Tag Archives: fda approval

How often do drugs obtaining accelerated approval fulfill their potential?

Tara Haelle

About Tara Haelle

Tara Haelle (@TaraHaelle) is AHCJ's medical studies core topic leader, guiding journalists through the jargon-filled shorthand of science and research and enabling them to translate the evidence into accurate information.

Image: Seattle Genetics

If a new drug appears to show remarkable success in curing patients with a specific, aggressive cancer type that has few or no other effective treatments, there can be a compelling reason to get it on the market as soon as possible.

However, evidence requirements for drugs to receive accelerated FDA approval often rely on surrogate endpoints. That’s particularly true for conditions such as cancer where the ideal primary endpoint — survival — isn’t possible to assess in a short period. Continue reading

Potential changes in regulation of medical devices would likely impact health care costs

Andrew Van Dam

About Andrew Van Dam

Andrew Van Dam of The Wall Street Journal previously worked at the AHCJ offices while earning his master’s degree at the Missouri School of Journalism.

As the medical device industry ramps up its campaign against further government regulation, Merrill Goozner takes stock of the regulatory and business environment in that arena and explains what is at stake. The key battleground at present is possible modifications to the 1976 law which allows devices to bypass some rigorous testing as long as they’re similar to something that has already been approved for market. The problem? That similarity doesn’t always mean they’re safe, as Goozner points out.

glucose-testing

Photo by AlishaV via Flickr

A study published earlier this year in Archives of Internal Medicine found that of 113 major product recalls between 2005 and 2009, only 19 percent had gone through the more rigorous clinical trial testing required for new products, while 71 percent had used the follow-on process. There had been only 49 major recalls in the prior five years.

Despite slipping onto market through the similarity provision, many of these new products claim to be improvements over their predecessors and thus come with commensurately higher price tags. According to Goozner and his sources, this little disconnect has done quite a bit to increase the cost of health care in America.

“Requiring evidence of benefit of effectiveness for patients before device approval would prevent billions of dollars from being spent on technologies that are not helpful for patients and are even harmful,” said Rita Redberg, editor of the Archives of Internal Medicine and a cardiologist at the University of California, San Francisco. “There are many examples, such as vertebroplasty and kyphoplasty for back pain [compression fractures], on which Medicare spends approximately $1 billion annually. After they were FDA-approved, randomized clinical trials showed they were no more effective than a sham procedure in relieving symptoms.”

The device industry, often cited for its red-hot growth rates in the past, now posts numbers that, while huge, still lag behind the health sector at large. That may be why the industry is stepping up political pressure to reduce its regulatory burden and to sidestep a 2.3 percent excise tax that was passed as part of recent health care reform efforts. For more on the money and politics involved, see Goozner’s full piece, which was also published in The Fiscal Times.

Related

Medical device investigation unearths conflicts, regulatory issues

Andrew Van Dam

About Andrew Van Dam

Andrew Van Dam of The Wall Street Journal previously worked at the AHCJ offices while earning his master’s degree at the Missouri School of Journalism.

The latest investigation from the Chicago Tribune’s Jason Grotto and Deborah L. Shelton focuses on a single medical device, yet still hits many of the health beat’s biggest narratives, primarily conflicts of interest and government regulation. In their centerpiece, they dig into the tale of a cardiologist who may or may not have tested a device he invented on patients without consent or regulatory approval. It’s in those gray areas that the story takes shape.

The device in question is an annuloplasty ring, which in 2001 was reclassified from class III to class II. In practical medical device terms, this means that new models don’t need clinical trials if they’re similar enough to devices that have already been approved. The reporters write that the rings earned this change of status in part because of the relatively low number of adverse events reported between 1991 and 1995 (465). Unfortunately, as their use has increased, so have the events.

Carpentier-McCarthy-Adams IMR ETlogix annuloplasty ring

Now that you have some idea of what they were up against, here’s a healthy excerpt from their “how we did it” sidebar, which is almost always Covering Health’s favorite part of a major investigative package.

The first hurdle in stories like these is understanding the medical science behind the device in question, which required us to gather up dozens of studies and reports on annuloplasty rings and the heart valve disease the device is intended to cure. We sifted through government documents and researched regulations as well as the history of the rings.

But writing about heart valve repair rings involves more than researching a disease and a device.

We also spent a lot of time with the patients in our stories because there is a level of trust that must be built up before people are willing to share personal information about their health. The same goes for Northwestern Memorial Hospital and surgeon, Dr. Patrick McCarthy.

We had to assure each of them that their viewpoint would be reflected in the stories. It’s a difficult balancing act that we tried to buttress with reams of documents, studies and interviews with independent sources.

For more on how Grotto and Shelton reported the story, watch for a “How we did it” article on the AHCJ website in the next couple of weeks.

If the issue sounds familiar, the storyline is similar to a 2005 investigation from The Wall Street Journal‘s David Armstrong, which found a partnership between the Cleveland Clinic – and some prominent staff members – and companies manufacturing devices used in clinical trials there.

Skyrocketing drug price is chance to examine manufacturing, FDA approval process

Andrew Van Dam

About Andrew Van Dam

Andrew Van Dam of The Wall Street Journal previously worked at the AHCJ offices while earning his master’s degree at the Missouri School of Journalism.

St. Louis Post-Dispatch reporter (and Midwest Health Journalism Program Fellow) Jim Doyle has put together a series of stories on KV Pharmaceuticals that read like a primer on the confounding economics of drug manufacture and FDA approval. In the stories, especially the first and last installments, Doyle presents the big picture and helps readers understand why the systems work they way they do.

The first story shows how FDA approval could end up sending the price of a prenatal drug skyrocketing 16-fold and earn piles of money for a local pharmaceutical company. The second involves a U.S. District judge condemning a former head of that same company for “greed, abuse of power, recklessness.” Finally, he ends his tour of pharmaceutical avarice with a stern warning about the potential longterm costs, both monetary and medical, that could result from the fast-track approval of the drug whose approval formed the basis of the first story. For lots more about KV Pharmaceuticals, be sure to check the “Related Reading” box on this page.

Further reading

If you’re looking for more on KV Pharmaceuticals and the Orphan Drug Act, check out Ed Silverman’s post on Pharmalot. There, he interviews a nonprofit advocate who helps explain how KV’s manipulations were possible, how it could happen again and how the act should be modified.

Fauber: FDA to review data on jaw implants

Andrew Van Dam

About Andrew Van Dam

Andrew Van Dam of The Wall Street Journal previously worked at the AHCJ offices while earning his master’s degree at the Missouri School of Journalism.

The Milwaukee Journal Sentinel‘s reporter John Fauber reports on the FDA’s order to review the long-term performance of certain joint implants. Fauber’s story on the agency’s failure to adequately test and regulate these products came out in late November.

lower-jaw

Photo by Jacob Enos via Flickr

The implants in question treat certain ailments affecting the joint that connects jaw and head, a disorder Fauber says “affects more than 10 million Americans, including a small number who undergo radical surgery to implant an artificial jaw joint.” And those people, Fauber reported and the FDA now acknowledges, have had a rough time in the long term.

Among the medical device reports it has reviewed, 52% of the devices that had to be removed were taken out less than three years after they were implanted, the agency said in an e-mail to the Journal Sentinel.
Device problems included the need for removal or replacement, loosening, difficulty removing, noise, fracture and breaking, the FDA said.
Patient problems included pain, surgical procedure or repeat surgical procedure, additional therapy and nonsurgical treatment or treatment with medication, infection, swelling, hospitalization and headache, it said.

At this point, the FDA isn’t talking about taking the devices off the market, but it is reviewing the existing data and beefing up its repeated requests that device makers supply long-term market data about the implants they’ve sold.

Beginning in 1999, the FDA approved four such devices made by three companies. At the time of approval it ordered the firms to collect the market data it now is reordering.
That data was inadequate, the agency said, noting that it did not show why or how soon the devices were being replaced. In addition, the companies had lost contact with many of the patients who had received the devices.
Without knowing that information, the agency cannot determine the true safety and effectiveness of the products or whether any of them should be removed from the market.

Diana Zuckerman, president of the National Research Center for Women & Families, credited Fauber’s reports in the Journal Sentinel and MedPage Today, a medical news website that partners with the newspaper, with prompting the FDA’s action.

Flawed approval process led to flawed jaw implants

Andrew Van Dam

About Andrew Van Dam

Andrew Van Dam of The Wall Street Journal previously worked at the AHCJ offices while earning his master’s degree at the Missouri School of Journalism.

In the Milwaukee Journal Sentinel, reporter John Fauber proves that it’s possible to do cutting-edge reporting on conflicts of interest and tie those conflicts to clinical trials of devices that hit the market a few decades ago.

His targets? Jaw joint devices that were initially grandfathered in, and for which newer treatments were approved in the late ’90s and early 2000’s. These trials, Fauber found, were too small, too conflicted and too inconclusive to provide real data, yet the devices were approved anyway. Now, he has found, patients are paying the price.

I’ll just include the first sentence here, as I’m confident that nobody will be able to resist reading the rest of Fauber’s story.

Before implanting a third artificial jaw joint in Heidi Clark’s head, doctors had to remove particles of plastic from the second failed joint that had broken apart and become embedded in muscle.