The National Institute for Health and Care Excellence (NICE) is currently developing technology appraisal guidance for the National Health Service on the use of Spinraza (nusinersen) for the treatment of spinal muscular atrophy (SMA).

NICE is in discussion with Biogen (the company that manufactures Spinraza) to determine if there are any commercial flexibilities that could support the company in putting forward a cost-effective price in order for NICE to recommend use of the drug as a clinically and cost-effective use of NHS resources.

NHS England has published an Interim Policy Statement determining the circumstances in which existing patients will be supported to access Spinraza through the Expanded Access Programme (EAP) scheme sponsored by Biogen. The policy statement enables existing patients with type 1 SMA to access Spinraza in advance of NICE’s guidance, although the company has now withdrawn the EAP for newly diagnosed patients.

I refer the hon. Member to the answer I gave on 5^th November 2018 to Question UIN 186178

My Rt. hon. Friend the Secretary of State for Health and Social Care meets Cabinet colleagues regularly to discuss a number of different issues.

My officials and I have regular discussions with a range of stakeholders, including those representing individuals with Muscular Dystrophy to discuss benefits such as Personal Independence Payment (PIP).

For example, in regards to PIP, we regularly engage with stakeholders and one of the main avenues is through the national PIP Forum events. Muscular Dystrophy UK were invited to the most recent PIP Forum event that was held in Scotland.

Muscular Dystrophy UK are an active member of the Disability Benefits Consortium (DBC); who the Department meet with regularly.

The National Institute for Health and Care Excellence (NICE) evaluates all medicines referred to its highly specialised technologies (HST) programme, including medicines for Duchenne muscular dystrophy, consistently in accordance with its published methods and processes. In 2017, following public consultation, NICE introduced an assessment of cost-effectiveness into its HST methodology that provides a consistent and transparent mechanism for evaluating technologies while recognising the need for greater flexibility in the evaluation of very high cost drugs for the treatment of very rare diseases.

The changes to the National Institute for Health and Care Excellence’s (NICE) methodology for the development of highly specialised technology (HST) guidance were introduced following public consultation and create a framework that will enable truly transformative new drugs for patients with rare diseases to be made available to patients where companies are willing to set prices that fairly reflect the added benefit they bring. NICE is currently developing HST guidance on the use of eteplirsen for treating Duchenne muscular dystrophy.

The changes to the National Institute for Health and Care Excellence’s (NICE) methodology for the development of highly specialised technology (HST) guidance were introduced following public consultation and create a framework that will enable truly transformative new drugs for patients with rare diseases to be made available to patients where companies are willing to set prices that fairly reflect the added benefit they bring. NICE is currently developing HST guidance on the use of eteplirsen for treating Duchenne muscular dystrophy.

The National Institute for Health and Care Excellence (NICE) is the independent body responsible for the development of authoritative, evidence-based guidance for the National Health Service on whether drugs and treatments represent an effective use of resources.

Drugs for the treatment of Duchenne muscular dystrophy selected for referral to NICE are assessed using NICE’s standard methods and processes for the relevant guidance programme. Most of the treatments NICE assesses are appraised through its technology appraisal programme, but NICE also operates a separate highly specialised technology programme for the evaluation of a small number of very high cost drugs for very small patient populations. Decisions about the most appropriate programme for assessment of individual treatments, including for Duchenne muscular dystrophy, are taken following consideration against published criteria, including the size of the eligible patient population, and engagement with stakeholders.

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.

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:

https://www.england.nhs.uk/wp-content/uploads/2018/05/supporting-research-in-the-nhs-consultation-response.pdf

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.

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.

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.