All posts by CPTFeditor

Payment reform puts medical-device industry on the defensive

News Stories & Editorials
By Sabriya Rice. Modern Healthcare. October 7, 2014

Medical technology companies are warning that burgeoning pay-for-performance and risk-based reimbursement models will motivate providers to block access to clinically important innovations.

Healthcare economists and quality experts, though, counter that the new models appropriately put the onus on manufacturers to prove their products are worth the cost.

The tension was the centerpiece of a news conference held by the leaders of AdvaMed, the industry’s trade group, at the start of its annual conference in Chicago Monday.

“We have to ensure these new payment models include safeguards to protect patients from unintended consequences,”AdvaMed CEO Stephen Ubl said. Ubl, flanked by AdvaMed Chairman and Covidien CEO Joe Almeida, said too many of the contracts put too much emphasis on cost targets over quality benchmarks.

New payment models have proliferated quickly in recently years, nurtured by provisions of the Patient Protection and Affordable Care Act. More than 360 accountable care organizations have signed contracts with Medicare, agreeing to strive for cost and quality targets to earn some of the savings achieved (or in some cases return money to the government if they fail). Many providers are striking similar agreements with private payers—Blue Cross and Blue Shield Association recently said that it’s now paying one in five dollars under incentive-based contracts—or assuming financial risk with their own insurance products.

AdvaMed is promoting an industry-funded white paper based on the responses of nine unnamed health insurance companies interviewed about their movement toward pay-for-performance and risk-based contracts. Five said they had become more selective about approving coverage for new technologies in the past three years. Four said they plan to demand more evidence before covering products. All said costs were driving their organizations to explore new reimbursement models. The findings are published in the Journal of Medical Economics.

“They run the risk of really tipping too far, so physicians have incentive not to adopt things that really benefit patients,” AdvaMed Senior Vice Presdient David Nexon said in a phone interview.

Representatives for insurers Cigna and Aetna, which responded to requests for comments on AdvaMed’s conclusions, said new technology is embraced if it adds value. “Providers, members and payers deserve and increasingly want to have proof,” an Aetna spokeswoman said. Cigna said in a statement that “technology that simply adds cost without improving quality, health, satisfaction and productivity is counter to our goals.”

Mark Pauly, professor of Health Care Management at the University of Pennsylvania called it an overstatement to suggest that new payment models will inspire widespread failure to adopt products that promise significant benefits to patients. The models add an appropriate hurdle, he said. Manufacturers that can’t provide evidence “won’t go over the hurdle.”

Diana Zuckerman, a researcher who has been critical of the Food and Drug Administration’s procedures for approving and monitoring medical devices, said the industry isn’t accustomed to having to prove that a product is cost-effective. “Something can be clinically appropriate and not be necessary,” said Zuckerman, who is president of the National Center for Health Research.

Manufacturers, though, are in a difficult situation, acknowledged Tom Skorup, VP of applied solutions for the ECRI Institute, a not-for-profit organization that studies the quality and costs of medical technologies, procedures and drugs. Many manufacturers are finding ways to partner with providers to generate evidence. “The greater the collaboration in the creation of the evidence, the fewer the barriers to the potential for uptake,” he said.​

Medical company may be falling short of its patient safety ideals

News Stories & Editorials
by Marshall Allen and Annie Waldman, NPR
October 6, 2014

When medical device entrepreneur Joe Kiani announced his commitment to eliminating medical mistakes, he did it with panache. His medical device company, Masimo Corporation, funded the launch of a nonprofit called the Patient Safety Movement Foundation. And at its flashy inaugural summit in 2013 – featuring former President Bill Clinton as the keynote speaker – Kiani pledged to galvanize the medical industry to reduce the number of deaths from medical errors across the country from hundreds of thousands a year to zero.

“Of all the dreams I’ve had none seem as important as this dream: The dream of no more preventable patient death,” Kiani said to the gathering of health care leaders.

Now people will see whether Kiani, and his company, Masimo, walk the talk. Keeping patients safe requires device makers to respond appropriately to complaints. But an inspection by the Food and Drug Administration last year found Masimo didn’t adequately investigate dozens of reports that its devices may have malfunctioned.

The FDA didn’t find that Masimo’s devices were defective, but rather that the company wasn’t sufficiently looking into that possibility. The inspection report was obtained by ProPublica via public records request.

With over $500 million in annual sales, Masimo is one of the leading makers of noninvasive pulse oximeters — patient monitoring devices that track pulse and blood oxygen, often clipped gently onto fingers or toes. Doctors in emergency rooms and intensive care units rely on the monitors to alert them when a patient has abnormal readings. If the devices give inaccurate readings or fail to alert doctors to drastic changes in a patient’s vital signs, doctors could misdiagnose or fail to recognize the severity of a patient’s condition, which could lead to injury or death.

The complaints identified in the FDA inspection varied. In one case a patient suffered a burned toe, and in another there was a question about whether an alarm properly sounded before a patient died.

If a patient or health care provider sees a problem with one of Masimo’s products they can report the problem to the company, which is then required by law to investigate. If the device malfunctioned or was implicated in a patient injury or death, the company must in turn disclose that to the FDA.

After the agency’s inspection last year, Masimo disputed the FDA conclusions, saying the agency had misunderstood the circumstances surrounding each complaint. But after nearly a year of review, the FDA stuck to its findings. It reprimanded the company with an official warning letter this August, stating that Masimo was still not adequately responding to complaints about its devices.

FDA warning letters aren’t common – it’s the first Masimo has received in its 25-year history. They are generally reserved for significant violations that could result in an enforcement action, such as fines or a recall. The agency declined to offer further comment.

Diana Zuckerman, an expert on medical devices and president of the National Center for Health Research, reviewed the FDA’s findings and Masimo’s response at ProPublica’s request. She said it appears that Masimo is “not taking the care to investigate their own possible malfunctions.”

Since patients or doctors often don’t report problems with products, Zuckerman said, complaints that are filed are “always the tip of the iceberg.” Zuckerman added that she found it striking that Masimo disputed most of the complaints and “particularly troublesome” that the company challenged a complaint that involved questions about whether a Masimo device properly set off an alarm before a patient died.

“It may well be that it’s a user error,” Zuckerman said. “But you have to investigate that and show that it’s a user error and not a device error.”

Zuckerman found it striking that the company refused to admit any wrongdoing to the FDA. “When a company refuses to respond in any way to the FDA other than to say that the FDA is wrong on every issue, that’s not very credible,” Zuckerman said. “Especially when users made complaints that the company’s product put patients at risk,” she said.

In an interview with ProPublica, Kiani again disputed the FDA’s findings, saying Masimo has always followed up on complaints. He said the company is cooperating with the regulator, providing it with information “to show them we did nothing wrong.”

When asked how the FDA findings reflect on the ideals put forth by his nonprofit effort, the Patient Safety Movement Foundation, Kiani said that he never claimed to be perfect. “I’m just trying to do my best and get my other colleagues to do their best, and put processes in place to hopefully minimize preventable death,” he said.

The two-year-old foundation has attracted a who’s who of top health care quality experts to its summits, including decision-makers from Medicare. The second annual conference was in January at the Ritz Carlton Hotel in Laguna Nigel, Calif.

The foundation promotes a “culture of safety” and encourages the early identification of problems that can lead to patients being harmed. “The lack of safety culture results in concealment of errors and therefore a failure to learn from them,” the foundation’s guidelines state.

One member of the foundation’s board said he was disappointed about Masimo’s alleged lack of response. Masimo “has to improve because [companies] are a big part of our ability to get to zero preventable harm,” said the board member, Dr. David Mayer, also a vice president of quality and safety at Medstar Health in Maryland. Mayer said he gets no compensation to sit on the board and pays his own expenses to attend its meetings.

The foundation’s president, Jim Bialick, said the organization is primarily funded by a charitable offshoot of the company, the Masimo Foundation for Ethics, Innovation, and Competition in Healthcare.

In response to a question about whether the FDA’s concerns would be addressed at the upcoming summit, Bialick said that was Masimo’s choice. “I would imagine it would come up. Whether Masimo brings it up, that’s up to them.”

ProPublica is a nonprofit investigative reporting newsroom based in New York.

Read or listen to original article here.

Testimony of Dr. Laurén Doamekpor, senior fellow, on Sonoblate treatment for prostate cancer

Testimony & Briefings
October 1, 2014

Good morning. Thank you for the opportunity to speak today. My name is Dr. Laurén Doamekpor, and I am a senior fellow at the Cancer Prevention and Treatment Fund. Our research center scrutinizes scientific and medical data and provides objective health information to patients, providers and policy makers. We do not accept funding from device companies and therefore I have no conflicts of interest.

We all know that current treatments for prostate cancer have side effects that harm men’s quality of life. While most men diagnosed with localized, low-risk prostate cancer will likely die from something else, not from prostate cancer, recurrent prostate cancer frightens patients and needs to be treated. However, any new treatment should show clear evidence that the benefits outweigh the risks. We are not convinced that the HIFU device achieves this goal.

The analysis of the primary endpoint showed that 64% of the patients who were treated with this device had obtained local control of prostate cancer at 1 year after treatment. But that doesn’t tell us anything about long-term effectiveness, which is key for prostate cancer.  Proof of long-term effectiveness would also require a control group or comparison group, which this application lacks.

The sponsor’s goal was a 40% success rate at 1 year, and they achieved that. However, even a 64% success rate at one year is very low compared to the long-term success rate of prostate surgery.  We share the FDA’s skepticism that this device should be approved based on this low 1-year success rate.

In addition, the safety profile is not impressive. Incontinence was reported in 43% of patients, and was still 31% after one year. That is worse than most studies of prostate surgery, which has a much higher long-term success rate.

With only 100 patients in the study and no long-term data, the sponsor has not provided enough data to justify approval.  The small sample includes only 13 Black patients, even though prostate cancer is more deadly for Blacks. There are only 5 Hispanic patients.  The sponsor says they did subgroup analysis of effectiveness and that Blacks did well with the treatment.  The Advisory Committee should demand to see the safety and effectiveness outcomes separately for Blacks and Whites.

The sponsor is already enrolling a 2nd cohort of 100 patients, and FDA should delay an approval decision until those data are submitted.  The FDA should also require at least 2-3 years of data before the agency decides whether to approve this device. While we commend the sponsor for its plan to conduct a 5-year post treatment study, it would be unfair to patients to pay for an unproven device based on such a small, short-term study.

In summary, the study is too small, too short term, and has no comparison sample.  It has too few Blacks and Hispanics. Evidence of long-term effectiveness is needed BEFORE approval, not as a post-approval study.

That’s why we urge you to vote NO 3 times: the data from the study do NOT support a reasonable assurance of safety OR  effectiveness for this device and that therefore we can NOT conclude that the benefits outweigh the risks.

At the end of the meeting, the panel voted 10 to 0 with 1 abstention that benefits of the device do NOT outweigh the risks.

Medical devices lack safety evidence, study finds

News Stories & Editorials
By Thomas M. Burton, The Wall Street Journal
September 29, 2014
Researchers Say Public Data Unavailable on Majority of Newly Approved Devices

The majority of moderate- to high-risk medical devices approved by the U.S. Food and Drug Administration lack publicly available scientific evidence to verify their safety and effectiveness despite requirements in the law, according to a study released Monday.

Researchers reported in JAMA Internal Medicine that 42 of 50 selected medical devices cleared by the FDA over five years lacked such data, despite a 1990 law calling for sufficient detail to justify their FDA clearance. The law calls for public data about studies, which may include clinical studies, involving human patients.

“If wonderful studies are being done, there is no evidence of this, and there’s no way for the public to see it,” said Dr. Diana Zuckerman, a study author and president of the National Center for Health Research, a public health think tank in Washington. “It’s shocking how little information is available despite an FDA leadership that talks about transparency.”

The study’s authors, who were from the health-research center, called for better enforcement of that law.

In response to the findings, the FDA said that it “reviews a significant amount of data—far more than what is publicly available.” The agency said its approach “has served the American public well by balancing the need for robust evidence to assure safety, while expeditiously bringing new technologies” to the market.

At issue is a type of medical-device approval under which about 400 implanted devices annually—the majority of moderate- to high-risk ones—are cleared by the FDA for marketing.

Under that system, called a 510(K) review, the company doesn’t have to conduct studies in human patients, as is generally the case with drug approvals, although it may. Most devices can get FDA clearance simply by showing that they are roughly equivalent to another product, called a predicate device, that is already on the market. The theory is that if the older device has proved safe and effective, the new one should be also.

The 510(K) process has led to cases like metal-on-metal hips, which can leave metal filings in the body.

But the Safe Medical Devices Act of 1990 required companies to make publicly available evidence of why the new product truly is comparable to the old one; the evidence can include studies of patients, known as clinical trials, but it isn’t necessary.

The Institute of Medicine, the research branch of the National Academy of Sciences, in 2011 called for an overhaul of this type of FDA clearance, saying in a report that “reliance on substantial equivalence cannot assure that devices reaching the market are safe and effective.”

“A lot of these are high-risk devices that get on the market with no studies at all,” said Dr. Rita Redberg, medical professor at the University of California, San Francisco, and the chief editor of JAMA Internal Medicine. “When there are studies, they’re not available” for the public to see.

She said that she sees no evidence that the leadership of the FDA under the Obama administration has heightened the level of safety assurance required.

Read original article here.

Lack of publicly available scientific evidence on the safety and effectiveness of implanted medical devices

In the News, News Stories & Editorials
By Diana Zuckerman, PhD; Paul Brown, BA; Aditi Das, PhD

The US Food and Drug Administration (FDA) approves about 400 implanted medical devices each year through an abbreviated process called the 510(k) process, which only rarely requires clinical trials (studies of patients).  These implants include potentially high-risk devices such as heart valves, spinal implants, and hip and knee joint replacements.  In contrast, fewer than 20 implants each year are approved by FDA through the more rigorous Premarket Approval (PMA) process that requires that the impact on the patients’ health be studied in clinical trials

The law requires that all medical implants sold in the U.S. provide a reasonable assurance of safety and effectiveness based on scientific evidence, even if the company doesn’t provide data from studies of patients.  The scientific evidence could include bioengineering studies, for example, to determine if the new implant is expected to be similar to older implants on the market.  This groundbreaking study examined the scientific evidence provided to the FDA and the public for 1155 medical implants.

The study focuses on 50 representative medical implants newly cleared by the FDA 510(k) process from 2008-2012.  Since those implants were required to be substantially equivalent to implants already on the market, called “predicates,” the study also included the 1105 “predicates” for those 50 implants.  The implants included hip implants, surgical mesh, cardiac implants, spinal implants, and dental implants, among others.

By law, every 510(k) medical device application since March 15, 1995 must provide a summary that includes “sufficient evidence” to prove that the new device is substantially equivalent to a device already on the market.  This summary must be available online, linked to a letter on the FDA website granting the company clearance to sell their device in the U.S.  If a company doesn’t provide that summary, the law requires it to provide all the same information within 30 days of a written request from anyone.  This is especially important for implants, since they can cause permanent damage or even be life-threatening if they fail while inside the human body

RESULTS:  Just 3% of the 1155 implants in the study provided scientific evidence that the implant was substantially equivalent to its predicate or that it was safe and effective.   Even when predicates had been permanently recalled because of fatalities or serious injuries, there was no evidence of how the newly cleared implant was similar or different from those predicates.  Since 2009, the FDA leadership has repeatedly focused on the need for transparency at the FDA, but even in 2012, the 3rd year of new leadership at the FDA, only 20% of the implants had any kind of publicly available scientific data.

CONCLUSIONS: For the 1155 implants studied, “sufficient detail to provide an understanding of the basis for a determination of substantial equivalence,” as required by law, was unavailable to physicians, patients, providers, and others who care about patient safety or public health. Numerous newly cleared implants had predicates that had been recalled because of serious risks, calling into question the safety of the newer “substantially equivalent” implants. Regardless of how much effort physicians and providers would make to find out about the safety or effectiveness of new medical implants, they do not have access to such information for approximately 84% of medical implants that have been cleared in recent years, and more than 95% of those cleared in the last 2 decades.

IMPLICATIONS:  Previous criticisms of the FDA medical device approval process by the Institute of Medicine and public health researchers have focused on the lack of clinical trial data proving safety and effectiveness.  The FDA has repeatedly claimed that the agency has other rigorous scientific criteria for approval, such as bioengineering data proving that the new devices are so similar to the ones already on the market that they will be just as safe and effective.  This is the first study to examine the “other scientific data” that the FDA explains that it relies on.  The results indicate that either the companies are not providing solid scientific data proving that the new devices are substantially equivalent to the predicates, or that the companies are not making a sufficiently detailed summary of their scientific evidence publicly available, as required by law.  In cases where an earlier “predicate” was recalled because of serious or possibly fatal risks, the researchers found no publicly available scientific evidence to inform physicians or patients whether the newer implant would have the same potentially fatal flaw.  To safeguard the health of patients, the FDA should enforce the law.

From JAMA Internal Medicine, published online September 29, 2014.  A link to the official abstract of the article is available here. A podcast with Drs. Zuckerman and Sharfstein is available on the JAMA Internal Medicine website and can be found 

Can wearing a bra cause breast cancer?

Breast Cancer, Prevention
Caroline Novas

There are several persistent email and internet rumors about potential causes of breast cancer. One is that wearing a bra, or wearing an underwire bra, causes the disease.

The idea that bras may cause cancer was fueled by the 1995 book called Dressed to Kill by Sydney Ross Singer and Soma Grismaijer. It claims that women who wear underwire bras for 12 hours a day have a much higher risk of developing breast cancer than women who do not wear bras.They maintain that bras restrict the lymph system, which results in a build-up of toxins in the breasts.1 However, according to the American Cancer Society, there is no evidence that compression of the lymph nodes by bras causes breast cancer; in reality, body fluids travel up and into the underarm lymph nodes, not towards the underwire.2 Similarly, there is no sufficient evidence that any types of bras cause breast cancer.

In Dr. Susan Love’s Breast Book, Love claims that the hypothesis about bras causing cancer stems from our desire to have control over areas of life where we have a lot of uncertainty or fear. People want something to blame, and also hope that by avoiding bras they can avoid breast cancer.3 While there are geographic variations in breast cancer rates, there are many, many factors, including diet, exercise, lifestyle, childbearing practices, as well as other behaviors and exposures that are more plausible explanations for these regional differences in breast cancer than bras. In places where people have less access to medical care, breast cancer will not be diagnosed as often, even though it might be present. And because the risk of breast cancer increases as women get older, breast cancer rates will be lower in parts of the world where people die of other causes at younger ages, whether they have worn bras or not.

Even if women who wear underwire bras are more likely to be diagnosed with breast cancer, a likely explanation would be that many women with larger breasts also tend to be heavier. Being overweight or having a lot of body fat puts a woman at increased risk for breast cancer.4 It would make sense that women with larger breasts are both more likely to wear underwire bras and more likely to develop breast cancer. But this doesn’t mean that underwire bras cause breast cancer!

In a study published in 2014, researchers interviewed postmenopausal female participants about their lifetime bra wearing patterns. Evaluating more than 1,000 women with breast cancer and almost 500 who did not have breast cancer, the researchers found no evidence of a connection between the number of hours spent wearing a bra or wearing an underwire bra and increased breast cancer risk.5

The bottom line: well-designed studies have not convinced experts that wearing bras or underwire bras increase your chances of developing breast cancer. Here are some factors that are associated with increased risk of breast cancer:6

Risk factors you can’t control

  • Sex: Women represent 99% of all breast cancer patients and have a 12.1% chance of being diagnosed with breast cancer during their lifetime.
  • Age: The chances of getting breast cancer increase with age. About 65% of women are over 55 years old when they are diagnosed.
  • Race: After age 45, white women are more likely to get breast cancer than black women, but black women have a higher incidence before age 45 and are more likely to die from breast cancer.
  • Family history: Certain inherited gene mutations (BRCA1 and BRCA2) increase the risk of developing breast cancer. However, these genes account for only 5-10% of overall cases. Even without those genes, having a grandmother, mother, sister, or daughter diagnosed with breast or ovarian cancer increases the risk.
  • A previous history of breast cancer, abnormal breast cells (atypical hyperplasia) or certain non-invasive “pre-cancers” like lobular carcinoma in situ (LCIS) or ductal carcinoma in situ (DCIS) increase the risk of developing invasive cancer.
  • Beginning menstruation early (before age 12) increases the risk of breast cancer by affecting the level of reproductive hormones a woman is exposed to during her lifetime.
  • Starting menopause late (after age 55) increases the risk of breast cancer.
  • Dense breast tissue (including fibrocystic breasts) increases the risk of breast cancer

Risk factors you can (possibly) control

  • Women who delay having their first child until later in life or who never have children are at a higher risk for breast cancer.In contrast, having children at a younger age and breastfeeding decrease the risk of developing breast cancer
  • Women who take hormonal therapy for menopause are at an increased risk for breast cancer.
  • Being overweight or obese increases the risk of postmenopausal breast cancer
  • Physical inactivity increases risk.
  • Women who drink an average of 2 alcoholic beverages per day increase their breast cancer risk by 21%. The more a woman drinks, the greater her risk.
  • High levels of radiation in the chest area before the age of 30 increase the risk.
  • Women who took DES during pregnancy (this drug was mainly used in the 40s, 50s, 60s, and 70s) are at an increased risk of breast cancer. The risk to their daughters is still being studied.
  • There is growing evidence that smoking and exposure to tobacco smoke probably increase breast cancer risk.
  • The use of oral contraceptives may slightly increase the risk of developing breast cancer. Some studies have found no increased risk from taking birth control pills and others have shown an increased risk.

If you are worried about your risk of breast cancer, you should discuss your concerns with a health care professional and find out about ways to cut your risk. Knowing the real risk factors and making healthy lifestyle choices can help you reduce your risks. Going braless won’t.

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

Testimony for FDA meeting on sunscreen active ingredient safety testing

Testimony & Briefings
Delivered by Dr. Anna Mazzucco
September 4, 2014

 

Thank you for the opportunity to speak today on behalf of the Cancer Prevention and Treatment Fund. My name is Dr. Anna Mazzucco, and after completing my Ph.D. in Cell and Developmental Biology from Harvard Medical School I conducted research at the National Cancer Institute. I bring those perspectives today.

Our nonprofit research center conducts research and scrutinizes data to provide objective and reliable information to patients and providers.  Our president is on the Board of Directors of the Alliance for a Stronger FDA, which is a nonprofit dedicated to increasing the resources that the FDA needs to do its job.  Our organization does not accept funding from pharmaceutical companies, and therefore I have no conflict of interest.

In 2014 almost 10,000 Americans died from melanoma skin cancer, and 76,000 new cases were diagnosed.7  Despite warnings to avoid sun exposure, adults and children are spending a great deal of time in the sun, thus making safe and effective sunscreens essential to prevent skin cancer.  At the same time, Americans are using sunscreens more frequently and on a more long-term basis than ever before, as the FDA’s report noted.

Therefore, our safety and efficacy standards must reflect that Americans of all ages rely on these products regularly.  In its prepared questions, FDA scientists point out the lack of safety testing in pediatric populations.  I hope you’ll agree that we need conclusive evidence that these products are safe for children, and also for prenatal exposures when pregnant women use them.  Clinical studies must include separate analyses of these and other vulnerable populations before products can be marketed for widespread use.

Studies of several active ingredients currently on the market have raised concerns about photostability—how long the protections last under real-world conditions.8

Testing for photostability should be required as part of the pre-market testing of sunscreen active ingredients.  Consumers need to know how long products so that they can avoid increased skin cancer risk.

Several active ingredients currently on the market have also shown potential endocrine-disrupting or carcinogenic activity.9 10   We support the FDA scientists’ proposal to require that active ingredients be tested for carcinogenic and developmental and reproductive toxicity prior to marketing.  Research shows that endocrine-disrupting agents often have greater risks at low doses,11 so dose-response testing is not appropriate for these studies.  Without well-designed studies, children and others who are particularly susceptible could be harmed by these hormone-altering ingredients.

There is also research evidence that certain active ingredients demonstrate potentially carcinogenic activity when exposed to UV light, such as generating free radicals that damage DNA and can cause harmful mutations.  Testing for carcinogenic activity should be done under conditions that reflect real-world use as closely as possible, i.e. during UV light exposure.  Such carefully designed testing is needed to ensure that safety data is reliable and meaningful.  Without such information, products intended to help prevent cancer may do just the opposite.

As you consider FDA’s questions, please pay special attention to the issue of combining active ingredients with different vehicles, to make sure that does NOT result in final product formulations which are marketed over-the-counter without any additional testing.  We agree with FDA scientists that different vehicles could change the properties or absorption of active ingredients in ways that could affect safety or efficacy of the final product.  As medical professionals, please urge the FDA to directly confront this problem through additional testing requirements that can ensure products that will be safe and effective for all consumers.

Diversity in clinical trials

News Stories & Editorials, Uncategorized

We Need Better Clinical Trials

Our president explained to reporters why drugs have to be tested on all kinds of people to know who they work for and who can be harmed by them. Cancer patients deserve studies that include women and men, people under and over 65, and all major racial/ethnic groups.

 

FDA is Chastised Over its ‘Action Plan’ to Diversify Clinical Trial Participation

By Ed Silverman, The Wall Street Journal
August 28, 2014

In response to a law passed two years ago, the FDA was directed to assess the extent to which women and minorities are represented in clinical trials and also devise a plan to bolster their participation. The requirement was made in response to concerns that drugs and devices may often be used by subsets of the population on whom the products were not actually tested.

Well, the FDA released its plan the other day and it was met with what could best be described as faint praise. In particular, a pair of consumer advocacy groups says the biggest issue is that the so-called Action Plan lacks the sort of teeth needed to generate real change. They also complain the plan fails to require drug and device makers to contain specific demographic information in product labeling.

They acknowledged the plan does contain several constructive steps, such as working with drug and device makers to revise product applications with enhanced information on “demographic subgroups;” strengthening FDA reviewer training so the need for demographic data is communicated; improving FDA systems for collecting and analyzing such data; and updating or finalizing guidance for industry.

“The action plan has a lot of good things,” Diana Zuckerman, the president of the National Center for Health Research, tells us. “The problem is there is no incentive for industry to recruit more diverse groups of patients. As long as they continue to test mostly on white men under 65 [years old] and get drugs and devices approved, then they have no incentives” to diversify the pool of trial participants.

What does she suggest? The FDA ought to consider not approving drugs and devices for use in people on whom these products were not tested. As an example, if a drug is tested predominantly on men, then the agency should not endorse widespread use for women. “They need to put some muscle behind their actions. I think if FDA did this,” she says, “then the companies would find they’re able to miraculously find they can recruit all those groups.”

Read the original article here.

Surgery Studies Rarely Use Females

An analysis of papers published in several surgical journals reveals an overwhelming reliance on male subjects and male-derived cells.

By Kerry Grens, The Scientist

August 28, 2014

Sex biases are evidenced in many areas of science—from clinical trials in humans to basic neuroscience studies on animals. Often, male subjects are overrepresented, compromising the generalizability of findings. In a study published in the September issue of Surgery, researchers from Northwestern University analyzed more than 2,300 papers from five surgical journals, finding an overwhelming skew toward investigations involving male-only subjects and cells derived from males.

“Women make up half the population, but in surgical literature, 80 percent of the studies only use males,” Northwestern Medicine vascular surgeon Melina Kibbe, who led the study, said in a press release.

The studies Kibbe and her colleagues reviewed were published in the Annals of Surgery, the American Journal of Surgery, JAMA Surgery, the Journal of Surgical Research, and Surgery from 2011 to 2012. About a quarter of the studies involved animals or cells. Just 3 percent of the total reported using both male and female subjects or cells, while another 22 percent did not state the sex.

According to the release, “editors of the five major surgical journals reviewed in this study have responded to this finding and will now require authors to state the sex of animals and cells used in their studies. If they use only one sex in their studies, they will be asked to justify why.”

Although sex disparities in clinical trials have received considerable attention, such human studies still suffer from unsatisfactory diversity. Last week, for instance, the US Food and Drug Administration (FDA) released an action plan to address the need for more women and minorities in clinical trials. The Wall Street Journal’s Pharmalot blog pointed out that the plan was met with “faint praise,” lacking “the sort of teeth needed to generate real change.”

Speaking with MedPage Today, Diana Zuckerman, the president of the National Center for Health Research, said: “As long as the FDA is going to approve these products for everyone, when they haven’t been studied on everyone, then the [pharmaceutical] companies really have no incentive to improve.”

Read the original article here.

Sign on letter to Senator Nelson on child nicotine poisoning

Legislation
August 11, 2014

The Honorable Bill Nelson
United States Senate
Washington, DC 20510

Dear Senator Nelson:

On behalf of organizations dedicated to improving the health and safety of children, we write to express our support for the Child Nicotine Poisoning Prevention Act of 2014. This legislation recognizes the danger that liquid nicotine used to refill electronic cigarettes poses to small children and gives the U.S. Consumer Product Safety Commission (CPSC) the authority to require the use of child-proof packaging on liquid nicotine containers sold to consumers.

Recent data from the Centers for Disease Control and Prevention (CDC) indicate that there were 215 calls to poison control centers related to e-cigarettes in February 2014 alone, up from only one call per month in 2010. Liquid nicotine is highly toxic and sold in a highly concentrated form. It is common to find liquid nicotine containing upwards of 36 mg of nicotine per milliliter of liquid. At this concentration, a small 15 mL dropper bottle of liquid nicotine would contain over 500 mg of nicotine. Given that the estimated lethal dose of nicotine is 1 to 13 mg per kilogram of body weight, even at the high end of this range a bottle of liquid nicotine at this size and concentration would be enough to kill four toddlers.

In addition, these products come in bright colors and candy flavors that make these substances likely candidates for ingestion by young children. Alarmingly, these products are often sold in containers without any child-proofing, and there are currently no federal requirements for child-proof packaging for liquid nicotine despite the highly toxic nature of these products. Given that liquid nicotine may also be absorbed quickly through the skin as well as ingested, children are in extreme danger of being poisoned without simple safeguards to ensure that these liquids stay sealed in their containers.

CPSC has the expertise to quickly require child-proof packaging for liquid nicotine if given the authority by Congress. The Commission currently requires such packaging on toxic household substances like bleach, as well as Food and Drug Administration (FDA)-regulated products like prescription drugs. Parents have come to expect that household products that can cause serious harm to children come in child-resistant packaging. Liquid nicotine should be no different.

The FDA is in the process of extending its regulatory authority to include additional types of tobacco products, including e-cigarettes and liquid nicotine. S. 2581 preserves FDA’s authority to regulate the manufacture, marketing, sale, and distribution of tobacco products and the packages and containers in which they are sold. We also encourage action by the FDA to protect children from child poisoning and other dangers posed by tobacco products and their containers.

This is an urgent public health danger and quick action is needed to ensure that child poisoning from liquid nicotine is addressed as soon as possible. We look forward to working with you to pass this important legislation.

Sincerely,
American Academy of Otolaryngology – Head and Neck Surgery
American Academy of Pediatrics
American Association of Poison Control Centers
American Association for Respiratory Care
American College of Cardiology
American College of Physicians
American College of Preventive Medicine
American Public Health Association
Arizona Consumers Council
Association of Maternal and Child Health Programs
Association of State and Territorial Health Officials
Boston Public Health Commission
Campaign for Tobacco-Free Kids
Cancer Prevention and Treatment Fund
Chicago Consumer Coalition
Consumer Federation of America
Consumer Federation of California
Consumer Federation of the Southeast
Consumers Union
EverThrive Illinois
First Focus Campaign for Children
Kids in Danger
March of Dimes
Minnesota Department of Health
National Association of County and City Health Officials
Ohio Public Health Association
Partnership for Prevention
Public Citizen
U.S. PIRG
Virginia Citizens Consumer Council