Sickle cell disease: A long life with sickle cell disease, wherever a child is born.
About 7.74 million people live with sickle cell disease, most of them in sub-Saharan Africa. A daily medicine, hydroxyurea, with lab checks, costs about $336 a child a year there, our arithmetic.
Ryan K. Hertel · heyitskenton
The problem
What we're up against.
Common, and growing.
The number of people living with sickle cell disease rose to 7.74 million in 2021, and 515,000 babies were born with it that year.
Mostly in Africa.
Sub-Saharan Africa has 79% of the births, and 5.68 million people living with the disease.
Deadlier than the records show.
Counting every death it contributes to, sickle cell disease took about 376,000 lives in 2021, nearly eleven times the deaths recorded as its cause, including 81,100 children under five.
Childhood in danger.
In Africa, an estimated 50% to 90% of children born with the most severe form die in early childhood.
In America, too.
About 1 in every 350 Black newborns in America has sickle cell disease, about 100,000 Americans live with it, and life expectancy at birth with the disease is about 52.6 years.
What works
Proven, and ready to grow.
A daily medicine.
In a large trial in four African countries, deaths among children taking hydroxyurea fell from 3.6 to 1.1 per hundred patient-years, though without a separate comparison group, and 94.2% stayed on treatment for three years.
Safe where malaria is common.
In a randomized trial in Uganda, hydroxyurea was safe for children where malaria is common, and sickle cell complications struck 45% of children on it, against 69% on placebo.
The right dose matters.
In another randomized trial in Uganda, 86% of children on a gradually raised dose reached the treatment targets, against 37% on a fixed dose.
Screening and penicillin.
Penicillin protects young children with the disease from serious infections, and screening newborns, with prevention for those who have it, costs about $184 for each healthy year of life gained in sub-Saharan Africa.
Savings, and cures.
In Uganda, full-dose hydroxyurea saved about $191 a patient compared with care without it, and in America, a gene therapy freed nearly all its trial patients from pain crises for at least a year.
What a family could fund
Where the money goes furthest.
Screening that needs a next step.
The American Society of Hematology's newborn screening network in Africa pays for staff and lab supplies, but not for hydroxyurea, a gap a family could fill.
Medicine for everyone who needs it.
At about $336 a year, keeping all 5.68 million people with the disease in sub-Saharan Africa on hydroxyurea would cost roughly $1.9 billion a year, our arithmetic, and more for adults' larger doses.
Gene therapy, at a negotiated price.
In America, a federal program covering 84% of Medicaid patients with the disease negotiates gene therapy prices, and makes the makers pay for fertility preservation. A family's initiative can fund what it doesn't, like the people who help patients through care.
Honest limits
What money can't promise.
Screening alone saves no one.
Only about a third of babies who screened positive got a confirming test, and many never reached follow-up care, often because families couldn't afford to get to the clinic.
Medicine runs out.
78% of clinicians surveyed reported hydroxyurea running short, and at one Ugandan clinic, 84% of caregivers lived on less than $1.90 a day.
Gene therapy is hard, and still rare.
It needs chemotherapy that usually causes infertility, one version carries a blood cancer warning, and the other was given to just 64 people worldwide in 2025, by its maker's count.
The evidence has gaps.
Randomized trials of hydroxyurea found no difference in deaths, and its long-term risks, including to fertility, aren't yet known.
Drugs can fail, and go unused.
One sickle cell drug was withdrawn in 2024 because its benefits didn't outweigh its risks, and even in America, only 38% to 53% of children with the disease on Medicaid used hydroxyurea.
Hard questions
Asked and answered.
You might ask
Gene therapy is a cure. Why not just pay for cures?
Ryan
One list price, $2.2 million, would pay for about 6,548 child-years of hydroxyurea, our arithmetic. The therapy isn't approved in Africa, where most patients live, and medicines like hydroxyurea are within reach in many more places.
You might ask
Is the proof strong that hydroxyurea saves lives?
Ryan
Strong on safety and illness, weaker on deaths: it's safe where malaria is common, deaths fell sharply in the four-country trial without a separate comparison group, and randomized trials found no difference in deaths.
You might ask
Won't programs collapse when the donor leaves?
Ryan
That's the main risk. But full-dose hydroxyurea saves health systems about $191 a patient in Uganda, which gives governments a reason to take it over.
In the slider
See what your fortune could do here.
Sickle cell disease
The slider prices this cause with these numbers:
- About $336 keeps a child with sickle cell disease in sub-Saharan Africa on hydroxyurea, with lab checks, for a year: our arithmetic from African drug prices and a Ugandan costing.
- The whole need here is everyone with the disease in sub-Saharan Africa, about 5.68 million people.
- Finding children and keeping them in care is the real bottleneck, and costs extra: in one screening network, only about a third of babies who screened positive got a confirming test.
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Sources
Every number in this chapter, and where it came from.
The Lancet Haematology, 15 June 2023. Checked 16 October 2026.
A global model. Its numbers use thin spaces and a raised decimal point, which the checks keep as printed.
Blood (American Society of Hematology annual meeting), 2 November 2023. Checked 16 October 2026.
A survey of 120 clinicians, presented as a conference abstract.
PharmacoEconomics, 18 July 2023. Checked 16 October 2026.
A model costed at a clinic in Kampala, in 2021 dollars. Lab monitoring costs $104 a year after the first, in its cost table.
American Journal of Preventive Medicine, December 2011. Checked 16 October 2026.
Centers for Disease Control and Prevention (MMWR), 28 March 2024. Checked 16 October 2026.
Source 6: FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease (opens in a new tab)
US Food and Drug Administration, 8 December 2023. Checked 16 October 2026.
Blood Advances, 1 July 2023. Checked 16 October 2026.
Source 8: Hydroxyurea for Children with Sickle Cell Anemia in Sub-Saharan Africa (REACH) (opens in a new tab)
New England Journal of Medicine, 1 December 2018. Checked 16 October 2026.
Children in four countries, compared with their own time before treatment, not with a separate group.
Blood, 19 October 2017. Checked 16 October 2026.
A randomized, placebo-controlled trial in Uganda.
Source 10: Hydroxyurea Dose Escalation for Sickle Cell Anemia in Sub-Saharan Africa (opens in a new tab)
New England Journal of Medicine, June 2020. Checked 16 October 2026.
A randomized trial in Uganda.
Cochrane Database of Systematic Reviews, 8 March 2021. Checked 16 October 2026.
Trials in the USA and Jamaica.
BMC Health Services Research, 26 July 2016. Checked 16 October 2026.
Its lead author worked for a drug company.
Source 13: Exagamglogene Autotemcel for Severe Sickle Cell Disease (Frangoul and others) (opens in a new tab)
New England Journal of Medicine, 24 April 2024. Checked 16 October 2026.
A single-group trial without a comparison group.
Blood Advances, 1 May 2026. Checked 16 October 2026.
The society's report on its own program.
Source 15: CGT Access Model Frequently Asked Questions (opens in a new tab)
Centers for Medicare & Medicaid Services, 22 September 2026. Checked 16 October 2026.
Source 16: Cell and Gene Therapy Access Model (opens in a new tab)
Centers for Medicare & Medicaid Services, 30 September 2026. Checked 16 October 2026.
Source 17: Vertex Reports Fourth Quarter and Full Year 2025 Financial Results (opens in a new tab)
Vertex Pharmaceuticals, via BioSpace, 12 February 2026. Checked 16 October 2026.
The maker's own count, for sickle cell disease and a related blood disorder together.
Source 18: Hydroxyurea (hydroxycarbamide) for sickle cell disease (opens in a new tab)
Cochrane Database of Systematic Reviews, September 2022. Checked 16 October 2026.
US Food and Drug Administration, 26 September 2024. Checked 16 October 2026.
Centers for Disease Control and Prevention (MMWR), 30 September 2022. Checked 16 October 2026.
Source 21: Vertex Reports Second Quarter 2026 Financial Results (opens in a new tab)
Vertex Pharmaceuticals, via BioSpace, 3 August 2026. Checked 16 October 2026.
American Journal of Hematology, 1 April 2026. Checked 16 October 2026.