bluebird bios beta-thalassaemia gene therapy betibeglogene autotemcel (beti-cel) has not been recommended by the UKs National Institute of Health and Care Excellence (NICE) for use on the NHS.<\/p>\n
Beti-cel, marketed as Zynteglo in Europe, is a gene therapy intended for the treatment of transfusion-dependent beta-thalassaemia (TDT) in people aged 12 years and older who do not have a beta0\/beta0 genotype.<\/p>\n
It is indicated for TDT patients when haematopoietic stem cell transplantation (HSCT) is appropriate but there is no suitable donor.<\/p>\n
TDT is the most severe form of thalassaemia, a condition wherein an inherited faulty gene leads to the inability of the body to produce normally functioning haemoglobin.<\/p>\n
People living with TDT require life-long blood transfusions every two to five weeks.<\/p>\n
In its draft recommendations, NICE commented that the follow-up on people included in clinical trials of beti-cel was not very long, adding that the population included was small.<\/p>\n
NICE also determined that there were uncertainties around the cost-effectiveness of beti-cel, with the estimate for the gene therapy considerably higher than what it usually deems an acceptable use of NHS resources.<\/p>\n
We are extremely disappointed with NICES decision not to recommend betibeglogene autotemcel as a treatment option in the UK, said Romaine Maharaj, executive director, UK Thalassaemia Society.<\/p>\n
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, she added.<\/p>\n
<\/p>\n
Read more:<\/p>\n
bluebird bio's beta-thalassaemia gene therapy rejected by NICE - PharmaTimes<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" bluebird bios beta-thalassaemia gene therapy betibeglogene autotemcel (beti-cel) has not been recommended by the UKs National Institute of Health and Care Excellence (NICE) for use on the NHS. Continue reading