FDA vows to combat high drug prices, companies ‘gaming the system’

Food and Drug Administration Commissioner Scott Gottlieb said he will do everything “within my lane” to combat high drug prices and that he sees drug companies “gaming the system to try to block competition” in a multitude of ways in the marketplace.

In an interview with Kaiser Health News on Thursday, Gottlieb also said that he wants to speed up the U.S. approval process for generic and “biosimilar” versions of biologic drugs, which are drugs comprised of living organisms, such as plant or animal cells.

“Where we see things that we can address, we’re going to take action,” Gottlieb said, adding that he is most bothered when brand-name companies use tactics to block makers of generics and biosimilars from developing drugs. He deflected questions about whether the FDA approves drugs of questionable value that carry exorbitant prices.

“I think we should have a free market for how products are priced,” Gottlieb said. A free market “provides proper incentives for entrepreneurs who are going to make the big investments needed to innovate. But that system is predicated on a premise that when patents have lapsed you’ll have vigorous competition from generic drugs.”

The FDA, Gottlieb said, worked with the White House on a proposal to bring generics to market faster by ensuring that a 180-day exclusivity period isn’t used by drugmakers to block competition. He said there are “situations where you see deals cut” in which a drugmaker will get the 180-day exclusivity and then be persuaded to sit on it without ever selling the drug — essentially delaying the brand drug from facing generic competition.

Currently, generics makers must buy large quantities of the brand-name product in the U.S. to run their own clinical trials. But the companies that make brand-name medicines, in some cases, are making it very difficult for makers of generics to purchase their drugs, he said.

“They are adopting all kinds of commercial restrictions with specialty pharma distributors and wholesalers” to prevent sales to generic companies, Gottlieb said, adding that not every branded company is using the tactic, but it is “going on across the board.”

To come up with a generic, a drugmaker needs 2,000 to 5,000 doses for testing, Gottlieb said. He said the companies were willing to pay sticker price but are being blocked in other ways.

The FDA is now exploring whether generics makers could buy the drugs they need in the less-expensive European market without having to do additional work to prove the biologics from Europe are the same — even though the American and European versions are often manufactured in the same plants. Gottlieb wants to get rid of such tests, known as “bridging” studies.

“I have lawyers now looking at this,” Gottlieb said. The FDA has been exploring the issue for a couple of months, he said, and he thinks it may be “hard for us to get there without legislation, but we’re not done yet looking at this; we’re still pressing on this.”

Last fall, Gottlieb said that he wanted to “end the shenanigans” that interfere with competition in the marketplace. Since then, the FDA has released a steady stream of action plans and new guidance that tinkers with the drug development system.

“All of these steps are going to have an impact, and I don’t think there’s one silver bullet,” Gottlieb said. “If anyone [thinks] there is one thing you can do with policy intervention that is going to dramatically change drug prices, that’s not true.”

Instead, he said, there are “layers of things that we can do to try to make sure the system is working.”

The agency has been approving drugs at a fast clip: The FDA’s Center for Drug Evaluation and Research approved a record 46 new drugs in 2017, including treatments for sickle cell disease and Batten disease and new cancer therapies. The list doesn’t include landmark gene and cellular therapies and vaccines that are regulated as biologics.

That rate of approvals has raised concerns about the value and quality of drugs being approved. Specifically, criticism of the FDA’s handling of cancer drugs has increased in recent years.

Although some patient advocates want the FDA to approve new drugs more quickly, others charge that the agency greenlights mediocre cancer drugs that do little to prolong survival or improve quality of life. A 2014 study found that the cancer drugs approved from 2002 to 2014 extended survival by an average of just 2.1 months. For many cancer drugs, there is no evidence showing they prolong life.

Once drugs are on the market, companies can charge whatever the market will bear; prices for cancer therapies now routinely top $100,000 a year.

But Gottlieb said it’s not his job to help insurance companies or government programs decide which drugs to cover. Health systems and insurers “have a difficult time saying no,” Gottlieb said, “so they want to put the regulator in the position of saying no.”

Gottlieb acknowledged that it can be difficult for insurance plans to decide which drugs they should include on their drug list. But insurance plans “ought to have the confidence to make [such decisions] and not say, ‘Well, it’s the job of the federal government to make those decisions for us.'”

Gottlieb defended his agency’s approval of drugs that help the average cancer patient live just two or three extra months, noting that some patients do much better than average on cancer drugs — perhaps living months or even years longer than expected. He also said it would be wrong to make cancer patients wait years to try a drug that has a chance to help them.

“We’re ultimately going to learn why some patients respond really well and some don’t,” he said. If you “try to have all that information upfront when you approve a drug, [you’ll] end up having a development process that is very long and very costly and a lot fewer products will be developed.”

Gottlieb maintains that the FDA sets a high standard for approving drugs.

“It is important that we have a rigorous bar” for approval, he said, “but a bar that doesn’t impede these products from coming to the market.”

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Fatal cases of meningitis connected to Miami day care unrelated, state says

Two children who attended the same Miami day care center died of meningitis within days of each other in December, but state health officials now say the cases are not related.

“These two unfortunate events appear to have been caused by different bacteria and are therefore, unrelated to each other,” Dr. Reynald Jean of the Florida Department of Health wrote In a letter sent to parents Tuesday.

Both boys attended WCA Carol Glassman Donaldson Childcare Center in downtown Miami.

On Dec. 3, 22-month-old Connor Mincey died of meningitis after falling ill in late November, his family said. A week later, a 2-year-old boy died of meningitis, an attorney for his family told the Miami Herald. 

State health officials quickly confirmed that Mincey had pneumococcal meningitis, which can be transmitted person to person. At the time, officials recommended preventative measures such frequently hand washing. 

The second child died in Belize and complicated efforts for state health officials to review the autopsy. On Feb. 7, state officials reviewed the report and determined that the boy died of pseudomonas aeruginosa meningitis, which is not typically spread person to person.

The Florida Department of Children and Families conducted a routine inspection Dec. 1 at the day care, just two days before Connor died. 

Among the multiple violations noted in DCF’s report were that the day care had no quarantine room for sick children.

The day care agreed to close shortly after the two deaths and said they would not to reopen the center until they were cleared by the health department and DCF.

The day care reopened Dec. 26 and the investigation into the center closed on Jan. 31.

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‘I looked at it, and it was moving’: Worm in woman’s eye leads to unique discovery

Imagine looking into your irritated eye for a pesky eyelash, only to pull out a translucent, wiggling worm nearly a half inch long.

“I looked at it, and it was moving,” recalled 28-year-old Abby Beckley of Grants Pass, Oregon. “And then it died within about five seconds.”

Now, imagine doing that not once but 14 times.

That’s what Beckley endured over a three-week period in August 2016. Her story, published Monday as a case report by the US Centers for Disease Control and Prevention, is a historic one:

“This is only the 11th time a person has been infected by eye worms in North America, ” explained lead author Richard Bradbury, who is the team lead for the CDC’s Parasite Diagnostics and Biology Laboratory. “But what was really exciting it that it is a new species that has never infected people before. It’s a cattle worm that somehow jumped into a human.”

A summer adventure

Growing up on a ranch in Brookings, Oregon, surrounded by cattle and horses, Beckley loved the outdoors. She also had a burning desire to travel. So, in July 2016, she jumped at a chance to combine the two by working on a commercial salmon fishing boat in Craig, Alaska. It was only a couple of weeks into the job that the symptoms started.

“My left eye just got really irritated and red, and my eyelid was droopy,” Beckley remembered. “I was getting migraines too, and I was like, ‘What is going on?’ “

She’d been suffering for five days when the ship finally returned to port. Beckley found a good mirror and looked closely into her eye, never expecting what she would find.

“I pulled down the bottom of my eye and noticed that my skin looked weird there,” Beckley said. “So I put my fingers in with a sort of a plucking motion, and a worm came out!

“I was just in shock,” she said. “I ran into my crewmate Allison’s room, and I said, ‘I need you to see this! I just pulled a worm out of my eye!’ “

Believing it to be a salmon worm, the women feverishly searched for similar cases on the internet but could find nothing. Visits to a local doctor and ophthalmologist also proved fruitless.

“They said they had never seen anything like this,” Beckley said, adding that during that time, she pulled another four worms from her eye. “And then I could see them moving across my eye at that point, too. There were so many.”

Worried family and friends encouraged her to return home and set up an appointment at Oregon Health and Science University in Portland. She went directly there from the airport.

“There were several doctors examining my eye, and at first, they were a bit skeptical, because who comes in and claims they have a worm in their eye?” Beckley remembered. “I am thinking to myself, ‘Worms, please show up,’ because sometimes they would go behind my eye and under the eyelid, and you couldn’t see or feel them anymore.”

Luckily, she says, after a half-hour, the worms made an appearance.

“I felt one squiggle across my eye, and I told the doctors, ‘You need to look right now!’ ” Beckley said. “I’ll never forget the expression on their faces as they saw it move across my eye.”

‘I tried not to go to the darkest place’

While some of the worms Beckley removed were sent off to the CDC for identification, she frequently visited the university for vision tests and eye washes designed to flush out additional worms. Although her vision remained fine, the flushes were unsuccessful.

“I just kept pulling the worms out of my eye at home, but when I went to the office, they would flush, and nothing would come out,” Beckley said. “They were trying to figure out what to do because there was no road map, no protocol for this.”

The worst part, she says, was wondering what the worms might do to her body, “so close to my brain and eyes.”

“I tried not go to the darkest place, like, are these worms going to paralyze my face or infect my brain or impact my vision?” she said. When a doctor explained that the worms would remain on the surface of her eye, she calmed down.

“I was definitely in distress, for sure, but I also started making jokes, because I had to, to deal with it,” Beckley said. “It’s so gross to think about, but it was happening to me.”

‘Fascinating ecological niche’

Parasitic eye worms are common among dogs, cats, pigs, sheep, goats, cattle and wild carnivores like foxes and wolves. The larvae are transmitted by female “face flies” that feed on the animal’s eye secretions.

“Tears are full of proteins of various kinds, so the flies get a lot of nourishment from those tears,” explained Dr. William Schaffner, a professor of medicine at Vanderbilt University’s Division of Infectious Diseases, who was not involved in Beckley’s case. “For a scientist, it’s a fascinating ecological niche.”

The worm larvae grow into adulthood and reproduce between the eye and the eyelid. Their offspring leave the host’s body via more secretions from the inflamed eye, which the flies ingest, completing the life cycle.

“The early-stage larvae need to go through the fly’s digestive system to be able to develop to a more advanced stage to infect another host,” Bradbury explained. “It’s a complicated life cycle.”

Veterinarians treat the infection in pets and livestock with the anti-parasitic drug ivermectin. But in untreated animals, Bradbury says, the worms can live and reproduce up to 30 months, leading to vision loss or even blindness.

People infected by the parasite typically don’t suffer that fate, because, like Beckley, they can remove the worms from their eyes.

Unraveling a medical mystery

When the worms from Beckley’s eye arrived at the CDC’s diagnostic lab, scientists were expecting to find a species of the Thelazia parasite called californiensis. That’s what infected the eyes of the 10 cases found in the US: nine from California and one from Utah.

“It’s an eye worm that often infects dogs and very occasionally affects humans,” the CDC’s Bradbury explained. “Another type of eye worm called callipaeda, found in Asia and Europe, has also infected people, but it’s also rare, with only about 163 reported cases in the world.”

Most cases of human infection around the world occur in poor, rural communities among the very young and elderly, who may be less able to keep flies from their face. But not always. In 2015, a 21-year-old South Korean soldier developed eye worms from the Asian strain, callipaeda, after multiple brief contacts with a dog in his father’s factory.

“He recalled nothing abnormal about the dog,” said Kyungmin Huh, a South Korean doctor who wrote about the case in the New England Journal of Medicine. “But I should note that previous reports show that patients cannot remember how it was transmitted in the majority of cases.”

Beckley has no memory of any fly landing close to or in her eye.

“It makes me curious if there was someone else who had this happen but wasn’t seen by a doctor,” she mused. “The only reason that I knew the cause is that I physically pulled one out of my eye.”

Schaffner agreed: “Dollars to doughnuts, there were people in the past that had these infections but were never specifically diagnosed. Here, we have someone who developed this unusual infection, and the physicians were interested enough to send the materials to the CDC, where they have extraordinary diagnostic abilities.”

Without that expertise, says Schaffner, investigators may have never noticed the small differences in the anatomy of the worms from Beckley’s eye.

“Something was strange about it,” Bradbury said, “and we had to go digging to find out what it was. I finally found the microscopic pictures I needed to find the exact species in a paper written in German in 1928.”

Bradbury says the species, Thelazia gulosa, is unique to cattle and has never before been seen in a human eye. That means Beckley was infected by cattle near her home, before she left for Alaska.

“It’s possible that there are cases that were misdiagnosed as another species of the worm, californiensis, because people just assume that it will be,” Bradbury said. “But through our work, we were able to understand that a brand-new species can now infect people who are around cattle.”

The end of a nightmare

Beckley was not treated with anti-parasitic medicine because doctors were worried that a dead worm might remain in her eye, possibly causing scarring. Instead, she was told to continue to monitor her eyes and remove any worms she found. How did she handle the uncertainty?

“You can go into ‘Poor me, Oh, my God, I’m going to let this destroy me,’ or you can just think, ‘OK, these are worms, and now I know the life cycle, and I know that they will die, and they are just sharing space,’ ” she said. “Doesn’t mean I wasn’t grossed out! It doesn’t mean I wasn’t angry! But I would try to self-soothe and put it in perspective.”

Twenty days after pulling the first worm from her eye, Beckley discovered the final wiggling worm. Once that was out, her ordeal was over. She knows because she’s not found another since. Her vision remains good, with no other complications.

But why go public with her story?

“Part of the reason I’m speaking out is that I had wished I could find one article or source that would reassure me this happened to someone else and they are fine,” Beckley said. “If this does happen again, I’m hoping my story will be out there for the next person to find.”

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How Trump plans to cut prescription drug prices

President Donald Trump finally explained how he plans to fulfill his promise of lowering prescription drug prices — at least in part.

On Monday, his administration released its 2019 budget blueprint, which proposed changes to payment policies in Medicare and Medicaid and fast-tracking the development of generic drugs as ways to help accomplish that goal.

“In particular, the budget’s efforts to reduce the high cost of prescription drugs, especially for America’s seniors, are a reflection of President Trump’s deep commitment to addressing this important issue,” said Health Secretary Alex Azar.

The budget calls for giving up to five states greater leeway to test drug coverage and payment models in their Medicaid programs. These states would be allowed to determine which drugs would be covered, known as a formulary, which would in theory let them negotiate bigger discounts directly with manufacturers.

Currently, any medications a manufacturer includes in the federal Medicaid drug rebate program are automatically on the formulary. Some states negotiate supplemental discounts for certain drugs. But if states could decide which drugs were covered, they could theoretically obtain even lower prices.

The budget also recommends changes to Medicare to lower drug prices for senior citizens. The administration would require insurers and pharmacy benefit managers to share with Medicare enrollees more of the discounts they receive from drug manufacturers in the Part D drug program. Also, it would give insurers more negotiating power by requiring them to only cover one drug per category, rather than two.

In addition, it would eliminate cost-sharing for generic drugs for low-income seniors and create a new out-of-pocket spending cap.

One proposal that’s not in the budget is allowing the federal government to negotiate Medicare drug prices directly. Many health policy experts and consumer advocates say that the federal government could use its heft to negotiate much lower prices for the more than 57 million Americans in the program, rather than having insurers that provide Medicare Part D prescription coverage wring discounts from manufacturers for their enrollees. Drug companies, however, have lobbied hard to prevent government involvement, and Republicans have generally not supported such a proposal.

The budget would also adjust payments for Part B drugs, which are administered at a doctor’s office or hospital outpatient clinic, to discourage manufacturers from raising prices faster than inflation. Plus it would modify the 340B program — in which hospitals with a lot of low-income patients get deeply discounted drugs from manufacturers — to reward hospitals that provide more charity care.

The administration also wants to speed the development of generic drugs to enhance competition. It would make sure that generic manufacturers do not try to block their peers from entering the market.

The drug price proposals in the budget come just days after Trump’s Council of Economic Advisers released a report on how to reduce pharmaceutical costs while stimulating innovation. That report blamed high prices in part on foreign countries that control drug prices, thereby taking advantage of American innovation without paying for it. The council suggested changing trade policy to address this problem.

It also contained several broad suggestions for enhancing competition in the generic market and curbing prices in the U.S., including some also contained in the budget blueprint.

The administration’s proposals to reduce drug prices were met with skepticism by some consumer advocacy groups. They say the suggestions do nothing to lower the actual prices of the medications — instead, they just shift the burden of who has to pay for it, said Will Holley, spokesman for Campaign for Sustainable Rx Pricing, a coalition of health care providers, consumers, insurers, pharmacy benefit managers and others.

“None of these proposals get at the price,” Holley said.

Also, the budget doesn’t deal with ensuring brand-name manufacturers don’t interfere with the creation of generic versions of their products, Holley said.

The main lobbying group for pharmaceutical companies, Phrma, did not immediately return a request for comment.

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Broward County doctor accused of running pill mill out of office

A Broward County doctor and three of his employees were arrested last week after federal agents raided his medical office, the U.S. Department of Justice announced Monday.Dr. Andres Mencia, 64, of Fort Lauderdale, and his associates are accused of bein…

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Purdue Pharma says it will stop promoting OxyContin to doctors

More than a decade after Purdue Pharma was first criticized by the federal government for its “aggressive” marketing of the addictive painkiller OxyContin, the company says it will stop promoting the opioid to doctors.

The company told CNN on Sunday that it has cut its sales force in half to 200 representatives and will turn its focus to marketing non-opioid drugs. The news was reported earlier by Bloomberg.

Purdue will continue to sell OxyContin, but sales reps will no longer visit doctors’ offices to promote the drug.

For the past couple of years Purdue has routinely directed doctors to the CDC’s “Guideline for Prescribing Opioids for Chronic Pain,” the company said.

Opioids, a class of pharmaceuticals that include prescription pain killers like OxyContin as well as illicit drugs like heroin and fentanyl, are at the root of an ongoing public health crisis in America.

In 2016, there were 42,249 opioid-linked drug fatalities in the U.S. — more than the number of deaths linked to breast cancer, according to the Centers for Disease Control and Prevention.

The opioid crisis has raised significant concern about prescription painkillers. Between 1999 and 2009, overdoses from such drugs rose about 13% annually, though the increase has since slowed to 3% per year.

Sales of OxyContin, which is a long-acting version of the drug oxycodone that was designed to deliver medicine over 12 hours, grew rapidly after it hit the market in 1996.

Reports about OxyContin abuse began to surface by early 2000, according to a 2003 government report.

The Drug Enforcement Administration “expressed concern that Purdue’s aggressive marketing of OxyContin focused on promoting the drug to treat a wide range of conditions to physicians who may not have been adequately trained in pain management,” the report states.

Purdue collaborated with the Food and Drug Administration on a “risk management plan” aimed at preventing abuse of the drug, according to the report.

Then, in 2007, the federal government brought criminal charges against Purdue for misleadingly advertising OxyContin as less addictive than other opioids. Purdue and three executives pleaded guilty and agreed to pay $634.5 million in civil and criminal fines.

Purdue says it has since been involved in various measures to curb opioid addiction. In 2010, the firm released a new version of OxyContin that is more difficult to crush — and therefore more difficult to abuse by snorting or injecting it.

— Nadia Kounang and Sonia Moghe contributed to this report.

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