gene therapies

How will governments face the cost of medical miracles?


Have you read the NY Times article published early this year?

A Mother’s plight:

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 her case to court — and won. A judge ruled that the government had to buy the therapy for her 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 SMA, 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.

The impact:

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.

Worldwide ramifications:

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.

A numbers game:

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.

Brazil’s experience with Zolgensma shows the challenges that the breath-taking prices of these therapies will pose for governments and insurers with limited budgets. Those challenges are poised to multiply in the next few years as more such treatments become available for larger groups of patients.

Read next:

Overcoming the affordability hurdle: Are payers willing to pay for innovation for gene therapies and CAR-Ts and does a high price lead to commercial success?


How to face the cost of high medical miracles ( Accessed 16/02/2023

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