Macariwho took over as commissioner 13 months ago, has changed the agency’s culture and damaged trust built over decades while regulating 20 percent of U.S. consumer spending, said Stephen Grossmanregulatory consultant and former health and human services official.
“People have to make assumptions about the standards and processes by which the agency makes decisions,” he said. “And that uncertainty is bad for everyone — patients, sponsors and investors.”
They have senior officials under Makari, who resigned on May 12or suppressedorpushed forwardsomedrug approval and politicsby order of the president Donald Trump or the Secretary of HHS Robert Kennedy Jr.not paying attention to the advice of agency specialists. In defending his actions, Macari often avoided the traditional language of measuring the agency’s decisions.
In response to criticism for rejecting a melanoma treatment, for example, Macari accused its maker, Replimune, of “corruption” and said it was “engaging in corporate spin” to make the FDA look bad.
“I don’t work for Replimune, I work for the American people,” Macari said in a televised interview on May 5. CNBC. Kennedy supported her during a congressional budget hearing in which Kennedy mistakenly stated that patients in the Replimun clinical trial also received chemotherapy.
Macari did not respond to requests for comment.
“All criteria have been thrown out the window, so we don’t know what the agency’s decision is.”Paul Kimformer FDA official and aide to the late US Senator. Edward Kennedy who is now a consultant to the pharmaceutical industry in Boston. “Even when there are legitimate scientific and regulatory reasons why a drug isn’t approved, we assume it’s legitimate reasons or just a political game.”
A cure for cancer
Melanoma It is the fifth most common cancer in the United States, accounting for approximately 112,000 new cases each year. The American Cancer Society projects thatabout 8500 people will dieWhitman said that if Replimune’s treatment, RP1, works well in a clinical trial, up to 2,500 of these patients could be saved.
RP1 is a genetically engineered virus designed to destroy tumor cells and alert the immune system to act against them. Replimune sought accelerated approval — a kind of shortcut that allows a product to enter the market while a larger confirmatory trial takes place — by presenting data showing that a third of the 140 people in the trial had their tumors shrink or disappear.
But the agency warned Replimune in July that it would decline the risk unless it changed its development plans. In particular, the FDA noted that the trial had no control arm to compare RP1 with an approved melanoma treatment. Instead, all patients were given RP1 in combination Opdivoa type of immunotherapy.
Replimune scientists don’t fully understand how the drug works, but research shows that in addition to killing cancer cells, it releases chemicals that stimulate Opdivo’s ability to stimulate the immune system. The company argued that giving Opdivo alone as a control arm would be unusual because all patients enrolled in the trial stopped improving when given Opdivo alone or other drugs in its class.
“Having a control arm would be unethical,” Wise-Draper said. Some of her patients have responded very well to RP1 and no longer have evidence of melanoma, she said.
Replimune currently has a larger trial that includes a control arm, but “the bigger question is whether the company will survive,” Whitman said. Fast-track approval by the FDA would have prompted investors to put up enough money to complete a large trial, he said.
Replimune did not respond to repeated requests for comment. Butthe company told reportersis laying off more than half of its employees and closing some operations after the FDA ruling.
RP1 was not the first melanoma drug to be approved based on a single-arm trial. KeytrudaMerck’s top-selling cancer drug was approved to treat melanoma about 12 years ago under a similar trial design. But in its disclaimer, the FDA said it was not convinced that the positive effects of the combination regimen were all due to RP1 and not in part to Opdivo.
Kim said that Replimune could find an ethical way to establish a control team for its treatment. On the other hand, the FDA could give them a “temporary yes” with quick approval, he said.
The whole point of the three-decade-old accelerated approval program is to “play games,” Kim said. The agency’s statement, emphasizing the company’s methodology as a result, “reconsiders how much sponsors can trust with similar studies,” he said.
Vinay Prasad’s last days at FDA
Much of the criticism of the FDA under Trump has focused on it Dr. Vinay Prasadwho was fired, was rehired last summer and held various leadership positions at the agency. Prasad, an oncologist known for criticizing the statistical foundations of research, has repeatedly intervened in drug and vaccine approval processes normally handled by lower-level FDA officials.
Prasad, who did not respond to requests for comment, resigned on May 1, three weeks after Replimune’s decision. “There is a lingering question as to whether this was Vinay’s last stand or the objective decision of careful scholars,” Kim said.
Macari has criticized Trump administration officials for various decisions, the latest of which was the refusal to approve flavored vapes for smoking cessation. Anti-abortion advocates wanted Trump fired for allowing a generic form of mifepristone on the market and for not speeding up research they hoped would lead to the abortion drug being pulled from the market.
But in areas regulated by the FDA, from gene therapy to vaccines to cancer, officials are frustrated by the agency’s uncertain direction. In previous administrations, the agency generally walked a tightrope between loosening and tightening drug approval requirements. Under Macari’s leadership, “it was moving in every possible direction,” Grossman said.
“It’s so inconsistent; it’s all over the place,” Whitman said. “Independence is part of the concern.”
During his tenure, Macari made a series of sweeping statements that either overstated the progress made during previous administrations or exaggerated the agency’s ability to make progress on goals.
For example, he set a goal for himselfend animal testingwhich is considered unexpected at the moment, said Kim and moved to itthey do it rudelyartificial intelligence in the FDA – premature, according to critics. Makari and Prasad also promised thatstandard number of clinical trials requiredtwo to one. FDA regulations require two well-controlled clinical trials to approve a drug, but exceptions to this rule are already common.
“The FDA is sending signals that it wants to further reduce the evidence required for drug approval,” he said. Dr. Aaron KesselheimHarvard Medical School professor and drug expert. “Of course, if we’re talking about vaccines, it’s the exact opposite. The FDA is taking real steps to make it harder to approve vaccines.”
The FDA laid off nearly 4,000 employees at the start of the Trump administration. Macari has promised to put thousands back to work, but given the developments at HHS and the FDA, those positions may be difficult to fill. “What magic trick does that?” Grossman asked.
“Unfortunately, there has been so much confusion at the FDA that this Replimune decision, which probably should have happened, has been mired in controversy,” he said. Evan Seigermanhead of healthcare research at BMO Capital Markets.
More information
Learn how a new drug is launched, reviewed, and ultimately approved or rejected Drugs.com.
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What does this mean for you?
A decision to deny approval of a new skin cancer treatment could affect the development of other new drug treatments.
