Lord Botham

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, and has not yet published final guidance. Brineura is currently available for eligible patients via the managed access agreement.

Working under the UK Rare Diseases Framework, the Government is committed to improving the lives of those living with rare diseases. The framework sets out improving access to specialist care, treatments, and drugs as a priority, and health equity as a cross-cutting theme. As part of our work to address health inequities we commissioned a scoping review to further develop the evidence base which was published alongside the 2025 England Rare Diseases Action Plan. NHS England has developed a health inequalities toolkit to support people working within highly specialised services to improve equity in their everyday service delivery.

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.

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.

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.

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.

Ending tax breaks for private schools will secure additional funding to help the government deliver its commitments to the 94% of children who attend state schools.

The Government has published a detailed response to the consultation conducted between July and September. The Government carefully considered over 17,000 responses from a range of stakeholders including schools, school associations, and parents. This was published online and can be found on gov.uk.

Ending tax breaks on VAT and business rates for private schools will secure additional funding to help deliver the government’s commitments relating to education and young people. Where a school in England has charitable status, the government will remove their eligibility to business rates charitable rates relief from April 2025.

Transitional relief limits how much ratepayers’ bills can change each year as a result of changes to a property's rateable value and changes to the multiplier at a revaluation. As such, support provided through transitional relief is unaffected by changes in other business rates reliefs and their eligibility.

Ending tax breaks for private schools will secure additional funding to help the government deliver its commitments to the 94% of children who attend state schools.

As a result of the January start date, the VAT policy is forecast to raise £460 million in 2024/25.

The government will use this funding to help deliver its commitments relating to education and young people, where the government has announced at Budget a £2.3 billion increase to the core schools budget for financial year 2025/26, increasing per pupil funding in real terms.

Since the announcement on 29 July, HMRC has taken action to support private schools through the change: providing detailed guidance; and allocating additional resource to process VAT registration applications. HMRC continues to engage with schools and the organisations that represent them.