The Cost of Medical Miracles
Governments grapple with how to pay for patients’ million-dollar therapies.
Suhellen Oliveira Da Silva was six months pregnant when she learned that the child she was carrying had the same disease that had left her firstborn son paralyzed. But this time, there was a treatment that could make a profound difference. This baby could live a normal life.
The problem was the price: The treatment cost the equivalent of $1.7 million, and the public health system in Brazil, where the family lives, was refusing to pay for it.
So Ms. Da Silva went to court — and won.
A judge ruled that the government had to buy the therapy for her younger son, Levi. Today, Levi, 2, chats and claps and crawls, all things his brother Lorenzo, 10, cannot do.
The treatment, Zolgensma, a one-time infusion, is among the first in a new class of gene therapies that offer enormous promise for people with fatal or debilitating conditions — at huge prices. Its maker, the pharmaceutical company Novartis, has negotiated deals with national health systems and insurers to get the drug covered in many rich countries.
With Zolgensma, which treats a rare genetic disorder known as spinal muscular atrophy, or S.M.A., experiencing slowing sales, Novartis is pushing to get broad coverage in middle-income countries like Brazil, where public health systems are often underfunded. It has become a test of whether such therapies can win broad coverage around the globe.
After more than 100 successful lawsuits forcing the Brazilian public health system to pay for the treatment, the government announced in December that it would begin covering Zolgensma by default for infants with the most severe cases of S.M.A. later this
year. The government has agreed to pay the equivalent of about $1 million for each treatment.
At a hearing on the coverage issue, a congresswoman, Adriana Ventura, expressed sympathy for the families seeking the treatment, but said, “We also cannot be irresponsible and approve something that is not sustainable in the long term.” She added the concern is that “in order to give to one, you have to take the basics from millions of others.”
An analysis led by a researcher at Brazil’s drug regulator found that the court-ordered expenditures in Zolgensma’s first 14 months of availability in Brazil could have paid for over four million doses of the Covid-19 vaccine.
Over the past decade, wealthy countries have devoted a growing share of their pharmaceutical budgets to paying for expensive drugs that treat a tiny fraction of their citizens. Their spending on so-called specialty drugs, which treat rare and often serious diseases, now represents about half of their drug expenditures. And it is expected to grow.
Among the most unaffordable are gene therapies that promise to transform inherited disorders with a one-time treatment. Zolgensma’s list price of $2.1 million in the United States in 2019, believed to be the highest ever when it was set, has since been surpassed four times, and many more treatments expected to be as expensive are on the horizon.
In Europe, a product approved for a deadly neurological disorder known as metachromatic leukodystrophy was given list prices of up to $3.9 million. Last year, Germany’s health system agreed to pay a discounted $2.6 million. In the United States, the biotechnology company Bluebird Bio set prices last year of $2.8 million when it won approvals to treat an inherited blood disorder called beta thalassemia and $3 million to treat a fatal neurological condition known as cerebral adrenoleukodystrophy. When European systems refused to pay what Bluebird was asking, it withdrew the products from the continent.
Record-setting price tags for gene therapies have largely escaped the criticism that has followed other industry pricing decisions. The sentiment reflects just how powerful many of the therapies are and their unique position as one-time treatments. In some cases, such a therapy can replace chronic treatments that would otherwise be given for the rest of a patient’s life at a much higher cumulative cost.
Still, for middle-income countries, “if benefits of these therapies are immediate in terms of health but the potential savings happen in the future, that math may not work for them,” said Rena Conti, a health economist at Boston University.
Tay Salimullah, a Novartis executive, said the company works with governments and health plans considering whether to cover Zolgensma, in some cases allowing them to spread out payments over time, or offering a price cut if the treatment does not work.
‘This Kid Has a Future’
Until six years ago, there were no approved treatments for S.M.A., which affects about one in 10,000 newborns. Infants with the most severe form of it were sent home and their families were told to prepare for them to die.
Zolgensma and two other drugs have opened up once unimaginable possibilities for S.M.A. patients. “I’m telling parents to keep putting money in their college fund because this kid has a future,” said Dr. Thomas Crawford, who treats S.M.A. patients at Johns Hopkins Medicine in Maryland.
Zolgensma works by replacing the missing or nonfunctioning gene causing S.M.A. with a working copy of the gene. It has been given to more than 2,500 children and approved for use in 46 countries. Studies show Zolgensma can stop infants and toddlers from continuing to lose nerve cells that control muscle movement, preventing further decline, but it cannot restore motor or muscle function already lost. If given soon after birth, children may develop no significant disabilities. Children who are a little older may avoid a feeding or breathing tube, and may be capable of some movement.
A Right to Health
Ms. Da Silva had never heard of S.M.A. when Lorenzo was diagnosed at 6 months old, in 2013. He had been developing normally, and then his progress stopped. Doctors said nothing could be done.
When Ms. Da Silva learned in 2019 that she was unexpectedly pregnant with Levi, doctors said he might never have disabilities from S.M.A. if she could get Zolgensma for him soon after he was born.
The family had been struggling to make ends meet. Ms. Da Silva had quit her job as a travel agent in order to fight for care and therapies for Lorenzo; her husband, Azen Balbino, had spells of unemployment in Brazil’s recession. They knew they would need to rely on a process called judicilizacão, in which they effectively sue the federal government by invoking the constitutionally protected right to health to force the public system to pay for a therapy that it would not otherwise provide.
While Ms. Da Silva was still pregnant, she enlisted the help of Viviane Guimarães, a lawyer in her city, Recife, who agreed to take her case. When Ms. Guimarães stood before the judge to make the case for a therapy with a huge cost, she argued that it would save the government money compared with other drugs and care Levi would need without it.
Lorenzo receives three kinds of therapy every day. He watches cartoons while a physiotherapist makes a twice-daily visit to pummel on his chest and suction the secretions that would otherwise suffocate him. He communicates with Ms. Da Silva through a whisper and by blinking. While this happens, Levi clambers over his inert legs, singing along with the television and imperiously giving orders for things such as Popsicles to the health aide, one of three who take shifts by Lorenzo’s bed 24 hours a day.
While Levi has not met all of the milestones of his age, he is mobile and talkative. He has daily occupational and physical therapy and continues to progress in development.
Every time she sees her two boys together, Ms. Da Silva is reminded of the stunning power of Zolgensma. Her happiest moments, she said, come when Levi pushes on her cheeks to get her attention and demands, “Mamãe, Mamãe” like any other toddler. “Levi speaks like a child who doesn’t have S.M.A.,” she said.
Few Resources to Spare
Novartis has so far brought in $3.7 billion in revenue from Zolgensma, charging different prices in different places.
The treatment has not become a mega-blockbuster in part because so few patients are eligible for it. And sales have begun to slow. Middleincome countries like Brazil could bring many more patients. Novartis has already won coverage for Zolgensma in Russia, Egypt and, most recently, Argentina. It remains in negotiations in more than 10 countries, including Ecuador.
In Brazil, the government has agreed to cover the treatment only for babies with the most severe form of S.M.A. who can breathe independently for at least most of the day. They must also be under 6 months old. Brazil has agreed to pay for Zolgensma for no more than 250 babies over the next two years. If demand is higher than that, Novartis will offer 40 free treatments. At most, the government would spend about $50 million for this first group of children this year and a total of $200 million over the four years after that.
In comparison, a program known as the people’s drugstore had a budget last year equivalent to about $380 million to serve over 21 million people, providing drugs for conditions such as asthma, diabetes and high blood pressure. The public health system provides care to more than half of the roughly 216 million people in Brazil. Clinics and hospitals in lowincome areas lack basic supplies and technology more sophisticated than X-ray machines.
“We’re talking about a super expensive medication,” said Ms. Guimarães, the Da Silva family lawyer. “But when you go to the hospital, the nurse doesn’t even have a pair of latex gloves.”
“People are going to have to get comfortable with the debate,” she added, “because there are going to be many more therapies like this.”