Health Care

Free drug saved the life of JoCo family’s son. But soon it will cost $375,000 a year

Ann Schuller vividly remembers the first time her son, Will, took the medication that saved his life.

Will Schuller has Lambert-Eaton-myasthenic syndrome, or LEMS, a rare autoimmune condition. His symptoms began in 2014, around Halloween, during his senior year at Blue Valley Northwest High School. He went downhill quickly, losing feeling in his limbs. By Christmas he was in a wheelchair, too weak to walk, and had to be pulled out of school.

During a trip to the Mayo Clinic he received an experimental drug called 3,4-Diaminopyridine.

At first nothing happened. Then, all of a sudden, the medicine took effect.

“I kid you not, and I can show it to you on video, he jumps off the table and starts doing kicks and deep knee bends,” Ann Schuller said. “It’s a pretty amazing drug.”

It’s also poised to get much more expensive soon.

Will Schuller (center) no longer needs this wheelchair because of a new, experimental drug. His parents, Bob and Ann Schuller, are fighting the drug’s imminent price increase of $375,000 a year. Keith Myers

Will Schuller and a significant chunk of the estimated 3,000 Americans with LEMS currently get their medication from Jacobus Pharmaceutical, a small, family-owned company in New Jersey. Because the drug is not FDA-approved, Jacobus has doled it out for years to hundreds of LEMS patients for nothing but the price of shipping.

Now another company, Catalyst Pharmaceuticals, is set to start selling a slightly modified, FDA-approved version for a whole lot more — $375,000 a year before discounts and rebates. And because of the FDA process the company used, it will have exclusive rights to sell the drug for seven years.

Catalyst CEO Patrick McEnany said on a Dec. 13 conference call that the price of the drug, called Firdapse, reflects the “tens of millions of dollars” the company spent on 70 research studies over the last nine years to get it to market, plus ongoing research to see if it might work for other conditions.

McEnany said most insurance companies have already signaled they will cover the drug, and there will be assistance programs “designed so that a patient is out of pocket a minimal amount of money.” The effort to commercialize the drug, he said, was meant to allow more people to get it, not fewer.

“Our rationale for pursuing FDA approval for Firdapse was to ensure that all — all patients — could have access to an evidence-based treatment, with safety and efficacy properly studied and characterized,” McEnany said, “helping more than just the 10 percent of patients who are currently in an early access investigational program.”

Firdapse is expected to hit the market in January.

The Schullers are in touch with their insurance company, and they think the new drug will be covered. But they don’t know what price their insurer will negotiate with Catalyst or what their out-of-pocket obligation will be.

Ann Schuller said her famiy and others who have been getting the meds from Jacobus are now getting an unwelcome crash course in the complex world of U.S. drug development, testing, marketing and profits

“Obviously the small LEMS community is frightened that they are now not going to be able to afford a drug they have been receiving for free for years,” she said.

Pricing controversy

The experimental drug 3,4-Diaminopyridine or DAP has been provided to patients by Jacobus Pharmaceutical for only the cost of shipping. Now Catalyst Pharmaceuticals is set to sell a slightly modified, FDA-approved version, for $375,000 a year before discounts and rebates. Jacobus will no longer be allowed to produce the free version. Keith Myers

The Firdapse pricing controversy has been years in the making.

Laura Jacobus, one of the co-owners of the pharmaceutical company that bears her name, told NPR in 2015 that the process of seeking FDA approval was too expensive for a family-owned business. So, after developing the LEMS drug at the request of university physicians in the late 1980s, the company just gave it away under a program for doctors who have special FDA permits to prescribe investigational drugs.

It was a highly unusual arrangement.

But for a couple decades it worked for patients who could find those doctors, including the specialist at the Mayo Clinic who treated Will Schuller.

But in 2010 a much bigger company, publicly traded BioMarin Pharmaceutical, began to develop its own version and spend the money to put it through the FDA pipeline.

After the initial testing showed promise, Catalyst bought the rights from BioMarin in 2012, and continued the process.

Catalyst was still testing in December 2015. But it was also courting investors, and in a presentation at a health care conference that month, Catalyst executives said they projected Firdapse could be worth $300 million to $900 million in revenue every year.

That set off alarm bells in the medical community, because given how few people had LEMS, the per-patient price would have to be sky-high to hit those targets.

More than 100 neurologists from across the country, including Richard Barohn of the University of Kansas Medical Center, signed on to an op-ed expressing concern about a “potentially harmful price increase” in 3,4-diaminopyrine.

The op-ed, published in a neurology medical journal, said Catalyst was exploiting a loophole in the U.S. Orphan Drug Act, the bill that allows the company seven years of exclusive marketing rights once the drug is approved.

That legislation was supposed to be an incentive for pharmaceutical companies to spend big on developing new drugs for rare conditions. But the doctors argued that Firdapse was not truly new, but a version of the Jacobus formula slightly modified so that it doesn’t require refrigeration. It’s also not particularly expensive to make, they wrote.

“Thus, it differs dramatically from some innovative drugs now in or coming to the market,” the op-ed said. “It has been reported to be effective and safe since 1983.”

But on the conference call McEnany said Catalyst’s product is different, and putting it through the FDA approval process means it’s proven safe and effective.

He commended Jacobus for providing the drug free for years under the investigational drug program, while also noting that Catalyst had been doing the same since it started developing Firdapse.

But he said restricting it only to physicians with the special FDA permit had forced some patients to travel hundreds of miles to get a prescription, if they could get one at all.

“This presented a challenging situation for many physicians and patients who were either unaware of or not able to access this potentially beneficial treatment,” McEnany said.

He said the company also plans to sink some of the revenue from Firdapse into developing a longer-acting version of the drug, so patients can take it fewer times a day.

Meanwhile Jacobus will likely have to stop providing the drug. After the company learned of BioMarin’s plans to market it, Jacobus launched its own FDA approval bid under the Orphan Drug Act. But that application is still under review. BioMarin/Catalyst beat Jacobus to market.

“We are uncertain of our path forward at this time,” Laura Jacobus said via email.

Schuller’s future

Will Schuller, now 22 and a senior at the University of Tulsa, said the approval of Firdapse has some upsides.

He was diagnosed with LEMS relatively soon after his symptoms emerged by a local doctor, David Clark of Johnson County Neurology and Menorah Medical Center in Overland Park. But to get hooked up with the Jacobus drug he had to go to Mayo in Minnesota, and had to be there for awhile.

“For me it was really hard,” he said. “There was only, like, one doctor at Mayo Clinic who could prescribe it, and you would have to get the diagnosis confirmed and see him in person.”

He said he’s keenly aware that not every family is like his, with the financial means and the medical literacy to get an appointment at Mayo.

He’ll also have to take the medicine for the rest of his life unless a cure is developed, so the prospect of being able to see any doctor for his prescription is appealing.

But he is worried about the cost. He’s on his parents’ insurance now and can stay on it until he turns 26. But who knows what the future will bring?

“I know in Europe people are paying quite a bit for the medicine,” Schuller said, “and every doctor I’ve talked to says it costs maybe $3 to make a bottle of it.”

Still, Schuller said right now his parents are probably more concerned than he is and are following the controversy more closely.

Thanks to the Jacobus drug, Schuller is happily immersed in the life of a college student, after missing out on some major experiences, including a band trip to Spain, when he was a high school senior unable to walk. He’s studying mechanical engineering at Tulsa, jogging regularly and playing on the school’s Ultimate (Frisbee) team.

He’s become an expert on how to lead a full life with LEMS and he’s hopeful that Firdapse’s price won’t derail that when it hits the market.

His family’s next step is to voice their concerns about the pharmaceutical industry to Congress.

“Fundamentally, the question we need to ask as voters is, ‘Is this a system we want to have?” said Will’s dad, Bob Schuller. “Because this does not seem like a great example of developing a moonshot new treatment. We seem to be in kind of a runaway cost situation that needs to be fixed.”

Kansas City Star health reporter Andy Marso was part of a Pulitzer Prize-finalist team at The Star and previously won state and regional awards at the Topeka Capital-Journal and Kansas Health Institute News Service. He has written two books, including one about his near-fatal bout with meningitis.