Diana Zuckerman, PhD, Spectrum: December 1, 2016

Why would anyone vote against “cures,” especially “21^st century cures?” That question is the key to understanding how the U.S. House of Representatives, whose members usually can’t agree on anything, overwhelmingly passed a 996-page health bill yesterday — just a few days after the bill, the 21^st Century Cures Act, was written behind closed doors.

Here’s why many health policy and consumer advocacy groups — including the National Center for Health Research, where I work — strongly oppose the bill and are asking senators not to pass the bill next week: On the bright side, the bill promises more than $6 billion dollars over the next few years for medical research and to fight the opioid epidemic. On the other hand, the ‘promise’ of that money does not include anything resembling a guarantee that the money will be provided for that purpose.

If the bill passes, those 996 pages of mostly unintelligible legislative language will influence important issues that affect all of us. Most importantly, the bill instructs the U.S. Food and Drug Administration (FDA) to help drug and device companies get their products on the market more quickly. Unfortunately, it does that by loosening and lowering the very scientific standards that have made FDA approval the gold standard for countries around the world.

A Perfect Storm of Lobbying:

More than 1,450 lobbyists pushed to get this bill passed, including many patient groups and others funded by the companies that make prescription drugs and medical devices. Universities and medical schools also lobbied for the bill because they want more funding for medical research.

But physician groups concerned about patient safety, such as the National Physicians Alliance, the American Medical Women’s Association and the American Medical Student Association, oppose the bill. Dozens of nonprofit groups such as the Consumer Reports Safe Patient Project and National Consumers League also strongly oppose the bill. In one surprising twist, some of the same HIV/AIDS activists who have criticized the FDA in years past for being too rigid are lobbying against the bill because it doesn’t do enough to make sure new treatments are safe and effective.

None of these groups oppose all aspects of the bill. They just want Congress to take the time to fix it. That would mean waiting until next year instead of rushing through a bill that nobody has time to read.

The FDA’s job is to review new medical products to make sure they are safe and effective. ‘Safe’ doesn’t offer a 100 percent guarantee: Any treatment can harm some people; if the benefits are substantial enough, the FDA may even approve a treatment that can be lethal.

But on balance, the treatment’s benefits must outweigh the risks for most people. The company that makes a treatment provides all of the studies and information about it to the FDA, and scientists there review that information. For prescription drugs, that review usually entails the FDA scientists scrutinizing data from clinical trials that compare people taking the new drug with those taking a placebo or a different treatment, such as behavioral therapy.

Lower Standards:

Traditionally, the goal of clinical trials is to see if a treatment improves the outcome for the participants. Depending on the condition being treated, better can mean living longer (for cancer, for example) or losing a few pounds (for a weight-loss drug) or being more able to enjoy life (for many psychotropic drugs and devices).

However, the new bill wants the FDA to make it easier for companies to prove a ‘benefit,’ by relying on ‘surrogate markers’ of effectiveness. Surrogate markers don’t measure benefits directly, but rather a secondary trait that is believed to predict the benefits. For diabetes, for example, blood glucose level is the surrogate endpoint, but the treatment’s goal is to avoid amputation or extend life. Better glucose levels don’t always predict those health outcomes.

Cancer drugs are a perfect example. Last December, researchers reported that the FDA approved 36 of 54 new cancer drugs more quickly by basing approval on surrogate markers such as tumor shrinkage, rather than on survival benefit¹. Years after approval, only 5 of the 36 drugs had been proven to help people live longer, 18 definitely did not, and the manufacturers of the other 13 had not made the results of their studies publicly available (a sign that the drugs probably don’t work).

Our center looked at those same 18 ineffective cancer drugs and found that only 1 improves quality of life, 15 have no proven impact on quality of life and 2 makes quality of life worse. The most expensive of these drugs, costing $170,000 per person, is one of these last two.

Spiraling Costs:

The standards for medical devices, including implants and devices that send electromagnetic pulses to the brain, are even lower than for cancer drugs. Device companies are rarely required to conduct clinical trials to prove their product is safe or effective. More than 90 percent of the time, all they need to do is explain to the FDA that their device is similar to another device already on the market.

Even when device studies are required, the companies rarely use a placebo group as a comparison to the new device. Wishful thinking can make almost any new treatment seem effective at first. That’s why so many people pay so much for treatments that don’t seem to do any good at all.

The new bill would make that situation worse, by pressuring the FDA to make it even easier for new, unproven treatments to be sold in the United States. People might end up paying billions of dollars for a wide range of treatments that were approved based on shorter studies of fewer participants and surrogate endpoints. That would increase the already-spiraling cost of insurance coverage and challenge the financial stability of Medicaid and Medicare even more than is the case today.

Don’t be distracted by the false promises of funds for medical research. The reality is that there is too much in this bill that would dismantle the structures that help physicians make informed decisions and keep us all safe.

Read original post here.

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