How are you taking part in this consultation?

You will not be able to change how you comment later.

You must be signed in to answer questions

  • Question on Consultation

    Has all of the relevant evidence been taken into account?

  • Question on Consultation

    Are the summaries of clinical and and cost effectiveness reasonable interpretations of the evidence?

  • Question on Consultation

    Are the recommendations sound and a suitable basis for guidance to the NHS?

  • Question on Consultation

    Are there any aspects of the recommendations that need particular consideration to ensure we avoid unlawful discrimination against any group of people on the grounds of race, gender, disability, religion or belief, sexual orientation, age, gender reassignment, pregnancy and maternity?
The content on this page is not current guidance and is only for the purposes of the consultation process.

Betibeglogene autotemcel for treating transfusion-dependent beta-thalassaemia

Closed for comments This consultation ended on at   Request commenting lead permission

2 Information about betibeglogene autotemcel

Marketing authorisation indication

2.1 Betibeglogene autotemcel (Zynteglo, bluebird bio) is indicated for 'the treatment of patients 12 years and older with transfusion-dependent beta-thalassaemia (TDT) who do not have a beta0/beta0 genotype, for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available'.

Dosage in the marketing authorisation

2.2 The dosage schedule is available in the summary of product characteristics.

Price

2.3 The cost of betibeglogene autotemcel 1.2 to 20x106 cells/ml dispersion for a one-time infusion is £1,450,000 per patient (list price, excluding VAT). The company has a commercial arrangement, which would have applied if the technology had been recommended.