Category Archives: In the News

Morcellation devices: a surgical tool that can spread cancer? What you need to know

In the News, Medical Treatments with Cancer Risks, Other Cancers
By Anna E. Mazzucco, Ph.D.
2014

If you have uterine fibroids and are considering treatment or if you’ve heard about uterine morcellation in the news, this article will help you understand the issue.

What are power morcellation devices?

A power morcellation device is a small surgical tool which cut tissue into smaller pieces.  This allows organs or other tissues to be removed through smaller incisions, making surgery shorter and leaving smaller scars behind.  The front end of a power morcellator has a spinning blade that cuts the tissue into tiny pieces (pulverizes it), and the back end is connected to a tube which sucks the tissue through the device (see image below).

morcellator

Power morcellation devices were originally designed for removal of the uterus (or the womb), but are currently used for many different types of surgical procedures because they make it easier for a physician to perform surgery using smaller incisions.

What is the controversy about morcellation devices?

In 2012, two Harvard doctors had their lives tragically affected by these devices.  Dr. Amy Reed, an anesthesiologist at Beth Israel Deaconess Medical Center, which is affiliated with Harvard Medical School, had surgery in the fall of 2013 to remove her uterus due to fibroids.  After the surgery she was diagnosed with advanced (stage IV) uterine cancer, spread by the use of power morcellation during her surgery.  The morcellation left behind tiny pieces of tumor throughout her pelvic cavity, which allowed the cancer to spread.  While morcellation devices are sometimes used with a bag to contain the fibroid or tissue so that it won’t spread, the bags can be difficult to use so not all surgeons use them.   Dr. Reed was never told that morcellation would be performed during her surgery, or about the potential risks.

As a result of this surgical procedure with morcellation, a small cancer that could have been easily and completely removed through surgery has been spread and is now considered fatal.

Dr. Reed, who is now undergoing aggressive treatment for her metastasized uterine cancer, and her husband, Dr. Hooman Noorchashm, who was a surgeon at nearby Brigham and Women’s Hospital (also affiliated with Harvard), began a campaign to raise awareness of this issue and prevent other patients from being harmed.  As a result, Brigham and Women’s Hospital has since changed its policies on use of morcellation.  Since their story came out, other similar stories have surfaced, confirming that cancer has been spread by these devices, that it is not a rare event, and that it can have fatal results.  Moreover, many of these patients had not been told about these potential risks before their surgeries, and in some cases weren’t even told that morcellation would be used.  In May 2015, the Wall Street Journalreported that the FBI is investigating whether information about the risks of morcellation was not reported by hospitals, doctors, and device makers, as required by law.

What are uterine fibroids?

Uterine fibroids are non-cancerous (benign) growths on the uterus.  They are very common among women, especially during and after the reproductive years.  In many women, they do not cause any noticeable symptoms or problems.  But in some women, uterine fibroids can cause pain, discomfort during sex, and heavy bleeding.  To read more about fibroids and treatment options for them, read our in-depth article here.

What are the risks and benefits of using power morcellators?

Morcellation can allow surgeons to do shorter, less invasive surgeries.  This can reduce the chances of excessive blood loss and infection, and can reduce the amount of time spent in the hospital and result in an easier recovery afterwards.  However, in the case of fibroids, there is no way to rule out the chance of hidden cancer which could be spread by morcellation.  The Food and Drug Administration (FDA) estimates that 1 in 350 women receiving surgery for uterine fibroids has a hidden cancer that could be spread by morcellation. This is why the FDA released a warning in April 2014 recommending against morcellation for uterine fibroids.

On July 10-11, 2014, the FDA held a public meeting to discuss the risks and benefits of these devices.  Our testimony before the FDA panel on uterine morcellation, is here.

On November 24, 2014 the FDA announced that they were issuing an immediate change in the label for power morcellation devices, which will now include a black box warning as follows:

“Uterine tissue may contain unsuspected cancer. The use of laparoscopic power morcellators during fibroid surgery may spread cancer and decrease the long-term survival of patients. This information should be shared with patients when considering surgery with the use of these devices.”

These tragic events involving power morcellation devices raise questions about how medical devices are approved and monitored, and how a tragedy like this can be prevented in the future.  If larger studies had been done before the FDA allowed these devices to be used, and if cases of cancer being spread by morcellation had been reported to the FDA by doctors and companies, the FDA could have warned doctors and patients much earlier and prevented women from being exposed to these risks.

What do patients need to know?

If you are considering surgical treatment for uterine fibroids, be sure to discuss morcellation with your surgeon and make your wishes clearly known.  Depending on your particular situation, there are alternative surgical procedures, such as vaginal hysterectomy, which can be done without morcellation.  For any surgical procedure, make sure you have a clear and thorough discussion with your doctor about exactly how the procedure will be done, what choices you have, and what the risks and benefits of different options are.   If you feel that you are not getting enough information from your doctor, consider getting a second opinion.  Be sure to ask your doctor how often they perform the procedure you will have, because patients usually have a better outcome from surgery if the doctor performs the exact same surgery frequently.  If the physician does not have many years of frequent experience with the surgery, seek out a doctor who does.  For more tips on how to make smart decisions about medical treatments, especially use of medical devices, read our article here.

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.

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 

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.

Free patient booklet on ductal carcinoma in situ (DCIS)

Breast Cancer, In the News, news-you-can-use, Uncategorized

To view, download, or print a free copy of our patient booklet, here is a PDF of DCIS: What You Need to Know.

The Cancer Prevention and Treatment Fund has developed a free, easy-to-read 32-page color booklet for women diagnosed with ductal carcinoma in situ (DCIS). It explains DCIS and commonly used medical terms in plain language and helps women make informed decisions about their treatment.

If a woman has a particularly low-risk type of DCIS, she may choose “active surveillance” instead of surgery and other treatment. Active surveillance consists of closely watching the patient’s DCIS to make sure it does not develop into breast cancer. The patient can choose surgery and other treatments if the DCIS develops into breast cancer, or if she decides she wants surgery for any other reason. Another option for women with particularly low-risk types of DCIS is to take the hormone pills tamoxifen to prevent breast cancer, rather than surgery.

Experts estimate that at least half of all women diagnosed with DCIS would never develop breast cancer even if they never received any treatment for their DCIS. Since no one knows for sure which women with DCIS will develop breast cancer and which won’t, most women with DCIS choose to get some form of surgery. This booklet focuses on helping women decide what kind of surgery to get and what other kinds of treatment they might want or need.  Patients should keep in mind that if their physician tells them that they have a particularly low-risk type of DCIS, they may want to consider active surveillance or tamoxifen only, rather than surgery in addition to other treatments.

The current booklet was approved and funded by a grant from the D.C. Cancer Consortium through the Department of Health, Government of the District of Columbia, and a grant from the Jacob and Hilda Blaustein Foundation.  To request copies of the free patient booklet, write  info@stopcancerfund.org   or call    202-223-4000.

We are currently updating the booklet to include information about active surveillance. 

AIDS activist takes up a new fight: defending FDA

In the News

This Associated Press article quotes Gregg Gonsalves’ testimony at the Senate briefing hosted by the National Center for Health Research on June 12, 2014.

By Matthew Perrone

WASHINGTON (AP) — As an AIDS activist in the early 1990s, Gregg Gonsalves traveled to Washington to challenge the Food and Drug Administration.

Photo from Associated Press. In this July 22, 2014 photo, AIDS activist Gregg Gonsalves walks outside Grand Central Terminal during a photo session, in New York. In the early 1990s, Gonsalves traveled to Washington to confront, provoke and challenge officials at the Food and Drug Administration. A quarter century later, he still travels to Washington, but with a very different agenda: to defend the FDA. (AP Photo/Richard Drew)
Photo from Associated Press.
In this July 22, 2014 photo, AIDS activist Gregg Gonsalves walks outside Grand Central Terminal during a photo session, in New York. In the early 1990s, Gonsalves traveled to Washington to confront, provoke and challenge officials at the Food and Drug Administration. A quarter century later, he still travels to Washington, but with a very different agenda: to defend the FDA. (AP Photo/Richard Drew)

Gonsalves was part of the confrontational group AIDS Coalition to Unleash Power, which staged protests outside the FDA’s headquarters, disrupted its public meetings and pressured its leaders into speeding up the approval of experimental drugs for patients dying of AIDS.

A quarter century later, Gonsalves still travels to Washington, but with a different agenda: to defend the FDA.

At a recent forum on FDA issues, Gonsalves implored congressional staffers to protect the agency from growing anti-regulatory sentiment that he worries will roll back safety and effectiveness standards for all types of drugs. The efforts include new state laws designed to undercut the FDA’s authority by giving patients early access to unapproved drugs and a lobbying push by industry groups to speed up the time it takes the FDA to review new treatments.

Both initiatives come at a time when researchers who study the FDA say the caricature of a slow, outdated bureaucracy is inaccurate. The FDA reviews most drugs in 10 months and high-priority drugs in six months or less. And a 2012 review in the New England Journal of Medicine showed that FDA regulators approve new drugs faster than their counterparts in Europe and Canada.

“The rhetoric we hear today is that FDA stifles innovation, that FDA keeps drugs out of patients’ hands. And you know, that was our rhetoric in 1989,” says 50-year-old Gonsalves, now a program director at Yale University. “But there’s no countervailing narrative that we need a strong FDA.”

The story of how Gonsalves went from FDA critic to supporter is intertwined with the AIDS movement’s impact — and its unintended consequences — on the agency.

Groups like ACT UP showed that FDA’s bureaucracy could be influenced by outside pressure. Following protests by ACT UP, the FDA went from taking over two years to approve most drugs to clearing HIV drugs in a few months. In the early 1990s, those shorter review times were written into laws that have governed FDA procedures ever since. But while the push for ever-faster reviews was kicked off by AIDS activists, it is now primarily driven by pharmaceutical lobbying groups and libertarian think tanks.

Since May, three states — Colorado, Louisiana and Missouri — have passed laws designed to allow terminally ill patients to receive experimental drugs that have not been cleared by the FDA. Arizona will vote on its own so-called “right to try” initiative in November and lawmakers in Florida, Oklahoma and Utah are set to introduce similar bills. All of these efforts are driven by lobbyists from the Goldwater Institute, a libertarian think tank.

Supporters have dubbed the measures “Dallas Buyers Club” laws, after the Oscar-winning movie about an AIDS patient who thwarts FDA regulators by smuggling in HIV drugs from overseas. And lawyers for the Goldwater Institute acknowledge that groups like ACT UP helped pave the way for their strategy, though they say more deregulation is needed.

“Many, many groups have been trying to get FDA reform for decades and the only real successful movement was the AIDS movement,” says Christina Sandefur, an attorney with the Arizona-based Goldwater Institute.

Mark Harrington, executive director of the Treatment Action Group, which spun off from ACT UP in the 1990s, says AIDS activists have long tried to distance themselves from such anti-Washington efforts. By the mid-1990s, Harrington and Gonsalves were actually pushing for longer, larger studies of HIV drugs. That’s because the first drugs approved by the FDA were linked to dangerous side effects, including anemia and nerve damage.

Harrington says the Goldwater-backed state laws are political theater that will not help desperate patients. “They’re providing false hope and really pushing quack cures and medicines that could be unsafe and ineffective,” he says.

Federal law makes clear that patients do not have a right to experimental drugs and the Supreme Court has refused to hear challenges to FDA’s authority over the matter. For its part, the FDA already gives dying patients access to unapproved medicines if drugmakers are willing to provide them.

“The agency stands ready to work with companies that are interested in providing access to experimental drugs,” said FDA spokesman Stephanie Yao, in a statement.

But there’s nothing in the state laws that require companies to grant early access.

While the FDA faces pushback from conservative activists at the state level, it is also faces industry pressure at the federal level.

House lawmakers have held half a dozen hearings this year on “accelerating the pace of cures in America.” The push is part of a pharmaceutical industry-backed initiative dubbed “21st Century Cures,” designed to streamline the drug approval process.

“There is no doubt that our antiquated, patch-work clinical trial system makes developing new treatments a cumbersome, expensive and protracted process,” said the Friends of Cancer Research group in recent congressional testimony. The group receives funding from Pfizer Inc., GlaxoSmithKline and many other drugmakers.

Some FDA watchers say it’s too early to worry about a major overhaul of the agency. Congress is gridlocked and no major health legislation is expected to pass until after the next presidential election.

But Gonsalves isn’t wasting time. In closing his talk on Capitol Hill earlier this summer, Gonsalves warned Senate staffers that a political shift to the right in coming elections could “change the game” for drug safety and effectiveness.

“We will have a different FDA than we have had for the last 30 years.”

Photo credit to the Associated Press, click here for the original story online.

Johnson & Johnson praised for taking uterine surgery tools off market

In the News, News Stories & Editorials
By Katie Thomas, The New York Times
July 31, 2014

morcellator

Johnson & Johnson, which has come under withering criticism for its response to problems with some of its medical devices, won cautious praise from critics on Thursday for its decision to withdraw three products used in uterine surgery because of a risk of spreading cancerous tissue, only months after the safety issue became widely known.

Some experts continued to debate the medical value of the devices. A handful of other, smaller companies sell similar products.

The Ethicon unit of Johnson & Johnson said Wednesday that it was asking hospitals to return three types of power morcellators, devices that are commonly used in uterine surgery to remove fibroids by cutting the tissue into tiny pieces and extracting them through small incisions. In April, the Food and Drug Administration recommended that doctors stop using the procedure after the agency concluded that the risk of spreading cancer was higher than previously thought. That led Johnson & Johnson to announce that it would suspend sales and marketing of its products while it studied the issue, but it stopped short of withdrawing them from the market.

In a letter sent to health care providers, Ethicon asked that hospitals return three models of power morcellators made by the company: the Gynecare Morcellex and Gynecare X-Tract tissue morcellators, as well as the Morcellex Sigma tissue morcellator system.

About 50,000 operations a year involve power morcellation of tissue containing fibroid tumors, according to the F.D.A.

Some critics said they were pleasantly surprised that Johnson & Johnson acted as quickly as it did. “The company has had a rather abysmal track record on the public health front of ethical breaches in the last few years, so this is good that they’re doing this,” said Diana Zuckerman, president of the National Center for Health Research, a public health advocacy group that has criticized the company in the past over its safety record, especially concerning pelvic mesh implants. Dr. Zuckerman owns stock in Johnson & Johnson and her father, now retired, worked for many years in quality control at the company. Speaking of the decision on the power morcellators, she said, “At least it goes back to an earlier time when the company was seen as doing the right thing.”

The move comes as some in the gynecology field continue to disagree about the usefulness of power morcellators. Although the risk of spreading cancer through this procedure has been previously known, it was long believed that the chances for spreading cancer were lower, ranging from 1 in 10,000 to 1 in 500. In April, the F.D.A. concluded that the risk was closer to 1 in 350.

Still, some doctors cautioned against vilifying the procedure, saying that power morcellation allows minimally invasive surgery that, if carefully done, can be a better choice for some women. Without morcellation, more women will have to undergo serious abdominal surgery, which carries the risk of infection, bleeding, pain and blood clots. “These are things that people also die of,” said Dr. Barbara Goff, director of gynecological oncology at University of Washington. “So my concern is that we aren’t looking at this in balance.”

That medical debate is the reason Prof. Erik Gordon, who teaches business at the University of Michigan and has previously criticized the company for its safety record, said he was surprised that it acted as quickly as it did. “This is one of those things where it’s enough up in the air that you might have expected Johnson & Johnson to say, well, we’re going to keep it on the market because the evidence is inconclusive,” he said. “But that’s not what they did.”

The wrong approach to breast cancer

In the News, News Stories & Editorials
By Peggy Orenstein
New York Times
July 26, 2014

BERKELEY, Calif. — ONE of the nastier aspects of breast cancer is that it doesn’t have the five-year sell-by date of some other malignancies: you’re not considered “cured” until you die of something else. Although it becomes less likely, the disease can come back eight, 10, even 20 years after treatment. I fell on the wrong side of those odds.

I had a tiny, low-grade tumor in 1997; 15 years later, in the summer of 2012, while I was simultaneously watching “Breaking Bad,” chatting with my husband and changing into my pajamas, my finger grazed a hard knot beneath my lumpectomy scar. Just as before, time seemed to stop.

The recurrence appears to have been confined to my breast and was, like the original tumor, a slow-moving form of the disease. Since the lumpectomy and radiation I had in 1997 failed, however, this time the whole breast had to go. My first question to my oncologist (after “Am I going to die?” Answer: yes, someday, but probably not of this) was whether I should have the other breast removed, just to be safe.

It turns out, I’m not alone in that concern. After a decades-long trend toward less invasive surgery, patients’ interest in removing the unaffected breast through a procedure called contralateral prophylactic mastectomy (or C.P.M., as it’s known in the trade) is skyrocketing, and not just among women like me who have been through treatment before.

According to a study published in the Journal of Clinical Oncology in 2009, among those with ductal carcinoma in situ — a non-life-threatening, “stage 0” cancer — the rates of mastectomy with C.P.M. jumped 188 percent between 1998 and 2005. Among those with early-stage invasive disease, the rates went up 150 percent between 1998 and 2003. Most of these women did not carry a genetic mutation, like the actress Angelina Jolie, that predisposes them to the disease.

Researchers I’ve spoken with have called the spike an “epidemic” and “alarming,” driven by patients’ overestimation of their actual chances of contracting a second cancer. In a 2013 study conducted by the Dana-Farber Cancer Institute in Boston, for instance, women under 40 with no increased genetic risk and disease in one breast believed that within five years, 10 out of 100 of them would develop it in the other; the actual risk is about 2 to 4 percent.

Many of those same young women underestimated the potential complications and side effects of C.P.M. Breasts don’t just screw off, like jar lids: Infections can occur, implants can break through the skin or rupture, tissue relocated from elsewhere in the body can fail. Even if all goes well, a reconstructed breast has little sensation. Mine looks swell, and is a remarkably close match to its natural counterpart, but from the inside it feels pretty much like a glued-on tennis ball.

Of course, as any cancer patient will tell you, our fear is not simply of getting cancer, it’s of dying from it. What’s a mere mammary gland when, as Amy Robach, a journalist at ABC News, told People magazine last year after her own C.P.M., “I want to be at my daughters’ graduations. I want to be at their weddings. I want to hold my grandchildren.”

Unfortunately, for most women, C.P.M. is irrelevant to making those milestones. The most comprehensive study yet, published earlier this month in the Journal of the National Cancer Institute, showed virtually no survival benefit from the procedure — less than 1 percent over 20 years.

Researchers used the Surveillance, Epidemiology, and End Results registry and other databases to model survival chances for women who opted for C.P.M. and those who did not. They took into account a woman’s age at diagnosis, the stage and biology of her original tumor, the likelihood of dying from that cancer, the risk of developing cancer in the healthy breast, and the potential of dying from a new cancer. They even tweaked the numbers, nearly doubling the risk of contracting a second cancer and exaggerating the aggressiveness of a new tumor and the effectiveness of C.P.M.

“The story didn’t change,” Todd M. Tuttle, chief of surgical oncology at the University of Minnesota and the study’s senior author, told me. “Even if we used unrealistic figures, the conclusions were still the same. There was no group with a survival benefit of even 1 percent.”

How can that be? Well, first of all, it is extremely rare for a tumor on one side to spread to the other. Cancer doesn’t just leap from breast to breast. In any case, cancer confined to the breast is not deadly. The disease becomes lethal only if it metastasizes, spreading to the bones or other organs. Cutting off the healthy breast won’t prevent the original tumor from doing that. As for developing another cancer, Dr. Tuttle said, when that does happen (and remember, it’s far less common than patients believe), 91 percent will be early-stage lesions, so more readily treatable.

There’s some indication that patients understand that, yet choose C.P.M. anyway. The majority of the young women in the Dana-Farber survey knew the procedure wouldn’t prolong life; even so, they cited enhanced survival as the reason they had undergone it.

Such contradictions aren’t unusual, according to Steven J. Katz, a professor of medicine and health management and policy at the University of Michigan, who studies medical decision making. In exam rooms, all of us — men, women, cardiovascular patients, diabetics, cancer patients — tend to react from the gut rather than the head. “The general response to any diagnosis is, we want to flee it,” Dr. Katz explained. “It’s the kind of fast-flow decision making that we’re wired to perform. And it’s very difficult at that point to put data before a patient.”

I get that. When my cancer was first diagnosed, I felt as if a humongous cockroach had been dropped onto my chest. I could barely contain the urge to bat frantically at my breast screaming, “Get it off! Get it off!” Physicians, according to Dr. Katz, need to better understand how that visceral reaction affects treatment choices. They also need to recognize the power of “anticipated regret”: how people imagine they’d feel if their illness returned and they had not done “everything” to fight it when they’d had the chance. Patients will go to extremes to restore peace of mind, even undergoing surgery that, paradoxically, won’t change the medical basis for their fear.

Mothers inevitably cite their children as motivation for radical treatment; self-sacrifice has, after all, long defined good motherhood. It seems almost primal to offer up a healthy breast — with its connotations of maternal nurturance — to fate, as a symbol of our willingness to give all we have to and for our families. It’s hard to imagine, by contrast, that someone with a basal cell carcinoma on one ear would needlessly remove the other one “just in case” or for the sake of “symmetry.”

Treatment decisions are ultimately up to the individual. But physicians can frame options and educate patients in a way that incorporates psychology as well as statistics. Beyond that, doctors are not obliged to provide treatment that is not truly necessary.

The good news is that treatment to reduce the risk of metastasis has improved over the years. Not enough, but significantly. So those of us who dream of dancing at our children’s weddings? We may yet get there. But if — when — we do, it won’t be because of C.P.M.