Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what discussions she has had with (a) NICE and (b) NHS England on encouraging the adoption of new treatments for (i) sickle cell disease and (ii) beta-thalassaemia.

Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)

The Department regularly discusses a range of issues with colleagues in NHS England and the National Institute for Health and Care Excellence (NICE), related to patient access to new treatments.

The NICE appraises all new licensed medicines, and its recommendations are developed independently in line with its established methods and processes, on the basis of an assessment of the available evidence, and through extensive engagement with stakeholders. NHS England is legally required to fund treatments recommended in NICE technology appraisal guidance.

Question to the Foreign, Commonwealth & Development Office:

To ask His Majesty's Government what representations they have made to the government of Pakistan on ensuring the availability of thalassaemia treatment and awareness; and what allocations of the Official Development Aid is being put towards this.

Answered by Lord Ahmad of Wimbledon - Minister of State (Foreign, Commonwealth and Development Office)

The UK uses Official Development Assistance (ODA) to support Pakistan in realising its ambition of Universal Health Coverage (UHC) and ending preventable deaths of mothers, babies and children. We take a systems approach to this, providing technical assistance to strengthen decision making on health policy and financing. We also build the evidence base on effective sub-system interventions for UHC. No specific investments are made in Thalassaemia treatment or awareness raising in Pakistan. However, our support will strengthen core components of Pakistan's health system and increase coverage and service offers that should be of benefit to those with Thalassaemia.

Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what discussions she has had with NHS England and the National Institute for Health and Care Excellence on access to new treatments for sickle cell disease and beta thalassaemia.

Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)

The Department regularly discusses a range of issues with colleagues in NHS England and the National Institute for Health and Care Excellence (NICE) related to patient access to effective new treatments. NICE appraises all newly licensed medicines and aims to publish draft guidance around the time of licensing wherever possible. NICE works closely with the Medicines and Healthcare products Regulatory Agency and NHS England to ensure that there is a joined-up, timely approach to supporting access to new treatments for National Health Service patients.

NICE is currently appraising exagamglogene autotemcel for treating transfusion-dependent beta-thalassaemia and sickle cell disease, and expects to publish final guidance in March and April 2024 respectively.

Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what assessment he has made of the potential impact of the withdrawal of Bluebird Bio from the (a) UK and (b) European market on access to gene therapy treatment for people with (i) beta thalassemia and (ii) cerebral adrenoleukodystrophy.

Answered by Will Quince

No assessment has been made of the potential impact of the withdrawal of Bluebird Bio from the United Kingdom and European markets on access to gene therapy treatment for people with beta thalassemia and cerebral adrenoleukodystrophy. NHS England continues to make cost-effective treatments available to patients in England as determined by NICE’s technology appraisal and highly specialised technologies programmes.

Found: homozygous or double heterozygous sickle cell disease, hereditary spherocytosis, homozygous α or β thalassaemia

Found: homozygous or double heterozygous sickle cell disease, hereditary spherocytosis, homozygous α or β thalassaemia

Found: screening - coverage ST2 - Antenatal sickle cell and thalassaemia screening - timeliness of antenatal

Found: screening - coverage ST2 - Antenatal sickle cell and thalassaemia screening - timeliness of antenatal

Found: Updated England Region No additional breakdown All ages Yes OHID C24k - Sickle Cell and Thalassaemia

Found: screening - coverage ST2 - Antenatal sickle cell and thalassaemia screening - timeliness of antenatal

Found: coverage: antenatal screeningThe proportion of pregnant women eligible for antenatal sickle cell and thalassaemia

Found: offer of prenatal diagnosis (PND) to women at risk of having an infant with sickle cell disease or thalassaemia

Found: In people with β-thalassaemia, it can lead to severe anaemia.