Tagged Drug Costs

5 Reasons Why An $89K Drug Has Congress Fuming

The latest flashpoint in the ongoing debate over high drug prices is Emflaza, an $89,000-a-year drug that treats Duchenne muscular dystrophy.

People who have been watching the drug price issue closely, however, can reasonably ask why there is so much heat at that price tag? Late last year, two drugs went on the market for six-figure prices. Exondys 51 sells for $300,000 a year and Spinraza for a whopping $750,000.

While they did draw headlines, neither of those drugs sparked the bipartisan congressional firestorm and patient outcry that Emflaza ignited this week. Here are five reasons why:

1. It’s not a new drug.

Emflaza, the brand name for deflazacort, has never been approved for sale in the United States, but the steroid has been sold for decades in other countries — at much lower prices.

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Some Duchenne patients in the U.S. have imported deflazacort from Europe and Canada for years for just $1,000 to $1,600 annually. Mohammed Haider, 27, of Mount Laurel, N.J., said he’s been buying it from European pharmacies since he was seven years old.

In medical circles, deflazacort is often compared with the widely used steroid, prednisone, which has been around since 1955.

2. It’s not a scientific breakthrough.

Exondys 51 and Spinraza are medical breakthroughs that target genes to treat underlying diseases.

Emflaza, on the other hand, addresses symptoms of muscle weakness and deterioration by decreasing inflammation and suppressing the immune system in Duchenne patients.

Dr. Aaron Kesselheim, an associate professor of medicine at Harvard Medical School, questions why Marathon won approval using the Orphan Drug Act, which was created by Congress to motivate companies to develop drugs to treat rare diseases.

Instead, Kesselheim said, this is a steroid that could be used more broadly.

“There’s no indication to me that this is a steroid specific to muscular dystrophy,” he said.

Marathon had considered researching whether Emflaza could treat patients with juvenile arthritis, but the company said last week it has no plans to pursue that use.

3. Emflaza doesn’t work alone.

The drug needs to be part of a cocktail of drugs Duchenne patients take, said Pat Furlong, the founder of the advocacy group Parent Project Muscular Dystrophy. She questions how many high-priced drugs insurers will pay for.

“Where is the breaking point where any given insurer says [that’s] too much,” and stops covering the drugs, Furlong said.

Marathon said the price was set based on a number of factors, including recouping its research costs. The FDA required the drug to be submitted as a new drug — regardless of its approval in other countries. A company spokeswoman said Marathon funded 17 studies for Emflaza’s approval.

4. Marathon has cultivated relationships in the Duchenne patient community.

At a heated meeting with patient advocates on Monday, Marathon CEO Jeff Aronin announced a delay in the rollout of the drug and later released an open letter saying the company would “not move forward with commercialization” until discussing options with Duchenne community leaders.

The company, which is a corporate sponsor for Parent Project Muscular Dystrophy, announced it will continue offering an expanded access program to patients. He also said patients currently receiving the drug from other countries can continue importing.

Joel Wood, whose Duchenne foundation has received money from Marathon, said he believed the company will uphold financial promises to patients.

“I will be the first one to pick up a pitch fork” if the company fails to do so, said Wood, who has a son with Duchenne.

5. Timing is everything.

There was muted criticism when Spinraza’s $750,000 price was announced over the Christmas holiday.

Perhaps emboldened by President Donald Trump who recently said drug makers are “getting away with murder,” lawmakers from both sides of the aisle are expressing anger about Emflaza’s price and demanding answers from the drugmaker.

Rep. Robert Aderholt, a Republican from Alabama who chairs the subcommittee that oversees FDA’s appropriations, said Monday that Emflaza is a “tipping point” on the issue.

Last week, Republican Sen. Chuck Grassley, chairman of the Senate Judiciary Committee, announced he has opened an inquiry into potential abuses of the Orphan Drug Act that may have contributed to high prices on commonly used drugs.

Sen. Bernie Sanders (I-Vt.) and Rep. Elijah Cummings (D-Md.) sent a letter to Marathon Monday calling Emflaza’s price “unconscionable.” They asked the company to explain itself and lower the price.

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, Syndicate

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Former FDA Chief Cites 5 Things To Watch On Drug Approvals, And Keeping Drugs Safe

The just-departed commissioner of the Food and Drug Administration has concerns about plans to speed up drug approvals and dramatically reduce regulations at the agency, as advocated recently by President Donald Trump.

Dr. Robert Califf, who stepped down last month, shared his thoughts about keeping Americans safe — and making sure drugs actually work — after about a year overseeing the federal agency. His takeaways:

1. Faster drug approvals, being advocated by Trump and others, don’t necessarily mean less expensive drugs.

“What I’m concerned about is that when people hear ‘faster approval,’ you get an image of the FDA sitting on this application and, you know, twiddling thumbs,” Califf said. “That’s not what happens.”

Califf explained that the slow part of getting a drug into patients’ hands happens well before the drugmaker submits an application to the FDA. It can take decades to discover, tweak and test a new drug molecule, and that development process is what needs a boost.

“The real action [happens] before the application gets submitted and that can be a time frame from years to decades as people try to figure out what will work and what will not,” Califf said.

Although faster drug development could bring development costs down, Califf said, “There’s not a direct relationship between the cost of development and the price of drugs or devices.”

“The concept of safety is much more complex than most people think about until they look into it deeply,” said Robert Califf, just-departed FDA administrator. (Courtesy of the FDA)

“The concept of safety is much more complex than most people think about until they look into it deeply,” said Robert Califf, just-departed FDA administrator. (Courtesy of the FDA)

2. A law passed in December should streamline drug approvals — in a positive way.

The 21st Century Cures Act, a bipartisan bill signed by President Barack Obama late last year, is a “very well-rounded piece of legislation that will speed up product development.”

To begin with, it encourages the FDA to consider pieces of information in its approval process in addition to traditional clinical trials, including “real world evidence” and biomarkers. Biomarkers are used in studies in place of outcomes that are more difficult to measure. For example, tumor shrinkage is sometimes a biomarker for surviving cancer.

“Using real world evidence in clinical trials is one that I’m particularly excited about as a way to get better answers at a lower cost and faster at the same time,” Califf said.

3. But Califf has concerns about maintaining drug safety standards and ensuring thorough clinical trials.

“The concept of safety is much more complex than most people think about until they look into it deeply,” Califf said. “All drugs have risk. None of them are absolutely safe. And the actual safety risks are only revealed through clinical trials with the same quality and number of patients involved as it takes to look at efficacy.”

About 92 percent of drugs that get into human clinical trials don’t make it to market because they fail to show any benefit or, worse, they have unexpected toxicity, he said.

“Declaring a drug is safe after very little information is treacherous,” Califf said.

Califf recently wrote for JAMA on the FDA’s balancing act of protecting the public and encouraging innovation. And the FDA released a paper last month documenting examples of a promising drug, vaccine or device that each did well in a Phase 2 clinical trial but “bombed out” in Phase 3, Califf said. Drugs typically go through three “phases” of studies called clinical trials before gaining FDA approval. With each new phase, researchers test drugs or other products on more people and on more measures of safety and effectiveness.

“It’s 22 examples of why it’s a big mistake to think that you can judge the balance of risk and benefit from a small amount of data,” Califf said.

4. Faster, better drugs can’t be approved if the FDA is understaffed, he argued.

Califf called a potential FDA hiring freeze “unfortunate” and said new staff is needed to meet the faster approval timelines and to give advice to those developing drugs so “bad mistakes” aren’t made.

The FDA has been steadily hiring staff to keep up with the growing industry it oversees. But hundreds of openings are still left to be filled.

Califf said the drug industry’s most valued interaction with the FDA is “during the process of [drug] development,” which begins before a drug application is submitted for approval. FDA staff can provide timely feedback and advice that can help a company choose the right studies needed for approval and, effectively, speed up drug development.

“A hiring freeze at this time when we are just revving up with 21st Century Cures is unfortunate. I hope that the dust will settle soon and the FDA can get back to its hiring,” Califf said.

5. The FDA is using big data to track the safety of drugs already on the market — and it plans to do that a lot more.

The FDA has an established adverse event database but after pain reliever Vioxx was pulled from the market in 2004, the agency began working on a system called Sentinel.

The initiative uses claims data to look at drug safety. It has 180 million individual electronic records and tracks every dispensed prescription, hospitalization as well as serious outcomes, Califf said. In addition, Sentinel is now being opened up to industry and academia.

“It’s used every day by the FDA,” Califf said, adding that the system is “getting better and better.”

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

Categories: Pharmaceuticals, Syndicate

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Drugmaker Marathon ‘Pausing’ Delivery Of $89,000-a-year Muscular Dystrophy Drug

In a surprise move Monday, Marathon Pharmaceuticals told patient advocates that it would “pause” the launch of their drug Emflaza because of pricing concerns expressed by patients and advocacy groups.

The drugmaker had announced an $89,000 annual price tag for their newly-approved drug last week — but patients and lawmakers immediately cried foul.

“What you’re doing is robbing my insurance company,” said Dana Edwards, a mother from New Jersey whose 12-year-old has taken deflazacort, the generic version of the drug, since he was five years old.

Newly approved Emflaza treats Duchenne muscular dystrophy, a rare, devastating neuromuscular disorder. Patients have been importing the generic version of the drug from overseas for about $1,200 a year.

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A statement read to a conference of parents, patients and advocates in Washington, D.C., and attributed to Marathon CEO Jeffrey Aronin said: “Our goal in commercializing Emflaza all along has been to make it available to that broader set of patients who prior to FDA approval have not had access to the therapy… We are pausing our launch, which has not yet taken place. We have not sold any new product and will pause that process.”

Pat Furlong, the president and founder of Parent Project Muscular Dystrophy, which sponsored the Monday morning conference, read the statement to an outraged crowd in a conference room at the Mayflower Hotel.

The company will continue to offer patients an expanded access program, Furlong read, which allows about 800 patients to receive the drug from the company. More can join that program for free, and patients can continue importing drugs from Canada or “wherever they are getting it,” the statement said.

Furlong told the audience that complaints from patients helped to prompt Marathon’s action: “Since last week’s approval, they’ve heard from all of us,” he said.

Late Monday, Marathon released a statement and open letter to the Duchenne community, elaborating on its goals of patient access and promising that “price should not be a barrier.”

“Put simply we expect patients will pay a standard copay of typically $20 or less per prescription,” the statement reads.

A Marathon Pharmaceutical executive speaks at the Parent Project Muscular Dystrophy advocacy conference. (Sarah Jane Tribble/KHN)

A Marathon Pharmaceutical executive speaks at the Parent Project Muscular Dystrophy advocacy conference. (Sarah Jane Tribble/KHN)

The company also heard from Capitol Hill. On Monday morning, Sen. Bernie Sanders, I-Vt., and Rep. Elijah Cummings, D-Md., sent a letter to Marathon demanding answers about the $89,000 price tag for a drug that has been routinely used by Duchenne patients since at least 2005.

“We believe Marathon is abusing our nation’s ‘orphan drug’ program, which grants companies seven years of market exclusivity to encourage research into new treatments for rare diseases — not to provide companies like Marathon with lucrative market exclusivity rights for drugs that have been available for decades,” Sanders and Cummings wrote.

Orphan drugs are medicines targeted at people with rare diseases, which often carry very high price tags. Emflaza was approved last Thursday for treating Duchenne disease. The drug is a steroid that manages symptoms for Duchenne’s sufferers; it does not cure the underlying disease.

In January, Kaiser Health News published an investigation that found that the orphan drug program intended to help desperate patients is being manipulated by drugmakers. While the companies aren’t breaking the law, they are using the Orphan Drug Act to secure lucrative incentives and gain monopoly control of rare disease markets, the investigation found.

Republican Rep. Robert Aderholt, the chairman of the Appropriations subcommittee that funds the FDA, said the 6000 percent increase in deflazacort’s price “makes me question whether the current construct of how FDA approves orphan drugs does more harm than good if companies have found a way to game the system.”

In addition to orphan status, the FDA gave Marathon a rare pediatric disease priority review voucher, which the company can sell for millions of dollars to another drugmaker who can use it to expedite their own drug’s approval.

At Monday’s meeting, Marathon executives presented a slideshow to a packed room that detailed different patient financial assistance programs as well as the drug’s expected availability. Emflaza was expected to be on the market by mid-March, said Eric Messner, vice president of sales and marketing for Marathon.

Messner explained that the drug would cost between $50,000 to $54,000 after rebates and discounts. And he emphasized that Marathon did not expect patients to pay that price. “We expect that patients will experience a low or zero out-of-pocket experience. I know there has been a lot of discussion about that. How did we arrive at the price?” Messner said. An audience member interrupted Messner asking if the company had talked to patients and families about the price.

He offered assurance that they did and said there would be an ongoing dialogue with patient groups.

Edwards, the New Jersey mother, said she hopes President Donald Trump will fulfill his vow to bring down high drug prices.

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

Categories: Pharmaceuticals, Syndicate

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