Asked by: Peter Dowd (Labour - Bootle)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, whether his Department has made an assessment of the potential impact of suspending the medicines repurposing programme on people affected by rare conditions.
Answered by Karin Smyth - Minister of State (Department of Health and Social Care)
In April 2025, a decision was made to suspend the national Medicines Repurposing Programme. The decision was made with consideration to the following factors:
There are alternative sources of advice and support for clinicians or charities wanting to repurpose a medicine, including for a rare condition, such as the Medical Research Council, National Institute for Health and Care Research, and scientific advice from the Medicines and Healthcare products Regulatory Agency (MHRA).
Medicines repurposing means using an existing medicine in a new way, outside of its current marketing authorisation. Clinicians are very familiar with the option of prescribing medicines outside their licence, known as off-label use, particularly within specialised care. Off-label access is often agreed at a local level, informed by the available evidence and clinical guidelines, and where the clinician feels this best meets the needs of the patient. When appropriate and supported by the available evidence, national guidance from organisations including the National Institute for Health and Care Excellence or the British National Formulary could potentially support equitable patient access to repurposed medicines.
A further potential route to support patient access to a repurposed medicine used in specialised care in England is to develop a national clinical commissioning policy. A policy will confirm whether a specific treatment should be routinely available to eligible patients in the National Health Service. A policy can only be developed if the medicine is used within prescribed specialised services, namely services for which NHS England is the accountable commissioner, and not services where the accountable commissioners are integrated care boards. National clinical policies are based on the available research evidence and may be subject to a process of funding prioritisation, depending on the cost of the treatment concerned. A NHS clinician can propose a new or revised national clinical commissioning policy where there is sufficient supporting evidence. More information on the process is available at the following link:
https://www.youtube.com/watch?v=Ya1A9WTzQXQ
NHS England develops clinical commissioning policies in line with published methods and eligibility criteria, which have been subject to consultation. A clinical commissioning policy can recommend medicines that are used on-label or off-label. More information on the policies is available at the following link:
https://www.england.nhs.uk/publication/methods-national-clinical-policies/
The national medicines repurposing programme included a small number of medicines to treat rare diseases. In some cases, the programme supported academics to obtain MHRA scientific advice and gather evidence to support a licensing application. To date, no licensing applications have been received via the programme for medicines to treat rare disease.
Asked by: Peter Dowd (Labour - Bootle)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, whether the specific challenges of providing specialised care for rare conditions will be included in the NHS 10 year plan.
Answered by Ashley Dalton - Parliamentary Under-Secretary (Department of Health and Social Care)
The 10-Year Plan will reshape the National Health Service, and in many cases, will provide opportunities to address the challenges that are faced by patients, including those with rare conditions and the people who treat or support them.
Asked by: Peter Dowd (Labour - Bootle)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, how the NHS 10 year plan will improve the delivery of healthcare for people with rare conditions.
Answered by Ashley Dalton - Parliamentary Under-Secretary (Department of Health and Social Care)
The 10-Year Plan will reshape the National Health Service, and in many cases, will provide opportunities to address the challenges that are faced by patients, including those with rare conditions and the people who treat or support them.
Asked by: Peter Dowd (Labour - Bootle)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what steps his Department is taking to ensure that the (a) National Disease Registration Service and (b) National Congenital Anomaly and Rare Diseases Registration Service continue after the planned abolition of NHS England.
Answered by Ashley Dalton - Parliamentary Under-Secretary (Department of Health and Social Care)
Working under the UK Rare Diseases Framework, the Government is committed to improving the lives of those living with rare diseases. Digital data and technology are underpinning themes of the UK Rare Diseases Framework. We acknowledge the important role of the National Congenital Anomaly and Rare Disease Registration Service, part of the National Disease Registration Service, in underpinning the delivery of England’s Rare Diseases Action Plans. Further information about the National Congenital Anomaly and Rare Disease Registration Service is available at the following link:
https://digital.nhs.uk/ndrs/about/ncardrs
We are currently in the initial phases of scoping and designing a new integrated department that aims to enhance the efficiency and effectiveness of our healthcare system. The important role of the National Disease Registration Service will be taken into account as part of future plans.
Asked by: Peter Dowd (Labour - Bootle)
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 concerns raised by the Spinal Muscular Atrophy (SMA) stakeholder community on the speed of progress being made by the UK National Screening Committee in its work to start an in-service evaluation of screening for SMA in NHS services.
Answered by Ashley Dalton - Parliamentary Under-Secretary (Department of Health and Social Care)
My Rt Hon. Friend, the Secretary of State for Health and Social Care, is aware of the concerns raised by stakeholders. Plans for the in-service evaluation are being developed; however, its roll out and timeline will not be confirmed until after the 2025 Spending Review.
Asked by: Peter Dowd (Labour - Bootle)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what discussions he has had with the National Institute for Health and Care Research on expediting the publication of its research brief to inform the UK National Screening Committee’s in-service evaluation of newborn screening for spinal muscular atrophy.
Answered by Ashley Dalton - Parliamentary Under-Secretary (Department of Health and Social Care)
My Rt Hon. Friend, the Secretary of State for Health and Social Care is aware of the plans being developed for the National Institute for Health and Care Research’s research call and the in-service evaluation. However, its roll out and timeline will not be confirmed until after the conclusion of the 2025 Spending Review.
Asked by: Peter Dowd (Labour - Bootle)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what his timetable is for releasing funds to support the UK National Screening Committee’s review of newborn screening for Spinal Muscular Atrophy.
Answered by Ashley Dalton - Parliamentary Under-Secretary (Department of Health and Social Care)
Following the 2023 UK National Screening Committee (UK NSC) recommendation for an in-service evaluation (ISE) of spinal muscular atrophy (SMA), partnership planning between the Department, NHS England, the National Institute for Health and Care Research (NIHR), and other stakeholders has been in progress to implement an ISE in the National Health Service.
An ISE is a programme of work to develop high quality evidence in live NHS services. Successfully implementing the ISE will require funding, however, departmental budgets, including for the NHS beyond 2025/26, will be set through Phase 2 of the Spending Review, which will conclude and be published in June 2025.
The findings of the ISE, which will run over a few years, will provide the UK NSC with real-world evidence on feasibility, acceptability, and clinical and cost-effectiveness, as well as longer term health outcomes. Along with an SMA screening modelling study, these will inform a UK NSC recommendation on whether SMA should be added to the newborn blood spot screening programme.
Asked by: Peter Dowd (Labour - Bootle)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what the planned timetable is for the UK National Screening Committee's review of its advice on screening for spinal muscular atrophy.
Answered by Ashley Dalton - Parliamentary Under-Secretary (Department of Health and Social Care)
Following the 2023 UK National Screening Committee (UK NSC) recommendation for an in-service evaluation (ISE) of spinal muscular atrophy (SMA), partnership planning between the Department, NHS England, the National Institute for Health and Care Research (NIHR), and other stakeholders has been in progress to implement an ISE in the National Health Service.
An ISE is a programme of work to develop high quality evidence in live NHS services. Successfully implementing the ISE will require funding, however, departmental budgets, including for the NHS beyond 2025/26, will be set through Phase 2 of the Spending Review, which will conclude and be published in June 2025.
The findings of the ISE, which will run over a few years, will provide the UK NSC with real-world evidence on feasibility, acceptability, and clinical and cost-effectiveness, as well as longer term health outcomes. Along with an SMA screening modelling study, these will inform a UK NSC recommendation on whether SMA should be added to the newborn blood spot screening programme.
Asked by: Peter Dowd (Labour - Bootle)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what steps he is taking to ensure that there are an adequate number of health professionals trained in (a) identifying and (b) supporting people affected by Creutzfeldt-Jakob disease (CJD).
Answered by Ashley Dalton - Parliamentary Under-Secretary (Department of Health and Social Care)
Creutzfeldt-Jakob disease (CJD) is a very rare but rapidly progressive and fatal disease. Effective diagnostic and care services are important in ensuring that patients and their families get the support that they need. Unfortunately, there is no known cure for CJD. Treatment options focus on symptom control and palliative care.
Specialist diagnostic services are provided by the National CJD Research and Surveillance Unit. From April 2025, funding for these services is being transferred from the National Institute for Health and Care Research to NHS England.
Asked by: Peter Dowd (Labour - Bootle)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what collaboration there is between NHS England, the National Institute for Health and Care Research and the Medical Research Council on Creutzfeldt–Jakob disease funding; and what assessment he has made of the potential impact of withdrawal of funding on (a) Creutzfeldt–Jakob disease and (b) prion disease research by those bodies.
Answered by Ashley Dalton - Parliamentary Under-Secretary (Department of Health and Social Care)
The Department funds research through the National Institute of Health and Care Research (NIHR). Since 2002, the Department has made over £43 million of funding available for research and surveillance of Creutzfeldt-Jakob disease (CJD) and prion diseases.
The Department is working closely with NHS England on future funding for CJD diagnostic services. The Department also works closely with the Medical Research Council (MRC) to align funding for priority research, and has met specifically with the MRC Prion Unit recently.
Both the NIHR and the MRC welcome research proposals on all aspects of human health, including CJD and prion disease. Research applications are subject to peer review and judged in open competition, with awards being made on the basis of the importance of the topic to patients and health and care services, value for money, and scientific quality