DERWOOD, Md. — Kristen Lasko cried tears of relief in late December when she read online that an experimental drug that could help her 3 1/2-year-old son, Max, had finally been approved. Kristen and her husband, Jonathan, quickly pivoted to getting their cheerful, blond boy access to the only approved medicine for a devastating, muscle-wasting disease. The drug, Spinraza, would carry a staggering price: $750,000 for the first year.
Max’s rare, life-threatening disease, spinal muscular atrophy, required him to use a wheelchair and robbed him of muscles he needs to smile and swallow. Spinraza, developed by the Massachusetts biotech company Biogen, had helped other children with the disease sit, stand or even walk.
In the final week of 2016, the Laskos sat down with Max’s medical team and were told that Biogen was offering patients financial assistance and guidance — if they signed a sweeping authorization that gave the company access to private health information for “patient support services and marketing/other communications.”
The Laskos signed the form, and in walked a Biogen representative who was waiting at the hospital, to personally usher families into the program.
“Any of us who have been through an insurance battle know, when you get a denial your options are very limited. You can write back on your own, you can have medical practitioners write back — best-case scenario, find a lawyer, pro bono. At that point you’re a little bit charging at windmills,” Jonathan said. “These are pros. They should be able to get this through the system faster than us.”
The Laskos had seen tremendous medical bills before and appreciated more than most people the necessity of health insurance. As a baby, Max had been transported between hospitals by helicopter, a single bill that added up to $50,000.
Spinraza’s price was almost impossibly high: $750,000 for the first year and $375,000 annually after that. But the Laskos know it is expensive and risky for a company to develop a drug for a disease that affects one in 10,000 babies born each year. Drugs for tiny patient populations — called “orphan diseases” — are an increasingly attractive niche of drug development in part because of the high prices companies can charge.
The Laskos’ focus was on getting Max the drug. They aren’t calling for the price to be lowered. They saw that as a problem to be worked out between two giant industries that both say they work on behalf of patients: health insurers and drug companies.
After all, almost no person could actually afford to pay for such a drug.
“From the patient perspective, I don’t care who eats [the cost], but I don’t think the patient should eat it,” Jonathan said. “I don’t think the person whose life could be substantially improved by this drug should suffer because the other parties couldn’t decide who was going to be more magnanimous and pay for it.”
When pharmaceutical companies come under fire for the prices of their products, they almost inevitably point to the financial help they provide patients. That’s true not only for innovative companies like Biogen that create new drugs but also for those that jack up the price of older drugs. For instance, Mylan beefed up its coupons and assistance programs after it came under attack for boosting the price of the EpiPen.
Such programs have real benefits for families, but the alliance between drug companies and patients is a complex one that can make ethicists and health-policy specialists uneasy. In the rare disease space, where patients battle conditions many people have never heard of, patients depend critically on companies to develop treatments. But when diseases are rare and drug prices are sky-high, each individual patient becomes valuable to the company financially.
Wall Street analysts trying to project Biogen’s financial performance earlier this year paid close attention to individual patients’ experiences as it rolled out the drug. In a slide deck sent out to investors in March, Umer Raffat of Evercore ISI included a list of 29 unnamed patients who were taking the drug, including their start dates, what kind of insurance coverage they were thought to have and where they lived.
Brian O’Sullivan, a physician who sees patients who take extremely expensive drugs for cystic fibrosis — another rare disease with ultraexpensive treatments — sees the pros and cons. He knows patients of his might not be able to afford to cover their copays without help from the drug company. Still, he has reservations.
“I know the ulterior motive is not just to get the patient the medicine,” O’Sullivan said. “I’d like to think that’s a big piece of it, but there’s no question this is a moneymaking proposition” for the drug company.
Whatever drug companies do to help patients get access to the drug may help families — and companies’ bottom line. Because patient copays are typically a fraction of the cost of the drug, with the lion’s share paid by insurance (which means everyone pays through higher premiums), the companies can still make money and insulate their prices from the most potent critics.
“The best thing you can say for them is they’re plugging a hole the drug company created themselves with outrageous pricing,” said Ezekiel Emanuel, chairman of the department of medical ethics and health policy at the University of Pennsylvania. “It’s a Band-Aid.”
And Biogen’s assistance program had another twist: It asked the Laskos to sign a privacy authorization that handed over Max’s health information for a decade. In addition to saying the information could be used for patient support, Biogen also advises families that it could be used for “market research and other internal business activities.”
Parents must agree that “any information that I provide may be used by Biogen to help develop new products, services and programs,” according to the form.
Todd Cooper, a spokesman for Biogen, stressed that the program is optional and that patients’ health, insurance and financial information is used by Biogen staff to help get the drug covered.
But health-policy specialists questioned the marketing provision, the high price of the drug that may not make the program feel optional and the length of the agreement, especially considering the disease often affects vulnerable infants.
“These drugs are necessary to someone’s health and well-being, perhaps to someone’s life. Almost no one can afford them at these prices, so people who need these drugs have little choice,” said Bonnie Kaplan, a bioethicist at the Yale Interdisciplinary Center for Bioethics. “Their consent really cannot be given freely.”
Cooper said the company settled on the 10-year time frame so that patients wouldn’t have to sign the form each year for a drug they would be taking long-term. The authorization allows the company to provide information to families who often feel isolated and learn from them.
“None of us can know what it’s like to have a child or to be an adult with this disease,” Cooper said, “and it’s really important for us to be close to this community.”
Kenneth Hobby, president of the patient advocacy group Cure SMA, which receives financial support from pharmaceutical companies including Biogen, said he hadn’t heard any concerns or questions from the families about the program or its privacy authorizations.
Still ethicists are wary.
“It’s not clear if I might see my kid’s data presented in a meeting or in an ad campaign or a testimonial,” said Arthur Caplan, a bioethicist at the New York University Langone Medical Center. “I’m not saying they would, I just can’t tell.”
The Laskos have thought long and hard about the privacy that Max deserves. As new parents, Kristen and Jonathan heard a lot from friends that they were just lucky, they had a very calm baby. But they knew something was wrong soon after their son was born: Max didn’t lift his head off the floor during tummy time. He’d gurgle and have trouble clearing his throat. When he was 2 ½ months, they heard for the first time the words “spinal muscular atrophy.”
The doctor told the Laskos that their son would be in a wheelchair all his life. Okay, they thought. We can do this. Then, the doctor said he would need a machine to breathe. That he wouldn’t be able to eat on his own. That half of the children with this disease died before their second birthday.
Kristen and Jonathan looked at each other through tears. Their world was spinning.
A month later, the Laskos learned about a clinical trial of an experimental drug and signed up.
Then, Max got his first cold. For most babies, it would be an inconvenience, but Max landed in the intensive care unit for seven weeks.
The Laskos had to make a heartbreaking choice: They could give their son a breathing tube to help him get better, but it would disqualify him from the trial.
“He was 5 months old,” Jonathan said. “We gave ourselves time to grieve this missed opportunity.”
For a child with progressive neuromuscular weakness, even a routine sneeze can be an emergency. On a recent evening, Kristen and Jonathan were typical doting parents one moment, joking around with their son, helping him draw a picture. But his cheek twitched almost imperceptibly, and they transformed into an urgent, preternaturally calm medical team: Jonathan swiveled to switch on a machine to suction mucus out of Max’s nose. Kristen detached a ventilator tube at the base of his neck and hooked up yet another tube that provides a “cough assist” — an extra whoosh of air that helps Max clear out his lungs. Jonathan silenced an alarm on one of the stack of lunch-box-size devices that helps keep Max alive. They went back to playing with Max, as if nothing happened.
Kristen said that sometimes her job as a mom is to do something really mundane like make a mess with Max.
“You have to think, what would a typical 3-year-old do — and help him do it,” she said.
The Laskos watched through Facebook as families in the patient community benefited from Biogen’s experimental drug. It was profoundly encouraging to see others progress but crushing to feel Max was missing out.
Instead, they enjoyed every moment with Max and shared the joys and challenges of his life on a blog they call Max Strength. They wanted to let other families know what was possible. Max hasn’t had the facial muscles to smile since he was a baby, but he does a kind of full-body bob when he’s happy — or to say “yes.” He shows what he wants with his gaze and by fluttering his eyelids. He can’t form words with his mouth, but he makes a singsong, syllabic vocalization that his parents understand, conversing about music, a camera and his favorite cellist, Yo-Yo Ma, on a recent visit. He attends preschool through a telepresence robot.
“He deserves the right to privacy,” Jonathan said. “The flip side is, if we privately enjoyed our wonderful life with Max, many families would not even know that was possible.”
So when the Laskos finally had a chance to get access to the drug with some company help and they read the privacy authorization on the form, they didn’t hesitate.
“We made it our business to share what we learned,” Kristen said. “Why should people start from zero?”
On Tuesday, Max received his fourth dose of Spinraza. The sticker price for one treatment, which is administered through an injection into the fluid around the spinal cord, is $125,000.
The insurance company has covered the drug, and the Laskos were accepted into Biogen’s zero-dollar co-pay program.
That’s not to say things went seamlessly. Systems never move fast enough when a child faces a life-threatening disease, and the Laskos faced their share of delays and frustration this winter. Initially, the insurance company denied coverage. A treatment they thought would begin in February didn’t start until April.
Their experience — even having signed up for help — shows why patients often feel few qualms about signing away a little medical privacy if it can clear the logjam of obstacles that they can barely keep track of, between the hospital, the drug company and the insurer.
The Laskos are optimistic, but realistic. They know that Spinraza isn’t a miracle cure. But a little more strength in their son’s neck could help him control his head. Additional strength and dexterity in his hands and fingers could help him to steer his power wheelchair by himself. A bit more control of his jaw might help him communicate and vocalize.
It’s too soon to tell whether the drug is working or how, but a flicker of movement near the corner of Max’s mouth that means he is smiling seems to have become more frequent.
Jonathan and Kristen are thoughtful when asked about the privacy they signed away. Their greatest hope is that in the future, they’ll be explaining their decisions to their son. “We’ve erred on the side of being more open,” Jonathan said. “One day, we’ll have to answer to Max.”