The provision of cough assist machines is a matter for local clinical commissioning groups (CCGs). CCGs have the independence and autonomy to make commissioning decisions for local populations, taking into account the available evidence and individual circumstances as appropriate.

The evidence base to support the routine use of cough assist machines in muscle-wasting conditions over other cough assist methods is not well established. NHS England worked with Muscular Dystrophy UK through its Bridging the Gap project to address areas of concern raised by patients and their representatives, one of which was the provision of cough assist machines. Through this work, a number of CCGs developed commissioning policies which set out the circumstances in which to consider these devices. In addition, in February 2016 the National Institute for Health and Care Excellence published new guidance on the assessment and management of motor neurone disease, recommending that cough assist machines should be considered if assisted breath stacking is not effective, and/or during a respiratory tract infection.

What steps his Department is taking to ensure that clinical commissioning groups follow best practice commissioning policy on access to cough-assist machines for people with muscle-wasting conditions.

It is not for the Government to direct clinicians regarding the efficacy of particular treatments; it is for clinicians to decide, based on guidance from the National Institute for Health and Care Excellence and others. In developing its recent motor neurone disease guidance, NICE found that the evidence base for the routine use of cough-assistance machines was weak. However, the matter is kept under review, so that may change as and if new data emerge.

There are good examples of best practice cough-assist commissioning policies for muscle-wasting conditions that can be followed by health boards and CCGs. Given the hard work being done to extend the lives of those who suffer from muscular dystrophies, what support and assistance can the Department provide to Muscular Dystrophy UK to ensure that such policy is more widely adopted?

Cough-assist machines are one of a variety of respiratory treatments that may be appropriate for sufferers of conditions such as motor neurone disease or muscular dystrophy. In the end, it is a matter of clinical judgment.

I would be very happy to meet representatives from Muscular Dystrophy UK's Trailblazers to discuss accessibility at sports venues.

Our Sports Strategy sends a clear message to all sports that sports stadia should provide an inclusive environment that welcomes all spectators. While the spotlight on accessibility is rightly on football, we want all sports to ensure they fulfil their legal obligations towards disabled fans. We will be looking to other sports to follow football’s example, to ensure disabled spectators receive the legal requirements to which they are entitled.

The Equality and Human Rights Commission (EHRC) is the regulatory body responsible for enforcing the Equality Act 2010 The EHRC has powers to ensure service providers, including sports stadia, fulfil their legal obligations under the Act.

The Exon skipping therapy eteplirsen (Exondys 51) is currently being considered for referral to the National Institute for Health and Care Excellence’s (NICE) highly specialised technologies (HST) programme. Following discussion between NICE, NHS England and the Department as part of the topic selection process, NICE recently consulted stakeholders on its suitability for the HST process.

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.

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

The National Institute for Health and Care Excellence (NICE) has advised that, with NHS England, it is currently running a consultation which sets out proposals to enable faster access to therapies for patients with ultra-rare conditions. The proposals outline what conditions need to be met in order to receive a positive recommendation through the NICE highly specialised technologies programme and what mechanisms should be in place to introduce new technologies and manage the affordability challenge for NHS England.

NICE has also advised that a proportion of patients with Morqiuo syndrome who have met the criteria under the managed access agreement are already receiving elosulfase alfa (Vimizim) and are being monitored by their specialist centre. The remainder are being assessed to ensure that they fulfil the eligibility criteria and patients who do will start treatment within the first year of the managed access agreement coming into force. A very small proportion of the population has decided for personal reasons not to participate.

NHS England and PTC Therapeutics, with the brokering of NICE, agreed a managed access agreement that will enable access to translarna (Ataluren) for the treatment of Duchenne muscular dystrophy. Specialist centres are in the process of reviewing potentially eligible patients to assess their suitability to receive the drug under the terms agreed in the managed access agreement.

In the last 12 months, the National Institute for Health Research Horizon Scanning Research and Intelligence Centre has identified and added to its database the following technologies for which Duchenne muscular dystrophy is an indication:

- ezutromid;

- FG-3019;

- follistatin;

- halofuginone hydrobromide;

- SRP-4045;

- SRP-4053; and

- vamorolone.

Information on how many children are born each year in England with Duchenne muscular dystrophy is not collected. However, it is estimated that there are around 2,500 people living with Duchenne muscular dystrophy in the United Kingdom.

Following the recommendation made by the National Institute for Health and Care Excellence in April 2016 and the announcement made by NHS England on 7 July, Translarna is now routinely commissioned for those individuals who have Duchenne muscular dystrophy and meet the relevant clinical criteria. Currently, there are approximately 50 patients receiving Translarna.

I am very happy to join my right hon. Friend in congratulating all those who were involved in making sure that that innovative drug is available, and I thank her for raising awareness of this very important issue. I know that, as Prime Minister, my right hon. Friend the Member for Witney met Archie, the young man with Duchenne muscular dystrophy, and was inspired by him. I am sure that all Members across the House will welcome the fact that this innovative drug is now available on the NHS. We are committed to ensuring that patients with rare conditions get access to the latest medicines and we are taking some bold steps to speed up that process.

Today is World Duchenne Awareness Day, which is designed to draw attention to a terrible muscle wasting disease that affects a small number of young men. On this day, will the Prime Minister join me in welcoming the recent announcement that the drug Translarna will now be available to these young boys in NHS England, and will she congratulate my constituent Archie Hill, Muscular Dystrophy UK, and all those colleagues in this House and some former Ministers who have worked so hard to make this life-changing drug available in this country?

National Health Service funding decisions for treatments for rare and ultra-rare conditions, such as Translarna (ataluren) for Duchenne muscular dystrophy (DMD), are made by NHS England, as the responsible commissioner for specialised services.

I have been working with the National Institute for Health and Care Excellence (NICE) and NHS England to understand and expedite what can be done to help people access Translarna and have met with MPs, patient groups, drug companies, and parents of children with rare diseases, including DMD, to discuss their concerns about this specific issue.

Following stakeholders’ concerns regarding NHS England’s evaluation processes of treatments for rare conditions, NHS England decided in December 2014 to hold a 90-day consultation on prioritisation for specialised services (held from 27 January to 27 April 2015). This was to ensure that the principles and process for making such decisions were well informed, evidence-led and in line with the expectations of patients and the public.

The Department and the NICE took steps to ensure that Translarna was referred to the NICE as quickly as possible (March 2015) so that final guidance would be available without undue delay. NHS England announced in July that it would wait until the NICE’s guidance was available before developing a funding policy on Translarna.

On 15 April 2016, the NICE announced in its final draft guidance that it was recommending Translarna (ataluren) for the treatment of children aged five and over with DMD caused by a nonsense mutation.

Translarna was recommended only when a) the company provides it at a discounted price to the NHS under the patient access scheme agreed with the Department and b) NHS England and the manufacturer, PTC Therapeutics, in conjunction with patient representatives, agree a managed access agreement setting out (i) the criteria for starting and stopping treatment, which individual patients would be required to agree, (ii) data collection to address considerable uncertainty in the evidence base and (iii) additional confidential financial controls between the company and NHS England.

On 4 May 2016, the NICE announced that it was allowing extra time for further discussions on access arrangements for Translarna to take place before its final guidance is published. NHS England and PTC Therapeutics have been asked to reach agreement on the cost of the drug to the NHS by 7 July 2016.

If the NICE’s final guidance recommends Translarna, NHS England will be legally required to fund it.

Further to the answer given in Question 37646, NHS England has been undertaking the detailed preparatory work required to meet the National Institute of Health and Care Excellence’s request that NHS England and the supplier, PTC Therapeutics, work towards agreeing an acceptable managed access arrangement for Translarna (ataluren) by 7 July 2016.

NHS England will be meeting with the supplier when it has concluded this preparatory work. There have not been any commercial meetings with PTC Therapeutics in the period since 15th April 2016 - however, NHS England is in regular contact with the supplier and will be agreeing mutually convenient times to meet.

NHS England has advised that on 4 May 2016 the National Institute for Health and Care Excellence (NICE) asked NHS England and PTC Therapeutics to continue to work towards agreeing an acceptable managed access arrangement for Translarna (ataluren) for the treatment of children aged five and over with Duchenne muscular dystrophy caused by a nonsense mutation.

NHS England and the company have been asked to reach agreement on the cost of the drug to the National Health Service by 7 July.

NHS England has advised there have not been any commercial meetings with PTC Therapeutics in the period since 15 April 2016, however, it is in regular contact with the company.

NHS England will be meeting with PTC Therapeutics when it has concluded the detailed preparatory work required to effectively respond to NICE's request within the set timescale.

The National Institute for Health and Care Excellence (NICE) is currently evaluating Translarna (ataluren) for the treatment of Duchenne muscular dystrophy through its highly specialised technology programme. The expected publication date for NICE’s final guidance is July 2016.

National Health Service commissioners are legally required to fund drugs and treatments recommended in NICE highly specialised technology guidance within three months of its final guidance being issued.

On 15 April 2016, the National Institute for Health and Care Excellence (NICE) announced in its final draft guidance that it was recommending Translarna (ataluren) for the treatment of children aged five and over with Duchenne muscular dystrophy caused by a nonsense mutation.

Translarna was recommended only when (a) the company provides it at a discounted price to the National Health Service under the patient access scheme agreed with the Department and (b) that NHS England and the manufacturer, PTC Therapeutics, in conjunction with patient representatives, agree a managed access agreement setting out (i) the criteria for starting and stopping treatment, which individual patients would be required to agree, (ii) data collection to address considerable uncertainty in the evidence base and (iii) additional confidential financial controls between the company and NHS England.

On 4 May 2016, NICE announced that it was allowing extra time for further discussions on access arrangements for Translarna to take place before its final guidance is published. NHS England and PTC Therapeutics have been asked to reach agreement on the cost of the drug to the NHS by 7 July 2016.