Tagged FDA

FDA Approves Scope With Disposable Part Aimed At Reducing Superbug Infections

Seeking to prevent superbug outbreaks, federal health officials said they have approved the first gastrointestinal medical scope with a disposable cap for use in the U.S.

The Food and Drug Administration said that the design of the new duodenoscope by Japanese device maker Pentax should make it easier to remove dangerous bacteria that can become trapped inside these reusable instruments.

“We believe the new disposable distal cap represents a major step toward lowering the risk of future infections associated with these devices,” said Dr. William Maisel, the acting director of the FDA’s Office of Device Evaluation, in a statement Wednesday. “We encourage companies to continue to pursue innovations that will help reduce the risk to patients.”

But some medical experts questioned whether this design change goes far enough to protect patients.

“This new scope will probably reduce the infection risk, but I’m not sure by how much,” said Lawrence Muscarella, a hospital-safety consultant in Montgomeryville, Pa.

In addition, he and other experts said, this is just one scope, and its modest redesign will not address the shortcomings that plague many different medical scopes on the market and continue to put patients at risk of antibiotic-resistant infections.

Pentax didn’t provide any details Thursday about when the new duodenoscope will be available or what it may cost. In general, duodenoscopes can cost up to $40,000 apiece, and they can represent a major expense for hospitals that handle a large volume of procedures.

In a statement, the company said “the disposable distal cap design represents a significant advancement in infection control.”

One advantage of a removable cap is that it would allow hospital cleaning staff better access to tiny crevices and small parts at the tip of the scope. Some areas are hard to reach with brushes and washing machines, increasing the risk of bacteria being passed from one patient to another.

While the scope tip has proven troublesome, experts say harmful bacteria also have been found in other areas, such as biopsy ports and instrument channels.

“I think it may be a step in the right direction to have single-use components whenever possible. But we still have gaps here,” said Cori Ofstead, a researcher and epidemiologist in St. Paul, Minn.

She and other infection-control experts are urging manufacturers and regulators to move toward sterilization for all medical scopes, which would involve gas or chemicals and be a step above the current federal requirement for high-level disinfection. That change would likely require further design changes to enable complex scopes to withstand the process.

Other device manufacturers have gone in a different direction, developing scopes that are fully disposable, rather than just enabling the cap to be thrown away. The FDA recently approved two colonoscopes that are designed to be used just once. Other companies are promoting similar devices for use in the lungs and kidneys.

Doctors put duodenoscopes down a patient’s throat to diagnose and treat problems in the digestive tract, such as gallstones, cancers and blockages in the bile duct. There are about 700,000 of these ERCP procedures done annually in the U.S.

Since 2015, U.S. prosecutors, lawmakers and government regulators have been investigating dozens of infections and deaths tied to duodenoscopes.

The Los Angeles Times broke the news about a superbug outbreak at the Ronald Reagan UCLA Medical Center in February 2015, which triggered an FDA warning the next day. Those infections involved the industry’s leading scope maker, Tokyo-based Olympus Corp. The newspaper later reported that Olympus knew about infections and potential flaws with its duodenoscope as early as 2012 but failed to alert American hospitals or regulators.

Last year, the FDA said that as many as 350 patients at 41 medical facilities in the U.S. and worldwide were infected or exposed to tainted duodenoscopes from January 2010 to October 2015.

A U.S. Senate report in 2016 identified four outbreaks tied to Pentax duodenoscopes at hospitals and clinics in Illinois and Massachusetts.

More recently, in January, the FDA issued a safety alert about Pentax’s current duodenoscope model. The agency warned medical providers that cracks and gaps can develop in the device’s tip that “could allow fluids and tissue to leak into the duodenoscope.” The FDA urged hospitals to immediately remove scopes from service that showed signs of damage.

Muscarella, the hospital-safety consultant, said it was surprising the FDA “didn’t require removal of the product from the market considering the risk of superbug infections. It would seem Pentax was prodded by the FDA to come up with another solution, and this disposable cap design might be the company’s response.”

In a statement last year, Pentax said, “We are dedicated to patient safety and are consistently evaluating advancements in flexible endoscope design.”

A number of recent studies have found evidence of persistent contamination in a wide range of reusable devices, including colonoscopes and ureteroscopes.

This story was produced by Kaiser Health News, which publishes California Healthline, an editorially independent service of the California Health Care Foundation.

Categories: California Healthline, Cost and Quality, Health Industry

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FDA Moves To Guard Against Abuse Of ‘Orphan Drug’ Program

The Food and Drug Administration is changing the way it approves medicines known as “orphan drugs” after revelations that drugmakers may be abusing a law intended to help patients with rare diseases.

In a blog post Tuesday, FDA Commissioner Scott Gottlieb said he wants to ensure financial incentives are granted “in a way that’s consistent with the manner Congress intended” when the Orphan Drug Act was passed in 1983. That legislation gave drugmakers a package of incentives, including tax credits, user fee waivers and seven years of market exclusivity if they developed medicines for rare diseases.

A KHN investigation earlier this year, which was published and aired by NPR, found many drugs that now have orphan status aren’t entirely new. Of about 450 drugs that have won orphan approval since 1983, more than 70 were drugs first approved by the FDA for mass-market use. Those include cholesterol blockbuster Crestor, Abilify for psychiatric disorders and rheumatoid arthritis drug Humira, the world’s best-selling drug.

Gottlieb announced plans to close a loophole that allows manufacturers to skip pediatric testing requirements when developing a common-disease drug for orphan use in children. He also signaled that bigger changes are being considered, announcing a public meeting to explore issues raised by scientific advances, such as the increase in precision medicine and biologics.

“We need to make sure our policies take notice of all of these new challenges and opportunities,” he wrote. Gottlieb, through his agency, declined multiple requests for interviews.

Over the years, drugmakers have fueled a boom in orphan drugs, which often carry six-figure price tags. Nearly half of the new drugs approved by the FDA are now for rare diseases — even though many of them also treat and are marketed for common diseases.

Gottlieb became commissioner in May, a few months after three key Republican senators called for a federal investigation into potential abuses of the Orphan Drug Act, and the Government Accountability Office agreed to investigate.

The GAO has yet to begin its investigation, saying it doesn’t expect to start work until late this year, when staff is available. Regardless, in late June, Gottlieb announced what would be the first in a series of updates that shift the way the FDA handles orphan drugs.

Those include:

  • Eliminating a backlog in drug applications for orphan designation or status. Getting a “designation” is a critical first step if a company wants to win orphan incentives once the drug is approved for treatment use. And, much like the rise in approvals, the requests by companies to get drugs designated with orphan status has also skyrocketed. Gottlieb said in June that he wanted to get rid of the backlog; on Tuesday, he said the effort was complete. About half of the 200 applications from drugmakers won orphan status.
  • Mandating that drugmakers prove their medicine is clinically superior before getting the market exclusivity that comes with being an orphan. The agency had lost a lawsuit in which a company said it was owed the exclusivity period regardless of whether its medicine was better. And two more lawsuits had been filed by Eagle Pharmaceuticals and, more recently, another by United Therapeutics. The FDA Reauthorization Act, which passed last month, made it law that a drug has to be clinically superior to get the incentives.
  • Closing the loophole for pediatric orphan drugs by requiring all drugs approved for common adult diseases, like inflammatory bowel disease, undergo pediatric testing when getting approval as a pediatric orphan drug. Pediatric testing is not required for orphan drugs, and last month Congress mandated that orphan drugs for cancer be tested for children. Still, the American Academy of Pediatrics celebrated the proposed change but warned it was only a “first step.” Dr. Bridgette Jones, chair of American Academy of Pediatrics Committee on Drugs, said late Tuesday that orphan drugs are “still mostly exempt from pediatric study requirements … children deserve access to safe, effective medications.”

Dr. Martin Makary, who wrote a critical 2015 paper on orphan approvals, said the changes at the agency indicate that Gottlieb seems “concerned about all the right things.”

“The government does a lot of lip service in general,” Makary said. “This is not lip service.”

The restructuring has been swift in some ways.

Sandra Heibel, a senior consultant at Haffner Associates, a firm that helps companies submit orphan drug applications, noted that the approval process for designations definitely sped up over the summer, and “we are absolutely getting responses from the FDA back in 90 days. That has come through.”

Other changes to the agency, though, will evolve slowly. For example, the orphan drug office has begun reaching across the FDA’s divisions for help in reviewing drugs. In May, the FDA’s orphan reviews began to work with the office of pediatric therapeutics to review pediatric applications — ideally increasing the expertise applied when considering a company’s request for orphan drug use in children.

In an emailed note Tuesday, the agency confirmed that Gottlieb’s orphan modernization plan is part of a larger effort to increase competition and decrease drug prices. One focus is on targeted drugs — especially those that affect rare diseases or diseases for which there is no effective therapy, the agency said.

“Such drugs present some of the biggest opportunities in medicine to treat and cure debilitating and very costly diseases,” the agency stated.

KHN’s coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation.

Categories: Cost and Quality, Health Industry, Pharmaceuticals

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