New sickle cell therapies will be out of reach where they are needed most
There is no clear pathway for African patients to access the treatments, which cost several million dollars and are very complex to manufacture and to provide.< /p>
The Food and Drug Administration's approval Friday of two breakthrough gene therapy treatments for sickle cell disease brought a rare moment of hope and celebration to people with this distressing blood disease. But there is no clear path forward for new therapies – one-off treatments so effective in clinical trials that They have been hailed as cures – to reach countries where the vast majority of people with sickle cell disease live. Shortly after approval, their manufacturers announced multimillion-dollar prices: $3.1 million for Lyfgenia, made by Bluebird Bio, and $2.2 million for Casgevy, made by Vertex Pharmaceuticals.< /p>
Lyfgenia will launch in the United States. Vertex has prioritized gaining approval in six wealthy countries: the United States, Italy, Britain, France, Germany and Saudi Arabia - which, by one estimate, home to 2% of the world's sickle cell population.
Three-quarters of the world's sickle cell patients are found in sub-Saharan Africa. Several million are estimated to be sick enough to benefit from the new therapies, compared with about 20,000 in the United States.
Many African patients have been closely monitored. following online news of the treatments' success in clinical trials. In Tanzania, information about Casgevy spread a few months ago thanks to a WhatsApp group created by Shani Mgaraganza for mothers of children with sickle cell disease. Her son, Ramadhani, 12, and daughter Nasra, 10, suffer from a hereditary disease, which causes episodes of burning pain and damages their organs. She said the therapy felt like a miracle.
"Everyone was like, 'Thank God our kids are going to be OK,'" she said.
Then the mothers learned what it would likely cost. “This would amount to billions of Tanzanian shillings,” Ms Mgaraganza said. “No one can afford it. It was demoralizing. -editing method, CRISPR, which underpins Casgevy. “Today this will not be widely available,” she said. "Now that we have this approval, we really need to think about how we're going to open it up to more people."
Two key factors are preventing it from exist. the reach of patients in Africa.
First, the price: treatments are far too expensive for governments struggling to pay for basic health services. In some cases, there may be substantial additional costs, such as for a patient's extended hospital stay to receive gene therapy.
The second barrier is the medical infrastructure: Administering the treatment is a months-long process in medical centers capable of performing stem cell transplants. Patients must have their cells harvested and flown to a laboratory for manufacturing, undergo grueling chemotherapy and endure a lengthy hospitalization.
"A doctor...
There is no clear pathway for African patients to access the treatments, which cost several million dollars and are very complex to manufacture and to provide.< /p>
The Food and Drug Administration's approval Friday of two breakthrough gene therapy treatments for sickle cell disease brought a rare moment of hope and celebration to people with this distressing blood disease. But there is no clear path forward for new therapies – one-off treatments so effective in clinical trials that They have been hailed as cures – to reach countries where the vast majority of people with sickle cell disease live. Shortly after approval, their manufacturers announced multimillion-dollar prices: $3.1 million for Lyfgenia, made by Bluebird Bio, and $2.2 million for Casgevy, made by Vertex Pharmaceuticals.< /p>
Lyfgenia will launch in the United States. Vertex has prioritized gaining approval in six wealthy countries: the United States, Italy, Britain, France, Germany and Saudi Arabia - which, by one estimate, home to 2% of the world's sickle cell population.
Three-quarters of the world's sickle cell patients are found in sub-Saharan Africa. Several million are estimated to be sick enough to benefit from the new therapies, compared with about 20,000 in the United States.
Many African patients have been closely monitored. following online news of the treatments' success in clinical trials. In Tanzania, information about Casgevy spread a few months ago thanks to a WhatsApp group created by Shani Mgaraganza for mothers of children with sickle cell disease. Her son, Ramadhani, 12, and daughter Nasra, 10, suffer from a hereditary disease, which causes episodes of burning pain and damages their organs. She said the therapy felt like a miracle.
"Everyone was like, 'Thank God our kids are going to be OK,'" she said.
Then the mothers learned what it would likely cost. “This would amount to billions of Tanzanian shillings,” Ms Mgaraganza said. “No one can afford it. It was demoralizing. -editing method, CRISPR, which underpins Casgevy. “Today this will not be widely available,” she said. "Now that we have this approval, we really need to think about how we're going to open it up to more people."
Two key factors are preventing it from exist. the reach of patients in Africa.
First, the price: treatments are far too expensive for governments struggling to pay for basic health services. In some cases, there may be substantial additional costs, such as for a patient's extended hospital stay to receive gene therapy.
The second barrier is the medical infrastructure: Administering the treatment is a months-long process in medical centers capable of performing stem cell transplants. Patients must have their cells harvested and flown to a laboratory for manufacturing, undergo grueling chemotherapy and endure a lengthy hospitalization.
"A doctor...
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