Asked by: Lord Hunt of Kings Heath (Labour - Life peer)
Question to the Department of Health and Social Care:
To ask His Majesty's Government whether they intend to have any discussions with the National Institute for Health and Care Excellence about employing additional flexibility for rare condition medicines to take into consideration the implications of having a small population and potentially more expensive medicines.
Answered by Lord Markham - Parliamentary Under-Secretary (Department of Health and Social Care)
The Department regularly meets with the National Institute for Health and Care Excellence (NICE) to discuss a range of issues including access to and availability of medicines. NICE’s methods and processes for health technology evaluation have been proven to be suitable for medicines for rare diseases where companies price their products responsibly, and NICE introduced a number of changes to its methods and processes in 2022 that ensure that its processes are appropriate to the evaluation of emerging new technologies.
NICE also operates a highly specialised technologies programme for the evaluation of a small number of medicines for the treatment of very rare diseases. As of 31 March 2024, NICE has recommended 88% of medicines licensed for the treatment of rare diseases for some or all of the eligible patient population, which is comparable to NICE’s approval rate for all medicines.
The Innovative Medicines Fund, building on the success of the Cancer Drugs Fund, provides a mechanism for consistent and transparent managed access process for companies offering promising non-cancer medicines at a responsible price. The Fund has already provided early access for National Health Service patients to several innovative new treatments, including for patients with rare diseases such as graft-versus-host disease and Wolman disease.
Asked by: Lord Hunt of Kings Heath (Labour - Life peer)
Question to the Department of Health and Social Care:
To ask His Majesty's Government whether they plan to hold discussions with the National Institute for Health and Care Excellence about its appraisal system to ensure it is sufficiently flexible to respond to treatments and medicines for rare diseases.
Answered by Lord Markham - Parliamentary Under-Secretary (Department of Health and Social Care)
The Department regularly meets with the National Institute for Health and Care Excellence (NICE) to discuss a range of issues including access to and availability of medicines. NICE’s methods and processes for health technology evaluation have been proven to be suitable for medicines for rare diseases where companies price their products responsibly, and NICE introduced a number of changes to its methods and processes in 2022 that ensure that its processes are appropriate to the evaluation of emerging new technologies.
NICE also operates a highly specialised technologies programme for the evaluation of a small number of medicines for the treatment of very rare diseases. As of 31 March 2024, NICE has recommended 88% of medicines licensed for the treatment of rare diseases for some or all of the eligible patient population, which is comparable to NICE’s approval rate for all medicines.
The Innovative Medicines Fund, building on the success of the Cancer Drugs Fund, provides a mechanism for consistent and transparent managed access process for companies offering promising non-cancer medicines at a responsible price. The Fund has already provided early access for National Health Service patients to several innovative new treatments, including for patients with rare diseases such as graft-versus-host disease and Wolman disease.
Asked by: Cat Smith (Labour - Lancaster and Fleetwood)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, whether postural tachycardia syndrome is a rare disease as defined in the UK Rare Diseases Framework.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
The UK Rare Diseases Framework defines rare diseases as having a prevalence of lower than one in 2000 people. Postural Tachycardia Syndrome (PoTS) UK has estimated the prevalence of PoTS to be 0.2%, or four in 2000 people, which is higher than the definition of a rare disease under the framework.
The Government recognises the significant impact that PoTS symptoms can have on an individual’s life, and is committed to ensuring those with PoTS have timely access to a diagnosis and appropriate treatment and services.
Asked by: Lord Hay of Ballyore (Democratic Unionist Party - Life peer)
Question to the Department of Health and Social Care:
To ask His Majesty's Government what provisions are in place to support the families and caregivers of individuals with rare diseases.
Answered by Lord Markham - Parliamentary Under-Secretary (Department of Health and Social Care)
Each year since 2021, we have published an England Rare Diseases Action Plan. On 29 February 2024, we published the third England Rare Diseases Action Plan. In this plan we recognised that significant challenges exist with access to mental health and psychological support for people living with rare conditions, as well as their families and carers. During this year we have taken steps to further understand the challenges faced through a workshop, and have begun to address these needs by developing resources to better equip the workforce to provide support.
The enormous contribution of unpaid carers, including those caring for individuals with rare diseases, is reflected throughout the Next Steps to Put People at the Heart of Care, published in April 2023. Furthermore, the Better Care Fund in 2023/24 includes £327 million for carers support, including short breaks and respite services for carers. This also funds advice and support to carers, and a small number of additional local authority duties. The Accelerating Reform Fund also provides support for unpaid carers. The Government is developing a new survey of unpaid carers which will capture the wide range of experiences, circumstances, and needs of unpaid carers across England. Through the National Institute for Health and Care Research, an evaluation of the support provided to unpaid carers funded through the Better Care Fund has been commissioned, which will improve the understanding of what support works best for unpaid carers.
Asked by: Gill Furniss (Labour - Sheffield, Brightside and Hillsborough)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what steps her Department has taken to improve awareness of sickle cell disease among healthcare professionals.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
The Government is committed to improving the lives of those living with rare diseases such as sickle cell disease, and published the UK Rare Diseases Framework in January 2021, providing the high-level approach for rare diseases. The framework outlines future priorities including increasing awareness of rare diseases among healthcare professionals. In England we publish action plans annually to address these priorities. As part of this plan we committed to developing an innovative digital educational resource, ‘GeNotes’, providing healthcare professionals with relevant and concise information to support patient management, linking to the NHS Genomic Test Directories, and signposting to extended learning opportunities. This resource includes information on sickle cell disease and is updated regularly.
There have been recent improvements to the haematology medical curriculum, with understanding sickle cell disease now described in the curriculum as a core competency. The National Healthcare Inequalities Improvement Programme has launched several products to support NHS England in raising awareness of sickle cell disease among healthcare professionals including NHS England’s Can you tell it's Sickle Cell campaign and e-learning module and a communications campaign to raise awareness of existing NHS England arrangements to support people with sickle cell disease to save money on the costs of regular prescriptions.
Asked by: Alexander Stafford (Conservative - Rother Valley)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what training healthcare professionals receive in (a) diagnosing and (b) supporting people with Charcot-Marie-Tooth disease.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
In January 2021, the Government published the UK Rare Diseases Framework providing a national vision for how to improve the lives of those living with rare diseases, such as Charcot-Marie-Tooth disease. The framework lists the following four priorities, collaboratively developed with the rare disease community: helping patients get a final diagnosis faster; increasing awareness of rare diseases among healthcare professionals; better coordination of care; and improving access to specialist care, treatments and drugs.
To support with training for and awareness of rare diseases, National Genomics Education has developed a range of resources, including GeNotes. GeNotes puts innovative educational resources on genomics and rare diseases at the fingertips of healthcare professionals and includes information on Charcot-Marie-Tooth disease, which supports healthcare professionals to identify and manage this condition.
Asked by: Alexander Stafford (Conservative - Rother Valley)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what assessment she has made of the adequacy of treatment for Charcot-Marie-Tooth disease.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
In January 2021, the Government published the UK Rare Diseases Framework providing a national vision for how to improve the lives of those living with rare diseases, such as Charcot-Marie-Tooth disease. The framework lists the following four priorities, collaboratively developed with the rare disease community: helping patients get a final diagnosis faster; increasing awareness of rare diseases among healthcare professionals; better coordination of care; and improving access to specialist care, treatments and drugs.
To support with training for and awareness of rare diseases, National Genomics Education has developed a range of resources, including GeNotes. GeNotes puts innovative educational resources on genomics and rare diseases at the fingertips of healthcare professionals and includes information on Charcot-Marie-Tooth disease, which supports healthcare professionals to identify and manage this condition.
Asked by: Lord Hunt of Kings Heath (Labour - 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 changes to the National Institute for Health and Care Excellence's Methods and Process on patients with rare and ultra-rare diseases.
Answered by Lord Markham - Parliamentary Under-Secretary (Department of Health and Social Care)
A number of the changes made by the National Institute for Health and Care Excellence (NICE) to its methods and processes for health technology evaluation are expected to benefit medicines for rare diseases, including the introduction of a severity modifier and greater flexibility in responding to uncertainty. NICE has committed to assessing the impact of the changes on patients with rare diseases.
Due to length of the medicine’s evaluation process and number of rare disease topics using older methods or processes, the analysis of the impact of changes will take some time to assess. Data is being collected which will allow, for example, the percentage of positive NICE recommendations made following old methods and processes compared with new methods and processes for rare diseases, to be assessed. NICE will be in a position to publish the impact of the 2022 manual changes in a report by the end of 2024.
Asked by: Alexander Stafford (Conservative - Rother Valley)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what information her Department holds on the number of people with Charcot-Marie-Tooth disease in (a) Rother Valley constituency and (b) England.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
In January 2021, the Government published the UK Rare Diseases Framework providing a national vision for how to improve the lives of those living with rare diseases, such as Charcot-Marie-Tooth disease. The framework lists the following four priorities, collaboratively developed with the rare disease community: helping patients get a final diagnosis faster; increasing awareness of rare diseases among healthcare professionals; better coordination of care; and improving access to specialist care, treatments and drugs.
To support with training for and awareness of rare diseases, National Genomics Education has developed a range of resources, including GeNotes. GeNotes puts innovative educational resources on genomics and rare diseases at the fingertips of healthcare professionals and includes information on Charcot-Marie-Tooth disease, which supports healthcare professionals to identify and manage this condition.
Asked by: Daniel Zeichner (Labour - Cambridge)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what assessment her Department has made of the potential impact of the (a) methods and (b) processes used by the National Institute for Health and Care Excellence on access to medicines for patients with rarer diseases since January 2022; and whether this assessment will be reflected in the next Rare Diseases Action Plan for England.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
The National Institute for Health and Care Excellence (NICE) can recommend most rare disease medicines that it appraises through the technology appraisal programme for some or all eligible patients. NICE has no current plans to undertake a modular update of its methods and processes related specifically to rare diseases.
NICE specifically considered treatments for rare diseases during its methods review, and many of the changes introduced in NICE’s updated health technology evaluation manual in January 2022 will benefit treatments for rare diseases. These include the severity modifier, flexibility in uncertainty considerations and the emphasis on a comprehensive evidence base including real-world, qualitative, surrogate and expert evidence. These updates seek to mitigate the barriers faced by rare disease technologies while maintaining an evidence-based, robust and proportionate evaluation approach.
The 2022 England Rare Diseases Action Plan was published following the NICE methods and processes review. The 2022 Action Plan includes actions to capitalise on the changes made to NICE’s methods and processes to ensure that NICE continues to support the rapid adoption of effective new treatments for National Health Service patients with rare diseases. The impact of these changes is being assessed and an update will be provided in the 2024 England Rare Diseases Action Plan.