Response to the NICE Consultation on the Recommendation of Exa-cel for Severe Sickle Cell Treatment

I am writing on behalf of the All-Party Parliamentary Group for Ethnicity Transplantation and Transfusion to submit our response to the consultation on the draft recommendation concerning exa-cel gene therapy for the treatment of severe sickle cell disease. Many of the Groups stakeholders and supporters are sickle cell patients.

The Group recognises the critical role of NICE in ensuring that treatments recommended for use within the NHS are both effective and provide value for money and we appreciate the complexity and the rigorous evaluation processes involved in such decisions. However, we are concerned about the preliminary decision not to recommend exa-cel for NHS use, given its potential as a transformative treatment for individuals suffering from severe sickle cell disease.

Sickle cell disease significantly impacts the quality of life of those affected, with severe cases frequently experiencing excruciating pain, debilitating fatigue, and increased susceptibility to infections and organ damage. The introduction of exa-cel as a treatment option represents a significant advancement in potentially offering a curative solution to individuals who have lived with the condition’s severe implications on their daily lives.

For many patients with severe sickle cell disease, the lack of a suitable stem cell donor limits their treatment options. Exa-cel presents an innovative alternative that could circumvent this limitation, offering hope to those who currently have few, if any, options for a cure.

While we understand the concerns regarding the cost-effectiveness of exa-cel, we urge NICE to consider the long-term benefits of providing a cure for sickle cell disease. These benefits include the potential reduction in hospital admissions, the need for ongoing treatments, and the overall improvement in patients’ life expectancy and quality of life.

Additionally, offering a cure can have profound implications for mental health, employment opportunities, and the ability to lead a normal, productive life.

In light of these points, we respectfully request that NICE reconsider its draft recommendation on exa-cel. We believe it is essential to factor in the broader implications of this treatment, not only in terms of direct healthcare costs but also in its potential to significantly improve the lives of those affected by severe sickle cell disease.

We are committed to engaging with NICE, healthcare professionals, and patient advocacy groups to ensure that the voices and needs of sickle cell patients are adequately represented and considered in this crucial decision-making process.

Thank you for your consideration of our response.

Yours sincerely

Yaser Martini

For and on behalf of the All-Party Parliamentary Group : Ethnicity Transplantation and Transfusion