All posts by CPTFadmin

For Canadian Patients, Therapeutic Psychedelics Beset by Red Tape

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.

Can Daily Aspirin Prevent Both Heart Disease and Cancer?

Laura Gottschalk, Ph.D., Sasha Milbeck & Meg Seymour, PhD

Many adults take a low-dose aspirin (or “baby” aspirin) to prevent heart disease and possibly cancer. The most recent data indicates that in 2017, 23% of U.S. adults over 40 who did not have heart disease took low-dose aspirin to help prevent developing it.1 About 23% of people taking “baby” aspirin daily reported that they did so without a doctor recommending it. Should you take daily aspirin to prevent heart disease? The recommendations depend on your age, as well as other risk factors.

In April 2022, the United States Preventive Services Task Force (USPSTF) issued updated recommendations that people over 60 should not start taking low-dose aspirin, because the benefits do not outweigh the risks.2 For those ages 40-59 with a 10% or more chance of developing heart disease over the next 10 years, the USPSTF says the data are unclear. So, the decision to start taking low-dose aspirin should be made with one’s physician, weighing the risks (which vary among individuals) against the small benefits.

These recommendations are notably different from the USPSTF’s 2016 recommendations, which did not recommend against starting to take low-dose aspirin for those ages 60-69. The 2016 recommendations also recommended adults ages 50-59 with a 10% or more chance of developing heart disease over the next 10 years and who were willing to take daily aspirin for those 10 years begin to do so.3

What Are the Benefits and Risks?

Why have the recommendations changed over time, and what do we now know about the cardiovascular benefits of taking low-dose aspirin that we didn’t know a few years ago? A 2019 study combined the results of 13 clinical trials, all comparing daily aspirin to placebo or to no treatment.4 The combination study, called a meta-analysis, analyzed over 164,000 patients, who were followed for an average of five years. The researchers found that patients who used daily aspirin had fewer heart attacks and fewer ischemic strokes. However, the authors also found that people who took daily aspirin were more likely to have intracranial bleeding (inside the skull) and major gastrointestinal bleeding.

The USPSTF reviewed the research on low-dose aspirin in order to develop their recommendation, and drew similar conclusions.5 The analysis of over 161,000 patients found that people taking daily aspirin reduced their chances of a having at least one major cardiovascular event such as a heart attack or stroke by 2.5%. And yet, the analysis also found that people taking “baby” aspirin were not less likely to die from heart attacks or strokes. In addition, people taking daily aspirin were 1% more likely to experience major bleeding, such as gastrointestinal bleeding that resulted in a transfusion, hospital admission, intracranial bleeding, or death.

It is important to know who is most likely to experience harmful bleeding. Research has shown that the increased chances of bleeding begins relatively soon after starting to take “baby” aspirin on a daily basis, and it increases with age. Bleeding is more common among men, smokers, people with high blood pressure, people with liver disease, people with diabetes, and people with a history of gastrointestinal problems, such as peptic ulcer disease.5 Taking certain medications can also increase the risk of bleeding, including nonsteroidal anti-inflammatory drugs (such as ibuprofen or naproxen), steroids (such as prednisone), and anticoagulants (such as warfarin). We agree with the USPSTF recommendation that these factors make dangerous bleeding more likely for anyone taking daily aspirin.

In a 2023 meta-analysis of 16 studies, researchers compared the risks and benefits of low-dose aspirin in adults who did not have cardiovascular disease who use statins to those who did not use statins.6 Statins are a group of drugs that lower LDL or “bad” cholesterol by helping the body slow down cholesterol production and rev up cholesterol removal. Researchers found that at every level of cardiovascular disease risk, the chances of developing major bleeding complications from daily aspirin were greater than the chances of the aspirin preventing heart attacks or other cardiovascular problems. That was true for patients taking low-dose aspirin alone or with a statin. For that reason, statins alone are now considered safer than low-dose aspirin for preventing heart attacks even for those with little or no risk.

According to the USPSTF, the benefits of daily aspirin use are different for someone in their 60s or 70s who has already been taking low-dose aspirin compared to someone in their 60s or 70s who is considering whether to start to take daily aspirin. For people already taking daily aspirin, the benefits become smaller with age, because the chance of serious bleeding increases with age.  Modeling data suggest it may be a good choice to consider stopping taking regular aspirin after around 75. However, stopping daily aspirin can lead to a “rebound” effect where the chances of cardiovascular events increases slightly within the first few weeks that is maintained for at least one year.7 The increased risk means that about one out of every 74 patients (1.4%) who discontinue use will experience a cardiovascular event, such as heart attack or stroke. The risk is higher than 1.4% for people who previously had a CVD event and lower for those who took daily aspirin to prevent a first CVD event.8 If you are currently taking daily low-dose aspirin and want to consider stopping, speak with your doctor about how to safely taper your use.

Does Daily Aspirin Prevent Cancer?

The USPSTF also reviewed scientific studies of whether daily low-dose aspirin can help prevent colorectal cancer. They found that four studies following patients for up to 10 years found no evidence that taking daily aspirin reduced the chances of developing colorectal cancer.5

The 2019 paper mentioned above also found that daily aspirin did not reduce the chances of dying of cancer, compared with no aspirin.4 In fact, one clinical trial in that meta-analysis (called ASPREE: Aspirin in Reducing Events in the Elderly) found that individuals who took aspirin were slightly more likely to die from cancers. Researchers analyzed the overall chance of dying from 14 common cancers, including colorectal cancer, and found that 3.1% of the participants taking daily aspirin had died a cancer-related death at five years follow-up, compared with 2.3% of those taking a placebo.9 This is a small difference but is it conclusive? Most participants in the ASPREE trial had never used low-dose aspirin regularly before joining the study, and that made it impossible to determine if taking “baby” aspirin regularly for many years is likely to increase or decrease the chances of dying of cancer.

However, a more recent, longer-term study, published in 2021 found that people who started using low-dose aspirin regularly at a younger age and who continued after age 70 were less likely to develop colorectal cancer than people who didn’t use low-dose aspirin.10 That study combined 2 large studies that included almost 95,000 patients.

The USPSTF 2022 analysis notes that the evidence about daily aspirin and colorectal cancer is highly variable, with randomized clinical trials showing no benefit and some long-term observational studies showing some possible benefit.11 Given these inconsistent results, the USPSTF does not recommend that older patients begin taking low-dose aspirin for the sole purpose of preventing colorectal cancer.

The Bottom Line: Should I Take a Daily Aspirin?

Overall, the choice to begin taking low-dose aspirin (or to continue if you are already taking it) should be a decision that you make with your healthcare provider, after weighing the potential risks and benefits. The research described above is a good reminder that aspirin is a drug that has risks even at low doses. For many people, aspirin’s risk of serious bleeding may outweigh the benefits of decreased cardiovascular events. However, if you have been taking low-dose aspirin, stopping to take it also has risks, especially if you previously had a cardiovascular event such as a heart attack or stroke. When discussing with your healthcare provider the possibility of taking daily aspirin, let them know:

  •       Your medical history and all the medicines you are currently using, whether they are prescription or over-the-counter,
  •       Any allergies or sensitivities you may have to aspirin, and
  •       Any vitamins or dietary supplements you are currently taking

To read our comments made to the USPSTF about their 2021 updates to their recommendation statement, click here.

Other Ways to Prevent Heart Disease and Cancer

In 2022, heart disease and cancer were the leading causes of death in adults in the United States.

To reduce your risk of heart disease, don’t smoke, exercise regularly, keep your cholesterol and blood pressure under control, and do what you need to do to prevent diabetes.  Being a man and being older also put you at risk, but those are factors you can’t control.

To reduce your risk of colorectal cancer, don’t smoke, don’t drink alcohol in excess, have a healthy diet, stay physically active, and maintain a healthy weight.  Being older, and having a family history of colon cancer, Crohn’s disease, or ulcerative colitis are the risk factors you can’t control.

All articles are reviewed and approved by Dr. Diana Zuckerman and other senior staff.

The National Center for Health Research is a nonprofit, nonpartisan research, education and advocacy organization that analyzes and explains the latest medical research and speaks out on policies and programs. We do not accept funding from pharmaceutical companies or medical device manufacturers. Find out how you can support us here.


  1. O’Brien CW, Juraschek SP, Wee CC. Prevalence of aspirin use for primary prevention of cardiovascular disease in the United States: results from the 2017 National Health Interview Survey. Annals of Internal Medicine. 2019 Oct 15;171(8):596-8.
  2. Final Recommendation Statement: Aspirin Use to Prevent Cardiovascular Disease: Preventive Medication. April 26, 2022
  3. Final Recommendation Statement: Aspirin Use to Prevent Cardiovascular Disease and Colorectal Cancer: Preventive Medication. April 11, 2016.
  4. Zheng SL, Roddick AJ. Association of aspirin use for primary prevention with cardiovascular events and bleeding events: a systematic review and meta-analysis. JAMA. 2019;321(3):277-87.
  5. Guirguis-Blake JM, Evans CV, Perdue LA, Bean SI, Senger CA. Aspirin Use to Prevent Cardiovascular Disease and Colorectal Cancer: An Evidence Update for the U.S. Preventive Services Task Force. Evidence Synthesis No. 211. Rockville, MD: Agency for Healthcare Research and Quality; 2021. AHRQ publication no. 21-05283-EF-1.
  6. Khan SU, Lone AN, Kleiman NS, Arshad A, Jain V, Al Rifai M, et al. Aspirin With or Without Statin in Individuals Without Atherosclerotic Cardiovascular Disease Across Risk Categories. JACC: Advances. 2023 Feb;100197.
  7.  Maulaz AB, Bezerra DC, Michel P, Bogousslavsky J. Effect of discontinuing aspirin therapy on the risk of brain ischemic stroke. Archives of Neurology. 2005; 62(8):1217-20.
  8. Sundström J, Hedberg J, Thuresson M, Aarskog P, Johannesen KM, Oldgren J. Low-dose aspirin discontinuation and risk of cardiovascular events: a Swedish nationwide, population-based cohort study. Circulation. 2017; 136(13):1183-92.
  9. McNeil JJ, Nelson MR, Woods RL, Lockery JE, Wolfe R, Reid CM, Kirpach B, Shah RC, Ives DG, Storey E, Ryan J. Effect of aspirin on all-cause mortality in the healthy elderly. New England Journal of Medicine. 2018; 379(16):1519-28.
  10.  Guo C, Ma W, Drew DA, et al. Aspirin Use and Risk of Colorectal Cancer Among Older Adults. JAMA Oncology. Published online January 21, 2021. doi:10.1001/jamaoncol.2020.7338
  11. Mora S, Shufelt CL, Manson JE. Whom to Treat for Primary Prevention of Atherosclerotic Cardiovascular Disease: The Aspirin Dilemma. JAMA Intern Med. Published online April 26, 2022. doi:10.1001/jamainternmed.2022.1365

Statement of Dr. Diana Zuckerman, President, National Center for Health Research, March 8, 2023

Today the FDA announced that 19 women were reported in medical publications who developed squamous cell carcinoma (SCC) in the capsule around breast implants. This is more than the 10 women that FDA reported in September. Several of the women died.  It is important to know that 24 cases of SCC have been reported to the FDA, but it is not known if these cases overlap with the 19 that were published or if some of the 24 cases were reported more than once. Some of the women had silicone gel implants, some had saline implants, some textured, some smooth.  Some of the women got breast implants for augmentation, others for reconstruction after mastectomy.  That means that all women with breast implants need to be aware of this risk, even though it may be rare.

Last September, the FDA also reported 12 cases of lymphomas different from anaplastic large cell lymphoma (ALCL) that were also caused by breast implants.  The agency has not updated those numbers.

Meeting of Patient, Consumer, and Public Health Coalition with Commissioner Califf and Key FDA Officials

March 1, 2023

Location: FDA White Oak Campus, Silver Spring, MD-Building 1, Great Room

FDA attendees: Robert Califf (Commissioner, virtual due to exposure to viruses), Peter Marks (Director, CBER, virtual), Owen Faris (OPEQ Principal Deputy Director, CDRH, virtual); In person: Julia Tierney (Chief of Staff, Office of the Commissioner), Jacqueline Corrigan-Curay, (Principal Deputy Director, CDER), Dayle Cristinizio (Director, Stakeholder Engagement, Office of External Affairs), Hilary Maston (Chief Medical Officer), Namandje Bumpus (Chief Scientist, AdCom responsibilities), Kaveeta Vasisht (Assoc Commissioner for Women’s Health), Jennifer Dooren (Director Communications and Public Engagement, Food Safety).

Coalition Attendees: In Person: Thomas Eagen (NCHR), Maria Gmitro (BISA), Denise Hyater-Lindenmuth (NWHN), Patricia Kelmar (U.S. PIRG), Suzanne Robotti (MedShadow), Kim Witczak (WoodyMatters), Diana Zuckerman (NCHR).  Virtual: Tahir Amin (I-MAK), Wendy Dolin (MISSD), Helen Haskell (MAME), Rex Johnson (WAPS), Katherine Leon (SCAD Alliance), Judy Norsigan (OBOS), Linda Radach (PSAN), Reshma Ramachandran (DFA), Brian Ronholm (Consumer Reports), Tess Schulman (Medical Device Problems), Robin Strongin (NCL), Dru West (USAPN), Sophia Phillips (on behalf of the Coalition).

On behalf of the Coalition members at the meeting, Dr. Diana Zuckerman offered our support to the Commissioner and the FDA, and thanked the Commissioner for his efforts to improve the Accelerated Approval program by requiring confirmatory trials be started prior to granting accelerated approval. Patricia Kelmar discussed members’ support for the FDA to regulate lab-developed tests to ensure their accuracy. Kim Witczak and Suzanne Robotti discussed possible improvements to the FDA Advisory Committee process, based on their perspectives as consumer representatives on two FDA Advisory Committees.  After the meeting was completed, Coalition members who were attending in person had informal discussions with several FDA officials.

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

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, 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 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

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.

Joint Letter from from Cancer Groups and Patient-Centered Nonprofits about VALID Act in Omnibus

December 16, 2022

The Honorable Patty Murray
Senate Health, Education, Labor & Pensions Committee
Washington, DC 20510

The Honorable Richard Burr
Ranking Member
Senate Health, Education, Labor & Pensions Committee
Washington, DC 20510

The Honorable Frank Pallone
House Energy & Commerce Committee
Washington, DC 20515

The Honorable Cathy McMorris Rodgers
Ranking Member
House Energy & Commerce Committee
Washington, DC 20515

Dear Chair Murray, Ranking Member Burr, Chair Pallone, and Ranking Member Rodgers:

We are writing to express our strong support for your bipartisan support for the VALID Act earlier this year, and to urge you to include it in the Omnibus Spending bill with a few small improvements. Our concern is that women and men who are at high risk of breast cancer or other types of cancer are not currently able to have confidence that genetic tests used for screening and diagnosis are accurate. Our members were shocked to learn that the FDA only regulates diagnostic tests sold by companies, not those sold by laboratories. We have many examples of patients who have been terribly harmed when lab-developed diagnostic tests that were widely sold have been dangerously inaccurate. There are currently approximately 100,000 different lab developed tests that would be grandfathered under the current bill language, and none of them would be required to submit evidence now or in the future to prove that their tests are accurate. Even the highest risk lab developed tests that are currently being sold could continue to be sold without any restrictions, and it is unclear if serious adverse events caused by inaccurate tests would be reported to the FDA. That is unacceptable to us as patient-centered organizations.

Your constituents deserve better. In addition to the extensive grandfathering, the bill would categorize lab-developed tests as low, moderate, or high-risk. Low-risk would be essentially unregulated, moderate risk would  require some evidence but not be tested for accuracy (similar to the 510(k) review process). As you know, the 510(k) pathway is used to review approximately 95% of all medical devices, including most implanted devices, and it has been strongly criticized by physicians, patients, and public health experts because many of those devices are high risk, not moderate risk as the FDA claims.

As described in the current VALID provisions in the bill, only the highest risk devices would be required to provide clear evidence of accuracy, and the definition of high risk is unclear and based on the FDA’s track record is likely to exclude many tests that could cause irreparable harm, such as genetic tests that are used to inform people that they are at very high risk of breast cancer, ovarian cancer, stomach cancer, and other very serious diseases. Since many patients who are told that they tested positive for these genetic mutations have those important bodily organs surgically removed, those genetic tests should be considered high-risk. It would be equally tragic for a person to have an organ unnecessarily removed due to an inaccurate positive test result, or for a person to be incorrectly told they did not have a life-threatening genetic mutation, due to a false test negative result. Although we are focusing on breast cancer in this letter, it is well-known that most prenatal genetic testing is inaccurate, thus resulting in terminating wanted pregnancies because they were erroneously told their baby had a fatal birth defect. For that reason we strongly urge you to include genetic testing in the definition of “high risk” tests in the bill, and not allow grandfathering of high risk tests.

In addition to these shortcomings, we are concerned that academic medical centers are lobbying to be excluded from FDA regulation. While academic medical centers have an important role to play in U.S. healthcare, their tests are not monitored by the FDA, CMS, or any other independent entity to ensure they are accurate.  As a result, there are clear examples of tests that were dangerously inaccurate.  Any exemption for academic medical centers’ lab-developed tests should be extremely narrow, to target tests used for individuals, not for large numbers of patients.  High-risk tests should always be regulated.

In conclusion, we strongly recommend including an improved version of the VALID Act, as described above, in the Omnibus bill, in order to better protect patients and consumers from inaccurate and unreliable lab-developed tests.


Breast Implant Safety Alliance

Cancer Prevention and Treatment Fund

National Women’s Health Network

Not Putting on a Shirt

Our Bodies Ourselves

Patient Safety Action Network

USA Patient Network

Washington Advocates for Patient Safety



Cc: Members of Senate Health, Education, Labor, and Pension Committee and Members of Energy & Commerce Health Subcommittee

Breast Implant Illness: What’s the Evidence?

Debate swirls over the risks of breast implants, and physicians and patients are justifiably confused by the conflicting information available.  Despite surgeons’ claims that implants are proven safe, tens of thousands of women with breast implants have reported that they have serious symptoms that they refer to as “breast implant illness.”  Many women with these symptoms report that they recovered when their implants are removed.  Our report finds clear scientific evidence that implants increase the chances of those symptoms and removing implants usually improves’ their health. Women considering breast implants after mastectomy or for cosmetic reasons will want to know about our report.


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

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.

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Testimony of Dr. Diana Zuckerman About I-omburtamab FDA Advisory Committee Meeting

October 28, 2022

I’m Dr. Diana Zuckerman, president of the National Center for Health Research. We scrutinize the safety and effectiveness of medical products, and we don’t accept funding from companies that make those products.  

My expertise is based on post-doc training in epidemiology and public health, my previous policy positions at Congressional Committees with oversight over FDA, my previous position at the US Department of Health and Human Services, and as a faculty member and researcher at Harvard and Yale. 

This is a terrible disease and the personal stories are very important.  What is the evidence that this treatment actually works?

The sponsor has provided data from 2 studies:

  1. A single center, single arm trial (Study 03-133) included 94 pediatric patients ages 0.9 to 13 years with CNS/LM relapsed neuroblastoma who received intracerebroventricular infusions.  Three doses were used: 25 mCi, 33.5 mCi, or 50 mCi, based on age. The Primary Endpoint was overall survival and the results showed 45% alive at 3 year.
  2. The second study is not completed yet.  The Interim report of this multi-center single arm study (Study 101) shows 20 patients with imaging evidence of CNS/LM disease.  The Sponsor reports 7 responders, but 3 of these are not confirmed.  That matters because transient responses are not necessarily meaningful, and therefore can’t be considered evidence.  The Primary Endpoint is 3-year overall survival, but those data are not yet available.


It is important to note that 19% of patients permanently discontinued using the drug due to an adverse reaction in Study 03-133 and 28% of patients in Study 101.  Most were due to myelosuppression.  However, 3% were due to chemical meningitis (3 cases in 03-133 and 1 case in Study 101), and there was one case of fatal intracranial hemorrhage

Shortcomings of the Studies

Only one study was completed and it wasn’t randomized or blinded or controlled.  The external control group was very small and they differed from the patients receiving the treatment, because the latter had more intensive prior treatment.  There were significant population differences as well as treatment differences between the treatment group and the external controls.  In addition, overall survival for this condition has improved since the control data sample was collected.

Therefore, we agree with FDA’s conclusion that differences in overall survival can’t be “reliably attributed” to the drug.  We also agree that “the application does not include reliable response rate data to provide supportive evidence.”

In Study 101, no patient demonstrated a response that can be unequivocally   attributed to the drug.  And there was no overall response rate calculated for Study 03-133  and only very limited overall response rate data in Study 101.

FDA has been the Gold Standard?  Do these data meet that standard?

The FDA Summary states: The comparator is too dissimilar to the treatment group.  There is no reliable information on tumor response rate.  Therefore, the submitted study cannot be considered an adequate and well-controlled trial necessary to establish effectiveness. That’s the standard that the law requires for FDA approval.

On the other hand, there is an unmet need. These children need treatment.  Should FDA be flexible even though the data are clearly inadequate?  Why isn’t this an accelerated approval application instead of a regular application? Are the data even good enough for accelerated approval?

Are FDA Decisions Perpetuating Poor Studies?

Why didn’t the sponsor conduct a randomized, double blind controlled trial?  This isn’t a rhetorical question.  What’s the incentive for any company to conduct a well-designed study if a poorly conducted study with questionable findings results in approval?  Those controversial decisions set a dangerous precedent.

Unfortunately, when FDA approves a drug based on inadequate data, all companies lose the incentive to conduct well-designed studies.  Not just the company whose product is approved, but all other companies as well.

Patients Deserve Better

We do patients and their families no favors by approving treatments that aren’t proven to work. 

FDA’s Expanded access program is the way to give patients access to experimental drugs, because it provides free treatments in a well monitored situation where the patients and families know that they are taking a risk using an experimental drug.  Why should patients pay to take an experimental drug, thinking it is proven safe because it is FDA approved?

I can’t believe I have to say this: Without an appropriate control group, it is not possible to provide evidence that patients and doctors need to make informed decisions.  Even a small study with a small well matched control group is better than nothing.

Unfortunately, the preponderance of evidence doesn’t support approval for this drug.

The Advisory Committee agreed with our assessment and voted 16-0 that the studies did not prove the drug improved overall survival. The FDA subsequently rejected the application.