Suhellen Oliveira Da Silva was six months pregnant when she found out that the child she was carrying had the same disease that had left her firstborn paralyzed. But this time, there was a treatment that could make a world of difference. This baby could live a normal life.
The problem was the price: the treatment cost the equivalent of 1.7 million dollars and the public health system in Brazil, where the family lives, refused to pay for it.
So da Silva took his case to court — and won. A judge ruled that the government had to buy the therapy for his youngest son, Levi. Today, 2-year-old Levi talks, claps and crawls, things his 10-year-old brother Lorenzo can’t do.
The treatment, Zolgensma, a single-dose infusion, is among the first in a new class of gene therapies that offer enormous promise for people with fatal or debilitating diseases—at astronomical prices. Its maker, the pharmaceutical company Novartis, has negotiated deals with national health systems and insurers so that the drug is covered in many wealthy countries.
With Zolgensma, which treats a rare genetic disorder known as spinal muscular atrophy, or AME, experiencing a slowdown in sales, Novartis is pushing for 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 gain wide coverage around the world.
After more than 100 successful lawsuits that forced the Brazilian public health system to pay for the treatment, the government announced in December that it would start covering Zolgensma for babies with more severe cases of SMA later this year. The government has agreed to pay the equivalent of around $1 million for each treatment.
At a hearing on the issue of coverage, one congresswoman, Adriana Ventura, expressed sympathy for families seeking treatment, but said, “We also can’t be irresponsible and pass something that’s not sustainable in the long run.” She added that the concern is that “to give to one, you have to take the basics away from millions of people.”
An analysis by a researcher at Brazil’s drug regulatory agency found that court-ordered spending in the first 14 months of Zolgensma’s availability in Brazil they could have solved more than 4 million covid-19 vaccines.
Among the most expensive therapies are gene therapies that show promise to transform inherited disorders with a single dose. Zolgensma’s 2019 US list price of $2.1 million, believed to be the most expensive when it was established, has since been exceeded four times, and many more treatments are on the horizon that are anticipated to be just as expensive.
In Europe, a product approved for a deadly neurological disorder known as metachromatic leukodystrophy has received list prices of up to $3.9 million. Last year, Germany’s healthcare system agreed to pay $2.6 million at a discount. In the United States, biotech company Bluebird Bio set prices last year of $2.8 million when it won approval to treat an inherited blood disorder called beta thalassemia and $3 million to treat a deadly neurological condition known as cerebral adrenoleukodystrophy. When European systems refused to pay what Bluebird was asking, it withdrew the products from the continent.
Record prices for gene therapies have largely escaped criticism of other industry pricing decisions. The sentiment reflects how powerful many of the therapies are and their unique position as single-dose treatments. In some cases, such a therapy can replace chronic treatments that would otherwise be administered for the rest of the patient’s life at a much higher cumulative cost.
Still, for middle-income countries, “if the potential savings occur in the future, the 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, in some cases allowing them a payment scheme or offering a price reduction if Zolgensma doesn’t work.
Until six years ago, there were no approved treatments for SMA, which affects one in 10,000 newborns. Babies with the most severe form were sent home and their families were told to prepare for their death.
Zolgensma and two other drugs have opened up previously unimagined possibilities for SMA patients. “I tell parents to keep putting money into their college fund because this kid has a future,” said Thomas Crawford, who cares for SMA patients at Johns Hopkins Medicine in Maryland.
Zolgensma works by replacing the missing or non-functioning gene that causes SMA with a working copy of the gene. It has been administered to more than 2,500 children and has been approved for use in 46 countries. Studies show that Zolgensma can prevent further loss of nerve cells that control muscle movement in infants and young children, preventing further deterioration, but it cannot restore already lost muscle or motor function. If given soon after birth, children may not develop significant disabilities. Slightly older children may avoid requiring a feeding or breathing tube and may be capable of some movement.
Da Silva had never heard of SMA when Lorenzo was diagnosed at 6 months of age, in 2013. His development had been normal, when his progress suddenly stopped. The doctors said nothing could be done.
When Da Silva found out in 2019 that she was unexpectedly pregnant with Levi, doctors said it was possible she might never have SMA disabilities if she could get him Zolgensma soon after he was born. The family had been struggling to support themselves. Da Silva had resigned from her job as a travel agent to fight for care and therapy for Lorenzo; her husband, Azen Balbino, had spells of unemployment in Brazil’s recession. They knew they would have 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 therapy it would not otherwise provide.
While da Silva was still pregnant, she recruited Viviane Guimarães, a lawyer from her hometown of Recife, who agreed to take her case. When Guimarães pleaded with the judge for a very expensive therapy, she argued that she would save the government money compared to the other drugs and care Levi would need without her.
Lorenzo receives three types of therapy every day. A physical therapist visits him twice a day to beat on his chest and suck out secretions that would otherwise suffocate him. He has health assistants by his side 24 hours a day.
While Levi hasn’t reached all of his age milestones, he is mobile and talks. He has daily physical and occupational therapy and continues to progress in his development.
Every time he sees his two sons together, Da Silva is reminded of Zolgensma’s awesome power. “Levi talks like a kid who doesn’t have SMA,” he said.
Until now, Novartis has booked $3.7 billion in revenue from Zolgensma, charging different prices in different places. The treatment has not become a mega-success in part because so few patients are eligible for it. And sales have started to decline. Middle-income countries like Brazil could bring many more patients. Novartis has already gained coverage for Zolgensma in Russia, Egypt and, most recently, Argentina. It continues in negotiations in more than 10 countries, including Ecuador.
In Brazil, the government has agreed to cover treatment only for babies with the most severe form of SMA who can breathe independently for at least most of the day. They must also be less than 6 months old. Brazil has agreed to pay for Zolgensma for no more than 250 babies over the next two years. If the demand is greater than that, Novartis will offer 40 free treatments. At most, the government would spend about $50 million on this first group of children this year and a total of $200 million over the next four years.
By comparison, a program known as the village pharmacy had a budget last year equivalent to about $380 million to serve more than 21 million people, providing medicines for conditions such as asthma, diabetes and high blood pressure. The public health system provides care to more than half of Brazil’s approximately 216 million inhabitants. Clinics and hospitals in low-income areas lack basic supplies and more sophisticated technology than X-ray machines.
“We are talking about a super expensive medicine,” said 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 will have to feel comfortable with the discussion, because there will be many more therapies like this,” he added.
By: REBECCA ROBBINS and STEPHANIE NOLEN
BBC-NEWS-SRC: https://www.nytimes.com/2023/01/24/health/gene-therapies-cost-zolgensma.html, IMPORTING DATE: 2023-02-02 22:10:05
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