Asked by: Jim Shannon (Democratic Unionist Party - Strangford)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, when he last met charities representing the interests of people living with duchenne muscular dystrophy.
Answered by Steve Brine
The last meeting between a Minister of the Department and a charity representing the interests of people living with Duchenne muscular dystrophy took place in October 2017, when the Parliamentary Under-Secretary of State for Health (Lord O’Shaughnessy), met a number of charities representing people with neurological disorders, including the Neurological Alliance. The Alliance provides a collective voice for over 80 neurological condition charities, including Action Duchenne.
Asked by: Derek Thomas (Conservative - St Ives)
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 increase capacity at muscle centres to conduct clinical trials.
Answered by Caroline Dinenage
The National Institute for Health Research (NIHR) works closely with its partners - including charities, other public funders and industry – to ensure the best possible environment for supporting clinical trials. In particular, the NIHR funds infrastructure in the National Health Service to support research and trials, providing the expertise and facilities for high quality research which aims to speed up translation from clinical research to patient benefits.
To aid in the delivery of early phase musculoskeletal research, the NIHR has a number of Biomedical Research Centres (BRCs) with musculoskeletal research themes. These are partnerships between leading NHS organisations and universities, which focus on conducting early translational research. There were 351 musculoskeletal research studies across the BRCs and Biomedical Research Units in 2016-17. The NIHR Translational Research Collaboration in joint and related inflammatory diseases brings together internationally recognised investigators in the United Kingdom's leading centres of excellence to carry out early phase research.
In addition, NIHR Medtech and In vitro diagnostics Co-operatives build expertise and capacity in the NHS to develop new medical technologies and provide evidence on commercially-supplied in vitro diagnostic tests. The Leeds In Vitro Diagnostics Co-operative has a musculoskeletal theme.
Support for muscular wasting disease research is one of the NIHR’s great success stories. Laboratory findings have been taken into NIHR early phase research facilities and developed target products to be trialled in NIHR Paediatric Clinical Research Facilities. These earlier trials are spread across all NHS England Specialised Commissioning funded Muscle Centres and using the support of the NIHR Clinical Research Network have resulted in translating new products for approval by regulators. This work has been done in collaboration with charities and provides a model for building capacity in rare diseases. There is also a further rich vein of commercial clinical trials becoming available for rare muscle wasting diseases. The NIHR and NHS England are committed to ensuring these studies are prioritised through the centres in line with the NIHR/NHS England joint statement on research from November 2017. Further information and the response to the subsequent consultation was published on 4 May 2018:
Asked by: Cheryl Gillan (Conservative - Chesham and Amersham)
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 the suitability of the Single Technology Appraisal process for new treatments for patients with Duchenne Muscular Dystrophy.
Answered by Steve Brine
The National Institute for Health and Care Excellence (NICE) is the independent body responsible for developing authoritative, evidence-based guidance for the National Health Service on whether drugs and other treatments represent an effective use of NHS resources.
NICE assesses most significant new drugs through its technology appraisal programme and has been able to recommend a number of drugs licenced for the treatment of rare diseases for routine use on the NHS. NICE also operates a separate highly specialised technology (HST) evaluation programme for the assessment of very high cost drugs for the treatment of very small numbers of patients. Decisions on the most appropriate programme for individual drugs and other treatments are made through an established topic selection process that includes a public consultation and thorough consideration against a set of published criteria.
NICE published HST guidance recommending ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene in July 2016 and is currently developing guidance, through its technology appraisal and HST programmes, on other drugs for the treatment of Duchenne muscular dystrophy.
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 steps his Department is taking to ensure that patients with Duchenne Muscular Dystrophy have access to new treatments in the NHS.
Answered by Steve Brine
NHS England is responsible for commissioning specialised neurological services, including services for patients with neuromuscular disorders such as Duchenne Muscular Dystrophy. NHS England is working with the Neurological Alliance in support of the new national Neurology Advisory Group, which is considering ways to reduce variation and drive improvement in neurological care. Specialised neuromuscular care may include referral to local physiotherapy, hydrotherapy or rehabilitation if appropriate.
Further to this, the Royal College of General Practitioners has developed a learning module with Muscular Dystrophy UK to help general practitioners have a better understanding of their role in the management of neuromuscular conditions. It will also help them recognise the key moments when a patient needs to be referred to a specialist neuromuscular service.
The National Institute of Care and Health and Care Excellence is currently developing guidance on the use of eteplirsen (Exondys 51) for Duchenne Muscular Dystrophy through its highly specialised technology evaluation programme.
Asked by: Baroness Masham of Ilton (Crossbench - Life peer)
Question to the Department of Health and Social Care:
To ask Her Majesty's Government what assessment they have made of services to monitor and support patients with Duchenne Muscular Dystrophy who are experiencing respiratory decline.
Answered by Lord O'Shaughnessy
No specific assessment has been made.
NHS England commissions specialised neurological services from 24 centres at a national level, including some services for patients with neuromuscular disorder such as Duchenne muscular dystrophy. Two service specifications, one for adults and one for children, have been developed by NHS England to set out what these providers must have in place to deliver high quality neurological and neuromuscular care. A requirement for neuromuscular services is that patient clinics ensure they identify those at risk of respiratory problems and refer them for specialist respiratory assessment and monitoring. The specifications set out the breadth of these services and the objectives for their delivery, including clinical assessment and monitoring in respiratory care and respiratory failure. Copies of 2013/14 NHS Standard Contract for Neurosciences: Specialised Neurology (Adult) and 2013/14 NHS Standard Contract for Paediatric Neurosciences: Neurology are attached.
Asked by: Baroness Masham of Ilton (Crossbench - Life peer)
Question to the Department of Health and Social Care:
To ask Her Majesty's Government how many treatments with an orphan designation have been subject to a Highly Specialised Technology evaluation by the National Institute for Health and Care Excellence since 2013; what was the recommendation for each assessment; and what was the estimated populated size for each treatment.
Answered by Lord O'Shaughnessy
The National Institute for Health and Care Excellence (NICE) has evaluated six treatments with an orphan designation through its Highly Specialised Technology (HST) programme since 2013. The table below details the guidance, patient population size and the recommendation for each evaluation.
Guidance | Estimated patient population | Recommended |
Asfotase alfa for treating paediatric-onset hypophosphatasia | Not known | Yes |
Eliglustat for treating type 1 Gaucher disease | 50 to 100 | Yes - for long-term treatment in adults who are cytochrome P450 2D6 poor, intermediate or extensive metabolisers. |
Migalastat for treating Fabry disease | 142 | Yes – for people over 16 years of age with an amenable mutation, only if migalastat is provided with the discount agreed in the patient access scheme, and only if enzyme replacement therapy would otherwise be offered. |
Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene | Not known | Yes |
Elosulfase alfa for treating mucopolysaccharidosis type Iva | 74-77 | Yes |
Eculizumab for treating atypical haemolytic uraemic syndrome | Not known | Yes |
Asked by: Michelle Donelan (Conservative - Chippenham)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health, what recent assessment he has made of the provision of psychological support for adults with muscular dystrophy and neuromuscular conditions.
Answered by Steve Brine
No specific assessment has been made. Guidance from the National Institute for Health and Care Excellence has been published for a number of neuromuscular disorders, and where appropriate, the guidance makes recommendations about access to psychological and counselling support for patients. In addition, NHS England has also set out that specialised care for patients with neuromuscular disorders, such as muscular dystrophy, should provide access to psychologists and/or counsellors.
NHS England is also working with the Neurological Alliance in support of the new national Neurology Advisory Group, which is considering ways to reduce variation and drive improvement in neurological care. This includes looking at issues such as psychological support, which were recently raised in the report Parity of esteem for people affected by neurological conditions: meeting the emotional, cognitive & mental health needs of neurology patients, published by the Neurological Alliance on 5 July 2017.
Finally, as set out in Implementing the Five Year Forward View for Mental Health, published in July 2016, the expansion of psychological therapies services will require building skills and capacity in the workforce. This includes: top-up training in new competencies for long-term conditions (relevant to people with neuromuscular disorders and other long term health problems) and medically unexplained symptoms for current staff; targeted training in working with older people; and training new staff to increase overall capacity – such as the 3,000 additional mental health therapists located in primary care.
Asked by: Jim Shannon (Democratic Unionist Party - Strangford)
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 the provision of psychological support for adults with (a) muscular dystrophy and (b) neuromuscular conditions.
Answered by Steve Brine
No specific assessment has been made. Guidance from the National Institute for Health and Care Excellence has been published for a number of neuromuscular disorders, and where appropriate, the guidance makes recommendations about access to psychological and counselling support for patients. In addition, NHS England has also set out that specialised care for patients with neuromuscular disorders, such as muscular dystrophy, should provide access to psychologists and/or counsellors.
NHS England is also working with the Neurological Alliance in support of the new national Neurology Advisory Group, which is considering ways to reduce variation and drive improvement in neurological care. This includes looking at issues such as psychological support, which were recently raised in the report Parity of esteem for people affected by neurological conditions: meeting the emotional, cognitive & mental health needs of neurology patients, published by the Neurological Alliance on 5 July 2017.
Finally, as set out in Implementing the Five Year Forward View for Mental Health, published in July 2016, the expansion of psychological therapies services will require building skills and capacity in the workforce. This includes: top-up training in new competencies for long-term conditions (relevant to people with neuromuscular disorders and other long term health problems) and medically unexplained symptoms for current staff; targeted training in working with older people; and training new staff to increase overall capacity – such as the 3,000 additional mental health therapists located in primary care.
Asked by: Julian Lewis (Conservative - New Forest East)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health, what the timetable is for the National Institute for Health and Care Excellence evaluation of the Duchenne muscular dystrophy treatment Exondys 51.
Answered by Steve Brine
The National Institute for Health and Care Excellence (NICE) is developing guidance on the use of eteplirsen (Exondys 51) through its highly specialised technology (HST) evaluation programme. NICE’s HST evaluation committee is scheduled to discuss this topic in May 2018 with final guidance expected to be issued in October 2018. The provisional timeline for eteplirsen is based upon the anticipated licensing dates for the drug.
Asked by: Julian Lewis (Conservative - New Forest East)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health, if he will make it his policy to ensure that NHS England and Sarepta Therapeutics conduct pricing negotiations at an early stage in the evaluation of Exondys 51.
Answered by Steve Brine
The National Institute for Health and Care Excellence (NICE) highly specialised technology (HST) evaluation process for eteplirsen (Exondys 51) allows the company to propose a Patient Access Scheme to the Department, or to discuss commercial arrangements with NHS England, if required. The process provides sufficient time to ensure the evaluation is completed on schedule.