Category Archives: News Stories & Editorials

Two AstraZeneca Drugs To Be Scrutinized in First FDA Cancer Advisory Panel in 9 Months

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Tristan Manalac, BioSpace, March 9, 2026

The FDA’s cancer advisors will discuss AstraZeneca’s application for the oral SERD camizestrant in breast cancer and the AKT inhibitor Truqap in prostate cancer.

The FDA’s Oncologic Drugs Advisory Committee will meet at the end of April to discuss two of AstraZeneca’s cancer drug applications—one seeking approval in breast cancer and another requesting a label expansion in prostate cancer.

The panel of outside experts is scheduled to convene on April 30, according to a Federal Registry posting, breaking what will be more than nine months of silence for the Committee. The ODAC, as the board is more commonly known, last met in July 2025.

The April meeting will be a whole-day affair, according to the registry notice. The morning session will focus on AstraZeneca’s new drug application for the oral SERD drug camizestrant, in combination with a CDK4/6 blocker such as Pfizer’s Ibrance or Novartis’ Kisqali, for HR-positive, HER2-negative breast cancer in the first-line setting.

[….]

Despite acing its target outcomes, analysts at Leerink Partners expressed concerns about how SERENA-6 was designed. “Importantly, the trial does not answer if intervening earlier, leveraging serial diagnostics to detect the early emergence of [ESR1 mutations], provides a longer-term benefit to patients rather than front-loading a benefit that could have eventually occurred in a later line,” they wrote in a note to investors on Friday.

In addition, while oral SERDs have become the standard of care for second-line therapy in ESR1m breast cancer patients, SERENA-6 “did not have an oral SERD widely used in the 2L and crossover to [camizestrant] was not permitted.”

[….]

During the afternoon session on April 30, the FDA’s cancer committee will discuss AstraZeneca’s bid to expand its AKT inhibitor Truqap to treat metastatic hormone-sensitive prostate cancer. Data from the Phase 3 CAPItello-281 trial showed that the drug plus abiraterone significantly improved radiographic progression-free survival versus placebo plus abiraterone.

The Phase 3 CAPItello-280 study of Truqap was halted in April 2025, however, as an independent data monitoring committee concluded that a combination regimen of the drug with docetaxel and androgen-deprivation therapy would miss its primary endpoints of overall survival and progression-free survival in patients with metastatic castration-resistant prostate cancer.

The ODAC meeting in April comes amid mounting criticism of FDA panels, which according to experts, have grown increasingly unbalanced and devoid of nuance.

In a September 2025 interview with BioSpace, for instance, Diana Zuckerman, president of the nonprofit National Center for Health Research, blasted a July 2025 expert panel on the use of selective serotonin reuptake inhibitors in pregnancy, which she said put too much emphasis on the potential harms of these drugs and not enough on their benefits.

“They didn’t want any nuance. It seemed they didn’t want any real difference of opinion,” she said.

To read the entire article, click here Two AstraZeneca Drugs To Be Scrutinized in First FDA Cancer Advisory Panel in 9 Months – BioSpace

Prasad Out At FDA, Turning Critics’ Focus Back To Makary

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Jessica Karins and Maaisha Osman, Inside Health Policy, March 06, 2026

FDA’s polarizing and high-profile biologics chief Vinay Prasad is leaving the agency for the second time since his tenure began a year ago, and this time his departure may be permanent, after yet another controversy over rare disease approvals spilled into public view and cast new doubts on Prasad and Commissioner Marty Makary’s leadership. Makary’s next pick to run the biologics center is viewed as a make-or-break decision for his commissionership.

A House Energy & Commerce Committee Democrat who is vocal on FDA issues told Inside Health Policy the next goal for the agency’s critics could be ousting the commissioner. “Prasad is Makary’s man and Makary’s failure. Makary’s seat is hot and now we need a hearing to make it hotter,” Rep. Jake Auchincloss (MA) said.

In a social media post, Makary spun the departure as a planned return to Prasad’s family and academic committees after a one-year leave of absence from the University of California San Francisco, saying he has accomplished lasting reforms at the agency. Prasad had not previously mentioned plans to spend only a year at FDA.

The latest controversy for the Center for Biologics Evaluation and Research was its decision not to approve a Huntington’s disease gene therapy from UniQure, followed by anonymous statements from FDA officials to the media that left industry fuming. But that was one in a string of rare disease decisions under Prasad that angered industry and other FDA stakeholders. He also made several decisions on vaccines that were unpopular with regulated industry and public health stakeholders, including refusing to review Moderna’s mRNA influenza vaccine. He made headlines for claiming FDA had linked COVID-19 vaccines to multiple pediatric deaths, then never publicized promised data.

[….]

Many members of Congress have already criticized the agency under Makary’s leadership for what they see as inconsistent or inappropriate demands placed on rare disease drugmakers. Auchincloss said at an event earlier this week that Makary and Prasad should both be fired.

The dispute with UniQure centers around FDA asking the company to do a follow-up trial of its gene therapy that would require placebo surgery. FDA officials say the surgery isn’t risky or invasive; but UniQure and many Huntington’s disease patient advocates say it is. The anonymous FDA official said on the call with press UniQure’s original trial offered a “distorted or manipulated comparison,” and an HHS spokesperson told the Washington Post UniQure “lied” about what FDA wants from a second trial.

[….]

The dispute was viewed as not boding well for Prasad’s leadership at the biologics center. He had already left FDA for a short period once, amid controversy over a different gene therapy, before rejoining the agency. Another Makary appointee, George Tidmarsh, was accused of using his position to pursue a “bizarre personal vendetta” against a regulated company and left the agency permanently.

The backlash to the UniQure situation from rare disease stakeholders also isn’t a good sign for buy-in to FDA’s plausible mechanism pathway, which Makary has touted as evidence of a flexible regulatory flexibility but appears to offer more flexibility in theory than the agency is offering in practice.

Diana Zuckerman, president of the National Center for Health Research, told IHP Prasad’s departure from CBER represents a setback for efforts to strengthen scientific rigor in the agency’s regulatory decisions. She said Prasad brought a focus on scientific evidence that many researchers and policy experts have long argued has been missing at senior levels of FDA.

Zuckerman said that across several administrations, the agency has increasingly emphasized “customer service,” with industry too often treated as the primary customer rather than patients and public health. While she acknowledged that Prasad’s decisions and outspoken criticism of the agency drew opposition from industry groups, some rare disease advocates and even FDA staff, she said independent researchers have repeatedly raised concerns that FDA has approved costly medical products that are ineffective or unsafe.

In her view, the agency’s reliance on more “flexible” approval standards for products that lack clear evidence of benefit is contributing to rising health care costs and straining federal programs such as Medicare and Medicaid. “I think Dr. Prasad leaving is a loss for the FDA, for patients, and for Medicare and Medicaid,” Zuckerman said. 

To read the entire article, click here: https://insidehealthpolicy.com/inside-drug-pricing-daily-news/prasad-out-fda-turning-critics-focus-back-makary

Divisive F.D.A. Vaccine Regulator Is Resigning

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Christina Jewett, The New York Times (and syndicated at many other media outlets), March 6, 2026

Dr. Vinay Prasad, a polarizing figure at the Food and Drug Administration who oversaw vaccines, is leaving the agency at the end of April, according to a Health and Human Services spokesman.

As the agency’s chief science and medical officer, Dr. Prasad had wide-ranging authority over vaccines, drugs and gene therapies. He issued several controversial decisions, including overruling career scientists on some vaccine approvals and cracking down on a biotech company linked to two teenagers’s deaths.

In one of the most highly publicized moves, Dr. Prasad refused to accept Moderna’s application for a new mRNA flu vaccine, causing an uproar among companies and some experts who complained he was too often moving the goal posts on studies that had been OK’d by the agency. Within days, Dr. Marty Makary, the agency’s commissioner, reversed the decision after the company agreed to conduct another study.

And in recent months, he had issued a series of rejections for treatments of rare diseases, increasingly upsetting patients who have few options and biotech companies invested in developing cures.

Many of those decisions were made with little warning. He shied way from public advisory panel meetings on drugs under review, rebuffing calls for greater transparency. Dr. Prasad has criticized those forums, saying that the drug industry manipulated public opinion.

[….]

“[His]reforms represented a tremendous body of work achieved in a remarkably short period of time,” Dr. Makary wrote. “They are substantive, durable changes that will shape the agency’s approach for years to come and stand as part of Vinay’s lasting legacy here.”

Leaders in the biotech and investor communities had long pressed the White House for his ouster.

However, Diana Zuckerman, a close observer of the F.D.A. and president of the nonprofit National Center for Health Research, said Dr. Prasad’s decision to leave was a loss to independent researchers who had “hoped he would help strengthen the public health mission of the F.D.A.”

“F.D.A.’s ‘flexible’ standards for approving products that are not proven to work are resulting in an unsustainable health care system,” she said, noting the cost of failed therapies to Medicare and to patients.

Before joining the agency, Dr. Prasad was an academic at the University of California, San Francisco. He was known for criticizing the F.D.A., saying its drug review officials were too permissive in issuing approvals.

He has also been described as a Covid contrarian after complaining on podcasts and on his YouTube channel about public health measures that he deemed ill-informed by medical evidence.

But he and Dr. Makary broke with Mr. Kennedy on whether pregnant women should get the vaccine, including pregnancy as a condition warranting vaccination, in an article in The New England Journal of Medicine.

[….]

Last summer, after a second teenager died from liver-related complications from a therapy for Duchenne muscular dystrophy, Dr. Prasad demanded that the manufacturer, Sarepta, stop distributing the drug. The company pushed back, and the agency settled on keeping the drug from older children who were more likely to be harmed by it.

[….]

The Department of Health and Human Services held a background briefing on Thursday about the latest controversial decision, against a treatment for Huntington’s disease.

[….]

In a social media post Friday evening, Dr. Makary said that Dr. Prasad would be resuming his work in California and that a successor would be announced before he leaves.

To read the entire article, click https://www.nytimes.com/2026/03/06/health/fda-prasad-resigns.html?nl=breaking-news&segment_id=216275

Top drug regulator Richard Pazdur set to leave the FDA

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STAT News
December 2, 2025. Lizzy Lawrence

WASHINGTON — Top drug regulator Richard Pazdur has filed papers to retire from the Food and Drug Administration at the end of this month, adding to the turmoil atop the agency.

Pazdur informed leaders at the FDA’s drug center of his intention to leave the agency at a meeting on Tuesday, according to two agency sources familiar with the matter. The move comes less than a month after he took the role of top drug regulator at the urging of FDA Commissioner Marty Makary.

A spokesperson for the FDA confirmed the news and said Pazdur’s decision is final. Pazdur did not respond to a request for comment.

“We respect Dr. Pazdur’s decision to retire and honor his 26 years of distinguished service at the FDA,” the FDA spokesperson told STAT. “As the founding director of the Oncology Center of Excellence, he leaves a legacy of cross-center regulatory innovation that strengthened the agency and advanced care for countless patients.”

Pazdur is the fourth person to lead the Center for Drug Evaluation and Research this year. The previous director, George Tidmarsh, left the agency in November amid allegations that he used his position to advance a personal vendetta against a business associate, and amid conflict with Center for Biologics Evaluation and Research Director Vinay Prasad.

Three sources close to Pazdur told STAT that concerns about the legality of a new drug review program, Makary and Prasad inserting themselves into drug review decisions, and Makary’s efforts to handpick hires for CDER contributed to Pazdur’s decision to leave.

The elevation of Pazdur, a longtime FDA staffer who was previously the director of the cancer center, was intended to stabilize the agency. Biotech stocks gave up gains after STAT reported on Pazdur’s plan, with the S&P Biotech ETF sliding 1%.

“Really bad news here,” John Maraganore, the founder of Alnylam Pharmaceuticals, posted on social media. “Rick Pazdur has been a National Treasure for efforts in bringing safe and effective cancer medicines to patients.”

[….]

Even patient advocates who sometimes disagreed with Pazdur’s decisions to approve certain cancer drugs expressed dismay at the news.

“Dr. Pazdur’s retirement is a huge loss for the FDA and for patients across our country,” said Diana Zuckerman, president of the National Center for Health Research. “Many of us appreciate that he was willing to express his concerns about FDA’s recent plans to speed up the approval process at a time when FDA had lost staff and expertise.”

Pazdur took the job on assurances that he would be protected from political influence, sources close to him previously told STAT. Political pressure has increasingly affected science at the agency, especially for products of particular interest to health secretary Robert F. Kennedy Jr., like vaccines, antidepressants, and autism treatments.

But it seems that Pazdur ran into issues almost immediately. The Washington Post reported that Pazdur raised concerns about a new program spearheaded by Makary to offer expedited review to drugs that align with national priorities. He noted that the program, called the Commissioner’s National Priority Voucher, was not transparent and could be illegal. Tidmarsh also raised concerns about the program on his way out of the FDA.

[….]

Experts told STAT that the personnel drama at the FDA threatens to undermine the credibility of the agency, which is charged with protecting and promoting public health.

To read the entire article, click here 

The FDA Commissioner Is Missing the Point of Advisory Committees

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Makary’s hand-picked panels lack diversity of opinion, robust evidence reviews, and credibility

by Diana Zuckerman, PhD, November 22, 2025

Zuckerman is the founding president of a nonprofit think tank, and a former Congressional Committee staffer who investigated FDA policies.

The recent controversies about the safety of antidepressants during pregnancy and hormone therapy for menopause were heightened by how decisions are being made. Earlier this month, HHS officials announced that boxed warnings would be removed from hormone therapy drugs to treat symptoms of menopause, several months after a group of 12 experts handpicked by FDA Commissioner Marty Makary, MD, MPH, recommended removal of the warnings.

Makary asserted that traditional FDA advisory committees — which have historically been convened before making such a change — are outdated because they are “bureaucratic, long, often conflicted, and very expensive.” He said he plans to instead rely more on his expert panels, which he described as more “spontaneous” but still “robust.”

There are many differences between advisory committees and the “expert panels” held this year. As someone who has participated in hundreds of FDA advisory committee meetings and watched several of these expert panels, I believe Makary is missing the point and undermining the credibility of FDA decisions.

What Is the Purpose of Advisory Committees?

FDA advisory committee meetings are scheduled for drugs, devices, or tobacco products when there is conflicting evidence or a contentious difference of opinion within the agency or between the agency’s point of view and other interested parties (such as the company that makes the product).

Typically, the meeting focuses on whether a new drug or device merits FDA approval, whether there is persuasive evidence that an approved product is less safe or effective than FDA previously concluded, or if a type of product may require more/less rigorous regulatory standards than currently required.

Most meetings are around 8 hours long (although this varies): approximately 2 hours for the company to provide evidence on safety and efficacy; 1-2 hours for FDA scientists to express their views of the evidence; 1 hour for an “open public hearing” for patients, family members, or health professionals or organizations to express their views; and 3 hours of advisory committee questions, discussion, and voting. A full meeting transcript is made publicly available afterwards and typically the meetings are all livestreamed now as well.

The members typically include about a dozen medical experts (mostly clinicians) who do not work for a federal agency; at least one patient or consumer representative; and one industry representative (non-voting). Many serve on the committee for several years after being nominated and selected based on their medical specialty; at each meeting, a few additional experts are added because of their relevant expertise. FDA staff are not on the committee, but NIH or other agencies may have one or two non-voting members with relevant expertise.

Weeks before the meeting, advisory committee members are provided with the company’s description of their evidence (often 100+ pages long) and the FDA’s detailed critique of that evidence. These documents, the agenda, names and affiliations of committee members, and information about possible conflicts of interest are made publicly available on FDA.gov 48 hours in advance and stay online for years afterwards.

How Are Expert Panels Different?

Compare that to a 2-hour expert panel meeting, comprised of approximately 8 to 12 experts selected by the FDA, each speaking for a few minutes and answering softball questions asked by the FDA commissioner or other official. The list of speakers is not necessarily released in advance (perhaps in part due to the “spontaneous” nature of the panels), no written documentation regarding the evidence has been provided to the public, and the panel members’ presentations appear mostly consistent with the commissioner’s opinion. The public can attend in person or remotely but can’t ask questions or express their views.

One unfortunate trait that the advisory committees and expert panels have in common is that some members are less “expert” than others. Many are clinicians with limited understanding of statistics or the implications of the FDA regulatory process. Meanwhile, the academic physicians participating do not acknowledge that many clinicians who write these prescriptions may rely on drug and device representatives for information rather than regularly reading studies in medical journals or closely studying very long drug labels.

Another unfortunate trait they have in common is conflicts of interest. Financial ties to industry are discouraged but still exist and are not always disclosed. Both panels include clinicians whose quoted statements at these meetings may influence their salaries by generating positive or negative publicity. An additional possible conflict unique to researchers on expert panels is the assumption that pleasing HHS officials could improve their chances of future federal funding or recognition.

Shortcomings of Advisory Committees

To be fair, during advisory committee meetings, some committee members appear to have not read most of the meeting materials provided in advance, and it sometimes seems the FDA has “stacked the deck” by cherry-picking committee members. Also, the votes may at times be unanimous even when numerous members express great concerns about the product. For example, I once heard a committee member share substantial doubts and then say, “I certainly would not want my mother to take it” — but he voted in favor of the product anyway.

In addition to companies’ hours-long presentations, many recruit and train public comment speakers. Most are patients desperate for a cure who may not realize the product performed no better than placebo. Their stories are often tragic and compelling, and numerous advisory committee members have told me they voted in favor of a product of questionable efficacy because they didn’t want to seem indifferent to the patients’ plight.

As President Biden’s FDA commissioner, Robert Califf, MD, openly expressed concerns about FDA advisory committee meetings. In a meeting with several nonprofit leaders, he made it clear to us that he did not like it when FDA was criticized for approving a product that the advisory committee had voted against. At the end of the day, the advisory committees serve as just that: advisors. They have not been a rubber-stamp; although in the future, FDA may try to take them in a different direction. But the value of this public forum for discussion and transparency cannot be overstated.

Don’t Let Perfect Be the Enemy of Good

Advisory committees are not perfect, but I’ve never seen one that lacks a diversity of opinion. FDA scientists almost always raise important concerns that help committee members consider the risks and benefits of products and strengths and weaknesses of the research evidence.

In contrast, during the expert panel on hormones for menopause, the commissioner and the panel cited studies that, in several cases, were outdated, based on rodents rather than women, or included too few patients or were too short-term to provide meaningful evidence. There were no public speakers and no panel members willing to challenge those flawed research citations, so reporters under tight deadlines had little opportunity to include expert opinions that conflicted with the preordained message of the meeting.

Important FDA decisions deserve scientific scrutiny, challenging questions, differences of opinion, and nuanced summaries. These have, to date, been lacking in the FDA expert panels, making critical information less available to patients and their physicians.

Diana Zuckerman, PhD, is president of the National Center for Health Research in Washington, D.C. She is a former Congressional committee staffer who investigated FDA policies.

You can read the original MedPage Today article here.

Healio: HHS revises hormone therapy black box warning for menopause treatment

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By Emma Bascom, Michael Monostra, and Andrew Rhoades, November 10, 2025

HHS has announced changes to estrogen hormone therapy black box labeling for menopause treatment in a move that it said corrects misleading warnings.

HHS Secretary Robert F. Kennedy Jr. said in a press conference on Monday that women have been “told to fear the very therapy that could have given them strength, peace and dignity through one of life’s most difficult transitions, menopause,” but “that ends today.”

“We’re challenging outdated thinking and recommitting to evidence-based medicine that empowers rather than restricts,” he added. “When prescribed responsibly and started early, hormone replacement therapy transforms the lives of women.”

The details

The role estrogen replacement should play in menopause treatment has long been controversial. Concerns about menopausal hormonal therapy stem back to the publication of findings from the Women’s Health Initiative in JAMA in 2002.

The study found women receiving estrogen plus progestin had a significantly increased risk for total CVD, stroke and pulmonary embolism, and a nominally increased risk for breast cancer. The trial was stopped early due to “health risks that exceeded health benefits over an average follow-up of 5.2 years,” and its results led to a large decrease in the use of hormone therapy among women.

In July, the FDA convened an expert panel to discuss the safety and efficacy of menopausal hormone therapy, and some members stated the boxed warning should be changed to better reflect the published evidence. In the press conference, FDA commissioner Martin A. Makary, MD, MPH, said the move is based on “a robust review of the latest scientific evidence” and the July panel.

“After 23 years of dogma, the FDA today is announcing that we are going to stop the fear machine steering women away from this life-changing, even life-saving, treatment,” Makary said. “We are also approving two new drugs for the treatment of menopausal symptoms. We are listening to doctors who have been waving the flag in the air saying, ‘Hey, we have this wrong.’”

[….]

In a viewpoint published today in JAMA, Makary and colleagues wrote that the revisions to the labels “signal a meaningful shift toward more nuanced, evidence-based communication of hormone therapy risks — one that prioritizes clinical relevance, distinguishes between different formulations and patient populations, and balances the narrative to reflect both safety and therapeutic value.”

According to the viewpoint, the label updates specifically include:

  • removing boxed warnings for CVD, breast cancer, probable dementia and stroke, but not the warning in systemic estrogen labels for endometrial cancer with unopposed estrogen, since “it is important to remind health care practitioners and patients that this serious risk can be mitigated by adding a progestogen”;
  • removing the recommendation that hormone therapy be prescribed at the lowest effective dose for the shortest possible duration, since “treatment decisions are individualized and fall within the clinical judgment of a clinician in discussion with a patient”;
  • updating timing information to include new guidance on treating women aged younger than 60 years or within 10 years of menopause onset “to optimize the benefit-risk balance”; and
  • tailoring safety information that reflects the risks most relevant to each specific type of hormone therapy product.

[….]

According to Makary and colleagues, current evidence suggests that starting hormone therapy within 10 years of perimenopause onset has long-term health benefits. It has been associated with a reduced risk for fatal cardiovascular events, bone fractures cognitive decline and Alzheimer’s disease, they wrote.

Expert insight

In a statement, Steven J. Fleischman, MD, MBA, FACOG, ACOG president, commended HHS, saying “the modifications to certain warning labels for estrogen products are years in the making, reflecting the dedicated advocacy of physicians and patients across the country.”

[…]

The Menopause Society also released a statement saying it agrees with the decision since “the boxed warning may have been a deterrent to the use of the low-dose vaginal estrogen, which is a safe and effective therapy for a condition that affects most menopausal women.”

“However, systemic estrogen still comes with potential risks that should be reviewed in detail with women initiating therapy,” the organization said. “Risks are greater when initiated in older women and in those who are further from menopause onset. Medical comorbidities, personal and family histories, symptoms, and personal preferences all need to be considered and reviewed with patients considering the use of hormone therapy for management of menopause symptoms or prevention of bone loss.” 

Diana Zuckerman, PhD, the president of National Center for Health Research, told Healio that “the warnings on hormone products for menopause had become outdated and it was time to update them.”

“However, these products still have clear risks, and the benefits are mostly for hot flashes and related symptoms,” Zuckerman said. “They are not the fountain of youth that some people claim, they absolutely are not proven to prevent dementia, and they are definitely not safe for everyone.”

Barbara DePree, MD, NCMP, MMM, director of the Women’s Midlife Services at Holland Hospital in Michigan, told Healio that the decision will likely increase hormone therapy uptake, which is “a win.” Yet she wondered how exactly HHS will tailor the safety information to reflect the most relevant risks for each type of hormone therapy.

“I’m not opposed to that, I’m just curious what data they’ll be using to tailor that information,” she said. “When the FDA panel happened, it felt like some of the benefits were cherry-picked from data.”

DePree was also concerned about claims that hormone therapy can prevent some very serious noncommunicable diseases.

“I’m fine with maybe taking some of the blinking red lights out of it. That gives women some more ease,” she said. “But I also think we’re not in a position to promote its use to prevent some conditions that we just don’t have the data to support, and I’m worried that it’s headed in that direction. With social media and what’s being overstated, I think this will maybe be another step in that direction, which is a little concerning.”

But she said this will likely give providers more confidence in offering the therapy as a safe alternative.

“We’ve had a whole generation of providers who’ve only grown up with data from the WHI, which basically put it completely in a risk category, and I think this brings it back to the middle for those people who’ve not really had the confidence or the understanding to properly screen women who can very safely and appropriately be using hormone therapy,” De Pree said. “We just don’t want it to say it’s the right option for every woman or just because it’s available everybody should do it because we’re going to save brains and hearts because I just don’t think we have the data to support that.”

Nanette Santoro, MD, professor and the E. Stewart Taylor Chair of OB/GYN at the University of Colorado School of Medicine told Healio that the changes “are a positive step for menopausal women and their physicians,” but was “a bit surprised the black box was completely removed.”

“The changes reflect current data, and it’s great that the route and types of hormones will be dealt with separately as the risks and benefits of estrogen alone compared to estrogen plus progestin are different. These changes should reduce physician anxiety about prescribing hormones, which is important because they are under-prescribed in general,” Santoro said. “And best of all, removing the black box from topical vaginal estrogen is a big relief, because the tiny incremental increase in circulating estradiol that occurs when women use these preparations should not be compared to the systemic dosing regimens and should not be considered to have the same risk.”

However, “some assumptions and statements made here that go way beyond the data that we have available,” which “raise a bunch of red flags for me.”  

“While hormone therapy can indeed be life-changing for symptomatic women, there is not randomized clinical trial evidence that it reduces heart disease, or immune and cognitive decline. There just isn’t,” Santoro said. “And when hormone therapy prescriptions abruptly dropped after the primary WHI publication in 2002, the only population health change that was seen, when hormone therapy went from about 25% of women to less than 2%, was a decrease in breast cancer mortality. There has not been any kind of massive morbidity or mortality as a result of this drop in hormones, although many women suffered with symptoms unnecessarily because of the fear.”

Overall, she said it is “a very good thing that women don’t have to try to decipher the estrogen black box warning (which includes the phrase ‘probable dementia’ and which confuses my patients who read it because they think it means they will probably get dementia if they take estrogen!) and their prescribing clinicians don’t have to have excessive fear of doing harm.”

“[But] the rebirth of the concept that estrogen and hormone therapy are a fountain of youth for women and will extend their lives feels a whole lot like a sharp swing of a pendulum back into the ‘feminine forever’ days.”
[….]

To read the entire article, click here 

Politico Prescription Plus: FDA Removes Black Box HRT Warning

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Politico Prescription Plus

BY DAVID LIM AND LAUREN GARDNER, OCTOBER 11, 2025

MAKARY’S PRIORITY — In the Hubert H. Humphrey Building’s atrium in front of a crowd of HHS officials, doctors and journalists Monday, FDA Commissioner Marty Makary declared the agency is
urging companies to remove black box warnings for hormone replacement therapy for menopausal women.

[….]

“With few exceptions, there may be no other medication in a modern era that can improve the health outcomes of women on a population level than hormone replacement,” Makary said. “After 23 years of dogma, the FDA today is announcing that we are going to stop the fear machine steering women away from this life-changing, life-saving treatment.”

But the push to remove the warnings of breast cancer, cardiovascular disease and possible dementia isn’t clear-cut, and the process will likely take several months. Makary said the updated package inserts accompanying the treatments will include a nuanced discussion of the recommended ages for HRT use.

An HHS fact sheet released Monday says the FDA will advise women to start hormone replacement therapy within 10 years of menopause or before 60 years old for use of systemic HRT, which delivers the hormone throughout the body instead of a single area. And the FDA is not seeking to change its black box warning for endometrial cancer for systemic estrogen-alone products.

The background: The Women’s Health Initiative — a National Institutes of Health-backed study that looked at the treatments’ benefits and risks — halted a clinical trial in 2002 after researchers said breast cancer, blood clot and stroke risks outweighed the treatments’ benefits in relieving menopausal symptoms. The action prompted many women to stop the therapy.

But Makary slammed the study, saying it was misrepresented and created a “fear machine” around HRT for menopausal women. He argued the study did not show a significant link to breast cancer.

Diana Zuckerman, president of the National Center for Health Research, said it is “well established” that menopausal hormone therapy can increase heart disease and cancer risks in some circumstances.

“I am glad that the Commissioner admitted that there are risks of some types of hormone therapy for some women, but his claim that hormones for menopause is the best way to improve the health of women sounds like a PR statement,
not a scientific one,” Zuckerman said in an email.

Makary dinged advisory committees on Monday as “bureaucratic,
long and often conflicted, and very expensive” when asked why the

FDA did not opt to convene a panel of its outside advisers before making the recommendation.

[….]

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AFP: US to remove warnings from menopause hormone therapy

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AFP (Agence France Presse); November 11, 2025

Hormone Replacement Therapy (HRT) replaces estrogen that the female body stops producing during menopause with the aim of alleviating symptoms including hot flashes, brain fog, insomnia, night sweats and joint pain.

Previously used routinely, prescription and use of the therapies have plummeted worldwide since a landmark trial in the early 2000s pointed to risks associated with specific HRT formulations.

Since then “black box warnings” — the strongest warning the US Food and Drug Administration can require on prescription drugs — have sounded alarm over increased HRT risks including of certain cancers, cardiovascular conditions and probable dementia.

But critics have pointed to flaws with the early 2000s Women’s Health Initiative, whose trials were halted as risks appeared: namely it focused on women who were a decade-post-menopause and in their 60s, when cardiovascular risks increase regardless.

Today guidance generally indicates that healthy newly menopausal or perimenopausal women — people broadly in their 40s or 50s — are among potential candidates for treatment.

There also are newer, more localized or lower-dose forms of the therapies available.

“We’re challenging outdated thinking and recommitting to evidence-based medicine that empowers rather than restricts,” US health chief Robert F. Kennedy Jr said in introducing the measure.

Many experts had urged revisiting the black box label, which they say can scare women for whom benefits may outweigh risks.

Others have voiced concern that changes shouldn’t come without a rigorous review process.

“The warnings on hormone products for menopause had become outdated and it was time to update them,” said Diana Zuckerman, president of the nonprofit National Center for Health Research.

But she told AFP “these products still have clear risks and the benefits are mostly for hot flashes and related symptoms of menopause, not for general health.”

FDA head Marty Makary dismissed that notion of an independent review committee, saying they are “bureaucratic, long, often conflicted and very expensive.”

Over the summer Makary convened a panel of experts overwhelmingly in favor of HRT, which included people with ties to pharmaceutical lobbying.

Adriane Fugh-Berman, who directs a project that promotes rational prescribing at Georgetown University, told AFP that Monday’s announcement was “embarrassing” as it was ahead of any consensus and was “not how regulation should happen.”

There could be benefits of HRT for some people, she told AFP, but cautioned that real risks remain, and more high-quality study is needed.

But the president of the American College of Obstetricians and Gynecologists, Steven Fleischman, commended the FDA’s move, saying “the updated labels will better allow patients and clinicians to engage in a shared decision-making process.”

[….]

The FDA said it is not seeking to remove the boxed warning for endometrial cancer for systemic estrogen-alone products.

[….]

To read the entire article, click here 

FDA ‘serious’ about hormone therapy changes

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LAUREN GARDNER and DAVID LIM, Politico, October 28, 2025

OUT OF THE BLACK BOX? The FDA could announce as soon as this week a change to the black box warning on estrogen products used to treat menopause symptoms — a policy move that would be the first to stem from Commissioner Marty Makary’s “expert panel” series.

Makary told CNN’s Sanjay Gupta on a podcast earlier this month that the FDA was having “serious discussions” about the warning with changes coming “very soon.” He focused a chapter of his 2024 book “Blind Spots” on an NIH-backed hormone therapy clinical trial that was halted in 2002 amid concerns about cancer and stroke risks, blasting the study’s administrators for misinterpreting the data.

It’s unclear how far the agency will go, according to advocates monitoring the issue. Some of the speakers at Makary’s July panel discussion are medical advisers to a group petitioning the FDA to remove the boxed warning from vaginal estrogen products, arguing that it deters providers and patients from using them.

[….]

At issue: The FDA could opt to remove the warning for topical, nonsystemic estrogen creams or ointments that treat dryness and other genitourinary symptoms that some women experience while going through menopause.

Existing evidence suggests those localized low doses are safe, though Diana Zuckerman, president of the National Center for Health Research, said there’s a dearth of data from long-term controlled trials. Any changes to the black box warning should distinguish between local and systemic therapies, the latter of which she said pose some risks depending on the timing and duration of treatment and the patient’s health profile.

“There’s a lot of nuance in the research,” said Zuckerman, who criticized the July panel as being skewed in favor of hormone therapy. “But when it is presented as if there is no risk here of cancer or heart disease or anything else, that is just completely untrue.”

The opportunity: But Anne Fulenwider, co-founder and co-CEO of telehealth platform Alloy, said removing the warning will help “millions and millions of women” gain greater access to those therapies.

“Even if it is [removed from] just one of them, that is a huge step for all women,” Fulenwider said. “Everyone is afraid of estrogen to their detriment.”

[….]

To read the entire article, click here https://www.politico.com/newsletters/prescription-pulse/2025/10/28/fda-serious-about-hormone-therapy-changes-00623982

New Blood Tests for Early Cancer Detection Get Some Love From House Members

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 Joyce Frieden, MedPage Today, September 18, 2025

House members seemed generally supportive Thursday of bills that would expand access to “breakthrough” medical devices, although Democrats complained that the focus on the topic was misguided at a time when the Trump administration and Congress are cutting funding for research on cancer and other diseases.

“We continue to fiddle in this subcommittee while Rome burns,” said Rep. Diana DeGette (D-Colo.), ranking member of the House Energy & Commerce Health Subcommittee, during a hearing on “Examining Policies to Enhance Seniors’ Access to Breakthrough Medical Technologiesopens in a new tab or window.” “We should be talking about the cuts to the NIH, FDA, CDC, and our nation’s other critical healthcare agencies. The committee should be examining directives from the administration that have delayed or completely halted critical work, and all of us should be talking about the impact this is having on our constituents.”

Rep. Marc Veasey (D-Texas) agreed. “Our healthcare system is being undermined right now in front of us, and American leadership and medical innovation, I believe, is on the line,” he said. “The [HHS] secretary has proposed cutting NIH funding by nearly half, and that will drag us back to 2007 levels. He’s pulling the rug from under researchers who make cancer breakthroughs possible, who run the clinical trials, and train the next generation of scientists. And those cuts are going to mean slower progress and higher costs and more Americans dying while waiting for cures that may never come.”

One of the bills discussed extensively at the hearing was the Nancy Gardner Sewell Medicare Multi-Cancer Early Detection Screening Coverage Actopens in a new tab or window, which would require multi-cancer early detection screening tests to be covered by Medicare relatively quickly upon FDA approval. The bipartisan measure currently has 304 co-sponsors.

Subcommittee members heard from patient advocate Roger Royse about his experience with a blood test that can detect up to 50 different kinds of cancers. “In June of 2022 I took a … multi-cancer early detection test,” Royse said. “I had no symptoms … I thought I had no risk factors, but I did have one really big one, and that’s age — I was 62 years old at the time. The test came back positive … and within a couple weeks, I was diagnosed with stage IIb pancreatic cancer. At that time, the 5-year survival rate for pancreatic cancer was 12%; it’s currently 13%.” However, “as it turns out, mine was caught in an early stage and was localized, meaning that my survival rate instead of 13% was 44%,” he added.

[….]

fSubcommittee member Rep. Neal Dunn, MD (R-Fla.), a surgeon specializing in advanced prostate cancer, co-sponsored the bill. “The status quo for cancer detection in America today is simply unacceptable,” he said. “Each day, more than 1,400 Medicare beneficiaries receive the devastating news that they have advanced-stage cancers. Further, over 70% of cancer tests occur from cancer for which there is no routine screening. This demands our attention.”

“While practicing I certainly experienced firsthand the difference between early-stage and late-stage cancer diagnosis,” he said. “Simply put, when it’s caught early, patient outcomes are dramatically better. [This bill] offers us a chance to do just that.”

But not everyone at the hearing was completely on board. “As a cancer survivor, I appreciate that the goal of [this] bill is to save lives,” said Diana Zuckerman, PhD, president of the National Center for Health Research. “Multi-cancer early detection tests are so promising, but they’re not quite ready for prime time yet. The most recent research — in a study that just came outopens in a new tab or window this week — has concluded that the existing tests are subject to bias, miss most early cancers in people who do not have symptoms, and may provide false positives to most patients. In one of these tests, test results indicating cancer was correct only 4% of the time.” 

“I agree with the article on the American Cancer Society websiteopens in a new tab or window that a test with many false positives, where many patients who are told they may have cancer do not have cancer, causes anxiety and results in additional testing that may be painful, harmful, expensive, time consuming, and stressful,” she added. “A test with many false negatives, in which patients are told that they do not have cancer when they actually do have cancer, is likely to result in patients who ignore signs and symptoms of cancer and thus delay needed treatment.”

Another bill discussed at the hearing was the Expanding Access to Diabetes Self-Management Training Actopens in a new tab or window, which would allow allied health professionals to provide the self-management training, in addition to physicians. It also specifies that Medicare coverage includes an initial 10 hours of training as well as an additional 2 hours of training per year. The bill also prohibits CMS from limiting training that is deemed medically necessary.

Several subcommittee members spoke in favor of the measure, including DeGette, who added one caveat. “[This] is a great bill to help Medicare beneficiaries with diabetes take better charge of the disease,” she said. “As the co-chair of the diabetes caucus, I love this bill, but meanwhile, the Trump administration has proposed to eliminate the National Diabetes Prevention Program at CDC, a program that is proven to help people with pre-diabetes avoid progression to type 2 diabetes through lifestyle changes.”

The bills must be approved by the subcommittee before moving to the full Energy & Commerce Committee for a vote; those that pass will then be sent to the full House to be voted on.

To read the entire article, click here