A Dilemma for Governments: How to Pay for 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 eldest son paralyzed and nearly mute. But this time there was a treatment available 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, refused to pay for it.
So Ms. Da Silva went to court — and won. A judge ruled the government should buy the therapy for her youngest son, Levi.
Today, 2-year-old Levi chatters, claps and crawls, all things his older brother Lorenzo, 10, never got to do.
The treatment, called Zolgensma, a unique infusion, is among the first in a new class of cutting-edge gene therapies that hold huge promise for people with life-threatening diseases or debilitating - at extremely high prices. Its maker, pharmaceutical company Novartis, has brokered deals with national health systems and insurers to ensure the drug is covered in many wealthier countries.
Now, with its sales slowing, the company is striving to gain broad coverage in middle-income countries like Brazil, where public health systems are often fragile and underfunded.
Zolgensma, which treats a rare genetic condition known as spinal muscular atrophy, or SMA, was for some time the most expensive treatment in the world. It has become a closely watched test of whether such therapies can gain widespread coverage around the world, and what the trade-offs might be.
The experience of Brazil with Zolgensma shows the challenges that the staggering prices of these therapies will place on governments and insurers with limited budgets. These challenges are set to multiply over the next few years as more and more such treatments become available for larger groups of patients.
After more than 100 successful lawsuits of families forcing the Brazilian public health system to pay for their children's 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 approximately $1 million for each treatment – much less than some other governments pay, but still a staggering sum for Brazil's strained healthcare system.
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 eldest son paralyzed and nearly mute. But this time there was a treatment available 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, refused to pay for it.
So Ms. Da Silva went to court — and won. A judge ruled the government should buy the therapy for her youngest son, Levi.
Today, 2-year-old Levi chatters, claps and crawls, all things his older brother Lorenzo, 10, never got to do.
The treatment, called Zolgensma, a unique infusion, is among the first in a new class of cutting-edge gene therapies that hold huge promise for people with life-threatening diseases or debilitating - at extremely high prices. Its maker, pharmaceutical company Novartis, has brokered deals with national health systems and insurers to ensure the drug is covered in many wealthier countries.
Now, with its sales slowing, the company is striving to gain broad coverage in middle-income countries like Brazil, where public health systems are often fragile and underfunded.
Zolgensma, which treats a rare genetic condition known as spinal muscular atrophy, or SMA, was for some time the most expensive treatment in the world. It has become a closely watched test of whether such therapies can gain widespread coverage around the world, and what the trade-offs might be.
The experience of Brazil with Zolgensma shows the challenges that the staggering prices of these therapies will place on governments and insurers with limited budgets. These challenges are set to multiply over the next few years as more and more such treatments become available for larger groups of patients.
After more than 100 successful lawsuits of families forcing the Brazilian public health system to pay for their children's 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 approximately $1 million for each treatment – much less than some other governments pay, but still a staggering sum for Brazil's strained healthcare system.
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