Category Archives: In the News

Consumer and Public Health Groups Support FDA Proposal to Ensure Accuracy of Lab-Developed Medical Tests

October 26, 2023Press Releases, We're In the NewsCPTFadmin

September 29, 2023

A coalition of consumer advocacy groups is welcoming a proposed rule released today by the Food and Drug Administration to regulate laboratory-developed tests (LDTs), a category of diagnostic tests developed and used in a single lab. LDTs are a subset of In Vitro Diagnostics (IVDs) which are FDA regulated, despite the fact that they have been regulated differently for decades. The proposal clarifies that LDTs are medical devices, meaning FDA will ensure they are safe and effective before they are sold to consumers. The tests have long been under “enforcement discretion,” meaning FDA has not enforced premarket approval and other requirements, but stakeholders and regulators alike have been calling for increased scrutiny of these tests for years.

The proposed regulation will take effect 60 days after a final rule is published and contemplates a phase-in over the subsequent four years. As these tests have become more complex and more important to patient care, ensuring their accuracy has become more crucial, according to the coalition, which includes the Center for Science in the Public Interest, the National Center for Health Research, Strathmore Health Strategy, and U.S. PIRG.

False-positive test results could lead patients to believe they have a serious medical condition that they do not have, and false-negative results may cause a patient’s life-threatening condition to be missed. Some tests have falsely diagnosed people with cancer or inaccurately provided results that lead directly to chemotherapy selection. Many of the tests have been found to be inaccurate, including some COVID-19 diagnostic tests, genetic non-invasive prenatal screening tests, and blood tests manufactured by the biotech company Theranos.

The group applauds many provisions of the proposed rule. These include:

Including Academic Medical Centers in the regulatory scheme. There is no reason that people treated or tested at one facility should be more at risk for inaccurate results than those tested at another facility. All tests should be evaluated based on the benefits and risks of the tests, not the building in which the test is run. The proposed rule notes that “[r]eview of the underlying science behind an [in vitro diagnostic] is based on what the IVD does and is in no way related to where the IVD is made.” The FDA requests additional information on this topic, but the coalition hopes the agency sticks to its guns.
Including tests for rare diseases in the regulatory scheme. Patients with rare diseases should be equally protected from inaccurate tests.
Registration and listing requirements for all tests, which exist for all other medical devices. This will allow FDA and the public to know which tests are available. Further, manufacturers are required to publish performance data on IVDs, which will provide much-needed transparency about the clinical and analytical validity of these tests, according to the coalition.

“This rule is a critical step forward for clinical medicine,” said Dr. Peter G. Lurie, President of Center for Science in the Public Interest and a former Associate Commissioner at the Food and Drug Administration where he worked on LDTs, including on a report demonstrating their potential dangers. “It will help ensure that when a patient receives a test, they can rely on the results to make essential decisions for their health. This rule will close a gaping hole in FDA’s current regulatory reach.”

In the absence of FDA oversight, LDTs have been regulated only by the Centers for Medicare and Medicaid Services, which does not require documentation that the test results accurately inform the diagnosis of patients, a concept known as “clinical validity.” CMS only requires laboratories to document the “analytical validity” of their tests, or their ability to reliably detect a biomarker.

Oversight under FDA would be much more comprehensive and would ensure that healthcare providers and patients can rely on results to make medical decisions, particularly the riskiest medical decisions, where inaccurate test results can cause harm to patients. A modern regulatory framework for LDTs will improve patient access to accurate tests and promote innovation in the diagnostic testing industry.

“We strongly support the decision by the FDA to do what is necessary to rectify a situation that has been so harmful to patients,” said Dr. Diana Zuckerman, President of the National Center for Health Research. “We understand the need for a transitional period but urge the FDA to address problems with existing high-risk LDTs as quickly as possible. We welcome the opportunity to work with FDA and other interested parties to ensure that FDA has the resources it needs to robustly regulate LDTs so that patients can make informed decisions based on test results.”

This comprehensive approach will require resources, including both user fees and Congressional appropriations. The coalition will continue working with all stakeholders, including Congress and the Agency, to ensure that the agency is adequately resourced to fulfill this critical function.

For more information, contact Dr. Zuckerman at dz@center4research.org

Are PIP rubber playgrounds safe for Kingston?

October 23, 2023We're In the NewsCPTFadmin

Dr. Diana Zuckerman, PH.D, Kingston Wire, October 23, 2023

When I first saw rubber playground surfaces under swings, slides, and children’s climbing equipment, I was impressed. They seemed very attractive and safe for active young children. I was wrong. As a scientist I learned that children all over the country are being exposed to unsafe chemicals without their parents’ knowledge or consent. That’s why I recently wrote to Kingston’s mayor, superintendent of schools, members of the Board of Education, and numerous principals to share scientific information about the lead and dangerous chemicals in artificial turf and playground surfaces, hoping it will help them make the right decisions about what is best for Kingston’s children. I want to share that information with you.

As president of the National Center for Health Research, I have testified about these products to local, state and federal agencies and legislators and met with parents and community leaders from coast to coast. Our nonprofit think tank includes scientists, physicians and health experts who conduct studies and scrutinize research conducted by others. We explain scientific and medical information that can be used to improve policies, programs, services and products.

What’s in those rubber playground surfaces?

In recent years, scientists have learned about the lead, cadmium, PFAS, and other chemicals that are in the rubber playground surfaces called PIP (Poured in Place) and recycled tire mulch (also called recycled rubber, since that sounds even more environmentally friendly). We now know that the rubber pieces made from recycled tires contain lead and heavy metals, as well as chemicals that increase the chances of developing obesity; early puberty; attention problems such as ADHD; exacerbate asthma; and eventually cause cancer. Although I’m focusing on playground surfaces, recycled rubber is also used as “infill” for many artificial turf fields and also the rubber mulch sold for your lawn at Home Depot and many other stores. (The plastic grass that makes up artificial turf also has dangerous levels of lead, PFAS, and other toxic chemicals.)

Pediatricians tell us that no level of lead exposure is safe. The solid rubber surface used on playgrounds looks safe, but whether or not the top is made from recycled tires, underneath is recycled tire crumb that causes lead dust on top of the surface. Children breathe that lead dust as they play. And, after a few months or years, the solid rubber surface wears off or cracks, revealing small pieces of recycled tires that young children (like those at George Washington Elementary School’s Children’s House) may be tempted to put in their mouths, exposing them to even more lead. Blood lead levels for Kingston residents are already higher than in most communities. That makes it especially essential to avoid additional exposures.

The PFAS in tire mulch are also dangerous because they enter the body and the environment as “forever chemicals.” PFAS are not metabolized and won’t deteriorate, accumulating over the years. PFAS can cause liver damage and other serious health problems. That’s why Governor Kathy Hochul signed a law this year banning PFAS from clothing and carpeting (they are used to make them stain resistant), including the plastic grass carpet used in artificial turf fields, such as the one at Dietz Stadium. Unfortunately, PFAS is not banned from rubber playgrounds, such as PIP.

There are also environmental risks from these materials. They retain heat, so that on a warm sunny day when the temperature above the grass is 85 degrees, it is often over 150 degrees for anyone on PIP and artificial turf fields. And, during heavy rains, the tire mulch washes off, contaminating nearby areas and your water supply.

Evidence of Harm vs. Evidence of Safety

Scientists at the National Institute of Environmental Health Sciences (which is part of NIH) have concluded that unlike most other chemicals, hormone-disrupting chemicals (found in tire mulch and artificial turf) can be dangerous at very low levels, and also when they combine with other exposures in our environment. That is why the U.S. Consumer Product Safety Commission has banned these chemicals from toys, pacifiers, teething toys and other products used by young children.

Companies that sell and install artificial turf and rubber playground surfaces often claim that there is “no evidence children are harmed” or that their products cause cancer. This is often misunderstood as meaning the products are safe or are proven to not cause harm. Neither is true.

It is true that there is no clear evidence that an artificial turf field has caused specific children to develop cancer. However, that statement is misleading because it is virtually impossible to prove any chemical exposure causes one specific individual to develop cancer.

As an epidemiologist, I can tell you that for many years there was no evidence that smoking or 9/11 exposures caused cancer. It took many years to develop that evidence, and the same will be true for products made from recycled tires.

We know that these materials contain carcinogens. When children are exposed to those carcinogens day after day, week after week, and year after year, it increases the chances of our children developing cancer, either in the next few years or later as adults. That should be adequate reason not to install them in your community.

What Should Kingston Do?

There have never been any safety tests required prior to sale that prove that any of these products are safe for children who play on them regularly. In many cases, the materials used are not publicly disclosed, making independent research difficult to conduct. None of these products are proven to be as safe as engineered wood fiber or natural grass in well-constructed fields.

Officials in communities all over the country have been misled by salespeople and scientists hired to lobby them to purchase these fields and playgrounds. They were erroneously told that these products are safe. In fact, there is clear scientific evidence that these materials are harmful. How much exposure is likely to be harmful to which children? Do you want to take that risk with your children? Don’t our children deserve better?

I am not paid to write this or speak up on this issue. I do so because I care about the health of my family and yours.

This oped is posted on the Kingston Wire website at https://kingstonwire.com/opinions/2023/10/23/opinion-are-pip-rubber-playgrounds-safe-for-kingston/361KUj and you can read a pdf version of the article here.

Who gets to decide who receives experimental medical treatments?

August 10, 2023In the News, News Stories & Editorials, We're In the NewsCPTFadmin

Jessica Hamzelou, MIT Technology Review, August 10, 2023

Max was only a toddler when his parents noticed there was “something different” about the way he moved. He was slower than other kids his age, and he struggled to jump. He couldn’t run.

Blood tests suggested he might have a genetic disease— one that affected a key muscle protein. Max’s dad, Tao Wang, a researcher for a climate philanthropy organization, says he and his wife were initially in denial. It took them a few months to take Max for the genetic test that confirmed their fears: he had Duchenne muscular dystrophy.

Duchenne is a rare disease that tends to affect young boys. It’s progressive—those affected lose muscle function as they get older. There is no cure. Many people with the disorder require wheelchairs well before they reach their 20s. Most do not survive beyond their 30s.

Max’s diagnosis hit Wang and his wife “like a tornado,” he says. But eventually one of his doctors mentioned a clinical trial that he was eligible for. The trial was for an experimental gene therapy designed to replace the missing muscle protein with a shortened, engineered version that might help slow his decline or even reverse it. Enrolling Max in the trial was a no-brainer for Wang. “We were willing to try anything that could change the course [of the disease] and give us some hope,” he says.

That was more than two years ago. Today, Max is an active eight-year-old, says Wang. He runs, jumps, climbs stairs without difficulty, and even enjoys hiking. “He’s a totally different kid,” says Wang.

The gene therapy he received was recently considered for accelerated approval by the US Food and Drug Administration. Such approvals, reserved for therapies targeting serious conditions that lack existing treatments, require less clinical trial data than standard approvals.

While the process can work well, it doesn’t always. And in this case, the data is not particularly compelling. The drug failed a randomized clinical trial—it was found to be no better than a placebo.

Still, many affected by Duchenne are clamoring for access to the treatment. At an FDA advisory committee meeting in May set up to evaluate its merits, multiple parents of children with Duchenne pleaded with the organization to approve the drug immediately—months before the results of another clinical trial were due. On June 22, the FDA granted conditional approval for the drug for four- and five-year-old boys.

Between 2009 and 2022, 48 cancer drugs received accelerated approval to treat 66 conditions—and 15 of those approvals have since been withdrawn.

This drug isn’t the only one to have been approved on weak evidence. There has been a trend toward lowering the bar for new medicines, and it is becoming easier for people to access treatments that might not help them—and could harm them. Anecdotes appear to be overpowering evidence in decisions on drug approval. As a result, we’re ending up with some drugs that don’t work.

[….]

Expanding access

There’s a difficult balance to be reached between protecting people from the unknown effects of a new treatment and enabling access to something potentially life-saving. Trying an experimental drug could cure a person’s disease. It could also end up making no difference, or even doing harm. And if companies struggle to get funding following a bad outcome, it could delay progress in an entire research field—perhaps slowing future drug approvals.

In the US, most experimental treatments are accessed through the FDA. Starting in the 1960s and ’70s, drug manufacturers had to prove to the agency that their products actually worked, and that the benefits of taking them would outweigh any risks. “That really closed the door on patients’ being able to access drugs on a speculative basis,” says Christopher Robertson, a specialist in health law at Boston University.

It makes sense to set a high bar of evidence for new medicines. But the way you weigh risks and benefits can change when you receive a devastating diagnosis. And it wasn’t long before people with terminal illnesses started asking for access to unapproved, experimental drugs.

[….]

Today, there are lots of ways people might access experimental drugs on an individual basis. Perhaps the most obvious way is by taking part in a clinical trial. Early-stage trials typically offer low doses to healthy volunteers to make sure new drugs are safe before they are offered to people with the condition the drugs are ultimately meant to treat. Some trials are “open label,” where everyone knows who is getting what. The gold standard is trials that are randomized, placebo controlled, and blinded: some volunteers get the drug, some get the placebo, and no one—not even the doctors administering the drugs—knows who is getting what until after the results have been collected. These are the kinds of studies you need to do to tell if a drug is really going to help people.

But clinical trials aren’t an option for everyone who might want to try an unproven treatment. Trials tend to have strict criteria about who is eligible depending on their age and health status, for example. Geography and timing matter, too—a person who wants to try a certain drug might live too far from where the trial is being conducted, or might have missed the enrollment window.

Instead, such people can apply to the FDA under the organization’s expanded access program, also known as “compassionate use.” The FDA approves almost all such requests. It then comes down to the drug manufacturer to decide whether to sell the person the drug at cost (it is not allowed to make a profit), offer it for free, or deny the request altogether.

Another option is to make a request under the Right to Try Act. The law, passed in 2018, establishes a new route for people with life-threatening conditions to access experimental drugs—one that bypasses the FDA. Its introduction was viewed by many as a political stunt, given that the FDA has rarely been the barrier to getting hold of such medicines. Under Right to Try, companies still have the choice of whether or not to provide the drug to a patient.

When a patient is denied access through one of these pathways, it can make headlines. “It’s almost always the same story,” says Alison Bateman-House, an ethicist who researches access to investigational medical products at New York University’s Grossman School of Medicine. In this story, someone is fighting for access to a drug and being denied it by “cold and heartless” pharma or the FDA, she says. The story is always about “patients valiantly struggling for something that would undoubtedly help them if they could just get to it.”

But in reality, things aren’t quite so simple. When companies decide not to offer someone a drug, you can’t really blame them for making that decision, says Bateman-House. After all, the people making such requests are usually incredibly ill. If someone were to die after taking that drug, not only would it look bad, but it could also put off investors from funding further development. “If you have a case in the media where somebody gets compassionate use and then something bad happens to them, investors run away,” says Bateman-House. “It’s a business risk.”

FDA approval of a drug means it can be sold and prescribed—crucially, it’s no longer experimental. Which is why many see approval as the best way to get hold of a promising new treatment.

As part of a standard approval process, which should take 10 months or less, the FDA will ask to see clinical trial evidence that the drug is both safe and effective. Collecting this kind of evidence can be a long and expensive process. But there are shortcuts for desperate situations, such as the outbreak of covid-19 or rare and fatal diseases—and for serious diseases with few treatment options, like Duchenne.

Anecdotes vs. evidence

Max accessed his drug through a clinical trial. The treatment, then called SRP-9001, was developed by the pharmaceutical company Sarepta and is designed to replace dystrophin, the protein missing in children with Duchenne muscular dystrophy. The protein is thought to protect muscle cells from damage when the muscles contract. Without it, muscles become damaged and start to degenerate.

The dystrophin protein has a huge genetic sequence—it’s too long for the entire thing to fit into a virus, the usual means of delivering new genetic material into a person’s body. So the team at Sarepta designed a shorter version, which they call micro-dystrophin. The code for the protein is delivered by means of a single intravenous infusion.

The company planned to develop the therapy to treat patients with Duchenne who could still walk. And it had a way to potentially fast-track the approval process.

Usually, before a drug can be approved, it will go through several clinical trials. But accelerated approval offers a shortcut for companies that can show that their drug is desperately needed, safe, and supported by compelling preliminary evidence.

For this kind of approval, drug companies don’t need to show that a treatment has improved anyone’s health—they just need to show improvement in some biomarker related to the disease (in Sarepta’s case, the levels of the micro-dystrophin protein in people’s muscle).

There’s an important proviso: the company must promise to continue studying the drug, and to provide “confirmatory trial evidence.”

This process can work well. But in recent years, it has been a “disaster,” says Diana Zuckerman, president of the National Center for Health Research, a nonprofit that assesses research on health issues. Zuckerman believes the bar of evidence for accelerated approval has been dropping.

Many drugs approved via this process are later found ineffective. Some have even been shown to leave people worse off. For example, between 2009 and 2022, 48 cancer drugs received accelerated approval to treat 66 conditions—and 15 of those approvals have since been withdrawn.

Melfulfen was one of these. The drug was granted accelerated approval for multiple myeloma in February 2021. Just five months later, the FDA issued an alert following the release of trial results suggesting that people taking the drug had a higher risk of death. In October 2021, the company that made the drug announced it was to be taken off the market.

There are other examples. Take Makena, a treatment meant to reduce the risk of preterm birth. The drug was granted accelerated approval in 2011 on the basis of results from a small trial. Larger, later studies suggested it didn’t work after all. Earlier this year, the FDA withdrew approval for the drug. But it had already been prescribed to hundreds of thousands of people—nearly 310,000 women were given the drug between 2011 and 2020 alone.

And then there’s Aduhelm. The drug was developed as a treatment for Alzheimer’s disease. When trial data was presented to an FDA advisory committee, 10 of 11 panel members voted against approval. The 11th was uncertain. There was no convincing evidence that the drug slowed cognitive decline, the majority of the members found. “There was not any real evidence that this drug was going to help patients,” says Zuckerman.

Despite that, the FDA gave Aduhelm accelerated approval in 2021. The drug went on the market at a price of $56,000 a year. Three of the committee members resigned in response to the FDA’s approval. And in April 2022, the Centers for Medicare & Medicaid Services announced that Medicare would only cover treatment that was administered as part of a clinical trial. The case demonstrates that accelerated approval is no guarantee a drug will become easier to access.

The other important issue is cost. Before a drug is approved, people might be able to get it through expanded access—usually for free. But once the drug is approved, many people who want it will have to pay. And new treatments—especially gene therapies—don’t tend to be cheap. We’re talking hundreds of thousands, or even millions, of dollars. “No patient or families should have to pay for a drug that’s not proven to work,” says Zuckerman.

What about SRP-9001? On May 12, the FDA held an advisory committee meeting to assess whether the data supported accelerated approval. During the nine-hour virtual meeting, scientists, doctors, statisticians, ethicists, and patient advocates presented the data collected so far, and shared their opinions.

Sarepta had results from three clinical trials of the drug in boys with Duchenne. Only one of the three—involving 41 volunteers aged four to seven—was randomized, blinded, and placebo controlled.

Scientists will tell you that’s the only study you can draw conclusions from. And unfortunately, that trial did not go particularly well—by the end of 48 weeks, the children who got the drug were not doing any better than those who got a placebo.

But videos presented by parents whose children had taken the drug told a different story.

[….]

But the difference is not statistically significant for the results the trial was designed to collect. And there are some safety concerns. While most of the boys developed only “mild” side effects, like vomiting, nausea, and fever, a few experienced more serious, although temporary, problems. There were a total of nine serious complications among the 85 volunteers. One boy had heart inflammation. Another developed an immune disease that damages muscle fibers.

On top of all that, as things currently stand, receiving one gene therapy limits future gene therapy options. That’s because the virus used to deliver the therapy causes the body to mount an immune response. Many gene therapies rely on a type called adeno-associated virus, or AAV. If a more effective gene therapy that uses the same virus comes along in the coming years, those who have taken this drug won’t be able to take the newer treatment.

Despite all this, the committee voted 8–6 in favor of granting the drug an accelerated approval. Many committee members highlighted the impact of the stories and videos shared by parents like Brent Furbee.

“Now, I don’t know whether those boys got placebo or whether they got the drug, but I suspect that they got the drug,” a neurologist named Anthony Amato told the audience.

“Those videos, anecdotal as they are … are substantial evidence of effectiveness,” said committee member Donald B. Kohn, a stem-cell biologist.

The drugs don’t work?

Powerful as they are, individual experiences are just that. “If you look at the evidentiary hierarchy, anecdote is considered the lowest level of evidence,” says Bateman-House. “It’s certainly nowhere near clinical-trial-level evidence.”

This is not the way we should be approving drugs, says Zuckerman. And it’s not the first time Sarepta has had a drug approved on the basis of weak evidence, either.

The company has already received FDA approval to sell three other drugs for Duchenne, all of them designed to skip over faulty exons—bits of DNA that code for a protein. Such drugs should allow cells to make a longer form of a protein that more closely resembles dystrophin.

The first of these “exon-skipping” drugs, Exondys 51, was granted accelerated approval in 2016—despite the fact that the clinical trial was not placebo controlled and included only 12 boys. “I’ve never seen anything like it,” says Zuckerman. She points out that the study was far too small to be able to prove the drug worked. In her view, 2016 was “a turning point” for FDA approvals based on low-quality evidence—“It was so extreme,” she says.

[….]

But for many in the scientific community, that data still needs to be confirmed. “The clinical benefit still has not been confirmed for any of the four,” Mike Singer, a clinical reviewer in the FDA’s Office of Therapeutic Products, told the advisory committee in May.

“All of them are wanted by the families, but none of them have ever been proven to work,” says Zuckerman.

[….]

Selling hope

On June 22, just over a month after the committee meeting, the FDA approved SRP-9001, now called Elevidys. It will cost $3.2 million for the one-off treatment, before any potential discounts. For the time being, the approval is restricted to four- and five-year-olds. It was granted with a reminder to the company to complete the ongoing trials and report back on the results.

[….]

Doctors may end up agreeing that a drug—even one that is unlikely to work—is better than nothing. “In the American psyche, that is the approach that [doctors and] patients are pushed toward,” says Holly Fernandez Lynch, a bioethicist at the University of Pennsylvania. “We have all this language that you’re ‘fighting against the disease,’ and that you should try everything.”

“I can’t tell you how many FDA advisory committee meetings I’ve been to where the public-comment patients are saying something like ‘This is giving me hope,’” says Zuckerman. “Sometimes hope helps people do better. It certainly helps them feel better. And we all want hope. But in medicine, isn’t it better to have hope based on evidence rather than hope based on hype?”

A desperate decision

A drug approved on weak data might offer nothing more than false hope at a high price, Zuckerman says: “It is not fair for patients and their families to [potentially] have to go into bankruptcy for a drug that isn’t even proven to work.”

The best way for people to access experimental treatments is still through clinical trials, says Bateman-House. Robertson, the health law expert, agrees, and adds that trials should be “bigger, faster, and more inclusive.” If a drug looks as if it’s working, perhaps companies could allow more volunteers to join the trial, for example.

Their reasoning is that people affected by devastating diseases should be protected from ineffective and possibly harmful treatments—even if they want them. Review boards assess how ethical clinical trials are before signing off on them. Participants can’t be charged for drugs they take in clinical trials. And they are carefully monitored by medical professionals during their participation.

That doesn’t mean people who are desperate for treatments are incapable of making good decisions. “They are stuck with bad choices,” says Fernandez Lynch.

To read the entire article, see here

Biden’s Crackdown on ‘Junk’ Plans: Minimal Impact on Payers

August 3, 2023In the News, Policy, We're In the NewsCPTFadmin

Jesus Mesal, Health Payer Specialist, July 14, 2023

The Biden administration’s proposed restrictions on short-term private health plans aim to protect consumers, but they raise questions about the future value potential of a thriving market segment and do little to quell the controversy about insurance criticized by some as “junk.”

Short-term plans offer flexible coverage periods, such as 30 days or three years, and cost 50% to 80% less than individual market coverage. They are not regulated to the same extent as plans offered in the Affordable Care Act insurance marketplaces and can, for example, exclude pre-existing conditions or limit the number of visits or coverage amounts.

The proposal from the Department of Health and Human Services, the Labor Department’s Employee Benefits Security Administration, and the Internal Revenue Service would restrict them to three months, or to four months at a maximum.

These “misleading insurance products” can “trick consumers into buying products that provide little or no coverage when they need it most,” says a joint statement from the agencies.

[….]

The plans, devised as a way for consumers to plug short-term gaps in coverage, have remained a source of political contention since the ACA was enacted in 2010. In 2016, the Obama administration limited their coverage period to three months to address concerns that consumers might choose these plans over comprehensive coverage under the ACA.

Two years later, former President Donald Trump reversed the rule, arguing that consumers should have choice. He extended the allowable duration of short-term plans to a year, with option for consumers to renew them for up to three years. Unlike the ACA’s once-a-year open-enrollment period, these plans are accessible at any time during the year.

Growing market

Since the policy swing, the short-term health insurance market has emerged as a thriving segment experiencing significant growth. The firm Persistence Market Research reports that its value reached $41.1billion in 2022, with the Trump administration rule change playing a substantial role in this expansion.

Mixed opinions

Democratic lawmakers have long advocated for measures to limit the impact of short-term plans. They argue that these lower-cost plans provide minimal coverage and have the potential to lure Americans away from more-comprehensive ACA plans.

The proposed rule could increase ACA marketplace enrollment by an estimated 60,000 individuals in the years2026, 2027, and 2028. Enrollment in ACA plans hit 16.3 million people this year, according to HHS.

“They are called ‘junk’ plans for a reason,” said Diana Zuckerman, president of the National Center for Health Research. Zuckerman questioned why the Biden administration took so long to take this step and does not agree with the argument that having one of these plans is better than having no coverage at all. Short-term plans end up being more expensive for Americans because many people cannot afford the bills they receive when they do not have coverage for emergencies, she said.

“When more people have high-quality health insurance, we are all better protected. These plans do not provide the 10 essential health benefits required by the ACA,” she told Health Payer Specialist. “People claim they have a choice, but what we have observed is that due to misleading marketing, many customers do not fully understand what they are purchasing, and it ends up costing millions of dollars for all Americans.”

[….]

Lilly battling rivals for breast-cancer patients

June 12, 2023In the News, We're In the NewsCPTFadmin

John Russell, Indianapolis Business Journal, June 9, 2023

Eli Lilly and Co. is pushing hard to gain a sales edge against two other drugmakers in the war against metastatic breast cancer. It is spending hundreds of millions of dollars to win over patients with an extensive advertising campaign.

The Indianapolis-based company, maker of cancer drug Verzenio, is blanketing airwaves with commercials that tout its drug’s track record in helping afflicted women live a little longer.

Metastatic breast cancer—also known as MBC or stage 4 breast cancer—is a tough, crippling disease without a cure that claims about 40,000 lives a year. The disease is the most severe form of breast cancer. Nearly three-quarters of all women diagnosed with the disease die within five years.

Last year alone, Lilly spent $111.8 million advertising Verzenio, according to ad-tracking specialist iSpot.tv, as reported by Fierce Pharma Marketing, an industry newsletter. That made Verzenio the 10th-most advertised drug in the U.S. by spending, up 60% from $70.1 million in 2021.

No other cancer drugs broke the top 10 list for advertising spending. (Lilly has two other drugs on the top 10 list—diabetes drug Jardiance, fifth-highest; and diabetes drug Trulicity, seventh-highest.)

Lilly declined to say how much it is spending on its Verzenio campaign, or to confirm the outside estimates. But it defended the use of the direct-to-consumer marketing effort.

[….]

The drugmaker has produced about 10 TV spots for Verzenio since 2018, and two of them are currently running as part of a “Future Photos” campaign for women with metastatic breast cancer. It is also running two spots highlighting use of Verzenio for treatment of early breast cancer as part of its “Make Your Way” campaign.

Lilly shows no sign of slowing its advertising push for Verzenio as it competes against Pfizer’s Ibrance and Novartis’ Kisqali in the war against metastatic breast cancer.

All three medicines belong to a class of drugs called CDK inhibitors, which work by blocking overactive enzymes that would otherwise allow cancer cells to proliferate.

[….]

The drugs are not cheap. List price for Verzenio or Ibrance is about $14,500 a month. For Kisqali, the price ranges from $6,000 to $15,000 a month, depending on the dosage. The actual price for all three drugs varies, depending on health plans and pharmacies.

Three-way fight

Some experts say the three-way advertising war is likely to confuse patients, as they try to figure out, with their doctors, which medicines are likely to help and what to expect from the side effects.

Common side effects for Verzenio, for example, are diarrhea, low white-blood-cell counts, anemia, nausea, headaches and tiredness.

“Competition can be good if it keeps prices down, but otherwise it can be confusing, because they all have different risks,” said Diana Zuckerman, an epidemiologist and president of the National Center for Health Research, a nonpartisan health think tank in Washington, D.C. “It’s rather impossible for the average person to make sense of the list of risks even if they read them.”

[….]

Last month, Lilly launched its latest commercial for Verzenio, an upbeat, 60-second spot that encourages patients to look ahead, not just back. The commercial opens with a 60-something, gray-haired woman sitting on a couch, flipping through a photo album.

“Living with metastatic breast cancer means I cherish my memories,” she says in a voiceover. “But I don’t just look back on them. I look forward to the chance to make new ones every day with Verzenio.”

The camera zooms in to show a new section of the album, titled “Future Memories.” The pages scroll by, showing pictures of the woman at an alumni reunion, a backyard cookout, a New Year’s Eve party and other celebrations, with everyone wearing big smiles.

“Verzenio is proven to help you live significantly longer when taken with fulvestrant,” the announcer says. How much longer? According to small type at the bottom, women who take Verzenio and fulvestrant (an older drug for breast cancer) lived for a median of 46.7 months, versus 37.3 months on fulvestrant alone.

Some experts raised an eyebrow when asked about the “significantly longer” claim, given that the additional survival benefit of Verzenio is about nine months, compared to taking an older drug alone.

“When people hear ‘significant,’ they probably think an extra year or two of life, at least,” Zuckerman said. “For cancer drugs, living nine months longer is considered a meaningful benefit, unless the side effects—nausea, vomiting, diarrhea, exhaustion, etc.—make a person’s life miserable. Wouldn’t you rather have 37 enjoyable months instead of 46 miserable months?”

Indeed, the announcer spends nearly half of the 60-second Verzenio spot listing common side effects and warning patients to see their doctors immediately. (“Blood clots that can lead to death have occurred.”)

Lilly said its direct-to consumer marketing campaign has been successful “at raising awareness and helping patients feel more prepared for discussions about Verzenio with their physicians.”

Two national patient-advocacy groups, Breast Cancer Action and the National Breast Cancer Coalition, declined to comment about the competition among the three drugmakers or the effectiveness of the drugs. Nor did they comment about whether direct-to-consumer marketing was helpful.

Some breast cancer patients who are taking Verzenio acknowledge that the drug has powerful side effects, including diarrhea, but they take it on the advice of their oncologist.

[….]

The ads run only in the United States, one of the few countries to allow direct-to-consumer drug advertising. IBJ asked a few patients who live overseas to look at the ads on the website iSpot.tv for their reaction.

Debbie Donnison, 61, of Worcester, England, who was diagnosed in 2022 with stage 4 breast cancer, said she has read the package insert sheets carefully but was alarmed when listening to the announcer rattling through them in the TV spot.

“They sound terrifying without context,” she said. “…They say them as fast as possible whilst your brain is saying, ‘Hey, hang on a minute.’ I realize they don’t want to focus on them, though, and time is short.”

To read the entire article, click here.

For Canadian Patients, Therapeutic Psychedelics Beset by Red Tape

March 13, 2023We're In the NewsCPTFadmin

Jonathan Moens, Undark, March 8, 2023

IN JANUARY OF 2022, Janis Hughes was told she had two years to live. After two prior bouts of cancer, she was now told she had stage 4 breast cancer, which had already spread to her sternum, ribs, and right lung. The news devastated her, making her feel “a great weight” pressing on her chest, robbing her of any joy. One day, she came across a documentary about how psilocybin mushrooms, commonly known as magic mushrooms, combined with therapy could help people with terminal cancers get relief from their existential dread. Hughes, now 66, was not one for taking what she considered “hard drugs,” but she was intrigued.

“I had nothing to lose at this point, and it just resonated with me — oh, that’s what I need,” she said.

Getting access to these drugs, however, was not going to be easy. In Canada, where Hughes lives, it is generally illegal to use psychedelics outside of a research setting. But since 2020, the government has allowed a small number of patients to seek medical exemptions. More recently, in January 2022, Canada reversed a regulatory restriction that had prohibited patients from accessing restricted drugs, including psychedelics, through its Special Access Program. The SAP, which is overseen by Health Canada, the nation’s public health agency, allows patients with serious or life-threatening conditions to obtain emergency access to unapproved medications when conventional therapies have failed or are otherwise unsuitable.

Canada’s recent policy change makes it among the few countries in the world to explicitly offer psychedelics on a compassionate or emergency use basis.

Hughes applied through the SAP, but soon ran into a hurdle: She had to find a doctor willing to fill out an eight-page form; administer psilocybin, the psychoactive compound in magic mushrooms; and oversee the entire process. The doctor would also have to take responsibility in case anything went wrong. She asked two oncologists at her cancer center and her family physician, but all of them declined. One oncologist had never heard of psilocybin-assisted therapy for terminal cancer patients, Hughes said, and the other told her that she preferred not to get involved because of the amount of paperwork. Her family physician was open to helping with her application, she said, but didn’t want to be responsible for overseeing her use. When Hughes finally found a willing doctor, she would have to travel more than 1,000 miles across Canada to an unfamiliar city to be treated. She decided against it.

[….]

“This SAP process is really a horrible process for getting patients access to experimental drugs like psilocybin,” said Spencer Hawkswell, CEO of TheraPsil, a nonprofit psychedelic advocacy group based in British Columbia that currently works on assisting patients in applying through the SAP. “It just doesn’t work.”

Not everyone sees the SAP so bleakly. While the program may be slow, some medical ethicists and physicians say the application form asks perfectly legitimate questions and should help ensure that the drugs are prescribed safely and effectively. Psychedelics hold a lot of promise, they argue, but the evidence surrounding their use is still preliminary, so safeguarding against misuse is critical.

These experts also say that the best way to get access to these drugs is through a clinical trial, a type of study that tests how well a treatment works in humans. Such trials guarantee a certain level of safety and help health regulators compile data to rigorously identify the benefits of the drugs. “Then all patients could ideally have access to them without going through these pathways,” said Holly Fernandez Lynch, an assistant professor of medical ethics and health policy at the University of Pennsylvania.

Clinical trials specific to these patients are rare, however, and conducting one is costly. And according to the SAP’s critics, patients are often left with few legal options.

THE FIRST STUDIES specifically looking at how psychedelics could help patients with existential distress took place in the late 1960s in the United States. While the studies mostly focused on drugs other than psilocybin, they generally seemed to suggest that psychedelics could help terminal cancer patients relieve deep-seated feelings of anxiety. But those studies came to a halt in 1970, when the country’s Controlled Substances Act banned psychedelics and classified these and other drugs as “Schedule I,” meaning they were now considered to have “no currently accepted medical use” and “a high potential for abuse” by federal law.

It was only about 30 years later that scientists picked up where they had left off. Of particular importance were two seminal studies published in 2016 by researchers at Johns Hopkins University and New York University, respectively, showing that terminally ill patients experiencing distress exhibited significant improvements in mood, anxiety, quality of life, and depressive symptoms after a single high dose of psilocybin. The studies had notable limitations: The sample sizes were relatively small, about 90 percent of participants were White, and about half had post-graduate education. But, for most of the NYU study participants, the drugs had quick and positive effects that lasted at least four and a half years.

End-of-life anxiety is not an official clinical diagnosis, however, so there was no pathway to approval by the U.S. Food and Drug Administration. Scientists soon pivoted to other conditions, including treatment-resistant depression. One of the latest studies on these patients found that psilocybin alleviated depressive symptoms as well as escitalopram, an often-used antidepressant. Last November, the biggest study on this group of patients to date, with more than 200 participants, showed that a high dose of psilocybin significantly reduced depression scores.

[….] And questions about how the drug actually works in the brain still remain. Scientists are just starting to map the specific receptors, pathways, and brain regions that seem to be altered when patients are on psychedelic drugs; there is currently no consensus on what biological mechanisms might explain the drugs’ anxiety-relieving or mood-boosting effects.

“The really quick answer,” said Albert Garcia-Romeu, an assistant professor and researcher in the psychedelics research center at Johns Hopkins: “We don’t know.”

[….]

SEVERAL HEALTH EXPERTS who do not have direct ties to Health Canada or to psychedelic advocacy groups reviewed the SAP application form for Undark and said they do see merit in the questions. Among other things, the form asks for patient and drug manufacturer information, the rationale for prescribing the drug, and treatments the patients have tried already.

“None of those things strike me as an outrageous ask,” said Fernandez Lynch, the University of Pennsylvania professor.

Quite the contrary, these questions are a necessary vetting tool to ensure the safe distribution of unapproved drugs, said James Downar, the head of palliative care at the University of Ottawa.

Hawkswell acknowledged that many of the questions posed are legitimate but said that much of the wasted time comes from physicians having to answer “absurd” follow-up questions by Health Canada, like asking doctors why symptoms like suicidal ideation, severe depression, and hopelessness are unacceptable.

In one example, Health Canada asked a medical professional to provide reasons why two of his patients, who were applying to use psilocybin through the SAP, couldn’t seek other legal treatments, including electroconvulsive therapy.

While ECT is approved for treatment of severe depression, mania, and schizophrenia in Canada, the therapy is controversial and, in some cases, can lead to serious adverse effects, including memory loss. Eventually, after some discussions with the health agency and pressure from the media, the SAP approved psilocybin for all four of his patients.

The controversy highlights what critics see as the heart of the issue: Unlike physicians, SAP health officials are trained to “check boxes” that follow internal guidelines and not to carefully evaluate what treatment is best for patients, Masuda said. Requests passing through the U.S. equivalent of the SAP, known as expanded access, are also vetted by health officials within the FDA, but the final say rests squarely with the medical professional, said Diana Zuckerman, president of the National Center for Health Research, who was involved in finding ways to improve the U.S. program. In fact, she said, the FDA approves expanded access requests in virtually all instances and the relevant forms are designed to be easy to complete and take about 20 minutes.

The SAP could follow a similar model. It might also benefit from creating a standardized form with pre-written answers that physicians could check off and determine whether their patients are eligible for psychedelic drugs, said Garcia-Romeu, the Johns Hopkins researcher. That way, the process would be streamlined, there would be fewer ambiguities, and drugs would likely be more fairly distributed.

[….]

To read the entire article, click here.

Many pediatric drug study results were never posted to a U.S. government database

February 17, 2023We're In the NewsCPTFadmin

Ed Silverman, Stat News, January 24, 2023

Amid ongoing controversy over clinical trial transparency, a new analysis found that results of 43 studies involving thousands of children were never reported to a U.S. government database or published in the scientific literature.

In some cases, medicines being studied were for such life-threatening conditions as congenital heart disease and Duchenne muscular dystrophy. One study explored the use of a particular painkiller for reducing the amount of opioids needed to treat pain following heart surgery. Another tested a drug for lowering aggression among children diagnosed with attention deficit hyperactivity disorder, or ADHD.

In each instance, the researchers scoured ClinicalTrials.gov, the federal database, and medical journals, but were unable to find results. More than 3,600 children participated across the 43 studies. There was partial information posted to the database or published in journals for another 22 studies, although the researchers noted that medical journal findings can be unreliable or omit relevant outcome data.

“When any adult or child participates in a clinical trial, they are taking a risk for the greater good, but if the results are not made public, those studies are of no help to anyone,” said Diana Zuckerman, who heads the National Center for Health Research, a nonprofit think tank that was one of three organizations that conducted the analysis. “Our next step is to work with Congress to fix this situation.”

The study has not yet been published in a scientific journal, but was made available on medRxiv, a preprint server. These platforms have become an increasingly popular destination for many studies before they have been peer-reviewed by medical journals.

In one instance, Astria Therapeutics failed to post results for an open label extension study of a drug to combat Duchenne muscular dystrophy. The study enrolled 130 boys. But after the Phase 3 trial failed, research into the drug was abandoned and the study was terminated. The company made the Phase 3 results available 110 days later than required by law.

But results for the extension study have still not been posted to ClinicalTrials.gov and are more than a year overdue. TranspariMED, another of the organizations that conducted the analysis, wrote that it was unable to locate final results from the extension study in scientific The company did not provide an explanation. We asked the company for an explanation and will update you accordingly.

In another instance, Phoenix Children’s Hospital ran a trial to determine whether a painkiller, ketorolac, could reduce opiates needed to treat pain in children after heart surgery. The hospital sought to enroll 166 infants and children aged between 3 months and 4 years. The primary completion date was December 2020, but results were never posted and the hospital did not respond to TranspariMED. We asked the hospital for comment and will pass along any reply.

[….]

To read the entire article, click here.

Lawmakers, advocates press for diversity in clinical trials

February 16, 2023We're In the NewsCPTFadmin

Erin Durkin, National Journal, February 15, 2023

Congress in December enacted new requirements that require drug- and device-makers to ensure there is sufficient diversity in their clinical trials. Yet lawmakers and advocates don’t want to stop there.

Among them is Democratic Rep. Robin Kelly, who plans to reintroduce a bill this month that would require sponsors of research funded by the National Institutes of Health to lay out recruitment goals that reflect the race, ethnicity, age, and sex of patients who have the condition being studied or that reflect the general population of the United States. Republican Rep. Brian Fitzpatrick cosponsored the measure. Republican Sen. Susan Collins and Democratic Sen. Bob Menendez sponsored a companion bill in the Senate.

“Racial and ethnic minorities are not properly represented in clinical research. That’s the bottom line,” Kelly told National Journal.

The proposal aligns with a measure enacted as part of the massive government-funding bill passed at the end of December. That provision requires drug and device sponsors to submit diversity action plans to the Food and Drug Administration. The agency is also required to develop new guidelines or update already existing guidance for these plans.

Kelly said her focus on NIH clinical trials can complement the FDA measures because there are “things that NIH will capture that FDA won’t capture.”

[….]

Eyes are now on the implementation of the new requirements for the FDA. Diana Zuckerman, president of the National Center for Health Research, said she is concerned that the new provisions lack teeth but that it is “potentially helpful” that Congress “reminds the agencies that they’re not happy with the lack of diversity.”

Ricki Fairley, cofounder and CEO of Touch, The Black Breast Cancer Alliance, said Kelly’s bill focusing on the NIH is a start, and that “we need more of this.” She said participation in a clinical trial should reflect “the burden of disease.”

“Who is getting this disease, who’s dying from it … that’s who should be in the trials. … Until we get that level of care and support, it’s not going to change,” said Fairley, who works on the campaign When We Tri(al), which aims to get more Black women to enroll in clinical trials for breast-cancer treatments.

“I know I can’t change the mind-set of a health care professional with illicit bias—I can’t change that,” she said. “What I can change—I can talk to Black women, educate Black women, and … teach them how to advocate for themselves.”

Kelly said her bill won’t fully solve the issue of diversity in clinical trials but that “it will take care of some things.” Along with submitting recruitment plans for clinical trials, the measure would require NIH and FDA to conduct a national awareness and educational campaign around the need for diverse clinical trials. She pointed to the use of “trusted messengers” during the COVID-19 pandemic to get people vaccinated.

To read the entire article, click here.

A Tiny Lab Finds Danger on Drugstore Shelves While the FDA Lags Behind

November 9, 2022Uncategorized, We're In the NewsCPTFadmin

Anna Edney, Bloomberg News, November 9, 2022

David Light can’t wait to show off his tchotchkes. The curly haired scientist lights up with boyish enthusiasm when he picks up a black coffee mug from the endless array of memorabilia in his office. It’s emblazoned with the trademark lettering of Zantac, the blockbuster heartburn drug. He quickly moves on to a Zantac wine glass from 1983, when the heartburn drug was approved for sale in the US, and then a white and blue Zantac Swiss army knife. A globe, then a t-shirt, next a hat — all stamped with the drug’s branding.

One floor above his office is the lab where groundbreaking Zantac research took place. But Light didn’t create Zantac — he nearly destroyed it. In the process, he’s also become a stand-in, protecting the American public from cancer-causing chemicals in place of a federal regulator that’s failed to do the job.

Light is the co-founder and chief executive officer of Valisure, the independent testing lab that first released research showing that Zantac and its generic forms were contaminated with a toxic chemical known to cause cancer. The findings, published in 2019, helped lead to massive recalls and eventual market withdrawal. Some two dozen companies were selling versions of the drug at the time.

Valisure truly shot into the public eye last year when it was the first to warn that some widely used hand sanitizers had high levels of carcinogens. Next came the lab’s evidence of leukemia-causing benzene in sunscreens. Then Valisure alerted consumers to dangerous chemicals in spray antiperspirants, and, more recently, dry shampoos. The lab has also warned of contaminants in a popular diabetes treatment. Procter & Gamble Co., Johnson & Johnson, Unilever Plc, CVS Health Corp. and Beiersdorf AG have all issued recalls or halted sales following Valisure’s findings.

In the course of just three years, Valisure’s quest to hunt down cancer-causing chemicals in everyday products has impacted pharmaceuticals and consumer goods in markets worth an estimated $9 billion that touch the lives of tens of millions of Americans.

“I feel that we’ve already saved many lives,” Light said. “When such big numbers of people are exposed, years of exposure with a well-defined carcinogen, there is no doubt there’s unacceptable risk.”

On every step of that journey, the Food and Drug Administration, the agency responsible for safeguarding consumers from these problems, has lagged behind the small lab.

[….]

Impurity Testing

The testing done by Valisure is routine and can be run by any company that makes pharmaceuticals. On a recent visit, a scientist in a white lab coat replicated the experiment on an older version of Zantac at Valisure’s lab in Connecticut. A vial of milky pink liquid disappears into a machine that costs more than some luxury cars. The liquid is vaporized and separated into its various components. After several minutes, there’s a readout on a nearby computer screen: There’s a tall peak, like an irregular rhythm on a heartrate monitor. The figure indicates high impurity levels.

Valisure first released its Zantac findings to the public in September 2019. It took roughly seven months for the FDA to finally force the drugmakers to pull Zantac products off the market.

GSK Plc, the creator and original seller of Zantac, maintains that there is “no consistent or reliable evidence” the drug “increases the risk for any type of cancer,” the company said in an emailed statement. Sanofi, the most recent seller of the non-prescription version of the drug, said it “stands by the safety of the medicine today.”

Powerful Regulator

As problems with carcinogen-laden medications and consumer products continue to fall through the cracks, the FDA maintains it is up to companies to ensure their products are safe. The situation highlights one of the biggest challenges at the agency: It doesn’t conduct much testing for these types of contaminants.

“FDA doesn’t do routine testing,” said Scott Knoer, who served as the CEO of the American Pharmacists Association for two years before stepping down in June.

“I had always believed anything in the US was safe,” Knoer said. “It was not as thorough as I guess previous perception was.”

The FDA takes a risk-based approach to quality testing, said Harrison, the agency spokeswoman. Each year it focuses on analyzing a few dozen products with already known issues. For example, the agency tested many hand sanitizers in the year that ended Sept. 30, 2021.

Pharmaceutical Fees

The FDA has an enormous purview, overseeing not just food and drugs, but also medical devices, tobacco and cosmetics. Despite that, its budget is just half that of the Environmental Protection Agency or the Internal Revenue Service. And about two-thirds of the funding the FDA receives for its drug activities comes from user fees paid by pharmaceutical companies. Since the early 1990s, the FDA and drugmakers have negotiated a deal every five years that Congress then approves. That agreement between the FDA and drugmakers dictates what the FDA can then do with those user fees.

It costs a drug company more than $3 million in fees to submit a new drug application to the FDA for review. In exchange, the agency has to meet review deadlines to help speed up the application process. This all means the regulator has far fewer funds to put toward making sure drugs already on the market are safe. It also means the industry gets a lot of deference.

“User fees provide access to FDA decision-makers in ways that foster a cozier relationship between FDA and industry,” said Diana Zuckerman, president of the National Center for Health Research, a think tank that focuses on the safety of medical and consumer products.

The agency has taken heat in the last year and a half for approving an Alzheimer’s disease drug that wasn’t fully proven to work, leading to accusations that the body is too beholden to the drug industry. The FDA also let clear signs of problems at an Abbott Laboratories’ infant formula plant slip by for almost five months before overseeing a recall and temporary closure of the factory in February, which ultimately led to a national formula crisis. These fiascoes have consumed the agency at a time when it’s been overwhelmed by the race to approve Covid-19 treatments and vaccines. All the while, recalls of sunscreen, antiperspirants, hand sanitizers and dry shampoo keep piling up.

[….]

Carcinogens and Where They’re Found

Rather than embrace Valisure as a partner in protecting the public, the FDA instead turned combative.

On May 24, 2021, Valisure released its findings showing cancer-causing chemicals in sunscreens. Two days later, two FDA inspectors showed up at the lab, according to agency documents. They brought an FDA lawyer with them for three of the 11 days they were at Valisure. The three FDA employees were deployed to the Valisure lab at a time when the regulator was focused on conducting its most critical inspections amid a pandemic backlog.

Valisure has about 20 employees who work in an office space of about 6,000 square feet in New Haven, Connecticut. The FDA, to compare, operates in 3.1 million square feet of office and lab space on a sprawling campus in Silver Spring, Maryland. It has 18,000 employees and a $6 billion annual budget.

To read entire article, click here.

A Special Report: Can Breast Implants Cause Chronic Disease?

October 26, 2022News Stories & Editorials, Uncategorized, We're In the NewsCPTFadmin

Julia Halpert, HealthCentral: October 25, 2022

With new FDA warnings, troubling research, and a growing online population sharing stories and symptoms, experts and women with implants weigh in.

JENNIFER JOHNSON, 43, of Wilcox, NE, underwent a preventative double-mastectomy—a surgical procedure that removes all tissue from both breasts—in July 2008 at age 29 after learning she carried the BRCA2 genetic mutation.

Research shows that having BRCA2 increases risk of developing breast cancer (BC) by 45%. Johnson’s family history didn’t make her keen to play the odds: Her mother died from the disease at 34, as did her sister Debbie at 39, while another sister, Valerie, was diagnosed with BC in her 40s and, thankfully, is still here. After Johnson’s doctor told her that her own chances of facing a similar fate were exceedingly high, she chose the double-mastectomy as the safer bet.

The surgery didn’t spare her, however. A post-op pathology report found that Johnson already had an aggressive type of breast cancer (“stage 1, triple-negative, grade 3”) in her right breast that required immediate treatment.

Her plastic surgeon was “adamant,” she says, that she get breast implants to return her body to normal, since she was so young. She got silicone implants on her 30th birthday. Within several months, she began experiencing intermittent, aching pain in her muscles and joints, as well as “shooting, stabbing pains” in her chest, she reports. She also battled rashes and severe fatigue. “I basically felt like I was dying a slow death, like my body was just giving out slowly over time,” she recalls.

A team of specialists told her nothing was wrong. But her symptoms continued—leaving her distraught. After four years of this, she had her implants removed (known as explant surgery). To her great relief, “I started feeling better right away,” she says. “Every single symptom disappeared within a year.”

Johnson is among those who have experienced what’s colloquially known as breast implant illness (BII), when significant health issues—fatigue, chest pain, hair loss, headaches, chills, photosensitivity, rash, chronic joint pain, among other symptoms—arise after getting implants.

[….]

We asked women who’ve undergone reconstruction or done elective breast implant surgery to share their experiences. We also polled breast health experts on their thoughts about this popular cosmetic surgery being done in the U.S. and around the world—and its potential implications for the chronic community.

SAFETY CONCERNS

Are Breast Implants Safe? Or Not?

In October 2021, the FDA issued new restrictions for breast implants, including a mandated box warning on the product label to inform patients of significant health risks, such as an increased cancer risk; a checklist of items that health care providers should discuss with patients as they consider implants; updated silicone gel-filled breast implant rupture screening recommendations; and a list of specific materials used to create the implant.

Then, this past September, the FDA issued a safety communication following reports of cancers, including squamous cell carcinoma (SCC) and various lymphomas in the scar tissue that had formed around breast implants, noting that “currently, the incidence rate and risk factors for SCC and various lymphomas in the capsule around the breast implants are unknown.” A spokesperson for the agency added, “The FDA recognizes that many patients’ symptoms may take years to develop, and patients may not be aware of the risk of SCC … We will keep the public informed as significant new information becomes available about SCC and lymphoma variants in the breast implant capsule.”

[….]

PATIENT REPORTS

Implants Remain Popular, Yet Some Patients Suffer

Safety issues haven’t dimmed enthusiasm for breast implants. According to a 2020 report by the American Society of Plastic Surgeons (ASPS), there were 137,808 implants provided for breast reconstruction and 193,073 for cosmetic surgery in this country alone. Silicone implants were used in 84% of breast augmentations, while saline implants were used in 16% of such procedures in 2020.

Mark Clemens, M.D., a professor of plastic surgery at MD Anderson in Houston, TX, who has led several MD Anderson-based safety studies on breast implants, says the recent FDA communication shouldn’t alter the perception of breast implant safety. He believes that it was done out of abundance of caution to inform, not frighten, women. When it comes to breast implants, “the vast majority of women will be completely healthy [after getting them] and won’t have any issues,” he says. However, he urges women who notice any signs of abnormality—asymmetry between breasts, the firming of a breast, or a palpable mass or a fluid collection—to consult a physician to ensure there’s nothing wrong.

Diana Zuckerman, Ph.D., president of the National Center for Health Research, a Washington, D.C.-based nonprofit organization that draws from scientific studies to improve public policy and medical oversight in the U.S., believes more independent research is required before an accurate safety assessment can be made. She says that nearly all the research being done on breast implant safety has been conducted by the very hospitals and plastic surgery organizations that either offer reconstruction and elective implant procedures as a service, or represent the surgeons who are paid to perform them—a big source of revenue and conflict of interest, leaving troubling questions of inherent bias being baked into the results.

“I can’t emphasize enough how much resistance there has been from the plastic surgeons’ medical societies and the implant manufacturers” to doing more and better research on implants, Zuckerman says. While some plastic surgeons have vocalized their concerns over the need for better information for their patients, “the medical societies—the major sources of information that FDA officials rely on—have been vehemently opposed,” she reports. “Their usual mantras are some variation of ‘breast implants are the most studied medical device in history’; hundreds of studies prove they are very safe’; and ‘so-called breast implant illness symptoms are common symptoms caused by aging and other factors, not by the implants.’” Implant manufacturers say the same thing—not coincidentally, Zuckerman adds.

Nicole Daruda, age 58 and living in Vancouver Island in Canada, openly doubts the industry’s safety claims. “Breast implants are linked to autoimmune symptoms and diseases and many other health problems,” she maintains. Daruda got cohesive gel implants in 2005 and saw her once excellent health “decimated by breast implants.” Within the first few years of having them she says she experienced fatigue, brain fog, various infections, food allergies, and hypothyroidism, with more symptoms appearing each year.

Daruda had her implants removed in 2013, and within four years she says all of her symptoms resolved. She started the Facebook group, Breast Implant Illness and Healing by Nicole, in April 2015 to provide a forum for women experiencing health issues after having implants to support and talk to each other. The group now has more than 170,000 members. Daruda says that she’s heard from thousands of women on her social media platform who report their health has improved after getting their implants removed.

[….]

IMPLANTS AND LYMPHOMA

What You Need to Know About Lymphoma

According to the FDA, as of September 2020, more than 700 people worldwide have been diagnosed with breast implant-associated anaplastic large cell lymphoma, an uncommon cancer. The agency found that the women with textured breast implants have a small but increased risk of developing this disease. The working theory, Dr. Glasberg explains, is that the texturing on the implant drives inflammation, which causes a change in the capsule around the implant that then develops into lymphoma.

Despite his belief that breast implants are safe for the vast majority of women, Dr. Clemens authored a 2021 study that examined eight cases of Epstein–Barr virus-positive large B-cell lymphoma associated with breast implants “and we’ve been trying to understand these better,” he says. (The eight women in the study were all patients at MD Anderson, a medical center that offers breast reconstruction and elective breast implant surgery, who were among the 30 known cases in the world of this type of lymphoma, per the FDA tally.) Increased awareness, combined with more pathology testing of scar tissue, plus physicians and patients being aware of breast implant-associated issues has “drawn our attention to looking for these other diseases,” he says.

IMPLANTS AND AUTOIMMUNE DISEASE

Breast Implants and Autoimmune Disease

Autoimmune issues arise when the body mistakenly attacks its own healthy tissue, causing damaging inflammation and often chronic pain and fatigue, among other symptoms, some of them disabling and/or permanent.

In 2018, MD Anderson conducted the largest study to date to explore long-term safety outcomes of breast implants, finding an association, though not a causation, with some rare diseases, including the autoimmune disorders Sjögren’s syndrome, rheumatoid arthritis (RA), and scleroderma. What’s more, researchers in the Netherlands found that more than two-thirds of women with autoimmune symptoms who had their breast implants removed experienced a reduction in symptoms.

That same year an Israeli study—research that Zuckerman says is both independent and well-designed—compared more than 24,000 breast implant patients to more than 98,000 women without breast implants but who shared similar demographic traits and reported a 22% increase in several autoimmune and rheumatic disorders, as diagnosed by their physicians and reported in their medical records. In addition, the same study reported a 60% increased risk of Sjögren’s syndrome, multiple sclerosis (MS), and sarcoidosis among those with implants, as well.

Dr. Clemens, the principal investigator of the large MD Anderson study, points out that some of those diseases in his study were self-reported by study participants, and not necessarily diagnosed by a physician—a limitation of the research. He doesn’t believe the findings are cause for concern. “The vast majority of patients with implants do not experience these symptoms or diseases,” he says. “However, it is important that they are aware of these conditions so that if they note any changes or have concerns, they can discuss with their treating physician.”

Then again, a 2021 study on breast implants and respiratory health found that 74% of participants who had their breast implants removed showed significant improvements on at least three of the six pulmonary function tests performed—an objective, not self-reported, medical tool.

For her part, Zuckerman notes that research is often funded by implant manufacturers and used to argue that breast implant illness is not real. A major weakness of most BII studies, a report by her organization found, is that they evaluate only diagnosed diseases. The reason why women decide to have their implants surgically removed and not replaced, she explains, is often due to symptoms of autoimmune and connective tissue diseases, rather than official diagnoses.

“The women and their doctors often report a constellation of symptoms that do not fit the exact criteria of known diseases,” she explains, adding that most people aren’t hospitalized for the autoimmune issues most associated with BII. Without symptoms that perfectly fit a specific diagnosis, many women will not have a diagnosis logged into medical records.

[….]

REMOVING YOUR IMPLANTS

Can Implant Removal Mean a Return to Health?

Some women, who can find no other explanation for their symptoms, like Johnson, are having their implants removed. In 2020, 22,676 explants were performed on reconstruction patients in the U.S., per the ASPS. Johnson says she was forced to find a different plastic surgeon to perform the procedure, since the one who put them in didn’t believe they caused health issues.

“He stood back looking at my chest and said, ‘I did an amazing job on those and really don’t want to take them out,’” she recalls.

[….]

Zuckerman believes the health rebound after explant surgery may be higher than the plastic surgery industry acknowledges. Since 2015, her organization has been contacted by more than 4,500 women who had breast implants they wanted removed due to rupture, breast pain, or medical symptoms caused, they believed, by their implants. NCHR was asked to advocate with health insurance companies, Medicare, and Medicaid to cover the costs of implant removal, she adds, since many of the women could not afford explant surgery.

[….]

To read the entire article, click here.