Asked by: Lord Lancaster of Kimbolton (Conservative - Life peer)
Question to the Department of Health and Social Care:
To ask His Majesty's Government what action they are taking to tackle childhood cancer.
Answered by Lord Markham - Parliamentary Under-Secretary (Department of Health and Social Care)
The Government is taking steps to tackle childhood cancer, by improving early diagnosis, delivering more research, and driving progress in genomic medicine.
Improving early diagnosis is essential for children with cancer. The National Health Service is working to deliver the ambition set in its Long Term Plan to diagnose 75% of cancers at stages one and two by 2028. This is backed by £2.3 billion funding to transform diagnostic services, with 123 new community diagnostic centres opened, giving millions of patients the chance to access quicker, more convenient checks. In addition, the NHS is expanding direct access to diagnostic scans across all general practitioner (GP) practices, helping GPs recognise cancer symptoms, cutting waiting times and speeding up diagnosis.
Delivering more research is key to understanding the causes of childhood cancer and increasing survival rates. Over the past five years, the National Institute for Health and Care Research (NIHR) has invested £13.9 million on 38 research projects into childhood cancers. In addition, alongside Cancer Research UK, health departments across the United Kingdom are jointly funding a network of 18 Experimental Cancer Medicine Centres, collectively investing more than £35 million between 2017 and 2022, driving the discovery, development, and testing of new treatments. This includes the Paediatric Network that the NIHR co-funds with The Little Princess Trust, dedicated to early-phase research on childhood cancers.
Ensuring all children with cancer get access to ground-breaking genomic medicine is vital. The NHS now offers all children with cancer whole genome sequencing, to enable comprehensive and precise diagnosis and personalised treatments. In July, the Government announced a multi-year partnership agreement with the pharmaceutical giant BioNTech which will accelerate the company's clinical trials in the UK and could provide up to 10,000 patients with personalised cancer immunotherapies by 2030.
In addition, the Department is taking steps to better understand the landscape of childhood cancer with experts, aided by Dame Caroline Dinenage MP.
Through improving diagnosis, encouraging research, and ensuring access to ground-breaking treatment, the Government will continue to take steps to tackle childhood cancer.
Asked by: Ben Lake (Plaid Cymru - Ceredigion)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, with reference to the APPG on Brain Tumours Inquiry Report entitled Pathway to A Cure, whether his Department plans to work with the Medicines and Healthcare Products Regulatory Agency to encourage the inclusion of brain tumour patients in early phase cancer trials.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
The Department of Health and Social Care welcomes the All-Party Parliamentary Group report, recommendations of which continue to be worked through with the Department of Science, Innovation and Technology, and UK Research and Innovation and the Medical Research Council.
A series of new measures are to be introduced by the Medicines and Healthcare products Regulatory Agency with support from partners to make it faster and easier to gain approval and to run clinical trials in the United Kingdom. As part of this, comprehensive new guidance, co-designed with various stakeholder groups, will be introduced to accompany the new legislative measures. This will ensure that UK clinical trials work in partnership with patients and the public and are representative of the diversity of people who may benefit from a medicine if the data generated ultimately lead to regulatory approval. The guidance will outline how to include patients meaningfully into the design and conduct of trials, and how to achieve diversity in trials in a way that is proportionate and achieves the best results.
Asked by: Dehenna Davison (Conservative - Bishop Auckland)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, if she will take steps to provide access to psilocybin for the treatment of (a) severe depression and (b) other health conditions.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
The Department commissions research through the National Institute for Health and Care Research (NIHR), who have invested £1.1 million in a randomised controlled trial to examine if it is feasible, safe and effective to use psilocybin to treat people with treatment-resistant depression. The NIHR is supporting psilocybin research via the NIHR King’s Clinical Research Facility and the NIHR Maudsley Biomedical Research Centre, where researchers are developing and evaluating the efficacy and safety of psilocybin therapy for use in the National Health Service and other healthcare settings. The NIHR welcomes funding applications for research into any aspect of human health, including the use of psilocybin in the treatment of severe depression and other chronic conditions.
Before any new medicine can be used to treat patients, it must go through a strictly monitored development process. Manufacturers of medicines are expected to conduct research and clinical trials, and medicines must undergo scrutiny by the Medicines and Healthcare Products Regulatory Agency which will assess the safety, quality and efficacy of a product before a marketing authorisation (licence) may be granted. For a licence to be granted for a medicine the MHRA must receive a full marketing application from the applicant. In addition, the National Institute for Health and Care Excellence (NICE) evaluates all new medicines for clinical and cost effectiveness and makes recommendations about routine funding and use on the NHS. This is the foundation of NHS decisions about routine access and funding of medicines.
Asked by: Mike Amesbury (Labour - Weaver Vale)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what assessment has he made of the potential merits of utilising medicinal cannabis to treat children with intractable epilepsy.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
The licensed cannabis-based medicine Epidyolex is prescribed and routinely funded by the National Health Service for the treatment of people aged two years old and over with Dravet syndrome, Lennox-Gastaut syndrome and Tuberous Sclerosis Complex. This follows approval by the Medicines and Healthcare products Regulatory Agency (MHRA) and assessment by the National Institute for Health and Care Excellence (NICE).
Clinical guidelines from NICE state that there is insufficient evidence of safety and efficacy to support a population-wide practice recommendation for the use of unlicensed cannabis-based products, for the treatment of adults and children with severe treatment-resistant epilepsy. NICE also made recommendations for further research to inform future commissioning decisions.
The Government encourages manufacturers to invest in research and clinical trials, and offers scientific and research advice from the MHRA and the National Institute for Health and Care Research.
Asked by: Baroness Randerson (Liberal Democrat - Life peer)
Question to the Department of Health and Social Care:
To ask His Majesty's Government what collaborative research they are currently funding in partnership with other countries into childhood brain tumours, including Diffuse Intrinsic Pontine Glioma; and whether this includes funding for access to clinical trials.
Answered by Lord Markham - Parliamentary Under-Secretary (Department of Health and Social Care)
The following table shows eight research studies around Diffuse Intrinsic Pontine Glioma, funded by research funding partners in the charity and public sectors since 2013 which have been supported by the National Institute for Health and Care Research (NIHR):
Study Title | Opening Date | Closure Date | Funders | Status |
Phase I trial of afatinib in paediatric patients with neuroectodermal tumours and rhabdomyosarcoma | 13/07/2015 | 05/05/2020 | Boehringer Ingelheim Ltd | Closed to Recruitment, Follow Up Complete |
A Phase I/II, Multicenter, Open-Label, Dose-Escalation Study of the Safety and Pharmacokinetics of Cobimetinib in Pediatric and Young Adult Patients with Previously Treated Solid Tumors | 25/08/2016 | 31/12/2018 | F. Hoffmann-La Roche Ltd | Closed to Recruitment, In Follow Up |
Biological Medicine for Diffuse Intrinsic Pontine Glioma Eradication (Biomede) | 14/04/2017 | 29/09/2019 | Cancer Research UK | Closed to Recruitment, In Follow Up |
Phase Ib /II Clinical Trial of Nivolumab Monotherapy and Nivolumab in Combination with Ipilimumab in Pediatric Subjects with High Grade Primary CNS Malignancies | 14/08/2017 | 22/02/2019 | Bristol-Myers Squibb International Corporation | Closed to Recruitment, In Follow Up |
A PHASE 2 CLINICAL STUDY OF POMALIDOMIDE (CC-4047) MONOTHERAPY FOR CHILDREN AND YOUNG ADULTS WITH RECURRENT OR PROGRESSIVE PRIMARY BRAIN TUMORS | 17/10/2017 | 27/09/2018 | CELGENE CORPORATION | Closed to Recruitment, In Follow Up |
A Phase 1/2, Single-arm Study to Evaluate the Safety, Pharmacokinetics, and Antitumor Activity of Avapritinib in Pediatric Patients with Solid Tumors Dependent on KIT or PDGFRA Signaling | 15/02/2023 | 31/01/2025 | Blueprint Medicines Corporation | Suspended |
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*** Please note that the 2 commercial studies, due to the commercial sensitivity, cannot be shared more widely or made public.
NIHR provides infrastructure support to studies taking place in the National Health Service. For example, staff, research nurses, local networks and NHS trusts will work across many studies, to varying degrees. As a result, NIHR cannot provide precise information on expenditure.
The NIHR Clinical Research Network supports the delivery of high-quality research across the NHS, community and social care and public health organisations. It does this by meeting the costs of additional staff, facilities, equipment and support services.
In May 2018 the Government announced £40 million for brain tumour research as part of the Tessa Jowell Brain Cancer Mission. The Government is committed to funding high-quality brain cancer research. The £40 million funding remains available.
Asked by: Baroness Randerson (Liberal Democrat - Life peer)
Question to the Department of Health and Social Care:
To ask His Majesty's Government how much funding they have allocated for research into the treatment of Diffuse Intrinsic Pontine Glioma brain tumours in children in each of the last 10 years; and to which organisations were those funds allocated.
Answered by Lord Markham - Parliamentary Under-Secretary (Department of Health and Social Care)
The following table shows eight research studies around Diffuse Intrinsic Pontine Glioma, funded by research funding partners in the charity and public sectors since 2013 which have been supported by the National Institute for Health and Care Research (NIHR):
Study Title | Opening Date | Closure Date | Funders | Status |
Phase I trial of afatinib in paediatric patients with neuroectodermal tumours and rhabdomyosarcoma | 13/07/2015 | 05/05/2020 | Boehringer Ingelheim Ltd | Closed to Recruitment, Follow Up Complete |
A Phase I/II, Multicenter, Open-Label, Dose-Escalation Study of the Safety and Pharmacokinetics of Cobimetinib in Pediatric and Young Adult Patients with Previously Treated Solid Tumors | 25/08/2016 | 31/12/2018 | F. Hoffmann-La Roche Ltd | Closed to Recruitment, In Follow Up |
Biological Medicine for Diffuse Intrinsic Pontine Glioma Eradication (Biomede) | 14/04/2017 | 29/09/2019 | Cancer Research UK | Closed to Recruitment, In Follow Up |
Phase Ib /II Clinical Trial of Nivolumab Monotherapy and Nivolumab in Combination with Ipilimumab in Pediatric Subjects with High Grade Primary CNS Malignancies | 14/08/2017 | 22/02/2019 | Bristol-Myers Squibb International Corporation | Closed to Recruitment, In Follow Up |
A PHASE 2 CLINICAL STUDY OF POMALIDOMIDE (CC-4047) MONOTHERAPY FOR CHILDREN AND YOUNG ADULTS WITH RECURRENT OR PROGRESSIVE PRIMARY BRAIN TUMORS | 17/10/2017 | 27/09/2018 | CELGENE CORPORATION | Closed to Recruitment, In Follow Up |
A Phase 1/2, Single-arm Study to Evaluate the Safety, Pharmacokinetics, and Antitumor Activity of Avapritinib in Pediatric Patients with Solid Tumors Dependent on KIT or PDGFRA Signaling | 15/02/2023 | 31/01/2025 | Blueprint Medicines Corporation | Suspended |
*** |
|
|
|
|
*** |
|
|
|
|
*** Please note that the 2 commercial studies, due to the commercial sensitivity, cannot be shared more widely or made public.
NIHR provides infrastructure support to studies taking place in the National Health Service. For example, staff, research nurses, local networks and NHS trusts will work across many studies, to varying degrees. As a result, NIHR cannot provide precise information on expenditure.
The NIHR Clinical Research Network supports the delivery of high-quality research across the NHS, community and social care and public health organisations. It does this by meeting the costs of additional staff, facilities, equipment and support services.
In May 2018 the Government announced £40 million for brain tumour research as part of the Tessa Jowell Brain Cancer Mission. The Government is committed to funding high-quality brain cancer research. The £40 million funding remains available.
Asked by: Chris Green (Conservative - Bolton West)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, how many single technology appraisals for medicines for rare diseases conducted by the National Institute for Health and Care Excellence resulted in (a) a positive recommendation, (b) an optimised recommendation, (c) a recommendation for managed access, (d) a negative recommendation and (e) termination in each financial year since 2018-19.
Answered by Will Quince
The following table shows a breakdown of technologies with designated orphan status appraised through the National Institute for Health and Care Excellence resulted single technology appraisal process by financial year from 2018/19:
Financial Year | 2018/19 | 2019/20 | 2020/21 | 2021/22 | 2022/23 | Total |
Recommended | 2 | 1 | 6 | 11 | 5 | 25 |
Recommended (Cancer Drugs Fund) | 2 | 2 | 4 | 0 | 0 | 8 |
Optimised | 3 | 9 | 3 | 3 | 8 | 26 |
Optimised (Cancer Drugs Fund) | 1 | 1 | 0 | 1 | 0 | 3 |
Research Only | 0 | 0 | 0 | 0 | 0 | 0 |
Not Recommended | 2 | 0 | 0 | 1 | 2 | 5 |
Total | 10 | 13 | 13 | 16 | 15 | 67 |
Terminated | 1 | 5 | 5 | 10 | 2 | 23 |
Medicines with an orphan designation are for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition that is rare, namely affecting not more than five in 10,000 people in Great Britain, or where the medicine is unlikely to generate sufficient profit to justify research and development costs.
Asked by: Tanmanjeet Singh Dhesi (Labour - Slough)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, whether he plans to make an assessment of the adequacy of (a) funding for and (b) research into breast cancer treatment.
Answered by Will Quince
The Department does not currently plan to make specific assessments as work is already underway. The Government is supporting the National Health Service with record funding, including investing an additional £3.3 billion in each of 2023/24 and 2024/25 to enable rapid action to improve emergency, elective and primary care performance towards pre-pandemic levels, bringing the NHS resource budget to £165.9 billion in 2024/25.
Under the Targeted Investment Fund, NHS England is investing in over 870 schemes across more than 180 hospital trusts to increase capacity through expanding wards, installing modular operating theatres, upgrading outpatient spaces, expanding mobile diagnostics for cancer and upgrading MRI and screening technology
Regarding research, the Department invests in health research through the National Institute for Health and Care Research (NIHR) and welcomes funding applications for research into any aspect of human health, including research into breast cancer. NIHR research expenditure for all cancers was over £101 million for 2021/22.
NIHR also supports research into breast cancer treatment via its infrastructure. This includes funding for the NIHR Biomedical Research Centres, NIHR Clinical Research Facilities, NIHR Clinical Research Network and Experimental Cancer Medicine Centres.
Asked by: Lord Weir of Ballyholme (Democratic Unionist Party - Life peer)
Question to the Department of Health and Social Care:
To ask His Majesty's Government what resource commitment they are providing to enable the translation of medical research into implementation in practice.
Answered by Lord Markham - Parliamentary Under-Secretary (Department of Health and Social Care)
The National Institute for Health and Care Research (NIHR) puts high quality research evidence at the heart of decision making in the National Health Service, public health, and social care. Through research, the NIHR helps policy makers, health & care professionals and patients make evidence-based decisions about which treatments and practices are most effective and which make best use of resources.
In 2022/23, the NIHR’s budget was approximately £1.25 billion, and resourcing to enable translation of research into practice is embedded across multiple workstreams in the NIHR portfolio. Through the Centre for Engagement and Dissemination, the NIHR also leads and coordinates activities which support the translation of NIHR evidence into practice, at a total five-year contract value of approximately £9.8 million.
Asked by: Baroness Bennett of Manor Castle (Green Party - Life peer)
Question to the Department of Health and Social Care:
To ask His Majesty's Government what assessment they have made of the potential of human-specific technologies, such as liver-on-a-chip, to improve the accuracy of tests to ascertain the safety of new drugs in humans.
Answered by Lord Markham - Parliamentary Under-Secretary (Department of Health and Social Care)
The Medicines and Healthcare products Regulatory Agency (MHRA) is aware of human-specific technologies, including organ-on-chip technologies, to better identify potential toxicity and safety of novel medicines and has engaged with other organisations active in this space such as the National Centre for the Replacement, Refinement & Reduction of Animals in Research which has hosted meetings on this theme. The MHRA has also provided scientific advice to at least one biotechnology company on the use of this technology to support proof of concept for a new medicine. The MHRA does not identify those with whom it may have had discussions who are active commercially in this space.
In relation to human specific methods, some medicines have been developed which only have activity in humans, such as eculizumab (Soliris), tebentafusp (Kimmtrak) or CAR T cell products (for instance, Kymriah, Yescarta and Tecartus). These medicines were developed using human specific methods; however, versions of these medicines that were active in animals were, in some cases, also used. The MHRA supports the developers of these products by its offer of scientific advice services, the Innovation Office and the Innovative Licensing and Access Pathway.