The Managed Access Agreement relating to Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy, including a list of signatories, is available at the following link:

https://www.nice.org.uk/guidance/hst3/resources

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.

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.

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 publication date for NICE’s final guidance is to be confirmed.

NHS England has advised that it is in constructive discussions with the pharmaceutical company about the development of a managed access agreement for Translarna and a progress update will be given at the next NICE Evaluation Committee meeting on 17 February 2016.

In the absence of guidance from NICE, it is for commissioners to make decisions on whether to fund medicines based on an assessment of the available evidence.

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 publication date for NICE’s final guidance is to be confirmed.

NHS England has advised that it is in constructive discussions with the pharmaceutical company about the development of a managed access agreement for Translarna and a progress update will be given at the next NICE Evaluation Committee meeting on 17 February 2016.

In the absence of guidance from NICE, it is for commissioners to make decisions on whether to fund medicines based on an assessment of the available evidence.

The National Institute for Health and Care Excellence (NICE) is the independent body that provides guidance for the National Health Service on whether to fund selected drugs and treatments. NICE is currently evaluating Translarna (ataluren) for the treatment of Duchenne muscular dystrophy through its highly specialised technology programme. The publication date for NICE’s final guidance is to be confirmed.

The NHS in England is legally required to fund drugs and treatments recommended in NICE highly specialised technology guidance within three months of its final guidance being issued. In the absence of guidance from NICE, it is for commissioners to make decisions on whether to fund medicines based on an assessment of the available evidence.

The National Institute for Health and Care Excellence (NICE) is the independent body that provides guidance on the clinical and cost effectiveness of drugs and treatments. NICE is currently evaluating Translarna (ataluren) for the treatment of Duchenne muscular dystrophy through its highly specialised technology programme. NICE currently expects to publish its final guidance in February 2016.

The NHS in England is legally required to fund drugs and treatments recommended in NICE highly specialised technology guidance within three months of its final guidance being issued. In the absence of guidance from NICE, it is for commissioners to make decisions on whether to fund medicines based on an assessment of the available evidence.

The National Institute for Health and Care Excellence (NICE) is evaluating Translarna (ataluren) for the treatment of Duchenne muscular dystrophy through its highly specialised technology programme. NICE currently expects to publish its final guidance in February 2016.

The National Health Service in England is legally required to fund drugs and treatments recommended in NICE highly specialised technology guidance within three months of its final guidance being issued. In the absence of guidance from NICE, it is for commissioners to make decisions on whether to fund medicines based on an assessment of the available evidence.

I met with Muscular Dystrophy UK and the parents of sons with Duchenne Muscular Dystrophy, alongside the hon. Member for Winchester on 8 September 2015.

I met again with the Muscular Dystrophy Campaign (MDC) alongside the hon. Member for Leeds North West (Greg Mulholland) on 26 March 2015.

The former Minister of State (Norman Lamb) met with the hon. Member for Blaydon (David Anderson) and a representative of the MDC group on 2 March 2015 where the issue of funding for Translarna was raised.

I met with the parents of children with Duchenne Muscular Dystrophy and Muscular Dystrophy UK, alongside the hon. Member for Winchester (Steve Brine) on 8 September 2015.

I also met with the Muscular Dystrophy Campaign (MDC) alongside the hon. Member for Leeds North West (Greg Mulholland) on 26 March 2015.

The former Minister of State for Care Services (Norman Lamb) met with the hon. Member for Blaydon (David Anderson) and a representative of the MDC group on 2 March 2015 where the issue of funding for Translarna was raised.

NHS England published its investment decisions for certain specialised services on 2 July 2015.

NHS England has agreed with the recommendations from NHS England’s Clinical Priorities Advisory Group that a final funding decision on Ataluren (Translarna) for Duchenne muscular dystrophy should be made after the National Institute for Health and Care Excellence (NICE) has concluded its Highly Specialised Technology Appraisal process.

NICE is expected to produce draft guidance on Ataluren (Translarna) in October, with final guidance to follow shortly after. The European medicines regulator has also asked the manufacturer to undertake further studies of the treatment for completion in October.

There is also a process which allows for the consideration of critically clinically urgent cases falling outside of routine commissioned provision.

I have had a number of meetings with patients and their families, hon. Members, charities and patient groups and I am keeping in close contact via correspondence with families and campaigners on this issue and on the availability of another drug, Vimizim for the treatment of Morquio syndrome. Whilst the key decisions are for the National Institute for Health and Care Excellence (NICE) and NHS England, I have also met with the manufacturers and NHS England and had discussions with NICE about ensuring as rapid a decision as possible and I am doing all I can to help those affected and get a swift resolution to this situation.

Following discussions between the Department, NHS England and the National Institute for Health and Care Excellence (NICE), it was decided that Translarna (ataluren) for treating Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene should be considered for formal referral to NICE’s highly specialised technologies (HST) programme which evaluates treatments for very rare conditions. NICE has recently consulted stakeholders on the draft remit and scope of its proposed HST evaluation of Translarna.

Ministers will take a decision on the formal referral of this topic following the outcome of NICE’s scoping work.

The review of the pathway for the development, assessment, and adoption of innovative medicines and medical technologies will consider how to speed up access for National Health Service patients to cost-effective new diagnostics, medicines and devices. We anticipate an initial report before the end of the year.