Final Neuroscience Specification released

After an incredibly hectic few weeks at the Scottish lobby, the Northern Ireland Lobby and in Westminster with Takin’ Charge, I’m finally getting back to writing up this blog.

On Monday 10th of June the final specialised service specification for England was released. You can find the final neurosciences service specification here.

What are Service Specifications?: The Service Specifications are government documents which have been created to remove the inconsistencies in service provision across England. They do this by setting out what specialised services need to be available in every region throughout the country. The 10 page Annex of the Neuroscience specification is dedicated to Neuromuscular conditions. There are very positive elements in the specification: It details the need for multi-disciplinary teams in every region, which will comprise of neuromuscular Consultants, neuromuscular physiotherapist, Neuromuscular Nurse, Occupational Therapist, Speech and Language Therapist (SALT), dietician, psychologist and neuromuscular care coordinator. It has specific sections on Dietary Care, Respiratory Care,  Cardiac Care, Orthopaedic and Spinal Care, Wheel Chair Services, Orthotic Provision, Pyschological Support and Transitional Care. Another key point is that they will map provision and through this process highlight measures for improving clinical outcomes, patient experience and integration of primary, secondary and tertiary services.

We have consulted on this document twice – you can request our response to the both rounds of consultations if you’d like by contacting myself at

This document is potentially a major step toward the provision of high quality care in every region of England. It is hoped that this national approach to commissioning will tie existing services together and map the provision of these services in the different regions – thereby addressing the inconsistency of provision of NMD services throughout England.

What will happen now?: The Service Specification will come into effect in October 2013. From then on, the care provision standards detailed in the specification will begin to be commissioned. Action Duchenne will ensure that gaps in services in every region is highlighted to commissioners and government so that everyone will access to the best standards of care available.


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Minister of State at the Department of Health Norman Lamb asked about Access to Drugs and Centres of Excellence

In Westminster today Norman Lamb MP, the Minister of State at the Department of Health, was questioned by Mark Durkan MP, Gary Streeter MP, George Freeman MP and Mary Glindon MP on access to high-cost drugs and Centres of Excellence.

Mark Durkan, MP for Foyle, referenced the Muscular Dystrophy APPG’s inquiry into regulation and cost of potential treatments, and requested the Minister’s presence at the all-party group to discuss measures for the removal of any impediments for new treatments. Mr Lamb agreed to this, and pointed out that NICE will now be responsible for the evaluation of new drugs for the treatment of rare conditions.

The MP for South West Devon, Mr Gary Streeter, mentioned a seven year old boy in his constituency who lives with Duchenne Muscular Dystrophy. He asked the Minister when ataluren may become available. Mr Lamb replied that no time scale had been set out but that the manufacturer of ataluren has now applied for conditional approval.

Following the meeting on the 15th April with ‘Empower:Access to Medicine Campaign‘, a supporter of the group George Freeman MP, requested a meeting with the Minister. The Minister agreed to meet with Mr Clifton-Brown MP and Mr Freeman MP to develop the campaign for greater patient involvement in accessing rare drugs.

Mrs Mary Glindon, another member of the APPG for Muscular Dystrophy, highlighted the importance of centres of excellence, such as the International Centre for Life in Newcastle,  in clinical trials. Mr Lamb also agreed to meet with Mrs Glindon to discuss the changes to specialist neuromuscular care with NHS North of England.

You can find these MPs on Twitter:

@Freeman_George, @normanlamb@garystreetermp@markdurkan

Please also sign Empower’s epetition by clicking HERE.

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Potential DMD treatments discussed in Science and Technology Committee

Science and Technology Committee, 13th March: Clinical trials
Witnesses: Professor Sir Michael Rawlins, Chair of Medical Sciences Regulation and Governance Review, Dr Keith Bragman, President, The Faculty of Pharmaceutical Medicine, and Dr Fiona Godlee, British Medical Journal

This discussion focused around barriers to clinical trials and included mentions of possible treatments for Duchenne Muscular Dystrophy.

It has been agreed that it takes more time in the UK to conduct trials than in other countries due to bureacratic systems.

The amount of bureacracy and the ‘one size fits all’ approach to all clinical trials led a an overburdensome system, Prof Rawlins argued giving an example of 62 CRB checks being done by 62 hospitals for one trial.

Graham Stringer MP, asked whether it was difficult to hold trials for Orphan drugs such as Duchenne, because it was so rare. Prof Rawlins answered that it depended on the situation, but that trials conducted by one centre of excellence can actually involve much less bureacracy.

The publishing of trial data, which is the focus of a current campaign by Ben Goldacre, was also discussed.

The Muscular Dystrophy APPG is currently holding an enquiry into research into the following issues:

  • Ways of speeding up access to high cost drugs for orphan diseases
  • The National Institute for Health and Clinical Excellence (NICE) taking on the assessment of very high cost, low volume drugs from the Advisory Group for National Specialised Services from April 2013
  • Setting in place a strategy to overcome potential barriers and delays
  • Impact of NHS reforms on the commissioning and funding arrangements
  • Challenges of delivering potentially costly therapies given the need for significant NHS efficiency savings
  • Organisations and determinants responsible for bringing drugs to the marketplace and their role
  • Views of people affected by muscular dystrophy and related neuromuscular conditions

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APPG for Disability on the Children and Families bill

Janet and Celine as part of the Takin’ Charge project  and representing Action Duchenne, attended the joint meeting of All Party Parliamentary Groups on Disability on Wednesday 20th March chaired by the Rt Hon. Tom Clarke MP and attended by  Mr Timpson parliamentary under secretary of State and the Department for Education who spoke  on aspirations for the SEN  in the Children and Families Bill, 2013.

The key proposals set out in the Bill cover children and young people from birth to 25 years:

  • Statements of SEN and Learning Difficulty Assessments will be replaced with Education, Health and Care Plans (EHCPs) the aim of which is to look holistically at the needs of children or young people with SEN from birth to 25;

  • Services will be JOINTLY commissioned by Education, Health and Social Care;

  • A Local Offer of published information about services in its area that it expects to be available for children and young people with SEN;

  • Extending statementing rights – now to be contained in EHCPs – into post-16 education for the first time;

  • Introducing personal budgets to secure a child or young person’s SEN provision that is set out in an EHCP;

  • Introducing new appeals and mediation measures to extend the right of appeal to the First Tier Tribunal to young people aged up to 25 and to introduce optional mediation before someone wishes to appeal against a decision in relation to SEN;

  • Publishing a new Code of Practice giving advice to education professionals, including those in the post-16 education sector.

We welcome these proposed changes. However, the issue for us as parents and professionals concerned with the needs of boys with Duchenne Muscular Dystrophy is that currently if a young disabled person does not have ‘learning needs’ they will not be eligible for an Education Health and Care Plan.  The Government is of the view that disabled people already have protections in the Children’s Act 1989 and the Equality Act, 2010 – however, we feel it is crucial that young people with a complex condition like Duchenne will need an EHC plan in order to access all the support that they need, whether they have learning needs or not.

This was being challenged yesterday in the APPG and Janet and Celine will be writing to Baroness Tani Grey and Lord Low, both cross bench peers to challenge this further and explain in detail the implications of this for young people and families living  with Duchenne Muscular Dystrophy.

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Welsh Cross Party Group on Muscular Dystrophy

On the 6th March I attended the Welsh Cross party Group meeting.

This meeting was chaired by Bethan Jenkins AM and was attended by Dr Andrew Goodall (Cheir Executive Aneurin bevan Health Board and Chair of WNMN) and Dr Eleanor Marsh (Specialist Registrar in Neurology and Clinical Coordinator for WNMN). Healthcare professionals, Care Advisors, families and patients were also in attendance.

The meeting began with an update on the ‘Welsh Neuromuscular Network Vision’. This ‘vision’ aims to develop neuromuscular services, determine a strategic direction for neuromuscular services, share good practice and act as a reference group and resource for professionals across Wales; that is, this network’s primary aim is to raise standards of care and support to create an ‘equitable and accessible service for patients and their families across Wales’.

The hope is that a business case can be made for specific developments. This could lead to the creation of Service Specifications incorporating published Standards of Care and quality measurements to ensure equitable access to best quality services.

The update of the Neuromuscular Vision document maps current provision and describes areas which have gaps. Areas singled out as highly problematic are Transition, specialist physiotherapy and clinical psychology and counselling involvement.

The document calls for full time care advisor roles, the appointment of specialist adult physiotherapists, specialist consultants and a specialist neuromuscular pyschologist.

There is also a case made for shared electronic health records supported by an administrative network manager.

If you’d like a copy of this vision document please contact

The Welsh Muscle Network are looking for feedback on this document over the next two weeks so please get in contact.

The meeting concluded with a discussion on Personal Independence Payments (PIP), which are replacing Disability Living Allowance (DLA) in April for those between 16-64 and not already on DLA. Those already on DLA, they will be reassessed for PIP after 2015.

You can find a lot of information on this changeover here.

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Northern Ireland All Party Group Meeting on the McCollum Report response

On Tuesday 5th March, I attended the All Party Group (APG) meeting in Northern Ireland. This meeting was chaired by Conall McDevitt MLA, Robin Swann MLA and attended by Pam Brown MLA and Kieran McCarthy MLA. Representatives from the Belfast Trust were also in attendance alongside families and people living with Muscular Dystrophy.

This meeting followed the response to the McCollum report released on the 13th February.

You can find this response here.

The response had a number of very positive elements. The response named Michelle Tennyson, the Assistant Director of Allied Health Professions and Public Patient Involvement at the Public Health Agency, as the Northern Ireland lead for muscular dystrophy and related neuromuscular conditions. This is a big step forward to ensure equitable and accountable development of services.

A neuromuscular registry has already started being set up and a Duchenne Muscular Dystrophy ‘patient pathway’ is going to be developed for patients and their carers.

Further interesting points include a clinical network which will incorporate Neuromuscular conditions. This is being set up to improve links with specialist neuromuscular centres. Increased clinical time and transition workers are also recommendations which have been given support.

The APG on Muscular Dystrophy met to discuss this response and the Welfare Reform changes from Disability Living Allowance (DLA) to Personal Independence Payments (PIP).

The response was generally welcomed and the work of Conall McDevitt MLA, Robin Swann MLA and other parliamentary supporters, as well as the patients and families who attended campaigning events, was recognised. However, there were areas where concerns were raised.

Recommendation 11 highlights the NICE accredited Standards of Care. The response accepts the possibility of moving them from ‘best practice’ to actual clinical guidelines. Janet Bailie pointed out that these Standards were created by the experts in DMD and should be followed where possible.

The response to Recommendation 5 discusses the extra links between Centres of Excellence in England and healthcare professionals in Northern Ireland, with the objective of filtering down expertise. However the structure of such links were seen as quite vague.

Recommendation 15’s response was also seen as a dissapointment, with the lack of integration and communication between Health Trusts not being fully addressed.

The main focus of the discussion centred around recommendation 12 on wheelchair services. The continual lack of improvement in wheelchair services, and numerous stories of large waiting times and having to battle the system, is an unacceptable state of affairs. The response to Recommendation 15 does not acknowledge these problems.

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Scotland CPG on Integration of Health and Social Care 19/02/2013

On the 19th of February, Jackie Bailie MSP chaired the very well attended Cross Party Group meeting on Muscular Dystrophy.

This meeting focused on the new legislation in Scotland which will bring together Health and Social Care services.

The Consultation response from the Scottish Government was released on the 13th February. This response did not address many of the issues surrounding disparity of care and how this integration will happen on the ground. Jackie Bailie MSP stated that more work needs to be done on how patient pathways can bring together health and social care services at a local level.

It was agreed that money may lie at the heart of this integration. Shared budgets to commission joint services was seen as incredibly important. There is need for more certainty in funding to put a stop to the to and fro between Health and Social Care services. Jackie Bailie MSP also drew attention to the ‘minimum range of functions’ of primary care which ‘must be included’ in the integrated budgets. It is vital that the ‘minimum range’ fulfills all the complex requirements of those living with Duchenne Muscular Dystrophy.

Empowering patients to assess their own needs and putting them at the heart of their care (via Self-Directed Support) was also seen as a vital aspect of integration. The cost of emergency admissions, at £2000 per day, must be avoided to save money, and this can only be done with proper monitoring of the condition and high quality care. These two strands compliment one another.

The final topic was Care Advisers. It was argued that three was not enough (although there are currently only two in post, with a third to be in post very soon). Arguments were put forward for more specialist nurses and social workers instead of Care Advisers as there was a feeling that Care Advisers roles often overlap into others and there role is not clearly defined. Possible Pilot Projects for Personal Assistants to be care coordinators was floated as an idea.

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Campaigning Update

A list of important dates which you should be aware of:
Scottish CPG on integration of Health and Social Care: Tuesday 19th February, 6pm – 7.30pm, Meeting Room TG-20-TG-21, Scottish Parliament
Westminster APPG for Muscular Dystrophy: future access to personalised medicines and the regulatory environment for orphan drugs: Tuesday 26th February, 12.30pm to 2pm, Committee Room 8, Houses of Parliament
Welsh CPG on Welfare Reform and accessing benefits: Wednesday 6th March, 6pm – 7.30pm, Conference Room 24, Ty Hywel, National Assembly for Wales, Cardiff, CF99 1NA
The Northern Ireland APG dates have yet to be announced.

To attend any of these events please contact me or

Rare Disease Days: Rare Disease events are being held all over the country to raise awareness of living with a rare disease. Please join us at the below events and write to your parliamentarian, so you can meet them and speak to them about the challenges you face:
Welsh Assembly, Cardiff – Wednesday 20th February 6pm – 8pm
Scottish Parliament, Edinburgh – Tuesday 26th February 6:30pm – 8:30pm
House of Commons, London – Wednesday 27th February 1pm – 3pm

Places are still available and you can register here.

Consultation Response to the Neuromuscular Service Specification. Action Duchenne’s consultation response can be requested.

The service specification sets out guidelines which aim to reduce the current ‘postcode lottery’ of provision. The focus on multi-disciplinary teams based at regional specialist centres is a huge step forward, after Action Duchenne has campaigned on these issues for many years.

The specification will continue to change over the coming months and it does not cover every aspect of care which is required by those living with DMD. However, it will come into use in April. This is a great step in the right direction and we hope to see the necessary changes implemented as soon as possible.

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Personal Independence Payments and Service Specification Consultations

Over the last week, there has been two big step towards the new-look NHS and benefits system. On the 12th of December, Service Specifications, which set out plans for nationally commissioned specialised services, have been released for consultation. This was followed by the Personal Independence Payment (PIP) regulations on the 14th of December.

The Service Specifications have been created to remove the inconsistencies in service provision across England. They also, for the first time, set out a national guide for provision; highlighting measures for improving clinical outcomes, patient experience and integration of primary, secondary and tertiary services.

The service specification which relates to Specialised Neuromuscular Services can be found from page 11 here. It is hoped that this national approach to commissioning will tie existing services together and map the provision of these services in the different regions – thereby addressing the inconsistency of provision of NMD services throughout England. This consultation is now under way  with the NHS seeking responses from families and organisations on these plans. Action Duchenne shall be responding, but please feel free to respond yourselves. If you’d like any help or information then please contact me. The consultation will close on the 25th of January, so please contact me before the 20th!

Personal Independence Payments (PIP) are replacing Disability Living Allowance (DLA) for disabled people ageing from 16-64 on the 8th April. People in receipt of DLA will not automatically qualify for PIP and all three million of them will need to be reassessed. This assessment is based on how your condition affects you, not what condition you have.

Around 500,000 current claimants of DLA will, it is estimated, have their benefits package reduced. 

All the information available on PIP can be found here. If you have any further questions, then please contact me:

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Scotland Parliamentary Debate

On the 20th November Action Duchenne’s motion from our lobby in June was debated in Holyrood. Jim Eadie MSP for Edinburgh Southern for lead the debate with Jackie Bailie MSP (Dumbarton), Graeme Dey MSP (Angus South), Nanette Milne MSP (North East Scotland), Mary Fee MSP (West Scotland), Malcolm Chisholm MSP (Edinburgh Northern and Leith) all contributing.

The debate focused on research breakthroughs and access to drug trials in Scotland. Jim Eadie MSP highlighted how Scotland should contribute to international research saying:

“The Scottish Government, through the chief scientist office and the health research strategy, has an important role to play [in funding research]. A co-funding arrangement would allow greater value and greater resource to be generated by the chief scientist office, working in partnership with medical research charities and potentially other health departments in the UK, to support this pioneering research. That has the potential to deliver the genetic medicines that will benefit boys and men with DMD.”

All MSPs made important points about the recent breakthroughs in research. With Malcolm Chisholm MSP stating: “the chief scientist’s office has a significant budget for research and its current chief scientist, the brilliant Professor Andrew Morris, has a particular interest in the genetics of medicine. I hope that the Scottish Government will respond positively to Jim Eadie’s call for the involvement of the Scottish Government and the chief scientist in partnership funding for research“.

Michael Matheson MSP, the Public Health Minister, responded by saying that “the Scottish Government’s chief scientist office is happy to consider co-funding arrangements with leading UK medical charities in particular areas of research. I also reassure members that a large amount of the research that takes place in Scotland involves international collaboration“.

This is fantastic news for research opportunities for Duchenne Muscular Dystrophy in Scotland and Action Duchenne hopes that this is the start of a very fruitful partnership.

Other areas pertinent to those living with Duchenne in Scotland were also addressed, with Nanette Milne MSP saying: “DMD sufferers deserve the best possible care and support and state-of-the-art equipment to help them cope with their disabilities.”

Michael Matheson also addressed the challenges of providing the best standards of care for Duchenne. After a question by Jackie Bailie MSP, Mr Matheson advised that there had been ‘recruitment difficulties’ with the hiring of a Care Advisor in the north of Scotland, but that they would ‘monitor’ the situation during the re-advertisement of the post.

Mr Matheson also stated that the has been a £1.5million investment into improving services for people with complex respiratory conditions. This investment shall “help to recruit new specialists, whether they are consultants, dieticians, physiotherapists, nurses or allied health professionals, so that people can be treated more effectively locally.”

Action Duchenne will be meeting with MSPs and the Chief Scientific Office in the coming weeks to ensure that this debate acts as a catalyst to further progress. We will contiinue to work alongside the Scottish Muscle Network and the Muscular Dystrophy Campaign to ensure that thsoe living with Duchenne Muscular Dystrophy have access to the care they need and that Scotland continues on its route to become a “an attractive place in which to conduct world-leading clinical research.

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