Sickle cell patients left behind as NICE declines gene editing therapy Exa-cel on NHS.
Join the fight for sickle cell treatment! The groundbreaking gene therapy, Exa-cel, could transform lives. Approved by regulators in both the UK and US, it’s a beacon of hope for the 17,000+ people with sickle cell in the UK. It is particularly important for the many people with severe sickle cell who do not have a suitable stem cell donor. However, NICE’s (The National Institute for Health and Care Excellence) decision not to fund it is a setback. Let’s urge for change. Act now to demand access to life-changing treatment. Exa-cel, is a new gene editing therapy for the treatment of sickle cell disease. NICE was considering whether to fund this treatment on the NHS in England.
What is Exa-cel?
Exa-cel (brand name Casgevy) is a new MHRA-licensed treatment for severe sickle cell in individuals aged 12 and above without a matched sibling donor for stem cell transplant. If successful, Exa-cel offers a potential cure for sickle cell and would mark the UK’s first gene therapy for this condition. Manufactured by Vertex, Exa-cel’s proposed cost to the NHS remains undisclosed, but it’s anticipated to be among the most expensive medicines ever reviewed by NICE.
Who are NICE and what did they decide?
The National Institute for Health & Care Excellence (NICE) is the independent public body that recommends whether new treatments should be funded by the NHS in England as a cost-effective use of NHS funds. Its decisions are usually also accepted by the NHS in Wales and Northern Ireland. Decisions about new medicines are made separately in Scotland, by the Scottish Medicines Consortium (SMC).
On March 14th, NICE released a preliminary decision against recommending Exa-cel for sickle cell treatment. According to NICE, based on the information provided by Vertex Pharmaceuticals and clinical/patient experts, Exa-cel may not be a cost-effective allocation of NHS resources. Additionally, they noted significant uncertainty regarding the evidence for Exa-cel, including uncertainty about the severity of sickle cell and the long-term benefits to patients.
It's not over just yet - we need you to campaign for the future of sickle cell patients...
Is this a final decision?
No, this is not a final decision. NICE has opened a public consultation to seek the public’s views on the draft decision. The public consultation is open until the 11th of April 2024 at 5pm. Once the consultation closes, NICE will meet again on the 8th of May 2024 alongside the selected clinical and patient experts and Vertex Pharmaceuticals and will issue its final decision.
NICE must hear from you and your loved ones, gathering as many voices from the community as possible before their upcoming discussion on Exa-cel. Here’s what to communicate with NICE:
- The significant negative impact of severe sickle cell on daily life.
- How sickle cell worsens over time.
- The effects of sickle cell on life expectancy and mortality risk.
What’s next?
Anthony Nolan and the Sickle Cell Society urge sickle cell patients, families, clinicians, and anyone with experience with sickle cell to share their perspectives on making Exa-cel available to the NHS.
How can I respond to the consultation?
The key action for members of the patient and clinical community is to respond to the NICE consultation which is open until 5pm on the 11th of April 2024. You can respond by email with your comments to public.affairs@anthonynolan.org or info@sicklecellsociety.org
Dear NICE,
As a member of the African and Caribbean community, I am writing to express my deep concern and disappointment regarding the recent decision by the National Institute for Health and Care Excellence (NICE) to potentially stop the availability of gene therapy for sickle cell patients in the United Kingdom.
This decision is a devastating blow to our community, which has long faced significant health inequalities, especially when it comes to sickle cell disease.
Sickle cell disease is a genetic blood disease that predominantly affects individuals of African and Caribbean descent.
In the UK, it is estimated that 1 in every 2,000 babies born are affected by sickle cell disease, with the majority being of Black African or Black Caribbean heritage.
This chronic condition causes immense pain, organ damage, and a significantly reduced life expectancy. Shockingly, the average life expectancy for someone with sickle cell disease in the UK is just 40-60 years, compared to the national average of 81 years.
The impact of sickle cell disease on the Black community is staggering. In England, Black people are over 80 times more likely to have sickle cell disease compared to White people.
Furthermore, the mortality rate for sickle cell patients in the UK is alarmingly high, with an estimated 1 in 16 individuals with sickle cell disease dying before the age of 18.
These statistics highlight the urgent need for effective treatments like the CRISPR gene editing therapy to be made available to the African and Caribbean community.
The health inequalities faced by the African and Caribbean community in the UK are well-documented.
Black people are more likely to experience poverty, discrimination, and barriers to accessing quality healthcare.
A report by the NHS Race and Health Observatory found that Black people in the UK have a life expectancy that is up to four years shorter than their White counterparts.
Furthermore, Black people are more likely to experience poor health outcomes across a range of conditions, including sickle cell disease.
Gene therapy offers a revolutionary approach to treating sickle cell disease by addressing the root cause of the condition.
By modifying the patient’s own stem cells to produce healthy red blood cells, gene therapy has the potential to alleviate the painful symptoms and complications associated with sickle cell disease.
This innovative treatment can greatly improve the quality of life for those affected and reduce the burden on the healthcare system in the long run.
It is crucial that NICE reconsiders its decision and ensures that gene editing therapy remains available to sickle cell patients in the UK, particularly those from the African and Caribbean community who are disproportionately affected by this condition.
Denying access to this potentially transformative treatment would be a significant setback in the fight against health inequalities and would leave our community without hope for a better future.
Moreover, the decision to stop the availability of gene editing therapy for sickle cell patients sends a troubling message about the priorities of our healthcare system.
It suggests that the lives and well-being of those affected by this condition, who are predominantly from the African and Caribbean community, are not valued as highly as they should be.
This is unacceptable and goes against the principles of equality and fairness that should underpin our society.
I urge NICE to reverse its decision and work towards making gene therapy for sickle cell disease more widely accessible.
The African and Caribbean community in the UK deserves the same opportunities for improved health outcomes as any other group.
By investing in and supporting this innovative treatment, we can take a significant step towards addressing the glaring health inequalities that have persisted for far too long.
Thank you for your attention to this critical matter.
I sincerely hope that NICE will reconsider its position and prioritise the health and well-being of the African and Caribbean community in the UK, especially those affected by sickle cell disease.
Yours sincerely,
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