AlphaMED Resources · https://alphamedicine.uk/Resources/cases/casgevy
Casgevy and NHS funding
Casgevy is a gene editing treatment for sickle cell disease and beta thalassaemia, priced at £1,651,000 per course. NICE recommends it with managed access for a narrow group while more evidence is collected.
- MHRA authorises the treatment for sickle cell and beta thalassaemia
- NHS England announces that patients can start receiving it
- NICE publishes guidance recommending it with managed access
- Vertex reinfuses 64 patients worldwide
- Vertex reports $76 million of Casgevy revenue for the second quarter
Summary
- Casgevy is a gene editing treatment for sickle cell disease and beta thalassaemia, made by Vertex and CRISPR Therapeutics. The Medicines and Healthcare products Regulatory Agency (MHRA) licensed it for people aged 12 and over.
- The problem is price against uncertainty. The list price is £1,651,000 per course. NICE, which advises the NHS, must decide whether that is worth paying. The discount is commercial in confidence.
- NICE's usual limit is £20,000 per quality adjusted life year. It accepted a higher estimate because of health inequalities in sickle cell, the treatment's innovation and benefits to carers.
- On 26 February 2025 NICE recommended it with managed access as an option for people with at least two crises a year for two years, one of three genotypes, and no matched donor for a transplant.
- Patients first receive busulfan conditioning, a chemotherapy with a fertility risk. The product information has no data on Casgevy's effect on fertility and advises considering freezing sperm or eggs if possible.
- NICE says more evidence is being collected, and the treatment is available with managed access until it decides. We found no published count of NHS patients treated, and Vertex reinfused 64 patients worldwide in 2025.
Dig deeper
- NHS England says sickle cell mainly affects Black African and Black Caribbean people, and that about 15,000 people live with it in England. A parliamentary group on sickle cell and thalassaemia reported in 2021 that the weight of evidence suggests negative attitudes towards patients are often underpinned by racism.
- A February 2026 study in Gene Therapy found Casgevy could be cost effective in Uganda at a scaled cost when societal benefits are counted. It assumed the treatment works in Ugandan patients as it did in the US trials. The authors are listed on the linked page.
- In its second quarter 2026 results, Vertex reported Casgevy revenue of $76 million, up 78% on the previous quarter.
- The product information asks for a check at least once a year for 15 years and notes a theoretical cancer risk from gene editing.
Ethical issues
| Principle | How it applies here |
|---|---|
| Autonomy | A child under 16 can consent with sufficient understanding and intelligence to understand fully what is proposed (Gillick, 1985). At 16 and 17, consent counts as an adult's under section 8 of the Family Law Reform Act 1969. The adult duty in Montgomery to explain material risks, including busulfan's fertility risk, then applies. |
| Beneficence | The aim is to stop recurrent crises. NICE noted the trial had no comparator and was small, so the benefit is uncertain. That benefit must be weighed against conditioning chemotherapy with a known fertility risk. |
| Non maleficence | The harms are busulfan chemotherapy and effects not yet known. Yearly checks for 15 years guard against a theoretical cancer risk from gene editing, and managed access means patients help gather the evidence. |
| Justice | NICE accepted a price above its usual limit partly for health inequalities in a condition that mainly affects Black African and Black Caribbean people. Justice asks whether that equity case justifies £1,651,000 per course, and whether a confidential discount can be held to account by the other patients whose care it competes with. |
Interview questions
- What do you know about Casgevy, and what does it treat?
- What did NICE recommend for sickle cell disease in 2025, and why is the recommendation narrow?
- A 14 year old with sickle cell disease asks whether to have Casgevy. What do you need to check first, and what do you tell them about fertility?
- Is it fair for the NHS to pay £1,651,000 for one course of treatment when the same money could fund care for many other patients?
- What is the case for paying a high price for a rare treatment, and what is the case against it?
- How would you explain the long term effects of a treatment to a patient when the evidence is still being collected?
- Why is sickle cell disease treated as a health inequalities issue, and how would you test that claim?
Sources
- GOV.UKgov.uk/…/mhra-authorises-world-first-gene-therapy-that-aims-to-cure-s
- NICEnice.org.uk/…/information-about-exagamglogene-autotemcel
- NICEnice.org.uk/…/committee-discussion
- NICEnice.org.uk/…/recommendations
- ema.europa.euema.europa.eu/…/casgevy-epar-product-information_en.pdf
- NICEnice.org.uk/…/ta1044
- biopharmadive.combiopharmadive.com/…/812243
- NHS Englandengland.nhs.uk/…/revolutionary-gene-editing-therapy-for-sickle-cell
- sicklecellsociety.orgsicklecellsociety.org/…/No-Ones-Listening-Final.pdf
- nature.comnature.com/…/s41434-026-00598-1
- biospace.combiospace.com/…/vertex-reports-second-quarter-2026-financial-results
- lawcases.netlawcases.net/…/gillick-v-west-norfolk-and-wisbech-aha-1985-ukhl-7-17-octobe
- legislation.gov.uklegislation.gov.uk/…/8
- UK Supreme Courtsupremecourt.uk/…/uksc_2013_0136_judgment_fd5635b4cd.pdf
Photo: Erhabor Osaro, CC BY-SA 3.0, via Wikimedia Commons, colour altered.
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