Asked by: Lord Botham (Crossbench - Life peer)
Question to the Department of Health and Social Care:
To ask His Majesty's Government what assessment they have made of the impact of ineligibility to receive cerliponase alfa for treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) on children born after January 2026; and what steps they are taking to ensure equitable access to treatment for all future patients who are diagnosed with CLN2.
Answered by Baroness Merron - Parliamentary Under-Secretary (Department of Health and Social Care)
In the absence of treatment with cerliponase alfa, clinical management of patients with neuronal ceroid lipofuscinosis type 2 (CLN2) focuses on symptom control, monitoring and preventing complications, and palliative care. The aim is to maintain function for as long as possible and to improve quality of life. Following the next National Institute for Health and Care Excellence Appraisal Committee meeting in July, the National Institute for Health and Care Excellence and NHS England will continue to try to reach an agreement with the manufacturer, based on the committee's preferred assumptions around modelling, that will provide access to cerliponase alfa for all future patients.
Asked by: Lord Botham (Crossbench - Life peer)
Question to the Department of Health and Social Care:
To ask His Majesty's Government what estimate they have made of the number of children born annually in the UK with neuronal ceroid lipofuscinosis type 2 after January 2026, and what alternative treatments will be available should they be ineligible for treatment with Brineura.
Answered by Baroness Merron - Parliamentary Under-Secretary (Department of Health and Social Care)
The National Institute for Health and Care Excellence (NICE) is currently updating its guidance on the use of cerliponase alfa (Brineura) for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) and has not yet published final guidance. NICE published its draft guidance for consultation in May 2025 and was unfortunately unable to recommend cerliponase alfa for routine use when taking into account all the evidence of costs and benefits, including real world evidence collected during the managed access period. We are, however, pleased that NICE and NHS England have been able to reach an agreement with the manufacturer of cerliponase alfa that ensures continued access to treatment for all patients who started on treatment before December 2025.
Officials in the Department have been in regular contact with NICE and NHS England about this appraisal. Following the next NICE Appraisal Committee meeting in July, NICE and NHS England will continue to try to reach an agreement with the manufacturer based on the committee's preferred assumptions around modelling that will provide access to cerliponase alfa for all future patients.
It is estimated that in the UK, approximately three to six children are diagnosed with CLN2 each year. In the absence of treatment with cerliponase alfa, clinical management options focus on symptom control, monitoring and preventing complications, and palliative care. Management aims to maintain function as long as possible and to improve quality of life.
Asked by: Lord Botham (Crossbench - Life peer)
Question to the Department of Health and Social Care:
To ask His Majesty's Government what discussions they have had with BioMarin and the National Institute for Health and Care Excellence regarding the long-term sustainability of access to Brineura for children with neuronal ceroid lipofuscinosis type 2 beyond the expiration of the current managed access agreement.
Answered by Baroness Merron - Parliamentary Under-Secretary (Department of Health and Social Care)
The National Institute for Health and Care Excellence (NICE) is currently updating its guidance on the use of cerliponase alfa (Brineura) for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) and has not yet published final guidance. NICE published its draft guidance for consultation in May 2025 and was unfortunately unable to recommend cerliponase alfa for routine use when taking into account all the evidence of costs and benefits, including real world evidence collected during the managed access period. We are, however, pleased that NICE and NHS England have been able to reach an agreement with the manufacturer of cerliponase alfa that ensures continued access to treatment for all patients who started on treatment before December 2025.
Officials in the Department have been in regular contact with NICE and NHS England about this appraisal. Following the next NICE Appraisal Committee meeting in July, NICE and NHS England will continue to try to reach an agreement with the manufacturer based on the committee's preferred assumptions around modelling that will provide access to cerliponase alfa for all future patients.
It is estimated that in the UK, approximately three to six children are diagnosed with CLN2 each year. In the absence of treatment with cerliponase alfa, clinical management options focus on symptom control, monitoring and preventing complications, and palliative care. Management aims to maintain function as long as possible and to improve quality of life.
Asked by: Lord Botham (Crossbench - Life peer)
Question to the Department of Health and Social Care:
To ask His Majesty's Government what equality impact assessment they have carried out in regard to the decision to end access to Brineura for patients diagnosed with neuronal ceroid lipofuscinosis type 2 after January 2026.
Answered by Baroness Merron - Parliamentary Under-Secretary (Department of Health and Social Care)
The Department has not undertaken any equality impact assessment.
The National Institute for Health and Care Excellence (NICE) is the independent body responsible for developing authoritative, evidence-based recommendations for the National Health Service on whether new medicines represent a clinically and cost-effective use of resources. Equality impact assessments are not undertaken for managed access agreements, but considerations of equality and health inequalities are integral to NICE’s guidance development process.
Managed access gives people faster access to promising new treatments which might otherwise not be recommended, because of uncertainties about their clinical or cost effectiveness.
The managed access agreement (MAA) for cerliponase alfa (Brineura) was put in place to allow eligible patients to be treated, while additional evidence was collected to address significant uncertainties identified by NICE’s independent committee. Without this agreement, the treatment could not be made available at all.
Patients who started treatment during the MAA will continue to be funded. However, if the committee ultimately concludes that the treatment is not a cost-effective use of NHS resources, it cannot be recommended for routine commissioning and new patients would not be eligible for NHS-funded access.
Asked by: Lord Botham (Crossbench - Life peer)
Question to the Department of Health and Social Care:
To ask His Majesty's Government why provision of cerliponase alfa to children with neuronal ceroid lipofuscinosis type 2 will be restricted from January 2026, and what engagement they have had with NHS England, the National Institute for Health and Care Excellence, and BioMarin regarding future access and funding arrangements.
Answered by Baroness Merron - Parliamentary Under-Secretary (Department of Health and Social Care)
The National Institute for Health and Care Excellence (NICE) is currently updating its guidance on the use of cerliponase alfa (Brineura) for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) and has not yet published final guidance. NICE published its draft guidance for consultation in May 2025 and was unfortunately unable to recommend cerliponase alfa for routine use when taking into account all the evidence of costs and benefits, including real world evidence collected during the managed access period. We are, however, pleased that NICE and NHS England have been able to reach an agreement with the manufacturer of cerliponase alfa that ensures continued access to treatment for all patients who started on treatment before December 2025.
Officials in the Department have been in regular contact with NICE and NHS England about this appraisal. Following the next NICE Appraisal Committee meeting in July, NICE and NHS England will continue to try to reach an agreement with the manufacturer based on the committee's preferred assumptions around modelling that will provide access to cerliponase alfa for all future patients.
It is estimated that in the UK, approximately three to six children are diagnosed with CLN2 each year. In the absence of treatment with cerliponase alfa, clinical management options focus on symptom control, monitoring and preventing complications, and palliative care. Management aims to maintain function as long as possible and to improve quality of life.
Asked by: Lord Botham (Crossbench - Life peer)
Question to the Department of Health and Social Care:
To ask His Majesty's Government whether they plan to revisit the Managed Access Agreement for Brineura for treating neuronal ceroid lipofuscinosis type 2 to ensure that children born after January 2026 are not excluded from the treatment.
Answered by Baroness Merron - Parliamentary Under-Secretary (Department of Health and Social Care)
The National Institute for Health and Care Excellence (NICE) is currently updating its guidance on the use of cerliponase alfa (Brineura) for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) and has not yet published final guidance. NICE published its draft guidance for consultation in May 2025 and was unfortunately unable to recommend cerliponase alfa for routine use when taking into account all the evidence of costs and benefits, including real world evidence collected during the managed access period. We are, however, pleased that NICE and NHS England have been able to reach an agreement with the manufacturer of cerliponase alfa that ensures continued access to treatment for all patients who started on treatment before December 2025.
Officials in the Department have been in regular contact with NICE and NHS England about this appraisal. Following the next NICE Appraisal Committee meeting in July, NICE and NHS England will continue to try to reach an agreement with the manufacturer based on the committee's preferred assumptions around modelling that will provide access to cerliponase alfa for all future patients.
It is estimated that in the UK, approximately three to six children are diagnosed with CLN2 each year. In the absence of treatment with cerliponase alfa, clinical management options focus on symptom control, monitoring and preventing complications, and palliative care. Management aims to maintain function as long as possible and to improve quality of life.