A new drug discovered in San Diego is giving hope to some patients with cystic fibrosis. It's giving others a severe case of sticker shock.
Approved by the FDA last month as the first drug to treat the most common genetic form of cystic fibrosis, Orkambi costs a quarter-million dollars per patient, per year. Some experts say our healthcare system can't afford it. Meanwhile, many patients are hoping their insurance will cover the drug's cost.
Klyn Elsbury is one of them. Leading a Zumba class one recent afternoon, she looked perfectly healthy. She guided everyone through a Latin dance-inspired workout without missing a beat.
But toward the end of the class, Elsbury couldn't stop coughing. It's one of the symptoms of her illness, cystic fibrosis. The disease causes mucus to build up in her lungs, making her prone to infections that leave her hospitalized every few weeks.
"Essentially, I'm spending half my life in the hospital," she said. "Because the mucus secretions in my lungs are so thick, and just covered in bacteria."
The average lifespan for patients with cystic fibrosis is just 41 years, according to the Cystic Fibrosis Foundation. There have been times when Elsbury didn't think she'd live through her 20s.
But today, at 27, Elsbury leads an active life despite her disease. She's now a fitness instructor and a certified personal trainer living in San Diego.
"I couldn't always wait for drugs to come through the development pipeline," she said.
"What I could do is I could get my body in the best shape possible. And it would hurt. It would be very grueling. I would get a lot of stares, but I knew I would walk into a gym and change my life, or my life was going to end very soon."
Elsbury thinks she could lead an even more active life if she could get on Orkambi. She knows it's not a cure. The drug has reportedly provided only "modest" improvements to lung function.
But data on the drug does give Elsbury hope that one day she could spend more of her life outside the hospital.
"Instead of being in the hospital every two months, maybe I could have three months," she said. "And if I could have three months — and I continue to be fit, and I continue to do all the right things day-in, day out — maybe I could go four months without a hospitalization."
She said with that kind of time, she could take a trip abroad, set new weight-lifting goals and take on more personal training clients.
"That's what the drug means to me," she said. "What could I do in four months, that I couldn't do in two?"
Orkambi is made by Vertex Pharmaceuticals. The company is based in Boston, but research on the drug was done at a Vertex site in San Diego.
Orkambi's origins date back to 1996, when San Diego-based Aurora Biosciences incorporated and began searching for chemical compounds that might treat cystic fibrosis. Vertex acquired Aurora in 2001.
Across town at UC San Diego, cystic fibrosis researcher Paul Quinton has been studying the disease for decades. When asked what he thinks of this new drug, he said, "It's incredibly, astoundingly expensive. And I think it's too expensive."
Orkambi has an annual per-patient cost of $259,000, a price Quinton and other cystic fibrosis experts call "unconscionable." Patients will have to stay on Orkambi for the rest of their lives.
In the early 1980s, Quinton helped identify basic cellular defects in cystic fibrosis by studying patients' sweat glands. Vertex built on these and other early discoveries to develop Orkambi.
Quinton is glad they did, but he said they're now charging too much for it. He believes Vertex signed a social contract when it accepted funding from a charity, the Cystic Fibrosis Foundation.
"I think a number of us feel a little bit betrayed," he said.
Quinton doesn't just study cystic fibrosis. He has cystic fibrosis. For the past few years, he's been on a different Vertex drug, Kalydeco. It targets a less common form of the disease, and it costs even more than Orkambi — upwards of $300,000 annually.
"This is the drug," Quinton said, displaying a blister pack containing a week's worth of pills. "Each of these little pills costs over $400. This is about $5,600 in this little tablet."
Quinton said soaring drug prices are not unique to cystic fibrosis. Lots of new drugs targeting small patient populations have been coming out with six-figure price tags. Quinton worries that "precision medicine" will end up costing the healthcare system more than it can bear.
"How can we sustain paying these kinds of costs for drugs if we are trying to develop a healthcare medical system that's efficient and takes care of us well? I don't think we can do that," he said.
Quinton said he doesn't have an easy solution for keeping drug prices in check. But he would like to see academic scientists get more involved in drug development, and he's in favor of giving Medicare more bargaining power to get drug companies to lower their prices.
Vertex would not agree to a taped interview, but a spokesman said Orkambi's price is based on nearly two decades of drug development. He said Vertex will use part of its revenue to fund work on new cystic fibrosis drugs for patients who still don't have any treatments.
However, critics say executive pay at Vertex is exorbitant. CEO Jeff Leiden earned $36.6 million last year, and other executives stand to take home bonuses totaling more than $50 million if Orkambi turns Vertex into a profitable company.
Of course, patients won't pay for Orkambi out of pocket. They'll get the drug if their health provider decides to cover it. Vertex's spokesman said it could take months of discussion with providers, but the company expects broad reimbursement.
Some patients have already been approved and are now taking the drug. Klyn Elsbury has not been so lucky. She's on a state-run program that has so far denied her repeated requests to get on Orkambi.
"The excitement factor is there," she said. "It's definitely there. But it's been a month now, and I'm still not on it. So I'm trying to keep an even keel."
California's Department of Health Care Services plans to establish guidelines later this month on whether or not to cover the drug for patients like Elsbury. Officials say cost will be considered as they craft those guidelines.
