Asked by: Greg Mulholland (Liberal Democrat - Leeds North West)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health, what steps he is taking to fund research to improve the care and treatment of people with the limb girdle 2b form of Duchenne muscular dystrophy.
Answered by Baroness Blackwood of North Oxford
The Department's National Institute for Health Research (NIHR) welcomes funding applications for research into any aspect of human health, including muscular dystrophy. These 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.
Research relating to limb-girdle muscular dystrophy is being funded by the NIHR Newcastle Biomedical Research Centre. Current funding for NIHR Biomedical Research Centres ends in March 2017.
In September the Government announced the largest ever investment into health research - £816 million over five years from April 2017 for 20 NIHR Biomedical Research Centres in England. We would expect some of this investment to support muscular dystrophy research.
The NIHR Clinical Research Network is currently recruiting patients to a study of acceptance and commitment therapy for muscle disease. Limb-girdle muscular dystrophy is one of four muscle diseases included in the study.
Asked by: Greg Mulholland (Liberal Democrat - Leeds North West)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health, what assessment he has made of the adequacy of care available to treat people with the limb girdle 2b form of Duchenne muscular dystrophy.
Answered by David Mowat
NHS England’s neurosciences services specifications for adults and children set out what providers must have in place to deliver evidence-based, safe and effective care for patients with neuromuscular disorders, including limb girdle 2b. Under the care of a consultant-led multidisciplinary team that includes a range of clinical, specialist and health professionals, patients can receive tailored care and support, access to specialist treatments and supportive therapies. The specifications can be found at the following links:
www.england.nhs.uk/wp-content/uploads/2013/06/d04-neurosci-spec-neuro.pdf
www.england.nhs.uk/wp-content/uploads/2013/06/e09-paedi-neurology.pdf
Asked by: Greg Mulholland (Liberal Democrat - Leeds North West)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health, what assessment NHS England has made of the effectiveness of the Translarna drug in treating the limb girdle 2b form of Duchenne muscular dystrophy.
Answered by David Mowat
NHS England has advised that limb girdle 2b is a form of muscular dystrophy distinct from Duchenne muscular dystrophy.
NHS England understands that Ataluren (Translarna) has not been trialed by the manufacturers of this drug, PTC Therapeutics, as a treatment for this disease and therefore no assessment has been made.
Asked by: Greg Mulholland (Liberal Democrat - Leeds North West)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health, with reference to the statement by the Parliamentary Under-Secretary of State for Life Sciences of 9 December 2015, Official Report, column 389-90WH, if NHS England will develop a commissioning policy on the use of anti-tumor necrosis factor therapy for adults with uveitis.
Answered by David Mowat
In November 2015 NHS England undertook to review the current adult policy for uveitis once the study, known as VISUAL II, was fully published in a peer reviewed journal. The results of this study were published in August 2016.
NHS England will be reviewing the clinical evidence as reported in VISUAL II and make a judgement on whether an interim policy should be published prior to the anticipated National Institute of Health and Care Excellence Guidance on treating uveitis expected in July 2017.
Asked by: Greg Mulholland (Liberal Democrat - Leeds North West)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health, what treatment options are available to adults with uveitis to access anti-TNF treatment therapy who do not meet the exceptionality criteria of the individual funding request process.
Answered by David Mowat
Due to a lack of clinical evidence anti-tumour necrosis factor (TNF) to treat uveitis in adults is not currently routinely commissioned by the National Health Service. Therefore patients can only access the treatment through the individual funding request process.
Asked by: Greg Mulholland (Liberal Democrat - Leeds North West)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health, how many adult patients with uveitis have had access to treatment with anti-TNF therapy through the individual funding request process since July 2015.
Answered by David Mowat
Between 1 July 2015 and 31 July 2016, fewer than 10 patients accessed anti-tumour necrosis factor (TNF) therapy through the individual funding request process.
Asked by: Greg Mulholland (Liberal Democrat - Leeds North West)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health, what steps he is taking to increase the transparency of NHS England's decision making process on the prioritisation of investment in specialised services.
Answered by David Mowat
Each year, a significant number of proposals are put to NHS England for investment in new drugs, medical devices or interventions for use by specialised services in England. NHS England has to make difficult decisions on behalf of tax-payers about how to prioritise the funding that is available for those new investments each year. In considering policies placed before it, the Clinical Priorities Advisory Group follows the published procedure. The published procedure can be found at the following address:
On 11 July 2016, for the first time, NHS England set out its provisional investment decisions for specialised services and the results of its annual process for deciding which new treatments and services it will make available to patients. This can be found here:
https://www.england.nhs.uk/2016/07/spec-services-investment/Background
Asked by: Greg Mulholland (Liberal Democrat - Leeds North West)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health, what support his Department makes available to families who are supporting relatives with Huntington's disease.
Answered by Baroness Blackwood of North Oxford
The Government published the UK Strategy for Rare Diseases in November 2013. The Strategy is signed up to by the four home countries of the United Kingdom. The Strategy contains 51 high level commitments which comprise a long term strategic vision for improving the lives of all those with rare diseases. The focus throughout is patients and their families.
The UK Strategy includes commitments to empower those affected by rare diseases including making sure that patients and their families have a say in decisions about treatment and in the planning, evaluation and monitoring of services.
The Strategy also recognises the important role played by patient organisations such as the Huntington’s Disease Association in the provision of support, information and advice and helping patients and their families feel less isolated.