NICE’s initial guidance does not recommend use of betibeglogene autotemcel for treating transfusion-dependent beta thalassaemia in the NHS in England

The National Institute for Health and Care Excellence (NICE) has published guidance on the use of betibeglogene autotemcel (beti-cel) for treating transfusion-dependent beta thalassaemia in the NHS in England. The guidance states that beti-cel is currently not recommended. This is not the final guidance and the appraisal committee is accepting comments and feedback until 4 March 2021. After this date a second appraisal committee meeting will be held (date not yet confirmed) to discuss any new feedback before releasing a final appraisal document. This final document will form the basis for NICE’s guidance on the use of beti-cel in the NHS in England.

The main reasons provided by NICE for not recommending the use of beti-cel are:

  • Haematopoietic stem cell transplantation (bone marrow transplant) is the preferred curative treatment option where possible.
  • Whilst the trial data is positive, the trial samples are small and are relatively new so the long-term effects are unknown.
  • Cost uncertainties and the perceived high cost of the treatment raises NHS resource allocation considerations.


The NICE publication acknowledges the debilitating nature of thalassaemia and the impact on a patient’s life of having frequent blood transfusions and chelation treatment. However, it refers to a UK patient preference report and states that “only 37% of respondents [with TDT] said they would immediately accept a referral to a transplant specialist and betibeglogene autotemcel if offered it.” This is inconsistent with the findings of United Kingdom Thalassaemia Society (UKTS). Romaine Maharaj, Executive Director at UKTS said: “Most of our members are very excited about the new therapy developments and are keen to explore these treatment options. Bone marrow transplant is only an option for a very small proportion of people with thalassaemia and so gene therapy offers a real potential alternative as a one-off resolution to this life-limiting condition.”


In terms of the charity’s response to the NICE report, Romaine Maharaj issued the following statement:

“Living with thalassaemia is a difficult and challenging experience not just for patients but for their entire families. Our main aim at the UKTS is to improve the quality of life and experience of people living with thalassaemia through education, one-to-one support, research, service improvements and policy changes. We aspire to provide our members with empathy and the necessary hope to be optimistic about their futures. We are extremely disappointed with NICE’S decision not to recommend Betibeglogene autotemcel as a treatment option in the UK. We also feel disheartened that our patient experts were misquoted and used out of context and feel that NICE needs to rectify this. Having an option and the access to a potentially curable treatment is vital and should be offered to patients”.


For further information, please visit  or contact Romaine Maharaj on 0208 882 0011.


Please  visit the following link  to have your say:

Consultation closes on March 4th 2021.


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