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Oral Testimony of Dr. Diana M. Zuckerman, Examining Policies to Enhance Seniors’ Access to Breakthrough Medical Technologies

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September 18, 2025, Energy & Commerce Subcommittee on Health 

Chairman Griffith, Ranking Member DeGette, Chairman Guthrie, Ranking Member Pallone, and distinguished members of the Committee, thank you for the invitation to testify. I’m Dr. Diana Zuckerman, president of the National Center for Health Research (NCHR), a nonprofit think tank that uses research to improve the quality of medical care in the United States.  

I was previously a faculty member at Vassar and Yale, a research director at Harvard, and most important, a Committee staffer in the House and Senate. 

Both the Breakthrough Devices Act and the Nancy Gardner Sewell Act require Medicare coverage for specific medical devices.  Keep in mind that the standard for Medicare coverage is “reasonable and necessary” for Medicare patients. FDA safety and effectiveness standards are not specific to Medicare-age patients. 

FDA requires prescription drugs be proven safe and effective, almost always based on patients in clinical trials. In contrast, FDA requires less than 5% of medical devices to be tested in clinical trials, while more than 95% go through a less stringent review called the 510(k) pathway. Those devices are cleared for market if the device is “substantially equivalent” to a device that’s already on the market, even though that older device also wasn’t required to be proven safe or effective in a clinical trial. 

Medicare almost always pays for prescription drugs approved by FDA. It does not pay for all medical devices, especially if there are no clinical trials or evidence that they are safe or effective for Medicare patients. 

When I served on CMS’ Advisory Committee that recommends whether products should be covered by Medicare, I saw many devices were not covered because they had never been studied on Medicare-age patients. Age is important for implants and many other devices because the older we are, the more likely we are to have chronic health conditions that make surgery, anesthesia, and other treatments riskier. 

Devices are designated as breakthrough before scientific evidence is available, based on the FDA’s belief that the device will be more effective than any other available devices. But if completed studies show the device is not more effective than other devices, it will still be sold as breakthrough devices.

Most of the 160 breakthrough devices available in the U.S. are therapeutic devices to treat a disease or condition. Thirty-eight percent of these treatment devices cleared the 510(k) review as substantially equivalent to devices already on the market.  Of the 34 distinct 510(k) devices that are relevant to patients 65 and over, 10 (29%) were studied in clinical trials that were listed in ClinicalTrials.gov, although the law requires these types of studies to be listed on that website.  

Most of those 10 devices have not yet publicly reported whether any of the patients studied were 65 and older. It is impossible to determine how many of those 10 devices included studies of sufficient numbers of patients ages 65 and older to be able to conclude that they would be considered reasonable and necessary for Medicare coverage.  

The other 55% of breakthrough 510(k) devices either had nonclinical data such as animal or mechanical data or had some kind of clinical data that was not a clinical trial. While some of those studies may provide very useful information, it will not be possible to conclude the data are relevant to patients over 65, who often have greater risks and fewer benefits from medical interventions. 

59 other breakthrough treatment devices went through the more stringent PMA or De Novo reviews and many were studied in clinical trials. However, numerous studies were small and had no placebo or comparison group.  Many studies did not include Medicare-aged patients or have not yet publicly reported the number of patients studied who were 65 or older. 

Nancy Gardner Sewell Medicare Multi-Cancer Early Detection Screening Coverage Act

As a cancer survivor, I appreciate that the goal of the Nancy Gardner Sewell Screening bill is to save lives. Multi-cancer early detection tests are promising, but they’re not ready for prime time. The most recent research 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.[2] In one of the tests, a test result indicating cancer was correct only 4% of the time.

  A test with many false positives, where most 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. A test with many false negatives, in which patients are told they do not have cancer when they actually do, is likely to result in patients who ignore signs and symptoms of cancer, thus delaying needed treatment.  

NCI recently launched the Vanguard Study of 24,000 people ages 45-75. The goals are to determine how accurate these tests are and whether any of the tests save lives. As stated in the study in Annals of Internal Medicine, it is unclear whether MCED tests “detect cancer types at later, untreatable stages or whether they detect very early-stage precancerous lesions that might never have developed into cancer.”

If in the future these tests are proven to have benefits that outweigh the risks for Medicare patients, then I strongly urge that the age restrictions be deleted from the bill. Age restrictions set a dangerous precedent for Medicare coverage decisions and would cause an uproar among patients who are excluded from coverage for reasons that are not scientific and will be perceived as unfair.

Conclusions

Both these bills are intended to help Medicare patients by increasing their access to medical devices. However, in both cases there is a lack of evidence that determines which of these devices have benefits that outweigh the risks for Medicare patients.  The goals are wonderful but requiring Medicare to pay for devices not proven to help its beneficiaries is not the best way to help patients. 

  1. Zuckerman D.M., Brown P. & Das A. (2014) Lack of Publicly Available Scientific Evidence on the Safety and Effectiveness of Implanted Medical Devices, JAMA Internal Medicine, 174(11): 1781-1787.  
  2. Kahwati, L. C., Avenarius, M., Brouwer, L., Crossnohere, N. L., Doubeni, C. A., Miller, C., Siddiqui, M., Voisin, C., Wines, R. C., & Jonas, D. E. (2025). Multicancer Detection Tests for Screening. Annals of Internal Medicine. https://doi.org/10.7326/ANNALS-25-01877

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

In the News, News Stories & Editorials, We're In the News

 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

FDA Leaders Moving to Abandon Advisory Committee Reviews of Specific New Drugs

In the News, News Stories & Editorials, We're In the News

KFF News, Arthur Allen, September 12-16, 2025

Under President Donald Trump, leaders at the US Food and Drug Administration (FDA) are moving to abandon a decades-old policy of asking outside experts to review drug applications, a move critics say would shield the agency’s decisions from public scrutiny.

The agency “would like to get away” from assembling panels of experts to examine and vote on individual drugs, because “I don’t think they’re needed,” said George Tidmarsh, head of the FDA’s Center for Drug Evaluation and Research. He relayed the message last week at a meeting of health care product makers and to an FDA advocacy group.

In addition to being redundant, Tidmarsh said, advisory meetings on specific drugs were “a tremendous amount of work for the company and for the FDA. We want to use that work and our time to focus on the big questions.”

The FDA’s advisory committees were created in their current form by a 1972 law aimed at expanding and regulating the government’s use of experts in technical decisions. They’re periodically summoned for advice, including to review evidence and vote on whether the FDA should approve drugs, vaccines, and medical devices, often when FDA officials face a difficult decision.

FDA actions have traditionally aligned with committee votes. A departure can provoke controversy and public debate, as was the case with the split 2021 decision on whether to approve the Biogen drug Aduhelm to treat Alzheimer’s disease.

The FDA approved the drug despite a “no” vote from its advisory committee, whose members felt the medicine did little to treat the disease. The conflict over Aduhelm laid bare the FDA’s struggle to reconcile pressure from industry and desperate patients with its rigorous evaluation of drug risks and benefits.

Tidmarsh said the committees would still be consulted on general issues like how to regulate different classes of drugs. But meetings on specific drugs, in which experts plow through piles of studies and hours of testimony from FDA and company officials, were mainly useful, he said, because they allowed the public to see how the FDA worked.

This month, the FDA began publishing the “complete response letters” it sends to companies when it declines to approve their products. Releasing the letters, which previously required filing requests under the federal Freedom of Information Act, promotes a level of transparency akin to the advisory meetings’, Tidmarsh said.

Advisory committee meetings on individual drugs “are redundant when you have the complete review letters,” he told KFF Health News in a brief interview after appearing at the health care products conference.

Former FDA officials and academics who study the agency disagree. The meetings help FDA scientists make decisions and increase public understanding of drug regulation, and abandoning them doesn’t make sense, they said.

Tidmarsh’s reasoning is “hard to follow,” former FDA Commissioner Robert Califf told KFF Health News. “It’s extremely useful for people inside FDA to find out what other experts think before they make their final decisions. And it’s important to do that in a way that enables the public to understand the points of view.”

“Experts might ask questions of the company or FDA that neither of them thought of on their own,” said Holly Fernandez Lynch, an associate professor of bioethics and law at the University of Pennsylvania. “The public has few other opportunities to comment about FDA decisions.”

Spokespeople for FDA and the Health and Human Services Department did not respond to repeated requests for elaboration on Tidmarsh’s comments.

Califf at times disagreed with advisory committees as commissioner of the agency and once floated the idea that it might be better if they deliberated but did not vote on products. Still, while “maybe someone can come up with a better one, I always thought it was an amazing system,” he said.

[….]

The advisory committees are “an important resource” for the FDA, said Sarah Ryan, a spokesperson for the Pharmaceutical Research and Manufacturers of America. “They can play an important part of the rigorous human drug review process we have in the U.S.”

[….]

The changes Tidmarsh described are already playing out on the ground. The FDA has held only seven advisory committee meetings since Trump reentered the White House, compared with 22 over the same time frame last year. Officials say they will now release complete response letters as they are sent, and published a batch of 89 earlier this month.

Makary has, to some extent, replaced the advisory committees, whose members have traditionally been vetted for expertise and biases and are required to deliberate in public, with panels of handpicked scientists who support his views on subjects such as hormone replacement therapy and antidepressants.

Diana Zuckerman, an FDA watchdog, attended the July hormone replacement therapy panel that considered the FDA’s black box warning listing dangers of the treatment. Makary had wanted the warning removed and packed the panel with like-minded experts.

The event was hastily called with no opportunity for the public to review discussion materials or comment on them, she said.

“All that was transparent was that they didn’t want to hear from anyone who disagreed with them,” said Zuckerman, who leads the National Center for Health Research.

Before becoming commissioner, Makary pushed for more advisory committee meetings. In early 2022, he blasted the FDA’s decision to approve COVID-19 boosters for children ages 12 to 15 without consulting its Vaccine and Related Biological Products Advisory Committee. Makary posted on the social platform X at the time, “It is a slap in the face to science for @US_FDA to circumvent the standard convening of the expert advisory board.”

But Tidmarsh seems to disagree.

Instead of asking an advisory committee to vote in favor of or against a Duchenne muscular dystrophy drug, for example, he said the FDA would be better served by a committee studying the best way to evaluate such drugs, such as which outcomes, or endpoints, to measure. “Is this endpoint correct for Duchenne muscular dystrophy? That’s an important question that cuts across many different companies,” he told KFF Health News.

FDA official Vinay Prasad canceled a planned July advisory committee meeting to discuss a Duchenne drug made by the biotech company Capricor Therapeutics. The FDA later published its complete response letter to Capricor, which then published its own letter of response to the FDA. Prasad was later pushed out and rehired with fewer powers.

An advisory committee meeting could have worked through the drug’s risks and benefits in a calmer, public, less politicized atmosphere, Ramachandran said.

[….] 

That’s why Tidmarsh’s comments “come as a complete surprise,” said Genevieve Kanter, an associate professor of public policy at the University of Southern California, who wrote commentary accompanying the study. 

[….]

“Another theory is that this decision is strategic,” she said, “in terms of consolidating power in the agencies so that you are no longer accountable to outside experts or the public.”

To read the entire article, click here https://www.cancertherapyadvisor.com/news/under-trump-fda-seeks-to-abandon-expert-reviews-of-new-drugs/

GDUFA IV Statement of Dr. Diana Zuckerman, President of NCHR

Policy, Testimony & Briefings

July 11, 2025

I’m Dr. Diana Zuckerman, president of the National Center for Health Research.  I appreciate the opportunity to speak today.

My perspective is based on my 35 years of working on issues pertaining to the safety and effectiveness of medical products. I have post-doctoral training in epidemiology and public health, and was a faculty member and researcher at Vassar, Yale, and Harvard before moving to Washington to work as a
Congressional investigator on FDA issues in the U.S. Congress. Prior to my current position, I also worked at HHS and the White House. Our research center is a nonprofit think tank that scrutinizes the safety and effectiveness of medical products, and we don’t accept funding from companies that make those products or have any financial interest in our work.

I am one of the FDA’s biggest fans, because I fully appreciate the agency’s importance.  As a founding Board member of the Alliance for a Stronger FDA, I work with nonprofits and industry to increase appropriations for the FDA. We all know that our healthcare system relies on generic drugs and frankly
would collapse without them. I wish appropriations would be sufficient to support all of FDA’s essential work, but we know that the FDA needs user fees to ensure getting safe and effective medical products to market in a timely manner. However, speed is not the most important part of that equation.

There have been many inspiring statements in the FDA this year about transparency and about the need for the FDA to regulate industry, rather than be influenced by unduly cozy relationships with industry. An important first step would be for user fee negotiations to include patient, consumer, and
public health advocates, instead of only industry and the FDA negotiating behind closed doors. Unfortunately, all user fee negotiations have focused on what industry wants and needs and what they are willing to pay for, and not on what patients and consumers want and need.

On a personal note, my life has depended on generic drugs, which I’ve taken for cancer treatment and for high blood pressure. Like most patients, we trust that generic medications work but we don’t always know for sure. We all depend on generic drugs and understand their importance, so all of us in this room are in this together, and we need to work together.

Trust in generic drugs is essential to help to make healthcare more affordable. Trust in the FDA and in generic drugs has eroded in recent years, and that’s why GDUFA needs to explicitly show that user fees will focus on ensuring that generic drugs are truly equivalent to brand name treatments in all the ways
that matter to patients. Speed should be secondary, because when patients realize that some generic drugs are ineffective or unsafe, it harms patients but also harms companies whose products are safe and effective.

Let’s talk about Performance Goals in GDUFA. Up until now, too few have been focused on safety or effectiveness. We are glad that metrics have included the number of inspections and timeliness of inspections and follow-up warning letters, import alerts, and regulatory meetings, and those metrics
should included. However, they are not sufficient.

Last summer, the FDA determined that Synapse (a company in India) “faked and forged” data submitted to the FDA. FDA withdrew the bioequivalency rating of 400 of their drugs, but they are still on the market. Neither patients nor pharmacists have access to the names of those drugs. Why is that? That
is terribly unfair to patients, but it is also unfair to companies whose safe and effective generic medications are competing with those 400 drugs. And it is also unfair to generic companies that make excellent medications when patients don’t know which generic drugs they can trust.

Valisure has also conducted research showing a sizable number of generic drugs are substandard, with doses that are too high, too low, or drugs that are contaminated or have other problems.

These are just some examples of why post-market surveillance, inspections, and re-inspections are so important and why GDUFA should include funding for those purposes and include those types of metrics in the Commitment Letter.

GDUFA should include metrics showing that these problems are being addressed and generic drugs are truly safe and equivalent to brand name drugs. That’s the promise that GDUFA and the FDA have made to patients and it needs to be kept.

Here is an important list of the kinds of metrics that are missing from previous GDUFA Commitment Letters and should be included in FDA monitoring under GDUFA IV. They are the exact same list that the FDA states are criteria for all generic drugs. They must be:

• Pharmaceutically equivalent
• Capable of making the drug correctly
• Capable of making the drug consistently
• The active ingredient is the same as the name brand and the
same amount gets in the body
• Inactive ingredients are safe
• Drug does not break down over time

We’ve heard about the progress that has been made at the FDA thanks to GDUFA. That progress is wonderful, but to regain trust, improvements should be measurable and included as metrics in GDUFA IV.

Thank you for the opportunity to share my views with you today.

PDUFA VIII Statement of Dr. Amanda Berhaupt, Health Policy Director of NCHR

Policy, Testimony & Briefings

July 14, 2025

Good morning. I’m Dr. Amanda Berhaupt, Health Policy Director at the National Center for Health Research. I appreciate the opportunity to speak on behalf of our nonprofit think tank. We scrutinize the safety and effectiveness of medical products and do not accept funding from companies that make those products or entities with a financial interest in our work.

Prior to my current position, I worked at the FDA and for the United States Senate. 

The appropriated funds for FDA are not sufficient to support all of its critical work, so the agency needs user fees to get safe and effective medical products to market in a timely manner. User fees are vital to ensure that reviewers have subject matter expertise, institutional knowledge, adequate time and uninterrupted access to the FDA library with peer-reviewed research, among other scientific resources. 

User fees have mainly supported faster reviews and more frequent meetings between the FDA and sponsors to address concerns about their applications. I want to emphasize that this is not what’s most important for most patients. Their greatest concern is to have access to safe and effective medical products to treat, maintain, and promote their health. 

With a renewed focus on transparency at FDA, will patients and healthcare professionals be represented at user fee negotiations? At minimum, they deserve to watch the negotiations virtually, and in real-time, if they are not participating. In the past, summaries of negotiations have been too vague, which has prevented key stakeholders from providing input. 

To date, the performance goals in the Commitment letters have outlined metrics for meetings and timelines in premarket reviews. These goals benefit industry, and may indirectly benefit patients, but are not patient-centered and do not focus on safety or efficacy. 

We urge the agency to include performance goals with metrics on quality post-market surveillance including confirmatory studies with clinically meaningful outcomes. This is especially important when drugs are approved based on data from short-term studies with a small sample size, or based on surrogate endpoints instead of measures for how a patient feels, functions, or their overall survival. 

Patient advocates, public health researchers and professionals without industry ties need representation during negotiations to ensure there are performance goals that directly benefit patients and consumers.  

The public’s trust in the FDA has eroded. PDUFA needs to show that user fees will do more to ensure that drugs are safe and effective in ways that matter to patients. Speed should be secondary, because when drugs are ineffective or unsafe, patients lose confidence in their doctors and the FDA, and look for advice from other sources like social media where they may find erroneous and harmful advice.

NCHR Written Comments on the Reauthorization of PDUFA

Policy, Testimony & Briefings

Food and Drug Administration Dockets Management Staff (HFA-305)

5630 Fishers Lane, Rm. 1061

Rockville, MD 20852

August 13, 2025

 

National Center for Health Research Comments on the Reauthorization of

The Prescription Drug User Fee Act [Docket No. FDA-2025-N-0816]

The National Center for Health Research appreciates the opportunity to share our views on needed improvements to the PDUFA negotiation process and policies for PDUFA VIII.  We are a nonprofit think tank that scrutinizes the safety and effectiveness of medical products and the policies that affect the U.S. healthcare system.  We do not accept funding from companies that make those products or entities with a financial interest in our work.

We wish that Congress provided sufficient appropriations for FDA’s essential work, and our president is a founding board member of the Alliance for a Stronger FDA, which includes industry and nonprofit leaders who work tirelessly to improve those appropriations.  However, we know that the agency needs user fees to get safe and effective medical products to market in a timely manner. User fees are vital to ensure that reviewers have subject matter expertise, institutional knowledge, adequate time, and uninterrupted access to the FDA library with peer-reviewed research, among other scientific resources. The agency also needs the staff and resources to revise indications and labeling as needed and to remove products from the market that are not proven to be safe or effective.

Unfortunately, user fees have mainly supported faster reviews and more frequent meetings between the FDA and sponsors to address concerns about their applications. That is not what’s most important for most patients. Most patients’ greatest concern is to have access to safe and effective medical products to treat, maintain, and promote their health.  Our own research shows, for example, that many years after FDA approved cancer drugs on the basis of surrogate endpoints, only one of the 18 drugs was proven to save lives or improve patients’ quality of life.[1]  It is important to note that our research confirmed and provided several years of follow-up data to a study of now-CBER director Dr. Vinay Prasad.

As a way of showing the FDA’s renewed commitment to transparency, we strongly urge that patients and healthcare professionals will be represented at PDUFA user fee negotiations. At a minimum, they deserve to watch the negotiations virtually if they are not permitted to actively participate. In the past, minutes of the negotiation meetings have been too vague to be informative, which has prevented key stakeholders from providing effective input.

Previous PDUFA Commitment letters have focused on performance goals for meetings and timelines for premarket reviews. These goals benefit industry, and may indirectly benefit patients, but are not patient-centered, and not focused on the public health mission of the FDA or the quality of drugs or biologics, because they do not focus on safety or efficacy.

We strongly urge the agency to include performance goals with metrics on quality post-market surveillance including confirmatory studies with clinically meaningful outcomes. This is especially important when drugs are approved based on data from short-term studies with a small sample size or based on surrogate endpoints instead of measures of how a patient feels, functions, or how long they live.

For example, user fees should be used to support for FDA staff and resources needed for essential work other than reviews of new drugs and biologics, especially for work that benefits companies as well as patients.  These include:

  • Confirmatory trials required for products granted accelerated approval
  • Post-market studies required for products approved based on surrogate endpoints or short-term preliminary data, or to follow-up on worrisome adverse event reports or unexpected findings from independently funded studies or testing
  • Inspections of manufacturing facilities and related work caused by problematic findings or failed inspections, since those are also required to ensure that companies can sell their products and that the products being manufactured are safe and effective.

To ensure that PDUFA makes CDER and CBER function more effectively, patient advocates, public health researchers, and professionals without industry ties need representation during negotiations to ensure there are performance goals that directly benefit patients and consumers as well as the overall functioning of these important Centers.

To regain the public trust in the FDA, PDUFA needs to show that user fees will focus more on ensuring that drugs are safe and effective in ways that matter to patients. Speed should be secondary, because when drugs are ineffective or unsafe, patients lose confidence in their doctors and the FDA.  That is not good for our country and will not help achieve our mutual goal of healthier adults and children.

PDUFA Commitment Letter

We’ve reviewed the 71-page PDUFA VII Commitment letter and were very disappointed at how infrequently any mention was made of the importance of FDA’s role ensuring that the drugs the agency is newly approving or previously approved are safe or effective.  There was more mention of “regulatory flexibility” aimed at making the regulatory process less stringent for the companies it regulates, than there was to ensuring that the benefits of newly approved drugs or previously approved drugs outweigh the risks for the American public.

Section K: Enhancing Regulatory Science and Expediting Drug Development 

As an example of how safety and efficacy were rarely mentioned, we will briefly examine Section K of the PDUFA VII Commitment Letter.  This section describes what FDA should do “to advance the use of biomarkers and pharmacogenomics, enhancing communications between FDA and sponsors during drug development, and advancing the development of drugs for rare diseases” to ensure that “new and innovative products are developed and available to patients in a timely manner.“

This section of the Commitment Letter describes numerous steps required of the FDA to facilitate approvals, with no mention of ensuring solid scientific evidence of safety or efficacy.  In PDUFA VIII, any such programs should include clear instructions that evidence from two well-designed studies is still the cornerstone of FDA approval, as well as the importance of clinically meaningful outcomes and statistical significance.

The Commitment Letter for PDUFA VIII should specify ways to increase safety and efficacy information that is publicly available and easy-to-understand, to reduce the burden on patients and providers that need to make potentially life-saving or life-threatening decisions regarding medical treatments.

For example, in #2 of Section K of the PDUFA VII Commitment Letter, entitled “Ensuring Sustained Success of Breakthrough Therapy Program,” the following addition in blue to the existing wording (in black) would have made it clear in PDUFA VII (and for information about the Breakthrough Program in a PDUFA VIII Commitment Letter) that, in addition to speedy review, the benefits of each approved Breakthrough drug should outweigh the risks:

Both FDA and the regulated industry are committed to ensuring the expedited development and review of innovative therapies for serious or life-threatening diseases or conditions by investing additional resources into the breakthrough therapy program to ensure that the therapies are proven to have benefits that outweigh the risks.

Another example is #3 in that same section in the PDUFA VII Commitment Letter, entitled “Early Consultation on the Use of New Surrogate Endpoints.”   The description should have made it clear that the FDA must agree to the use of a surrogate endpoint in order to use it as a primary endpoint.  This could have been clear (for PDUFA VII and in future descriptions of surrogate endpoints in PDUFA VIII) with the edits to the current wording (in black) which is in blue below:

Requests to engage with FDA on this topic will be considered a Type C meeting request. The purpose of this meeting is to discuss the feasibility of the surrogate as a primary endpoint and identify any gaps in knowledge and how they might be addressed. The outcome of this meeting may require further investigation by the sponsor and discussion and will require agreement with the agency before the surrogate endpoint could be used as the primary basis for product approval.

Our next example is #4, entitled “Advancing Development of Drugs for Rare Diseases.“ This current wording (in black) for PDUFA VII requires additional wording (in blue) to make it clear that evidence is important for treatment for rare diseases. This wording would have improved this section in the PDUFA VII Commitment Letter and in future information about regulating treatments for rare diseases in a PDUFA VIII Commitment Letter.

“FDA will continue to include information on rare disease approvals in its annual reports on innovative drug approvals, including utilization of expedited programs and regulatory flexibility and including metrics that indicate that approvals are based on convincing evidence that the drug or biologic has clinically meaningful benefits that outweigh the risks, and appropriate comparative metrics to non-rare disease approvals.

To support the advancement of rare disease treatments, FDA will establish a pilot program for supporting efficacy endpoint development for drugs that treat rare diseases by offering additional engagement opportunities with the Agency to sponsors of development programs that meet specific criteria. This pilot program will be strengthened to provide greater assurance that the endpoints indicate clinically meaningful benefits.

 

Section L.  Enhancing Regulatory Decision Tools to Support Drug Development and Review

The first item in this section is entitled “Enhancing the Incorporation of the Patient’s Voice in Drug Development and Decision-Making.”  We appreciate the FDA’s ongoing efforts to be more patient-centered, but we agree with the numerous patient groups who report that the FDA rarely shows interest in patients who ask the agency to help reduce harm to patients caused by taking drugs for approved or off-label indications. Many patients involved in these FDA efforts were recruited by industry and trained to support industry priorities, particularly getting drugs to market as quickly as possible.  Too often these patients focus on anecdotal evidence based on their own experience, without understanding scientific standards or the benefits of controlled clinical trials or clinical endpoints rather than surrogate endpoints.  Their views are important but need to be balanced by including patients with different priorities and the perspectives that comes from being harmed by FDA-approved prescribed drugs.

Section M. Enhancement and Modernization of the FDA Drug Safety System

This section is especially important to patients’ health and safety.  Unfortunately, “modernization” in PDUFA VII included the goal of reducing or eliminating REMS and on maintaining Sentinel, rather than improving either of these programs.

We agree that PDUFA VIII fees should support REMS and Sentinel again, but specific improvements are needed to make REMS more effective, rather than to eliminate ones that were designed to safeguard patients.

REMS

For example, FDA required a REMS program to train physicians who prescribe opioids, with the goal of reducing the opioid epidemic by reducing inappropriate prescribing.  That REMS underwent several changes after it was initiated in 2012.  As early as 2017, there was clear evidence that most physicians did not undergo the brief online REMS training that was required to be offered, and that many of those who started the training did not complete it or answer all key questions correctly.[2]  Despite those shortcomings, in 2020, the FDA announced that “the central component” of the REMS was still a voluntary continuing education (CE) program “for all health care providers, including nurses and pharmacists, who are involved in the management of patients with pain (in addition to doctors and others who prescribe these products).” The companies were required to offer the training through accredited CE providers, but the REMS does not require health professionals to take the training. Although Congress passed a law in 2023 that requires that physicians registered with the DEA take an 8 hour training on opioids, the physicians do need to get a passing grade on a test based on the training.[3]  PDUFA VIII should be used to strengthen the REMS to either require health professionals to pass a test to show knowledge of key information needed to safely prescribe opioids, or FDA should work with medical societies or other entities who have the authority to do so.  We also point out the conflict of interest of the FDA having the companies that sell opioids to be responsible for the training and for evaluating the effectiveness of the training.

Sentinel System

The Sentinel System was launched in 2008, with the goal of being an early warning system that could identify risks years earlier than would otherwise occur, by analyzing information from health records of millions of patients. Sentinel has resulted in several changes in risk information included in drug labeling, but the impact seems relatively modest considering the cost and scope of the program.

PDUFA VIII should include performance goals that quantify and specify how Sentinel should be used to provide additional information about risks on labels and to what extent it has resulted in changes to indications as more information becomes available about the risks of specific drugs and biologics. The enormous Sentinel “real world” datasets can be used to evaluate various adverse events on popular and less widely used drugs and to compare the safety of drugs used by similar patients for the same indication.  Sentinel can provide evidence of benefits when different treatments for the same indication are compared, but since it is based on real world evidence, the benefits of drugs cannot be compared to a placebo.  Sentinel has the data to make such comparisons possible, but it is not known how often that has been used.  PDUFA VIII should require metrics on how user fees help Sentinel meet these types of important performance goals.

Other PDUFA Issues

We have provided just a few examples of the specific ways that the PDUFA VIII Commitment letter should specify the types of performance goals and program expectations that will help patients, consumers, and health professionals make better informed decisions about FDA-approved drugs and biologics. PDUFA VII was too focused on making it easier to get drugs and biologics to market quickly and not focused enough on making sure “innovative” treatments were more beneficial to patients than previously approved treatments, or compared to no treatments at all.

We also want to express our concerns about the “non-user fee” spending trigger. The PDUFA statute outlines that if spending of appropriated funds for FDA reviewer salaries and expenses falls below inflation-adjusted 1997 levels, the agency must refund user fees. The intent of this clause was to ensure that user fees are used to supplement congressional funding but not replace it. However, given the dramatic downsizing of FDA staff this year, there is a risk that these “trigger” levels of non-user fee spending will not be met and could result in the agency refunding user fees regardless of how well the performance goals stipulated by industry. This would have a catastrophic impact on the agency’s ability to review prescription drugs effectively and ensure that they are safe and effective.

We are also concerned that whenever the Congress is unable to agree on appropriations levels and the government shuts down as a result, staff paid by user fees are the only ones who are allowed to work.  That is just one of many reasons that user fees should support safeguards as well as reviews of new drugs and biologics.

We also ask that the FDA to be transparent about the cost of artificial intelligence (AI) in the review of drug applications. FDA expects that AI can reduce review time, but since AI sometimes makes up information or citations, human reviewers will still need to review any information that AI provides.  It is not clear whether AI is funded by non-user fee appropriated funds as a reviewer salary or an expense. Historically, AI models at the agency have been designed and managed by third-party consultants. It is likely that review queries would come at a cost to the agency, and we ask that FDA be transparent about that. This funding distinction is important because an increased use of AI in the review process, in tandem to fewer FDA staff, could lead to a spending gap that influences user fees.

In conclusion, there are many ways that PDUFA VIII can provide great benefits to patients and our healthcare system, in addition to benefits to industry.  The U.S. taxpayers deserve to have their needs met with the help of user fees, since they are the ones who purchase the drugs and biologics that are approved by the FDA.

We would be glad to discuss our concerns or answer any questions and can be reached at info@center4research.org.

 

References

[1] Rupp, T., & Zuckerman, D. (2017). Quality of Life, Overall Survival, and Costs of Cancer Drugs Approved Based on Surrogate Endpoints. JAMA internal medicine, 177(2), 276–277. https://doi.org/10.1001/jamainternmed.2016.7761

[2] Cepeda, M.S., Coplan P.M., Kopper N.W. et al .ER/LA Opioid Analgesics REMD: Overview of Ongoing Assessments of Its Progress and Its Impact on Health Outcomes, Pain Medicine, 18:1, 78–85. https://doi.org/10.1093/pm/pnw129

[3] U.S. FDA, Opioid Analgesic Risk Evaluation and Mitigation Strategy (REMS) https://www.fda.gov/drugs/information-drug-class/opioid-analgesic-risk-evaluation-and-mitigation-strategy-rems

Testimony of Dr. Diana Zuckerman at the FDA Plastic Surgery Advisory Panel Meeting On Dermal Fillers for the Decolletage Area

Policy, Testimony & Briefings

August 13, 2025

I’m Dr. Diana Zuckerman, president of the National Center for Health Research, a nonprofit think tank. We do not accept funding from entities that have a financial interest in our work, so I have no conflicts of interest.

Thank you for the chance to share my perspective as a scientist who has looked at the data on dermal fillers for many years.

Dermal fillers are very popular despite numerous well-known risks. As PMA products, those risks should be quantified with meaningful statistical data on the short-term and long-term risks, rather than merely listing adverse events as either “common” or “rare.” Unfortunately, statistics on risks aren’t available.

As FDA considers whether these same dermal fillers, or new versions of them, should be approved for use in the dé cole taje area, I urge the FDA to improve the information available to patients about dermal fillers. FDA should require well-designed, controlled clinical trials so that patients have the information they need to make informed decisions. That should require data describing clearly defined short-term and long-term risks, because when the benefit is cosmetic, even short-term, mild or moderate adverse events such as weeks-long or months-long pain, swelling, rash, or bruising matter to patients. Specific information about the frequency and impact of migration also needs to be specified.

We agree with the FDA that use of dermal fillers injected in the de cole tage area has additional risks, such as potential interference with imaging and screening methods for cancer, which could result in false negative or false positive cancer diagnoses. The agency gives several good examples to back up that concern, based on post-approval studies and clinical experiences. How much better it would be to provide and quantify that type of risk information prior to approval.

As the FDA points out, potential harm to the vascular and/or lymphatic systems is of great concern for dermal fillers used in the de cole taje area. It has come to our attention that some major manufacturers of dermal fillers have stopped reporting vascular system impairment and instead categorizing those AEs as obstruction/occlusion. This is clearly intended to make their products seem safer than they are and is the kind of misleading reporting that makes it difficult to trust the data that some major companies are providing. As a result of manipulated data, patients are unable to compare which products are safer, or to make informed decisions about what risks they are willing to take for these cosmetic improvements.

I appreciate that the FDA is talking about studies to evaluate which risks are most important to patients. Unfortunately, merely listing possible risks on a label or a patient booklet is not enough. We’ve talked to thousands of patients who have told us they never saw the label, or the patient booklet that was supposedly required to be given to them. Not all physicians are as transparent as the experts on this panel. But even if patients read and understood the information, there are 2 problems: #1: What physicians tell patients is much more influential than anything provided in writing. Some physicians and their staff are unrealistically reassuring in person. The written informed consent protects them from legal liability if they make any overly optimistic assurances when talking to patients. And #2, research and clinical experience both tell us that patients tend to underestimate risk when they want something. Vague statements about cosmetic complications that are “short-term” may be misunderstood and those complications may last much longer than expected. Most patients assume that any risks that are referred to as “uncommon’ or “rare” won’t happen to them. They are willing to take the risk – but if they experience rare complications, they feel betrayed and devastated, especially for serious complications – but also for cosmetic problems. And for these PMA products, patients do not have the legal recourse for compensation that they would have with an unsafe prescription drug or 510k device.

We strongly agree with the FDA that we have little information about the impact of repeated use dermal fillers over the years. Since some fillers are already being used off label for the de cole tage area, FDA should analyze de-identified data from a registry. However, the FDA needs access to registry data to analyze it, but most registries in the U.S. are owned by medical societies that do not currently make all safety data available to the FDA or the public.

FDA notes that subpopulations of patients may be at higher risks for some potential adverse events, but research is lacking so informed consent isn’t possible for those patients.

The FDA states that “nearly every filler type has been associated with a severe complication” leading to stroke.

There are unique risks to the de cole tage area in addition to the skin necrosis, anaphalaxis, abscesses, migration, granulomas, and risk of intravascular injection which FDA describes as inherent to treatment with dermal filler
anywhere in the body. In addition to breast cancer misdiagnosis and interference with diagnostic imaging, the FDA lists unique risks of complications affecting breast feeding.

The FDA noted a newly recognized adverse event, bone resorption, which was identified on imaging that was conducted for reasons unrelated to the dermal injections.

Although the total number of MDR serious injury reports were less than 18,000 as of last Fall, we all know this is a voluntary reporting system and we know that few health professionals or technicians want to spend their valuable time reporting AEs. And they do not have an incentive to report problems experienced by their patients, especially if they might be considered responsible for them. Bottom line: MDR data are not helpful for quantifying risks.

In conclusion, PMA devices deserve good research for patients understand the risks. When the benefits cosmetic, the risks should not conflict with those benefits. When benefits are not life-saving, the risks should not be life-threatening. That’s why it is so important to require scientific evidence that the benefits outweigh the risks, so women can make informed decisions.

NCHR Public Comment on the Continued Implementation of the National Youth Tobacco Survey

Policy, Testimony & Briefings

August 15, 2025

Re: Proposed Data Collection Submitted for Public Comment and Recommendations; Docket No. CDC–2024–0106

The National Center for Health Research (NCHR) appreciates the opportunity to submit these comments in strong support of the continued implementation of the National Youth Tobacco Survey (NYTS) for the 2026-2028 cycle. 

NCHR is a nonprofit think tank committed to advancing evidence-based policies that improve the health of adults and children. We focus on evaluating medical products, prevention strategies, and public health surveillance systems to ensure they are safe and effective. We are very concerned about the health risks associated with youth tobacco and nicotine use.

We strongly support the continued implementation of the National Youth Tobacco Survey (NYTS) for the 2026-2028 cycle. NYTS is the cornerstone of public health surveillance in this field. Discontinuing or significantly weakening it would create an irreversible gap in the nation’s health data infrastructure. The sustained operation of NYTS is an essential prerequisite for the effective implementation of public health strategies, and an essential component of the MAHA movement.

NCHR Recommendations

We respectfully urge CDC and FDA to jointly support and implement the National Youth Tobacco Survey for the 2026–2028 cycle, including the following priorities:

  1. Retain core trend measures to ensure continuity in time-series analysis, while expanding items that address emerging products (e.g., nicotine pouches, synthetic nicotine);
  2. Ensure oversampling of key populations, including but not limited to under-represented age groups, racial/ethnic minorities, and adolescents with mental health risks;
  3. Release the full public use dataset for each wave promptly and restore datasets from 2020–2023 which were recently removed from public websites; and
  4. Preserve CDC’s scientific leadership in survey design and analysis, while supporting strong interagency collaboration with FDA to ensure funding and continuity.

NYTS Is Irreplaceable for National Youth Tobacco Surveillance

Since its annual implementation beginning in 2004, the NYTS has remained the sole nationally representative source on tobacco and nicotine use among middle school students (grades 6–8) and the most comprehensive dataset for high school students (grades 9–12) [1]. NYTS captures a full spectrum of tobacco and nicotine products. These include traditional cigarettes, cigars, e‑cigarettes, heated tobacco, nicotine pouches, and newer synthetic products [2]. This dataset allows for the tracking of both established and emerging patterns of use. NYTS also measures behavioral variables, such as exposure to pro‑ and anti‑tobacco messaging, social norms, perceived harms, peer influence, pathways of access, dependence indicators, and cessation behaviors, along with contextual factors like mental health and school connectedness [1]. 

The NYTS is the only national survey that provides this level of detail on an annual basis. Surveys such as the Population Assessment of Tobacco and Health (PATH), which focuses on longitudinal regulatory research; the Youth Risk Behavior Surveillance System (YRBSS), which is limited to high schoolers and conducted biennially; and the National Survey on Drug Use and Health (NSDUH), which covers broader substance use across age groups, offer valuable data. However, none of them match the NYTS in comprehensive youth-specific tobacco surveillance [3]. The rapid release cycles of NYTS support timely and responsive policy action. For example, when JUUL’s popularity surged among youth between 2017-2019, NYTS was the only system capturing the magnitude and speed of adoption, a responsiveness that remains unmatched in national surveillance [4] [5].

NYTS Has a Proven Track Record and Will Contribute to MAHA Goals

NYTS data have driven some of the most consequential youth tobacco policies. For example, between 2017 and 2019, NYTS data showed that current e-cigarette use among high school students more than doubled, rising from 11.7% to 27.5%, prompting the FDA to declare youth vaping an “epidemic” and initiate enforcement actions restricting flavored products [6] [7]. These findings also informed the federal Tobacco 21 law, raising the minimum purchase age to 21, which is a milestone policy supported by evidence showing youth nicotine use trends [1] [8]. Furthermore, NYTS data underpinned evaluation of FDA’s “The Real Cost” campaign by tracking shifts in youth perceptions and behavior over time [1] [9] [10]. NYTS has also provided evidence that some youth are more at risk than others, which provides state and local governments with the information they need to develop policies to improve the health of all their children [1]. Taken together, NYTS has served as the early warning system that benefits federal and state health strategies.

NYTS provides the empirical foundation the FDA needs to fulfill its statutory mandate under the Family Smoking Prevention and Tobacco Control Act of 2009 to prioritize youth protection in tobacco regulation [4] [11]. Without it, the agency lacks a reliable means to monitor youth use trends, assess product-specific risk, or measure regulatory impact. Additionally, NYTS informs at least seven Healthy People 2030 objectives [2]. These include tracking adolescent use of e‑cigarettes, cigarettes, flavored products, and other nicotine-containing products [2]. In addition, state Youth Tobacco Surveys can be compared with NYTS data to better evaluate local progress.

In conclusion, youth nicotine and tobacco use are serious and constantly changing public health concerns. NYTS is the most reliable national system for identifying emerging trends and guiding effective prevention strategies. Discontinuing or weakening this survey would severely undermine efforts to reduce youth exposure to nicotine and tobacco products and to prevent long-term addiction.

We strongly support the full approval and continued implementation of this essential survey. For questions or further discussion, we can be reached at info@center4research.org

 

References

 

  1. Gentzke AS, Wang TW, Cornelius M, et al. Tobacco product use and associated factors among middle and high school students — National Youth Tobacco Survey, United States, 2021. MMWR Surveill Summ. 2022;71(5):1-29. doi:10.15585/mmwr.ss7105a1
  2. Centers for Disease Control and Prevention. About National Youth Tobacco Survey (NYTS). CDC Smoking and Tobacco Use. Updated May 15, 2024. Accessed Aug 11, 2025. https://www.cdc.gov/tobacco/about-data/surveys/national-youth-tobacco-survey.html
  3. Boakye E, Erhabor J, Obisesan O, et al. Comprehensive review of the national surveys that assess E-cigarette use domains among youth and adults in the United States. Lancet Reg Health Am. 2023;23:100528. doi:10.1016/j.lana.2023.100528
  4. Wang TW, Gentzke AS, Creamer MR, et al. Tobacco product use and associated factors among middle and high school students — United States, 2019. MMWR Surveill Summ. 2019;68(12):1-22. doi:10.15585/mmwr.ss6812a1
  5. Cullen KA, Gentzke AS, Sawdey MD, et al. e-Cigarette use among youth in the United States, 2019. JAMA. 2019;322(21):2095-2103. doi:10.1001/jama.2019.18387
  6. Creamer MR, Jones SE, Gentzke AS, Jamal A, King BA. Tobacco product use among high school students — Youth Risk Behavior Survey, United States, 2019. MMWR Suppl. 2020;69(1):56-63. doi:10.15585/mmwr.su6901a7
  7. US Food and Drug Administration. Spotlight on Science – Winter 2020. Updated January 9, 2020. Accessed Aug 11, 2025. https://www.fda.gov/tobacco-products/ctp-newsroom/spotlight-science-winter-2020
  8. Agaku IT, Nkosi L, Agaku QD, Gwar J, Tsafa T. A rapid evaluation of the US Federal Tobacco 21 (T21) law and lessons from statewide T21 policies: findings from population-level surveys. Prev Chronic Dis. 2022;19:210430. doi:10.5888/pcd19.210430
  9. The ASCO Post Staff. The Real Cost campaign may have prevented thousands of youths from initiating e-cigarette use. The ASCO Post. March 18, 2025. Accessed Aug 11, 2025. https://ascopost.com/news/march-2025/the-real-cost-campaign-may-have-prevented-thousands-of-youths-from-initiating-e-cigarette-use/ 
  10. Kowitt SD, Sheldon JM, Vereen RN, et al. The impact of The Real Cost vaping and smoking ads across tobacco products. Nicotine Tob Res. 2023;25(3):430-437. doi:10.1093/ntr/ntac206
  11. US Food and Drug Administration. Family Smoking Prevention and Tobacco Control Act – An Overview. Updated August 29, 2024. Accessed Aug 11, 2025. https://www.fda.gov/tobacco-products/rules-regulations-and-guidance-related-tobacco-products/family-smoking-prevention-and-tobacco-control-act-overview

FDA Panel Urges Caution, More Data on Dermal Filler Use in Decolletage Area

We're In the News

By Alicia Ault, August 14, 2025

A FDA advisory panel recommended that manufacturers of dermal fillers collect more information on use in the decolletage area of the body and said that some patients might be at higher risk of complications from injections because of the proximity to breast tissue.

The FDA has not approved dermal fillers for use in the decolletage — a body area that advisory panel members said was not well-defined. It is generally considered the triangular area that runs from the neck and clavicle area to in between the breasts.

Agency officials and committee members noted that fillers are increasingly being used off-label to improve skin texture, crepiness, skin thickness, fine lines, and wrinkles in the decolletage. The most common fillers used in the neck and decolletage are made up of hyaluronic acid (HA), calcium hydroxylapatite (CaHA), or poly-L-lactic acid (PLLA), according to the American Academy of Dermatology Association (AADA).

At a meeting on August 13, the FDA’s General and Plastic Surgery Devices Panel was asked to review safety concerns, in anticipation that manufacturers will soon seek FDA approval of fillers for use in the decolletage area and need guidance on trial design and post-marketing studies. The agency raised the possibility that fillers could migrate from the injection site or form nodules and/or granulomas and interfere with mammograms, cause false positive readings on breast imaging or clinical exams, or impact breast feeding and lymphatic drainage.

The committee — made up of dermatologists, plastic surgeons, oncologists, and radiologists — did not formally vote. The panel members agreed that patients who are breastfeeding or pregnant should be excluded from receiving injections because of the unknowns. Individuals with darker skin types or known wound-healing issues — both of whom might easily form keloids or nodules — or those with a history of radiotherapy, lymphoma, or other blood cancers were also seen as potentially higher risk populations, said panel chairman Hobart Harris, MD, MPH, the J. Engelbert Dunphy endowed chair in surgery at the University of California, San Francisco.

Sandra R. Shuffett, MD, a breast imaging specialist in Lexington, Kentucky, and temporary panel member, said she was concerned that fillers could obscure tumors on breast imaging tests. “My focus is to find a cancer as small as possible,” she said, adding that an unseen tumor could quickly grow larger, necessitating more serious treatment.

The FDA has not received reports of problems with breast feeding or imaging but a post-approval study of Radiesse (CaHA) found that it obscured bone visualization. There have also been reports of lymph node enlargement near dermal filler injection sites.

[….]

Radiesse manufacturer, Merz Aesthetics, told the panel that, between 2018 and 2025, it received 44 reports of potential adverse events in the decolletage area, with none reporting migration of material or radiological interference. Radiesse is approved for decolletage wrinkles in the European Union and Canada.

Social media may be fueling more use of fillers in the decolletage, especially among those taking GLP-1 receptor agonists for weight loss who are seeking “to improve the skin rippling in the chest,” said Karol A. Gutowski, MD, a Chicago-based plastic surgeon who spoke to the committee.

Representatives from dermatology and plastic surgery organizations said they had crafted guidelines for safe use of fillers in the decolletage but warned that filler use was often unregulated.

“Filler adverse events are likely under reported, and they’re increasing in frequency as the popularity of injectable fillers increases,” said M. Laurin Council, MD, director of dermatologic surgery at Washington University School of Medicine, St. Louis, who spoke to the panel on behalf of the American Society for Dermatologic Surgery.

Many panelists suggested women undergo baseline breast imaging before receiving filler in the decolletage area and collecting more data — such as on the volume of filler used during procedures — and added that perhaps a registry should be created. But some were skeptical.

“Probably 75% of these injections are done by non-medical people,” such as attendants at medical spas or storefront wellness centers, said panelist Alan Matarasso, MD, a New York City-based plastic surgeon and past president of the American Society of Plastic Surgeons. Matarasso said that manufacturers should be responsible for tracking their products, not clinicians.

“When these things are being done in strip malls and other places, we’re not going to get the data that we need, because people are not going to cooperate with this,” said Gutowski.

There is no approved method of removing dermal fillers. That gave some panel members pause. But dermatologists and plastic surgeons said that HA-based fillers could be dissolved with hyaluronidase. Even so, CaHA and PLLA fillers can’t be dissolved and “must break down naturally over time,” said Natalie Curcio, MD, MPH, a Nashville-based dermatologist who spoke to the panel on behalf of the AADA.

Temporary committee member Karla V. Ballman, PhD, professor of biostatistics at the Mayo Clinic College of Medicine and Science, Rochester, Minnesota, said that patients should be informed, perhaps via wording on a product label that “at the current time, there is no approved method of removal” of a filler.

[….]

At the panel meeting, consumer advocate Diana Zuckerman, PhD, president of the National Center for Health Research, said that listing adverse events was not enough. “Risks should be quantified with meaningful statistical data on the short term and long term risks,” said Zuckerman, who spoke during the open public hearing. “FDA should require well designed and full clinical trials so that patients have the information they need to make informed decisions,” she said.

[….]

Read full article here.

After 10 Years, the FDA Is Still Letting Women Down

In the News, News Stories & Editorials
By Michelle Llamas, BCPA August 14, 2025

In Drugwatch’s 2015 investigation, How the FDA Let Women Down, we highlighted issues with drug and medical device approvals that posed greater risks to women.

Now, we’re diving deeper into regulatory processes to highlight how far they’ve come — and where the administration still falls short in terms of device testing and clinical trials for medical products marketed toward women.

The FDA’s 510(k) clearance process is still allowing moderate-risk devices on the market without clinical trials. Some of these products, such as pelvic mesh, continue to hurt women.

The agency has also been working to approve drugs faster than ever, offering fast-track options for new drugs for serious illnesses such as cancer.

However, mistakes can lead to devastating outcomes when drugs are approved based on lower-quality data. In some cases, the FDA proposed using one clinical trial with patients instead of two to approve drugs faster.

More recently, the FDA has championed AI to help achieve faster drug approvals, but AI has been known to produce false data.

As health care evolves, so do women’s needs and safety concerns. Stronger data and testing requirements can help protect women from dangerous medical devices and drugs.

Medical Approval Processes May Fall Short

While the FDA requires clinical trials for drugs to hit the market, a large number of medical devices are sold without trial data — exposing women and men to health risks.

The 510(k) clearance process allows medium-risk (Class II) medical devices like surgical mesh, some hip implants, catheters, pregnancy tests and others on the market without clinical trials as long as they are similar to devices already on the market.

[….]

Drug approvals, on the other hand, require more testing and clinical data for the FDA to approve them. However, in some cases, the quality of the data submitted may be an issue, and drugs could be approved based on lower standards.

Medical Devices: Inadequate Testing, Conflicts of Interest and Delayed Warnings

Donna Miser’s doctor implanted a surgical mesh bladder sling that was supposed to help her with stress urinary incontinence (SUI), a condition that causes urine to leak when there’s increased pressure on the bladder. Exercising, sneezing or coughing can all trigger these leaks. SUI affects 1 in 3 women.

But no one told her about the risks of mesh.

The implant is supposed to be permanent, but after a few years, the mesh eroded into her bladder and vaginal walls and cut into her urethra in multiple places. She’s since had several surgeries to remove the mesh.

“Someone’s really dropped the ball. I do not understand how so many women got implanted with [this] product. That surgeon looked at me with a smile on his face, telling me, ‘I have got the answer. I’ve got the cure for you. We’re going to put this in you,’” Miser told Drugwatch. “It wasn’t tested. It wasn’t approved.”

[….]
When Miser said her mesh wasn’t tested or approved, she wasn’t wrong. The 510(k) process allows devices to be approved without clinical trials if they are similar to products already on the market, which are called predicate devices.

The issue with 510(k) approvals is that products can enter the market based on similarities to decades-old devices. This was the case with the surgical mesh implanted in women for SUI or pelvic organ prolapse (POP), a condition where organs slip down and bulge into the vagina.

Another, more rigorous (but less frequently used) path to device approval, Premarket Approval (PMA), requires more scientific evidence. The PMA is intended for high-risk Class III medical devices, such as pacemakers or defibrillators.

Mesh implanted through the vagina for POP has since been reclassified to a Class III device and now requires more testing before it’s sold, but SUI mesh remains a Class II.

[….]

“Missing Safety Device Data May Delay FDA Warnings

The FDA’s Manufacturer and User Facility Device Experience (MAUDE) system is a searchable database for medical device complications. The FDA uses it to flag safety data and determine if it needs to take action.

Madris Kinard of Device Events used to work at the FDA as an adverse events subject matter expert for devices and unique device identification (UDI). Kinard spoke to Drugwatch and cited a report on a problematic birth control device called Essure. With Essure, doctors implanted two metal coils into the fallopian tubes. This caused scar tissue to develop, blocking the tubes and preventing sperm from reaching the egg.

Women reported thousands of complications from Essure that led them to get it surgically removed.

Kindard’s FDA database analysis showed that about 32,000 device complaints from inspections of Essure’s manufacturer in 2011 and 2013 hadn’t made it into the FDA’s database. Kinard said it’s not clear whether these complaints contained adverse event reports because the details haven’t been made public. The FDA still hasn’t responded to her Freedom of Information Act (FOIA) request.

If that data had been added to MAUDE, it might have given the FDA more information to warn women about Essure sooner.

“That set them back by probably 10 years in identifying these problems,” Kinard said.

[…]

Drugs: Poor Evidence for Approval, Improper Doses for Women and Underrepresentation in Clinical Trials

Unlike the 510(k) clearance pathway for medical devices, drugs require more clinical trial data for approval. One of the most important parts of the drug approval process is when the FDA looks at the risks and benefits from clinical trial data submitted by a manufacturer. The FDA expects the manufacturer to conduct two well-designed clinical trials, but in some cases, it will accept one.

To determine if drugs work safely, the FDA uses four minimum criteria to judge whether manufacturers have provided enough evidence for drug approval.

A new report from The Lever and the McGraw Center for Business Journalism at CUNY’s Newmark Graduate School of Journalism analyzed a government database and looked at 429 drugs approved by the FDA from January 2013 to December 31, 2022.

The report revealed that the FDA approved these drugs without clinical trials that met the minimum four criteria of having a control group, blinding, replication or clinical endpoints.

“More medical products have been allowed on the market in the last decade based on skimpier research, or research studying biological markers that patients can’t feel (such as plaques on the brain or bone density) rather than meaningful health benefits such as living longer or spending less time in a hospital,” Diana Zuckerman, President of the National Center for Health Research and a project advisor for The Lever, told Drugwatch.

Investigative journalists Jeanne Lenzer and Shannon Brownlee spearheaded the database project and found several surprises in the data.

“We knew going in that the FDA had relaxed its scientific standards over the years and that the result was drugs getting on the market without adequate evidence that they work,” Lenzer told Drugwatch. “We didn’t know just how bad it was.”

Lenzer and Brownlee were also surprised by how many cancer drugs in the data they pulled made it to the market without adequate proof they work. The exact cancer medications are included in the table above.

[….]

Improper Dosing Can Lead to More Side Effects For Women

Women experience side effects nearly twice as often as men, and one of the reasons is that medications take longer to leave women’s bodies.

Even with researchers recommending dose reductions for women, the FDA hasn’t taken meaningful action to require sex-specific dosing information on drug labels.

One study in Biology of Sex Differences looked at 86 drugs and found that (when compared to men), women generally had higher blood concentrations of the drugs, and the medications took longer to leave their bodies. This led to higher rates of side effects in women in 96% of cases.

The findings in this study suggest that women may have been prescribed higher doses of drugs than necessary, even when they take the dose recommended on the drug’s label or as directed.

Medications studied included common OTC and prescription medications such as aspirin and Zoloft (sertraline).

[….]

Older Women and Women of Color Still Underrepresented

When we interviewed Zuckerman in 2015, she highlighted the lack of women, people of color or people over age 65 in clinical trials. Over the past 10 years, the FDA has increased the number of women in clinical trials, but still lags behind with women of color and older women.

“Most trials submitted to the FDA include quite a few women, but they are not women of color or women over 65, even though many diseases are more common on people over 65 and at least as prevalent in people of color,” Zuckerman told Drugwatch.

While it’s great that more women are finally included in trials, the benefits might not be seen for a few years. Most drugs on the market today were approved during older clinical trials. The data from these trials were primarily gathered from men, leaving a gap in safety data for women.

Expert Opinion: How Can the FDA Improve Drug Safety?

When it comes to drug safety, the FDA needs to require more stringent clinical trial evidence before allowing drugs onto the market.

“The problem for the agency is it is now hamstrung by Congress, which has, over the years, steadily eroded the statutes governing drug regulation,” Lenzer said. “In addition, we believe that PDUFA has to be repealed.”

The PDUFA, or Prescription Drug User Fee Act, allows the FDA to collect fees from drugmakers in exchange for expediting their medications’ reviews and approvals.

“Nobody wants to talk about that because it would almost certainly require public funding, but an agency that is paid by the industry it is supposed to regulate — almost by definition — cannot be independent. What that means for the FDA is it no longer protects the public health and patients because it’s too busy protecting the commercial interests of its benefactors,” Brownlee added.

[….]

This excellent article has many examples of specific medical products that are unsafe, and provides more information about what needs to be done.  You can read the entire article at https://www.drugwatch.com/featured/fda-still-letting-women-down/
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