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, whether she has had discussions with the National Institute for Health and Care Excellence on the case for a rarity modifier in its standard technology appraisal programme as part of its modular updates to its methods and processes.
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.
Asked by: Andrew Lewer (Conservative - Northampton South)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what steps her Department is taking to support effective routes to market for precision medicines for (a) motor neurone disease and (b) other rare diseases in the UK.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
Should the Medicines and Healthcare products Regulatory Agency (MHRA) receive a marketing authorisation application (MAA) or applications, the MHRA will consider the drug with regards to quality, safety, and efficacy. The MHRA has procedures in place to consider drugs as part of an accelerated pathway. This is possible as part of national access collaboration, and international recognition. The MHRA would be able to consider motor neurone disease and other rare disease treatment products, under any of the procedures for the benefit of patients.
Asked by: George Howarth (Labour - Knowsley)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, whether the National Institute for Health and Care Excellence plans to launch a public consultation to take forward the commitment in the 2024 Rare Disease Action Plan for England to review the criteria for its Highly Specialised Technologies programme for very rare diseases.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
Department officials regularly discuss a range of issues with colleagues in the National Institute for Health and Care Excellence (NICE), including in relation to the criteria for routing topics to the highly specialised technologies programme. The aim of any change to the criteria would be to clarify the type of medicine that would be eligible for the programme, in line with the vision set out in the NICE’s published topic selection manual. The NICE will consult publicly on any proposed changes to the criteria.
Feb. 20 2024
Source Page: Update on MHRA safety review of medicines containing pseudoephedrineFound: There have been very rare reports of posterior reversible encephalopathy syndrome (PRES) and reversible
Asked by: Lisa Nandy (Labour - Wigan)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what steps her Department is taking to improve the (a) diagnosis rate and (b) management of Tumour Induced Osteomalacia.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
While the Department is not taking steps to ensure early diagnosis of tumour induced osteomalacia specifically, the 2021 UK Rare Diseases Framework aims to improve the awareness of all rare diseases which includes this condition. England’s second Rare Diseases Action Plan, published in February 2023, reports on progress made to help patients get a final diagnosis faster and outlines new actions such as commissioning research on how best to measure the diagnostic odyssey.
The Department asked the National Institute for Health and Care Excellence to carry out a single technology appraisal of burosumab for treating FGF23-related hypophosphataemia in tumour-induced osteomalacia, but the company was not in a position to apply for a marketing authorisation from the Medicines and Healthcare products Regulatory Agency for this indication and so assessment has been suspended.
Asked by: Lisa Nandy (Labour - Wigan)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what steps her Department is taking to improve the (a) diagnosis and (b) treatment of X-linked hypophosphatemia.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
Genomic testing in the National Health Service in England for hypophosphataemia is currently included in the National Genomic Test Directory under the clinical indication labelled R154. X-linked hypophosphatemia is one of 200 treatable rare conditions being included in the Generation Study, a landmark research study which will sequence the whole genomes of 100,000 newborn babies being led by Genomics England in partnership with the NHS.
The study will evaluate the utility and feasibility of using whole genome sequencing to screen newborn babies for a larger number of childhood-onset rare genetic conditions in the NHS, with a decision whether this should be rolled out now or in the future based on the relevant evidence.
National Genomics Education has also developed GeNotes, which puts innovative educational resources on genomics and rare diseases at the fingertips of healthcare professionals. GeNotes includes information on hypophosphatemia to support healthcare professionals to identify and manage forms of hypophosphatemia such as X-linked hypophosphatemia.
Burosumab is now recommended and available on the NHS for treating X-linked hypophosphataemia in children and young people who are still growing. This novel treatment addresses the underlying problem, rather than compensating for phosphate loss, so is effective in helping children to grow normally.
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, if she will make an assessment of the potential merits of publishing quarterly reports on progress made against the commitments set out in her Department's England Rare Diseases Action Plan 2023, last updated on 10 July 2023.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
While there are no plans to publish quarterly reports on progress made against the commitments of England’s second Rare Diseases Action Plan 2023. We have committed to publishing action plans annually and England’s third Rare Diseases Action Plan will detail progress made in the past year. In addition, minutes of the eight weekly England Rare Diseases Framework Delivery Group meetings, where progress is monitored, are published to the UK Rare Disease Forum on-line platform and the UK Rare Diseases Forum receives updates on progress across the four nations at its quarterly meetings.
Asked by: Ian Byrne (Labour - Liverpool, West Derby)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what recent assessment she has made of the efficacy of Esketamine in treating people with rare neurological conditions.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
Currently, authorised Esketamine products are not licenced specifically intended for treating people with rare neurological conditions. Before new medicines intended to treat a specific condition can be placed onto the United Kingdom market, they must first receive approval from the Medicines and Healthcare products Regulatory Agency (MHRA) via the granting of a marketing authorisation (MA), commonly known as a product licence. The MHRA reviews all MA applications it receives, assessing each new medical product for its safety, quality, and efficacy in treating a condition.
It is for an applicant, namely the drug manufacturer, to apply for a MA for a medicinal product, and the MHRA does not and cannot actively seek these applications for submission.
May. 22 2024
Source Page: England’s largest temperate rainforest a National Nature ReserveFound: Temperate rainforests are found in places that have high rainfall and humidity and a low annual variation
May. 22 2024
Source Page: England’s largest temperate rainforest a National Nature ReserveFound: Temperate rainforests are found in places that have high rainfall and humidity and a low annual variation