ACTION NEEDED : Your Voice Matters in the Decision on Exa-cel Gene Therapy 

News
04 Apr 2024

ACTION NEEDED : Your Voice Matters in the Decision on Exa-cel Gene Therapy 

Exa-cel (CASGEVY), a new gene therapy for sickle cell disorder treatment, is currently under consideration by the National Institute for Health and Care Excellence (NICE) for potential funding on the NHS in England. However, on 14 March, NICE released a draft decision not to recommend Exa-cel for NHS funding.

We were very disappointed to hear this, but there is positive news : this is not the final decision. Until 11 April you can put your views forward to help NICE to consider how challenging it is to live with sickle cell, and how needed this therapy, and potential functional cure, is – before they come to a final decision.

We’re working with the Anthony Nolan Charity to get as many voices in the sickle cell community as possible to be heard by NICE.

NICE’s decision comes from concerns around the cost-effectiveness of Exa-cel. They have also said that there is too much uncertainty in the evidence about exa-cel they have received. In particular, NICE are not certain about the severity of sickle cell or the magnitude of the long-term benefits to patients of exa-cel.

The crucial action for sickle cell patients, their families and clinical communities is to participate in the NICE consultation, open until 11 April 2024. It’s vital for NICE to hear from as many voices in the community as possible before their next meeting to discuss Exa-cel. The key things to share with NICE are:

  • The negative impact that severe sickle cell has on the day to day life of people living with the condition
  • The worsening impact that sickle cell has over time
  • The impact of sickle cell on life expectancy and the risk of mortality

Is this decision final? No, it’s not. NICE has started a public consultation to gather public feedback on the draft decision. The consultation remains open until 11 April 2024. Following the consultation period, NICE will convene again to make its final decision, although the exact date for this decision is currently unknown.

Your participation in the consultation can make a significant difference in shaping the outcome for Exa-cel and the future of sickle cell treatment. Let’s ensure NICE hears the voices of our community loud and clear. Please take action today. You can go straight to the consultation using the link below, or you can email your comments to us at info@sicklecellsociety.org and we’ll respond on your behalf.

NICE EXA CEL CONSULTATION PAGE

How to comment on a consultation using our online system (youtube.com)