By Jim Spencer and Joe Carlson, Star Tribune: November 10, 2016

SILVER SPRING, MD. – First, health policy expert Diana Zuckerman displayed huge photos of the disfigured, bruised faces of two babies who had been implanted with Medtronic’s Infuse Bone Graft product in their skulls.

Then Zuckerman, who is president of the National Center for Health Research, put up on the same projector screen the language of the Food and Drug Administration’s 2015 warning that the product had never been approved for use in children and could be dangerous for them. The warning does not include the commercial name of the Medtronic product, nor does it include a ban on using it in kids.

The perspectives of injured patients drove home the stakes in an ongoing legal and ethical debate over what companies can say and do when they sell products for uses not described on the FDA-approved labels.

Doctors are legally allowed to use devices and drugs in unapproved ways that they think will benefit patients. The question for the FDA is how far manufacturers should be allowed to go in explaining products for applications the FDA has neither approved nor cleared, a practice known as off-label use.

Zuckerman said the Infuse Bone Graft is “contraindicated” for children. But the FDA’s 2015 warning didn’t ban the product in kids because some children have such significant bone defects or such rare bone disorders that they would be willing to accept the risks.

Although both kids and adults are susceptible to risks from Infuse like excess bone growth, kids are more vulnerable because their bones are still growing and they have less space to absorb unexpected swelling.

Medtronic has repeatedly denied allegations in lawsuits that it promotes Infuse for off-label uses. […]

The parents of Hailey Reuter, whose injury photo was one of the two Zuckerman displayed at the FDA hearing Thursday, have said in a lawsuit that no one informed them Infuse was going to be used in what they called an “experimental” surgery on their 5-month-old daughter at a children’s hospital in Cincinnati.

“Most consumers have no idea when they are given a drug or device off-label,” Minnesotan Kim Witczak told the FDA panel Thursday. Witczak became a patient advocate after her husband killed himself in 2003 after being prescribed the antidepressant Zoloft for insomnia, which she blames for causing his death.

Witczak was among roughly 30 witnesses Thursday, most of whom said companies don’t need more leeway to spread information about product applications on which the FDA has not ruled. […]

Steven Francesco, who said his son died from careless off-label prescribing, believes the answer is a strong commitment to much more pediatric research.

“Seventy to 90 percent of medications prescribed to children is off-label,” said Francesco, a former pharmaceutical executive. “Where you have no data, you have the Wild West.”

For the full article, click here.

By Carolyn Y. Johnson, Washington Post: November 14, 2016

A single sentence in President-elect Donald Trump’s health-care platform sends a strong hint to the drug and medical device industry that they may have an easier time getting their products on the market under his administration.

“Reform the Food and Drug Administration, to put greater focus on the need of patients for new and innovative medical products,” his health plan states.

On the face of it, the bullet point may seem almost bland, but efforts to integrate patients’ preferences and encourage innovation often result in proposals aimed at speeding up the process for getting new medicines on the market by easing regulations. Critics argue that such efforts can erode standards that are in place to protect patients from drugs that don’t work and might even be harmful.

“The language … is industry code for deregulation and reducing of safety standards,” said Robert Weissman, president of Public Citizen, a consumer watchdog. “Of course, the general deregulatory rhetoric from candidate Trump is a worry for us, but as applied to FDA, it would be very troubling.”

No one is sure about the precise direction of policy under the Trump administration. But the idea of faster approval of medicines and devices has been popular, meaning this may be one of Trump’s health-plan goals to gain support from both sides of the aisle. The drug industry, which had been preparing to defend its business model and pricing under a possible Hillary Clinton presidency, may now see an opportunity instead to streamline the drug-approval process, which companies have complained can be onerous, bureaucratic and a barrier to competition.[…]

But it may be unwise to read too much into the sentence, given Trump’s unpredictability — and the lack of certainty about who will define his health policy.

“I think the honest answer is nobody knows” what to expect, said Diana Zuckerman, president of the National Center for Health Research. “Some members of Congress owe pharma a favor; we don’t know the Trump campaign is in that position, and they might not be — and that might give them a certain amount of flexibility. The Trump campaign is nothing if not iconoclastic.” […]

For the full article, click here. 

Shannon Firth and Joyce Frieden, MEDPAGETODAY: November 9, 2016

WASHINGTON — With the Republicans winning the White House and retaining control of both houses of Congress on Tuesday, healthcare scholars predict big changes in some healthcare policies, although perhaps not as much as feared.

MedPage Today spoke with several policy specialists experts who shared their views of what changes a Trump administration, coupled with a Republican Senate and House, could mean for the healthcare system, including the Affordable Care Act (ACA). […]

Offering a more liberal perspective, Diana Zuckerman, PhD, president of the National Center for Health Research, said,”I’m not sure what will happen to the Affordable Care Act.” She noted that full repeal of Obamacare would be hard with so many people now relying on it, many of whom live in red states.

“A year from now, we could be in a very different situation politically,” she said, acknowledging that the election showed a very divided electorate. “Every president wants to say they have a mandate … it is harder to make very dramatic changes when you’ve got less than half of the vote and when you have a party that is very truly as divided as the Republican party.” […]

For the full article, click here.

Amy Martyn, CONSUMERAFFAIRS: October 27, 2016

Makers of medical devices face such little scrutiny from the Food and Drug Administration that even a 2011 Institute of Medicine report, commissioned by none other than the Food and Drug Administration, described the agency’s medical device evaluation process as “fatally flawed.”

Even worse, the FDA has reportedly allowed device-makers to flout the few regulations that they are supposed to follow.

In a lengthy statement, the FDA tells ConsumerAffairs that it had granted an exemption to Medtronic and defended the company’s actions.

“FDA’s allowance of a summary report in certain circumstances, under the relevant regulation, is both appropriate and in the best interests of the public health,” the statement says in part. “Such summary reporting can create practical efficiencies by reducing data entry and FDA staff review time of information that is already well-understood about a particular device.”

Asked to comment, Medtronic referred ConsumerAffairs to a statement the company published online. 

Multiple companies

Zuckerman’s complaints aren’t new. In 2014, three years after the FDA’s Institute of Medicine panel called its regulatory process for devices flawed, Zuckerman lead a separate study claiming that there is scant public research to back up the safety of many FDA-approved medical devices. The agency has repeatedly contested such critical findings.

Criticism invited

But recent actions by the FDA now suggest the agency may finally be taking some of the criticisms of its device regulation to heart. On October 21, the FDA launched a new online program to encourage anyone, from patients to doctors, to report misconduct by medical device-makers.

“The webpage is not in response to any recent news articles,” FDA spokesman Stephanie Caccomo tells ConsumerAffairs via email. “The webpage was developed to provide the public with more information on allegations of regulatory misconduct related to medical devices and provide clear instructions for reporting to the FDA.” For public health watchdogs like Zuckerman, whether the FDA’s new program will have teeth remains to be seen.

The FDA’s new site, “Reporting Allegations of Regulatory Misconduct,”  specifically singles out medical devices and instructs people to report anonymously if they wish to do so. “Anyone may file a complaint reporting an allegation of regulatory misconduct,” the FDA says, with instructions on how to submit complaints via email or hard mail. […]

For watchdogs like Zuckerman, the FDA’s new site soliciting allegations of abuse in the medical device industry is an encouraging step, but only on paper for now.  Though the new policy “sounds great,” she says, “will it make a difference? Will the FDA finally stop treating device companies like their favorite customers and remember that patients and consumers are their most important customers? …More importantly, will FDA finally decide that they will no longer allow device companies to ignore patient safety?”

For the full article, click here. 

Brett Norman, Politico Pro: September 16, 2016

Drug companies and research institutions will have to publicly report more clinical trial data, including results that show their products or experiments failed, under new policies rolled out Friday by HHS.

The new rules address the fact that researchers have routinely flouted requirements to report data to ClinicalTrials.gov. A final rule from FDA and a complementary policy from NIH clarify which trials must be reported and project a new federal commitment to crack down on violators – threatening fines of $10,000 per day or disqualification from NIH funding.

The widespread failure by institutions to report the data is “simply unacceptable,” NIH Director Francis Collins said on a call with reporters.

“This is fundamentally an ethical issue,” FDA Commissioner Robert Califf said. “This is about maintaining the trust that we have with [clinical trial] participants … that if it doesn’t benefit them, it will benefit others.”

A 2014 review of the results of 400 clinical studies found that 30 percent had not been published or shared with ClinicalTrials.gov four years after they had been completed. A study earlier this year found that 43 percent of the trials conducted at 51 academic medical centers went unpublished two years after the trial was completed.

The new requirements, which will take effect in January, clarify that all NIH-funded studies must be published on ClinicalTrials.gov. The FDA will also require publication of studies being done on drugs and devices even if they have not yet been approved. Most studies must be reported within a year of completion, but those involving unapproved products could wait up to three years.

FDA and NIH are also expanding the range of information that must be published, including the demographic data on study participants, any adverse events and the original plan for statistical analysis. The latter is meant to combat so-called p-hacking – when the original hypothesis of an experiment fails and researchers massage the data to come up with some other positive finding that is statistically significant.

“We’ve had a problem where people do the trial and don’t like the result, so they do another analysis,” Califf said.

FDA and NIH decided not to require a lay summary of trial results that would enable the public – not trained in statistics – to more easily digest the information that is posted, angering some consumer advocates.

“That is an outrageous decision,” said Diana Zuckerman, president of the National Center for Health Research. “Without it, clinicaltrials.gov will not be providing useful information to patients.” The data won’t be useful to physicians either unless it is summarized, she said.

Collins and Califf said they had been uncertain how to keep “bias” from creeping into the summaries, adding that advocacy groups should be able to harness the relevant data and make it available in a way that would be more useful to patients. […]

To see the original article, click here

Anna Edney, Bloomberg: September 13, 2016

The U.S. Food and Drug Administration has a message for doctors: The money you’re taking from pharmaceutical companies may be clouding your judgment.

Research sites where Pfizer Inc. had paid doctors at least $25,000 in speaking, consulting or other fees reported sunnier results for its smoking-cessation drug Chantix, the FDA disclosed Monday. At those sites, doctors studying the drug’s possible link to suicide risk and other behavior changes reported fewer side effects than at locations where colleagues accepted lower or no payments.

The FDA’s findings — part of an agency review of Pfizer’s proposal to drop the most severe consumer warning on the drug’s label — demonstrate the federal government’s concern about the influence of consulting and speaking fees on medical decisions. President Obama’s 2010 health law requires drug makers to report such payments for posting to a public database. The law followed years of efforts by U.S. Senator Charles Grassley, an Iowa Republican, to make drugmakers publicly disclose financial ties to doctors. […]

Diana Zuckerman, president of the National Center for Health Research, part of a coalition of consumer and other groups that petitioned to keep the warning on Chantix, said the FDA staff report shows that it doesn’t trust the “integrity of the data.”

“FDA clearly seems to be saying we can’t trust the results of this study — the way it was coded, the way it was analyzed, and by the way there’s conflicts of interest,” Zuckerman said. […]

Outside experts are scheduled to meet Wednesday to advise the FDA about the necessity of the warning on Chantix. The pill’s “black box” label — the agency’s strongest — currently cites risk of “serious neuropsychiatric events” such as suicidal thoughts or behavior. Chantix generated $671 million in sales last year,according to Pfizer. […]

To see the original article, click here

Michael D. Williamson, Bloomberg BNA: September 8, 2016

High-risk medical devices are sometimes approved using low-quality clinical data and this increases Medicare costs, a member of a Medicare congressional advisory panel said Sept. 8.

The Medicare Payment Advisory Commission’s meeting session reflected its first major examination of the medical device industry. The session may portend a deeper examination of the device industry’s practices, which could cause Congress and/or the Department of Health and Human Services to change payment rates for devices and related services, as the commission’s recommendations are fairly influential among policy makers.

Commissioner Rita Redberg, a cardiologist at the University of California San Francisco Medical Center, called for further MedPAC examination of how to push the Centers for Medicare & Medicaid Services to more broadly consider quality over an entire episode of care when deciding whether to cover a device.

Commissioners didn’t vote on recommendations. However, they debated how MedPAC staff should further research device company practices and their role in driving Medicare costs. Results of that research could prompt the commission to draft recommendations in the future.

In a separate session, commissioners discussed MedPAC staff progress on developing a uniform set of quality measures that could be used under a unified Medicare payment system for all post-acute care providers.

Tie to Rising Medicare Costs

Devices with a high risk to patients are sometimes approved by the Food and Drug Administration using poor quality data, Redberg said. This can result in low-quality devices being implanted into patients. These low-quality devices then need to be deactivated or removed, which drives up Medicare costs. […]

Redberg’s stance received praise from the leader of a patient safety group. “I completely agree with Dr. Redberg,” Diana Zuckerman, president of National Center for Health Research, told Bloomberg BNA Sept. 8. The National Center for Health Research is a nonprofit that encourages new and more effective programs and medical treatments.

In addition, the CMS should advise the FDA about the lack of data and safety and efficacy analysis specifically on patients over age 65, Zuckerman said. The lack of data “is a major problem for drugs and devices,” she told Bloomberg BNA. […]

To see the original article, click here

Diana Zuckerman, Chicago Tribune: July 21, 2016
This article was published in the Chicago Tribune, Baltimore Sun, Orlando Sentinel, Sacramento Bee, and syndicated in many other newspapers across the country.

Imagine that you or someone you love has a potentially fatal disease with no proven treatment, but there is a new experimental treatment available.

Would you rather be given that treatment for free by a top physician who carefully monitors your treatment as part of a clinical trial to study whether it works, or, would you rather pay more than $100,000 a year for the same experimental drug and hope your doctor gives you the right dose?

For more than a year, a bill misnamed the 21st Century Cures Act has been a major focus of Congress, with grand promises that it would save the lives of desperate patients, including those with rare diseases.

Despite unusual bipartisan support from Republicans and Democrats, the bill has not become law. Here’s why you should be relieved, rather than disappointed.

The 21st Century Cures Act, which some experts refer to as the 19th Century Fraud Act, is the worst threat I’ve seen to the health of adults and children, whether rich or poor.

Simply put, this proposed legislation is based on hype and hope, not reality.

It has been marketed like a Super Bowl halftime ad campaign featuring families with dying children praising members of Congress for giving them hope. Supporters have included scientists, university officials and investors who are likely to benefit financially, should the legislation become law.

Desperate patients also support it, not realizing it would pave the way for unsafe and ineffective medical treatments. Worse yet, those patients would need to pay hundreds of thousands of dollars a year while being unwitting guinea pigs in experiments that are falsely hyped as “promising treatments.”

There are thousands of patients who urgently need new medical treatments. Many feel like they have nothing to lose, and assume that a promising new treatment is their best hope. But new isn’t always better which is why too many promising new drugs are subsequently found to be a harmful waste of money.

If a patient has only one year to live and the new drug or device kills them even sooner, or causes a stroke or unrelenting nausea or horrible pain that won’t ever stop, it becomes obvious that they had something to lose after all.

That’s why these medical products were still being studied because decades of disasters such as Vioxx, metal hip replacements, DES, and thalidomide have taught us that there is a right way to test medical products. And that testing them the right way and waiting for the results to be conclusive will save more lives than taking short cuts.

One of the most dangerous aspects of the 21st Century Cures Act is that it urges the Food and Drug Administration to settle for preliminary study results based on “surrogate endpoints” which are little more than well-educated guesses that a treatment works, rather than scientific evidence of real benefits such as living longer or having fewer complications. The FDA already relies on those surrogate endpoints for new medical products more often than they should.

For example, a study published in one of America’s most prestigious medical journals found that half of new cancer drugs are approved based on such short cuts, but many of those promising results were later reversed when more conclusive studies show that the drugs do not help patients live longer.

The National Center for Health Research has found that many of those ineffective often toxic drugs cost more than $100,000 per year a cost that can bankrupt individual patients and also bankrupt Medicare.

You’d think Congress would want to stop this travesty, but campaign contributions and the power of well-connected lobbyists have convinced Congress that this bill is a good idea.

If you or a loved one has a deadly disease, whether it is cancer or a rare disease, you deserve treatments that work. If experimental drugs are your only hope, they should be free as part of a study that you can choose to participate in.

Changing the rules so that patients have to buy experimental treatments that could harm them is a dream come true for snake oil salesmen. We all deserve better.

To see original article, click here.