Category Archives: We’re In the News

No more Pap smears?

Cervical Cancer & HPV, In the News, News Stories & Editorials, , ,
By Diana Zuckerman, PhD
2014

If you’re a woman over 21, that headline probably got your attention. After all, who likes Pap smears? Wouldn’t it be great to never need one again?

Well, don’t get too excited because the alternative could be worse, if a Food and Drug Administration (FDA) advisory committee gets its way. The committee proposes replacing Pap smears with an equally invasive but less conclusive test, the HPV test, when women reach the age of 25. And then, if the HPV test indicates the presence of HPV (or human papillomavirus, which is very common in sexually active young women and usually goes away by itself), the committee proposes following up the test with an even more invasive procedure called a colposcopy. Patients describe a colposcopy as being like a Pap smear that takes 20 minutes instead of a few seconds.

The purpose of a Pap smear is to test for abnormal cells in your cervix that could eventually turn into cervical cancer. Two strains of HPV virus are responsible for 70 percent of cervical cancers. It’s very common for sexually active women to be infected with HPV, but usually the body gets rid of the virus within a year or two all on its own. HPV can only cause cancer when it lingers in the body for several years and starts to damage the cervix.

Even if a woman has had an HPV vaccine, she could still potentially develop cervical cancer, so experts advise women to get a Pap smear every three years, starting at age 21 and ending at age 65.  Starting at age 30, women are given the option of asking their doctor to use the same sample for a Pap smear and the test for HPV. If nothing suspicious is found, they can get screened using both tests every five years instead of every three for the Pap smear alone.

Current guidelines recommend that if a woman has an abnormal Pap smear and an HPV test indicating that she has the types of HPV that can cause cervical cancer, she should undergo a colposcopy to see if she needs surgery. Most women get Pap smears to screen for problems, and typically only get an HPV test if their Pap results indicate abnormal cells.

That’s why not many women currently get the HPV test. The company that makes HPV tests would like to sell more of them. So it’s asked the FDA to change the agency’s instructions for using the HPV test to screen healthy women. Instead of an optional use with Pap smears, it wants FDA approval to use the HPV test on its own to screen all healthy women starting at age 25.

Unfortunately, the HPV test by itself isn’t very useful because so many young women have HPV that will disappear without any treatment. Having an HPV test without also getting a Pap smear to check for problems is going to scare a lot of women who are not developing cervical cancer.  Instead of waiting a few months to see if the HPV goes away by itself (which it typically does), the company wants those women to get a colposcopy, which is as painful as a Pap smear but the pain lasts longer and the procedure costs more. And like a Pap smear, the test isn’t always accurate.

Otherwise, it’s a great idea.

We’re not the only ones who believe that the current guidelines should not be changed. The unbiased experts at the U.S. Preventive Services Task Force recommend that the HPV test only be used on women 30 and over, and only in combination with a Pap smear. They point out that if the HPV test is used on younger women, the results can’t distinguish between HPV that would go away on its own and HPV that could cause cancer. This would lead to unnecessary worry for young women and many unnecessary colposcopies.

At an FDA Advisory Committee meeting this month, Anna Mazzucco, PhD, from our staff expressed her concerns about replacing Pap smears with HPV tests. She pointed out that Pap smears provide inexpensive and and effective screening. In fact, the women who get cervical cancer are usually women who did not regularly get Pap smears or follow-up.

Research indicates that the way to save lives is to help women get screened with Pap smears, not to put them through unnecessary follow-up procedures. The American College of Gynecologists—the doctors who do Pap smears, HPV tests, and colposcopies—also expressed concerns about changing current policies, since there is no evidence that the proposed changes would save as many lives.

In addition, we believe that the more expensive and painful procedures would discourage women from getting screened or following up after receiving suspicious results.

What is going on at the FDA? Why are they choosing advisors who ignore the research evidence in favor of a new, unproven screening strategy? The FDA needs advisors who focus on scientific evidence and who make recommendations based on facts, rather than embracing every new “innovation” in health care, regardless of whether it will work.

When the “old ways” are effective, let’s not throw them out unless there is proof that the new, more expensive way is better.

 

A version of this post appeared in Huffington Post and Maria’s Country Kitchen.

FDA warns against procedure to remove uterine fibroids; says it could spread hidden cancer

News Stories & Editorials
BRADY DENNIS, THE WASHINGTON POST
April 17, 2014

The Food and Drug Administration on Thursday took the rare step of urging doctors to stop performing a surgical procedure used on tens of thousands of women each year to remove uterine growths, saying the practice risks spreading hidden cancers within a woman’s body.

The procedure, known as power morcellation, has long been used in laparoscopic operations to remove fibroid tumors from the uterus, or to remove the uterus itself. It involves inserting an electric device into the abdomen and slicing tissue in order to remove it through a small incision. The surgery is far less invasive than traditional abdominal operations.

But the FDA on Thursday agreed with a growing chorus of researchers and clinicians who oppose the procedure, saying that it can recklessly spread undetected cancers throughout the body and make the disease more lethal in the process. The agency is not seeking to ban the practice or the roughly two dozen FDA-approved devices used to perform it, but hospitals and gynecologists are likely to abandon the procedure because of potential liabilities.

The FDA said its analysis determined that an estimated 1 in 350 women who undergo morcellation have an unsuspected form of uterine cancer called uterine sarcoma.

“The existence of the risk is not new,” said William Maisel, chief scientist at the FDA’s Center for Devices and Radiological Health. “What is new is that the magnitude of the risk appears to be higher than was appreciated by the clinical community.”

Maisel acknowledged that the agency was spurred to action — or at least moved more quickly — because of a high-profile campaign waged in recent months by a Massachusetts couple, both doctors, for a moratorium on the practice.

Anesthesiologist Amy Reed, a mother of six who last spring treated Boston Marathon bombing victims as well as one of the suspected bombers, underwent what was supposed to be a routine procedure last fall to end bleeding from fibroids. The procedure spread undetected, cancerous tumor fragments throughout her abdomen. Now she is battling stage-four leiomyosarcoma, a rare and aggressive form of uterine cancer.

Her husband, Hooman Noorchashm, a Harvard-affiliated cardiothoracic surgeon, responded by launching a campaign to ban the widely used procedure. He has e-mailed numerous regulators, doctors and lawmakers, written to medical journals and lobbied hospitals. The couple started a Change.org petition to end the practice.

Noorchashm said Thursday that he appreciated the FDA’s relative speed in addressing the problem. “You don’t even have to be a doctor to recognize that if tissue or a tumor has malignant potential, you should not mince it up inside someone’s body,” he said. “That’s just bad medicine.”

Many women develop uterine fibroids — benign growths that originate in the muscle tissue in the wall of the uterus. Although many fibroids do not cause problems, others can result in frequent urination, prolonged menstrual bleeding and pelvic pain.

Of the more than 500,000 hysterectomies performed in the United States each year, about 11 percent, or more than 50,000, involve morcellation, according to the American Congress of Obstetricians and Gynecologists. Some doctors advocate performing a morcellation only when using an “isolation bag” in an effort to minimize the spread of tissues, but that method is not foolproof, as the bags can break.

“When you consider what the benefit is, which is a shorter hospital stay and less pain, then consider what the risk is — this could kill you — most women would not choose that risk if they really understood what is at stake,” said Diana Zuckerman, president of the Cancer Prevention and Treatment Fund.

Even before Thursday’s announcement by the FDA, the push to limit uterine morcellations had gained traction.

In February, Temple University Hospital issued guidelines instructing surgeons not to perform the procedure on fibroids over a certain size. Doctors may use the procedure for smaller fibroids only after informing patients about the risks and, in most cases, using isolation bags. Even before that, prompted by Noorchashm’s campaign, Massachusetts General Hospital and Brigham and Women’s Hospital in Boston took similar measures.

On Thursday, the heads of obstetrics and gynecology at both Boston hospitals promptly notified their staffs to suspend use of power morcellation until further notice.
Margaret Jacobson, the medical director of Whatcom Hospice in Bellingham, Wash., cried upon hearing of FDA’s action on Thursday.

In March 2012, her sister, Elizabeth Jacobson, had a hysterectomy by morcellation. Elizabeth Jacobson did not want to miss much work, and the promise of a minimally invasive procedure appealed to her.

But inside the large fibroid that a doctor shredded lurked malignant cells that spread throughout her abdomen, her sister said. Soon, Elizabeth Jacobson was diagnosed with aggressive uterine cancer. She then had a second surgery, rounds of chemotherapy and long stretches of misery. She died Jan. 8, 2013.

“She suffered terribly,” Margaret Jacobson said. “It devastated our family.”
Thursday’s news brought a measure of relief, she said, that other women might avoid the same fate, especially given that safer surgical alternatives already exist.
“It’s a victory ,” Jacobson said. “It’s not okay to tolerate these deaths. . . . My sister’s life was extraordinary; she was beautiful and loving. She does not deserve to be an easily dismissed statistic.”

FDA advises halt of common uterine fibroid procedure citing cancer risks, overseer urges stop in use of morcellators to remove uterine growths

News Stories & Editorials
JON KAMP AND JENNIFER LEVITZ, WALL STREET JOURNAL
April 17, 2014
The U.S. Food and Drug Administration on Thursday advised doctors to stop using power morcellators in women’s abdomens to remove uterine growths called fibroids, citing the risk of spreading cancer.The popular devices—which typically use a tube-shaped blade to grind up and remove fibroids or the entire uterus to avoid the long surgical scars associated with traditional, open surgery—can also spread an often undetectable cancer known as a uterine sarcoma. In a rare safety alert for medical devices, the FDA cited estimates that this cancer affects one in 350 women undergoing such procedures and can significantly worsen the odds of long-term survival.”For this reason, and because there is no reliable method for predicting whether a woman with fibroids may have a uterine sarcoma, the FDA discourages the use of laparoscopic power morcellation during hysterectomy or myomectomy for uterine fibroids,” the agency said in a safety communication posted on its website Thursday. Myomectomy is the removal of just fibroids.”In general, the procedure should not be performed,” Dr. William Maisel, deputy director for science and chief scientist at the FDA’s Center for Devices and Radiological Health, said Thursday at a media briefing.If doctors do perform such procedures, the FDA said, they should advise patients of the cancer-spreading risk.The safety alert was addressed to doctors, medical associations, hospitals, women, device manufacturers and advocacy groups. The FDA’s alert follows a series of Wall Street Journal articles, starting in December, which documented the risk of spreading cancer and a campaign by two Boston-area physicians to halt the procedures.

Amy Reed, a 41-year-old mother of six and anesthesiologist at Boston’s Beth Israel Deaconess Medical Center, developed advanced uterine cancer shortly after a routine hysterectomy in October. The hospital where she was treated, Brigham and Women’s Hospital, acknowledge that use of morcellation worsened her cancer.

The FDA began its review in December, “when some high-profile cases covered in the media came to our attention,” Dr. Maisel said.

The FDA’s alert triggered immediate action on Thursday. Dr. Isaac Schiff, the chief of the Department of Obstetrics & Gynecology at Massachusetts General Hospital, said, “I have asked our doctors to stop the procedure immediately until we have more information.”

The FDA’s move could change the way many women are treated for symptomatic fibroids, which are common but often painful growths that spur about 40% of the roughly half-million hysterectomies performed in the U.S. each year, by some estimates.

Morcellators, first introduced in the 1990s, have helped gynecologists perform about 50,000 of these procedures each year through tiny holes, rather than longer incisions that can lead to bigger scars and a longer recovery.

Diana Zuckerman, president of the nonprofit National Research Center for Women & Families, and an advocate for stiffer medical device regulations, said the FDA’s statement should have a major impact. Going against the FDA’s recommendation could increase doctor’s liability if there are mishaps, she said.

“What surgeon is going to take the chance of using this device if they FDA has made such a strong warning?” she said. The FDA’s action “is going to save a lot of lives.”

Dr. Maisel said the clinical community has been aware of the risk of cancer since the advent of the procedure, although he said the rate of that risk is only now coming into focus. He also said it’s conceivable that some patients may believe the risks of alternative procedures outweigh the risks of laparoscopic surgery with morcellation. They should be explicitly told of the risks, he said.

With the issue gaining steam since December, gynecological societies had already been doing their own reviews of power morcellation. Commenting on the FDA move on Thursday, the American College of Obstetricians and Gynecologists said its own review, which includes an assessment of risks for various groups of patients, is ongoing. “We greatly appreciate the urgency behind the issue,” the group said.

Dr. Maisel said older women have a higher risk of having a hidden sarcoma than younger women.

Many gynecologists have argued there are other ways to perform these procedures without cutting up tissue in the open abdomen, including performing vaginal procedures or cutting tissue manually and inside protective bags.

Gynecologists have acknowledged they seldom used these bags during hysterectomies and fibroid procedures previously. But a number of top hospitals, including Brigham and Women’s and Massachusetts General, have recently told their doctors to only morcellate inside such bags for added protection.

The FDA noted these changes, although Dr. Maisel also said bags have some downside, including obscuring surgeons’ view during procedures, and are “not a panacea.”

The FDA on Thursday said it will convene a public advisory committee meeting this summer to discuss whether such bags can enhance the effective use of morcellators, amid other issues. The agency instructed manufacturers “to review their current product labeling for accurate risk information for patients and providers.”

The FDA recommended “routine follow-up” with physicians for women who have already had hysterectomies or myomectomies, don’t have symptoms and were told post-surgery tests were normal. Tissue is commonly checked for cancer afterward. But women with “persistent or recurrent symptoms or questions should consult their health-care provider,” the agency said.

Statement of Dr. Diana Zuckerman, President, Cancer Prevention and Treatment Fund, regarding FDA’s announcement about power morcellation devices for uterine fibroids and other uterine surgery

In the News, Press Releases
April 17, 2014

The FDA has saved lives today by announcing that they discourage surgeons using an FDA-approved device called a power morcellation device to remove the uterus or uterine fibroids.

This announcement is the result of excellent media coverage explaining that when uterine fibroids contain undiagnosed cancer, the use of power morcellation during laproscopic surgery  can spread the cancer.  The result is that a small contained cancer can spread and become a stage 4, fatal cancer.

This example indicates several major flaws in the system intended to protect patients from unsafe medical products and procedures:

  1. FDA standards for approving medical devices is much too low, resulting in unsafe devices such as the power morcellation.
  2. While many patients have been irreparably harmed by power morcellation devices that spread their undiagnosed cancer, surgeons and other health professionals rarely reported when that happened.  The voluntary system of reporting serious medical harm from devices, including deaths, is not working because most physicians are not reporting most incidents, regardless of how serious they are.
  3. The FDA apparently hardly noticed the problems with power morcellation until two Boston physicians, Amy J. Reed and her husband, Dr. Hooman Noorchashm, became outspoken advocates against it in the media.  Dr. Reed was diagnosed with a rare uterine cancer as a result of morcellation of what was thought to be a benign fibroid, and she and her husband have used their medical connections and the media to try to prevent that from happening to any other women.  We thank them for their courage in speaking out and our prayers are with them.

The FDA needs to do more to prevent these and similar tragedies due to inadequately tested medical products that too often are used by physicians who are inadequately trained to use them safely.  And patients need to be better informed of the risks of medical procedures, not just of the possible benefits.

To read a recent medical journal article on power morcellation, see http://jama.jamanetwork.com/article.aspx?articleid=1828692

F.D.A. panel recommends replacement for the Pap test

In the News, News Stories & Editorials
BY ANDREW POLLACK, THE NEW YORK TIMES
MARCH 12, 2014

The Pap test, a ritual for women that has been the mainstay of cervical cancer prevention for 60 years, may be about to play a less crucial role.

A federal advisory committee recommended unanimously on Wednesday that a DNA test developed by Roche be approved for use as a primary screening tool.

“Has our Pap, as we know it, outlived its time?” Dr. Dorothy Rosenthal, a professor at Johns Hopkins University, testified to the committee, which advises the Food and Drug Administration. She said deaths from cervical cancer in the United States had stopped declining and that there would be “a tremendous gain” by moving to the new test.

The Roche test detects the DNA of human papillomavirus, or HPV, which causes almost all cases of cervical cancer. Pap testing involves examining a cervical sample under the microscope looking for abnormalities.

Until now HPV testing has been used mainly as a follow-up test when the Pap results were ambiguous, or used jointly with Pap testing.

Wednesday’s 13-to-0 vote — by a committee mainly of academic pathologists, microbiologists and gynecologists — would allow Roche’s test to be used alone as the initial test for women 25 and older.

The Roche test is seen as better than Pap tests in finding precancerous lesions.

The Pap test, which is well entrenched and has been highly successful, will not go away quickly, if at all, however.

Assuming the F.D.A. itself agrees with its advisory committee and approves the new use of Roche’s test, it would become just another option, not a replacement for the older testing regimens. And many doctors will not adopt the new test unless professional societies recommend it in guidelines, which could take years.

Use as a primary screening tool could mean much wider sales of HPV tests. The United States market for such tests is more than $200 million, according to DeciBio, a market research firm.

Qiagen is the leading seller of such tests, with Roche and Hologic and BD also participating. Various laboratories also offer their own tests. But the approval as a primary screening tool would be only for Roche’s Cobas test, which could help it gain market share.

Current United States guidelines recommend that women 30 to 65 undergo either co-testing with both HPV and Pap every five years, or Pap testing alone every three years. Women 21 to 30 are supposed to have Pap testing every three years.

The American Cancer Society estimates that about 12,360 new cases of invasive cervical cancer will be diagnosed in 2014 and about 4,020 women will die from the disease. Between 1955 and 1992, the cervical cancer death rate declined by almost 70 percent, mainly because of Pap testing. The death rate has remained stable in recent years.

HPV testing has advantages over the Pap test. Studies have shown it is much more sensitive in detecting precancerous lesions. Proponents say it is more objective, its results varying less from lab to lab than those of the Pap tests, which rely on the judgments of people viewing slides under a microscope.

The main drawback of HPV testing is that most people get infected with the virus after they become sexually active, although in most cases their immune systems can clear the virus. So many women, particularly young women, would be sent for additional examinations or biopsies that might not be necessary. That is why co-testing with both HPV and Pap is recommended only for women 30 and over.

Roche says it sidesteps that problem because its test specifically detects two subtypes of the virus, known as genotypes 16 and 18, that account for 70 percent of the cancer cases.

In its study, Roche showed that its test outperformed Pap testing alone in various measures, like the ability to detect precancerous lesions. A negative result on the HPV test was also a better predictor than a negative Pap test that a woman would remain free of lesions for the next three years.

Most people who testified to the committee, which met in College Park, Md., urged approval, some saying it was time to move to modern molecular science.

“George Papanicolaou did not know about HPV,” said Lee Shulman, a professor at Northwestern University, referring to the initial developer of the Pap test. He compared replacing Pap smears with HPV testing to the transition from the horse to the automobile.

But there were objectors who said the data was not sufficient to justify such a change.

“The proposed indication represents a radical shift in clinical practice which would affect millions of women for most of their adult lives,” said Anna E. Mazzucco of the Cancer Prevention and Treatment Fund, a patient support and advocacy group.

She said that most cases of cervical cancer were in women who did not undergo screening, not in those whose disease was missed by screening.

While the committee vote was unanimous, some members had reservations about using HPV testing for women under 30. Some said that HPV testing may not be much better, if at all, than the currently recommended use of both tests.

“I think women are going to be well served by having more choices, but it’s going to be very interesting to watch over the next several years as this rolls out,” said Dr. Alan G. Waxman, a professor of obstetrics and gynecology at the University of New Mexico.

The worst new drug of 2014

In the News, News Stories & Editorials, , , ,
DIANA ZUCKERMAN, PHD, PRESIDENT OF The Cancer Prevention & Treatment Fund 

It’s only February, so it may seem early to be talking about one of the Food and Drug Administration’s (FDA’s) worst decisions of 2014. Yes, the year just started, but the FDA has already made a decision that could potentially harm thousands of patients.

The agency just approved a new diabetes medication that doesn’t noticeably improve health but may in fact cause cancer.

It’s called Farxiga (Dapagliflozin) and it will be available—and probably widely advertised—very soon. The good news is that the drug lowers blood sugar, which is a major symptom of type 2 diabetes. However, lowering blood sugar is not necessarily a way to improve health. The new drug is meant to reduce medical problems such as heart disease or kidney damage. Unfortunately, there is no evidence that it actually improves health—in fact, quite the opposite.

The FDA reports that the most common side effects of Farxiga are genital fungal infections and urinary tract infections. Not fun, but those aren’t its most serious side effects. If a patient has moderate or severe kidney damage (as many diabetics do), the drug is not beneficial and could cause further damage, possibly even renal failure.

But the more frightening news is that patients taking Farxiga in studies done for the FDA were more than five times more likely to contract bladder cancer than the patients who took an older diabetes drug. Based on the findings of the companies making and selling Farxiga, FDA scientists concluded that the drug might stimulate bladder cancer in patients already at risk. The fear is that patients take diabetes drugs for years, and the longer someone takes this drug, the greater their risk of cancer.

Bladder cancer can be fatal, and is especially dangerous in people who have diabetes.

The companies’ studies also found that patients taking the drug were more than twice as likely to get breast cancer as diabetics not taking the drug, but they say this could have happened by chance.

It’s important to mention that only a small number of African Americans were studied. Because African Americans are more likely to have diabetes than Caucasians, these studies should have included greater numbers of them to find out if the drug is safe or effective for them.

With so many other diabetes drugs already on the market, why would anyone want to take this drug, and why would FDA approve it?  Here’s the scorecard, based on information provided by FDA scientists and available on the FDA website:

  1. Is the drug new? Yes.
  2. Does it reduce the medical problems caused by type 2 diabetes, such as blindness, heart disease, kidney damage, or amputations? No.
  3. Can it cause kidney damage or make it worse? Yes.
  4. Does research show that patients taking it are more likely to get bladder cancer. Probably.
  5. Does research show that patients taking it are more likely to develop breast cancer? Maybe.
  6. Does research show it is more effective than most diabetes drugs on the market? No.
  7. Will it cost more than most other diabetes drugs on the market? No information on cost yet, but new drugs usually do cost more.

The FDA is requiring that the label for the drug warn patients with bladder cancer that they shouldn’t take Farxiga because it might make their cancer worse. Ya think? And, the agency is requiring the companies to study 17,000 diabetes patients for at least four years to determine whether and how often patients taking Farxiga are diagnosed with cancer, liver problems, or heart disease when the drug is taken for a longer period of time.

The FDA rejected this drug two years ago because of these cancer concerns and questions about how well the drug prevents the major risks of diabetes. But the FDA has been beaten up by many complaining congressmen since then, and by some patient groups, pushing it to approve more new drugs quickly rather than waiting for safety studies to be completed. Also, the pharmaceutical companies don’t like to take no for an answer, they spend lots of money on lobbying, and they are very effective at pushing FDA to reconsider rejections. Because of that, the FDA selected an advisory committee to review the data in December, and those doctors and scientists also expressed concerns about risks and benefits. I was at the meeting and pointed out that there were too many serious unanswered questions.

Despite my concerns, the concerns of FDA scientists and other experts, and the concerns of the advisory committee members themselves, the committee recommended that if the companies continued to study the drug after it was approved, in order to figure out what the actual risks are, the company could sell it now.

It’s too early to say whether this will be the worst decision the FDA makes in 2014. Maybe the diabetes patients who take it will be lucky and not be harmed by the drug. Or maybe only a few patients will develop cancer or kidney failure as a result of taking it. Or the FDA scientists who expressed their concerns could be wrong, and I could have made a mistake when I agreed with them. But regardless of what happens with this drug, it still would be a bad decision to allow the sale of a drug with such serious (potentially fatal) unanswered questions.

Given the epidemic of diabetes in this country, the risks are huge. For that reason, this drug worries me even more than some of FDA’s similarly mind-boggling decisions of 2013, such as:

  • A drug for hot flashes that has no meaningful benefits but can increase the risk of suicide among women who weren’t depressed
  • An antibiotic for pneumonia that seems to kill more patients than other antibiotics

I’ve always respected the scientists at the FDA for working so hard to try to keep all of us safe. I still do. But decisions are being made at the agency that seem to ignore scientists and science, and that is dangerous for all of us. Whether we have diabetes, hot flashes, pneumonia, cancer, or any other disease, we deserve better.

 

A version of this post appeared in The Huffington Post, Maria’s Farm Country Kitchen

 In 2018, the U.S. Food and Drug Administration (FDA) announced a new warning on Farxiga and other SGLT2 inhibitors because they can cause a rare but life-threatening infection of the genitals and area around the genitals. The infection is called necrotizing fasciitis of the perineum or Fournier’s gangrene.  

Clinical evidence in FDA drug approvals varies widely, study finds

News Stories & Editorials,
by Sabriya Rice, Modern Healthcare
January 21, 2014

Not every new drug approved by the U.S. Food and Drug Administration has undergone the rigorous clinical testing that physicians and their patients might expect, according to new research.

A study published Tuesday in the Journal of the American Medical Association finds that the FDA has “flexible standards” for approving of new therapies. Using publicly available information from the FDA drug database, investigators identified 188 novel therapies for treating 206 conditions approved between 2005 and 2012. Of these, 37% were approved on the basis of a single clinical trial, 38% on the basis of two trials and 25% had been tested in three or more trials.

Although most therapies were supported by at least one randomized, double-blind trial—the gold standard for clinical research—there was wide variation in the duration, size and completion rates. Comparative-effectiveness data was available for less than half of the indications.The purpose of the study, according to its authors, was not to suggest that the FDA is not rigorous in its approach to drug reviews. The regulatory flexibility allows for a customized approach, and the agency can rapidly approve potentially effective therapies for life-threatening diseases and those for which there is no existing, effective treatment, such as orphan diseases, said Dr. Joseph Ross, assistant professor of general internal medicine at Yale University School of Medicine and one of the study authors.

According to Ross, the drug approval variability is problematic in the sense that both patients and physicians feel the research is the same across all drugs approved.

The FDA, in a statement responding to the findings, explained that drugs may be tested in clinical trials that enroll hundreds of participants, while others, particularly those seeking to treat rare diseases, may be tested in trials that enroll only a handful of participants. “In all cases, however, the statutory standards of safety and efficacy must be met in order for the drugs to be marketed in the United States,” the agency said.

Whether or not the process has become too lenient has become a topic of debate among advocates.

The Progressive Policy Institute, a center-left think tank, acknowledged in a policy brief (PDF) that the FDA must strike a difficult balance. “If it is too lenient, (the FDA) will allow the sale of drugs and medical technology that could harm vulnerable Americans. Too tight, and the U.S. is being deprived of key innovations that could cut costs, increase health, and create jobs.”

Some argue, however, that physicians don’t have time to sift through statistical data on every new drug approved, and when they do, there is little information to choose from as they make decisions about the safety of new therapies.

“With new drugs, there is often an exaggeration of the benefits and underreporting of the risks,” said Diana Zuckerman, president of the National Research Center for Women & Families, who has testified at several hearings on drug safety. “There’s so much emphasis on drugs being the latest, the most innovative and novel—but unfortunately this usually means it’s just new, not necessarily better,” she said.

Zuckerman conceded that it’s a given that the FDA should have the flexibility to provide access to new treatments when there are no available options. “But the FDA shouldn’t be rushing studies for diseases that have good alternatives. It’s better to have an old treatment that is proven to be safe and effective than a new treatment that we don’t know is safe and may not improve health.”

“I think we can all agree that if you have a disease for which there are no available treatment options, that is the time to be flexible, although you’d still want the best possible research,” she said.

Ross, the Yale researcher who worked on the study, encourages physicians to be more nuanced with how new treatments are presented to patients. When prescribing newly approved drugs that have limited trial data, he said physicians should be clearer with patients about what the research shows. The physician should say, “There are not a lot of options, this drug was just approved, but we don’t know if it extends your life,” rather than giving the patient the impression the drug does things for which it has not been tested.

To view the original article in Modern Healthcare, click here.

October surprise

News Stories & Editorials,
by Diana Zuckerman, PhD
October 2, 2013

In politics, an October surprise is when a candidate has dirt on his or her opponent that is saved for just a few weeks before the election, so that the opponent’s media coverage will be negative just as people are deciding who to vote for.

This year’s October surprise was different, since there are very few elections. This year, the October surprise will be:

  1.  How long will the government be shut down?
  2. Will millions of Americans lose the opportunity to buy affordable health insurance in a bargain to re-open the government or pass the debt ceiling bill?

We live in a Democracy, but the decision about this October’s surprise is going to be made by a very small group of Congressman. How did 2-3 dozen Tea Party Members in the House of Representatives get that power in a Congress of 535 people, and should they be applauded for their principles or shunned for their bullying?

And let’s remember what’s at stake:

  • While the government is closed down, some essential services will continue (such as Social Security checks) but approximately a million workers won’t be paid (a blow to the economy) and services will be curtailed (national parks closed, Social Security, disability, food programs for poor families, and passport services delayed, etc.)
  • U.S. healthcare is the most expensive in the world, and Americans don’t live as long in the U.S. as they do in 16 other countries, including Canada, Portugal, and Japan. The purpose of the Affordable Care Act is to save lives by providing affordable health insurance to millions of Americans.

For those who (understandably) have tried not to think about the crisis, here’s a short “October Surprise for Dummies” version.

  •  The federal government and the services it funds (military, Social Security, cancer research, veterans’ health care, grants to public schools, etc.) is funded for one year at a time, starting Oct. 1 every year. This year, none of the funding bills have passed for the coming year, so the government closed down on October 1.
  • The funding bill for the year starting October 1, 2013 did not pass when it was supposed to (months ago) because 2-3 dozen Tea Party Republicans have refused to vote to support a budget unless it specifically removes funding for the Affordable Care Act, which they derisively call Obamacare.
  • You might ask: don’t we live in a Democracy where a bill in the House of Representatives needs a simple majority (218 votes) of the 435 Members to pass? How can even a few dozen people stop a bill if more than 218 want that law to pass? The answer is that Speaker of the House John Boehner is going by a different set of rules. Instead of allowing a vote for the entire House of Representatives, which would certainly pass a budget that the Senate and President would agree to, the Speaker won’t allow a vote on a bill unless it has the support of a majority of just the Republican Members of Congress – not including the Democrats. For hundreds of years, laws have passed only because of bipartisan cooperation, but that is not even possible now. Without support from enough Tea Party Members, the Republicans can’t get 218 votes to prevent the government from shutting down unless the bill cuts funding for Obamacare.
  • Hence, no bill yet, and a shutdown of most government activities. For example, the Congress made an exception at the last minute for pay for our military, but not disability payments for our veterans. The irony is that even as the government shuts down October 1, the new health care law will continue. In fact, on October 1, millions of people went online or in health centers to figure out which policies to sign up for. Those patients were from all over the country, including the red states that have officially opposed Obamacare.
  • If Congress can come to an agreement to fund the government, the next crisis date is October 17, when our country’s debt ceiling is reached. Our government borrows money every year, because we spend more than our taxes can pay for. Congress then votes for a new amount of debt that it considers acceptable. Like the annual budget, the debt ceiling has been held hostage by the same Tea Party Congressmen (and women) who refuse to vote for it unless – you guessed it – Obamacare is repealed, or at least loses the funding it needs to provide health insurance for people who can’t afford it.
  • And, if Congress comes to a compromise to fund the government temporarily (for 6 weeks, for example), then we will still need to go through this again when the temporary bill expires. That’s why the Democrats have told the Republicans that they want to meet to discuss a compromise for a 1-year funding bill, not a 6-week funding bill.

Is this any way to run a country?

Some people believe that the Tea Party opponents of the 2013 funding bill should be applauded for their principled stand. After all, they ran for office promising to gut the health care legislation. But, what is it about “Obamacare” that’s so terrible that it is worth shutting down the government or letting our country default on the money it owes.

Under Obamacare, health insurance plans now must provide:

  • Prescription discounts for seniors
  • Family insurance policies must cover all children under 26, even if they don’t live at home
  • Starting in January, insurance policies must cover all pre-existing conditions, such as cancer and heart disease. And they can’t stop paying for coverage when a disease gets very expensive (no yearly limits or lifetime limits on coverage, which in the past have bankrupted many families)

Starting in January:

  • States can get free Medicaid coverage for adults and children up to 133% of the poverty level (about $30,000 for a family of 4)
  • People who don’t have insurance through their employer or Medicaid will be able to go to state “Insurance Exchanges” that offer affordable health insurance. The federal government will help pay the annual cost of insurance for people earning up to 400% of the poverty level.

Sounds good, doesn’t it? There is a catch, though. The “mandate” in the law requires every American to buy health insurance, starting in January. The Tea Party hates the mandate, but the penalty if someone doesn’t comply is only $95 for the entire year. That means that those who don’t like the law and don’t want to buy health insurance would get away with the equivalent of a depressingly large parking ticket.

Is a mandate requiring insurance coverage (as is done with car insurance) fair? Unfortunately, it is the only way to keep prices down, because now that pre-existing conditions are covered for health insurance policies, a person could otherwise delay buying health insurance until they know they will have major medical expenses. (That would be like buying retroactive flood insurance to pay for flooding damage that already occurred a major saving for you, but untenable for insurance companies).

And, let’s not forget that Obamacare became law because of the affirmative votes of most Members of Congress, and that the numerous efforts to pass a law repealing it have been unsuccessful, because it does, in fact, benefit millions of Americans.

What will this year’s October Surprise be? Let’s hope it will be a pleasant surprise–a Congress that respects its citizens enough to preserve majority rule–the linchpin of our democracy.

 

This article appeared on the Huffington Post on October 2, 2013.

New concerns on robotic surgeries

News Stories & Editorials
by Rani Caryn Rabin, The New York Times
September 9, 2013

In early March 2009, Erin Izumi, a woman in her 30s from Tacoma, Wash., underwent robotically assisted surgery to treat endometriosis. The operation at St. Joseph Medical Center dragged on for nearly 11 hours.

Ten days later, Ms. Izumi was rushed to an emergency room, where doctors discovered that her colon and rectum had been torn during the operation. She was hospitalized for five weeks, undergoing a series of procedures to repair the damage, including a temporary colostomy, according to her attorney Chris Otorowski.

But even though medical device manufacturers and hospitals are required to report every device-related death and serious injury to a database maintained by the Food and Drug Administration within 30 days of learning about an incident, no report about the case was made in 2009. Hospital officials declined to comment, and a spokeswoman for the manufacturer said it became aware of the incident only when Ms. Izumi filed a lawsuit. It disputed the claim and settled the case in May 2012.

That was not the only lapse in reporting problems with robotic surgical equipment, a new study has found.

The equipment, called the da Vinci system, is made by Intuitive Surgical Inc. of Sunnyvale, Calif. It has been on the market for more than a decade; more than a million procedures have been performed with it. Between January 2000 and August 2012, thousands of mishaps were reported to the F.D.A. In the vast majority of cases, the patient was not harmed, but among the reports were 174 injuries and 71 deaths related to da Vinci surgery, according to a study published last week in The Journal for Healthcare Quality.

Yet by combing news reports and court records, researchers at Johns Hopkins were able to find examples of botched operations that were not reported to the agency. They concluded that adverse events associated with the da Vinci were “vastly underreported.”

It is fairly well known that reports made to the F.D.A. represent only “the tip of the iceberg” of surgical complications and adverse drug reactions, said Diana Zuckerman, the president of the National Research Center for Women and Families and an expert on the safety of medical devices, who was not involved in the study. The consequence is that little is known of the real disadvantages of the equipment, and the injuries and deaths it may cause, even as robotic surgery is widely marketed to consumers, Dr. Zuckerman said.

In a statement, Angela Wonson, vice president of corporate communications at Intuitive, said that the new study “gives the misleading impression that Intuitive Surgical has systematically failed in its obligation to timely report known adverse events to the F.D.A.” On the contrary, she said, “We take this requirement very seriously and make every effort to account for all reportable events — even those from several years prior.”

The new study follows a series of reports critical of robotically assisted surgery. Documents surfacing in the course of legal action against Intuitive have outlined the aggressive tactics used to market the equipment and raised questions about the quality of training provided to surgeons, as well as the pressure on doctors and hospitals to use it — even in cases where it is not the physician’s first choice and she has little hands-on experience.

Nevertheless, robotic surgery has grown dramatically, increasing more than 400 percent in the United States between 2007 and 2011. About 1,400 da Vinci systems, which cost $1.5 million to $2.5 million, have been purchased by hospitals, according to Intuitive’s investor reports.

The expansion has occurred without proper evaluation and monitoring of the benefits, said Dr. Martin A. Makary, an associate professor of surgery at Johns Hopkins and the senior author of the paper.

“This whole issue is symbolic of a larger problem in American health care, which is the lack of proper evaluation of what we do,” Dr. Makary said. “We adopt expensive new technologies, but we don’t even know what we’re getting for our money — if it’s of good value or harmful.”

Part of the problem is that the reporting mandate “has no teeth,” Dr. Makary added. “In health care, one fifth of the economy, we have this haphazard smattering of reports that relies on voluntary self-reporting with no oversight, no enforcement and no consequences.”

F.D.A. officials said in a statement that the agency has issued warning letters in the past when facilities have failed to report, and that the agency can take further regulatory actions like injunctions or imposing civil financial penalties.

A 2010 study found that 56.8 percent of surgeons surveyed anonymously said they had experienced irrecoverable operative malfunctions while using the da Vinci system, Dr. Makary noted.

Women were more likely to be harmed during the robotic procedures, Dr. Makary and his colleagues found. Nearly one-third of deaths that were reported to the F.D.A. database occurred during gynecologic procedures, and 43 percent of the injuries were associated with hysterectomies.

“Any time there is a serious problem with the da Vinci, it should be reported,” Dr. Zuckerman said. “It’s the F.D.A.’s job to figure out whether this is a problem related to the device or a doctor error.”

Click here to view the original article on nytimes.com.

Letter to the editor: Still working to understand cancer

News Stories & Editorials
Diana Zuckerman, PhD
August 5, 2013

Originally published in The Washington Post

The Post article on the overdiagnosis of cancer clearly reported the need to reduce patients’ fear and confusion about “cancers” that many experts now agree are not really cancer, because they don’t spread and aren’t harmful [“Panel addresses overdiagnosis of cancer,” news, July 30]. The major obstacle has been uncertainty about how likely these conditions are to predict cancer or change into an invasive cancer in the future. That is part of the reason many patients are choosing overtreatment, such as bilateral mastectomies that are not recommended and are not medically necessary.

In 2003, our center brought together nationally respected experts to the National Cancer Institute to address these issues. There were substantial disagreements, but for the sake of women across the country, none of us gave up. Let’s hope we make more progress in the coming months and years than we have in the past decade.