The UK Strategy for Rare Diseases commits all four countries of the United Kingdom to work to achieve reduced times for diagnosis of rare diseases and to work with the National Health Service and clinicians to establish appropriate diagnostic pathways which are accessible to, and understood by, professionals and patients.

NHS England commissions a highly specialised service for liver disease in children, including cirrhosis resulting from lysosomal acid lipase, from three expert centres in London, Birmingham and Leeds. These long established centres provide a clear referral pathway for expert diagnosis and management.

We have referred a number of high cost, low volume technologies to the National Institute for Health and Care Excellence (NICE) for evaluation under its highly specialised technologies work programme. We will continue to do so for technologies that meet the specified criteria and where a NICE evaluation would add value. Further information about the process by which topics are selected for referral to NICE’s highly specialised technologies work programme is available at:

www.nice.org.uk/about/what-we-do/our-programmes/topic-selection

NHS England has responsibility for the commissioning of specialised services and is currently consulting on changes to the principles and processes by which it makes decisions on prioritising investment in specialised services.

NHS England has provided assurance that any prioritisation which is urgent on clinical grounds will continue to be dealt with quickly through their existing procedures. The route for individual funding requests remains in place.

The consultation can be found at:

www.engage.england.nhs.uk/consultation/investing-in-specialised-commissioning

The Patient Experience of Diabetes Services (PEDS) pilot, a module of the National Diabetes Audit, was run last year. While the potential value of this survey is clear, there are other competing priorities for adding modules to existing national audits such as this. There is a robust process for assessing these, as well as which new national audits should be considered. As such, NHS England will review the potential to fund the PEDS survey alongside other Healthcare Quality Improvement Partnership commissioned national audit and associated modules.

Other NHS England surveys capture the experiences of people with a wide range of conditions including diabetes. However, with the exception of the cancer patient experience survey, the information is not collected by specific conditions. As part of a forthcoming review of patient experience surveys, NHS England will consider whether it is best to collect information as it does currently or by specific conditions.

NHS England has a selection of icons for use by staff, to highlight and add depth to a piece of work or to be used as a navigation tool.

NHS England has confirmed with the British Red Cross that the use of the symbol on the Consultation Guide: Investing in Specialised Services does not imitate their logo or breach their branding guidelines.

A copy of the document is attached.

Sodium valproate has been authorised since the early 1970’s for the treatment of epilepsy. It was known at the time of licensing that valproate was associated with an increased risk of birth defects and it was authorised for the treatment of women of childbearing potential only where other treatments were ineffective. The authorised product information (Summary of Product Characteristics or SPC and Patient Information Leaflet or PIL) contain warnings about the risks associated with use in pregnancy.

The Medicines and Healthcare products Regulatory Agency (MHRA) initiated a Europe-wide review of the risk of developmental disorders in October 2013 following publication of studies which provided further information on the magnitude and nature of the risk of developmental disorders in children born to mothers who took valproate in pregnancy. The Europe-wide review, led by the United Kingdom and the Netherlands, was completed in November 2014 and MHRA is working to fully implement the conclusions of that review which are binding in all Member States. There will be a mandatory requirement for all manufacturers to update the valproate SPC and PIL with strengthened warnings and in addition new educational materials will be provided to healthcare professionals and patients.

Sodium valproate has been authorised since the early 1970’s for the treatment of epilepsy. It was known at the time of licensing that valproate was associated with an increased risk of birth defects and it was authorised for the treatment of women of childbearing potential only where other treatments were ineffective. The authorised product information (Summary of Product Characteristics or SPC and Patient Information Leaflet or PIL) contain warnings about the risks associated with use in pregnancy.

The Medicines and Healthcare products Regulatory Agency (MHRA) initiated a Europe-wide review of the risk of developmental disorders in October 2013 following publication of studies which provided further information on the magnitude and nature of the risk of developmental disorders in children born to mothers who took valproate in pregnancy. The Europe-wide review, led by the United Kingdom and the Netherlands, was completed in November 2014 and MHRA is working to fully implement the conclusions of that review which are binding in all Member States. There will be a mandatory requirement for all manufacturers to update the valproate SPC and PIL with strengthened warnings and in addition new educational materials will be provided to healthcare professionals and patients.

The Government is committed to ensuring that patients have access to effective treatments, including those for pancreatic cancer, on terms that represent value to the National Health Service and the taxpayer.

The National Institute for Health and Care Excellence (NICE) is the independent body responsible for providing advice to the NHS on the clinical and cost-effectiveness of health technologies.

NICE has recommended gemcitabine as a treatment option for pancreatic cancer in technology appraisal guidance published in May 2001, subject to certain clinical criteria, and has been asked to appraise a number of other pancreatic cancer drugs. NHS commissioners are legally required to fund treatments recommended by NICE technology appraisal guidance.

Where a pancreatic cancer drug is not routinely available on the NHS, patients may be able to access it through the Cancer Drugs Fund.

NHS England’s Cancer Drugs Fund panel plans to assess, on the basis of the latest evidence, whether certain drugs, including Abraxane (albumin bound paclitaxel) for advanced pancreatic cancer, should continue to be made routinely available to new patients through the Fund and to consider a number of new drugs for potential addition to the Fund.

NHS England has assured the Department that no patient whose treatment is currently being funded through the Fund will have funding withdrawn, as long as it is clinically appropriate that they continue to receive that treatment. In addition, no drug will be removed from the Fund where it is the only therapy for that condition.

We are committed to maintaining the Cancer Drugs Fund until the end of March 2016. We will carefully consider with NHS England what arrangements should be put in place for the long term.

Surgery, radiotherapy and chemotherapy treatments that may be used for pancreatic cancer are commissioned by NHS England. NHS England’s pancreatic cancer service specification clearly defines what it expects to be in place for providers to offer evidence-based, safe and effective pancreatic cancer services.

NHS England has also committed to make up to £6 million available over the next three years to support six trials by Cancer Research UK - one of which will be on pancreatic cancer - into the use of Stereotactic Ablative Radiotherapy (SABR), an innovative radiotherapy treatment. This will allow patients to receive SABR treatment where clinicians think they could benefit. At the same time doctors can fully assess the effectiveness of this treatment so that, if it proves to be effective, it will be available for patients on the NHS where appropriate.

We are also commissioning an external review of the pathways for the development, assessment, and adoption of innovative medicines and medical technology. This review will consider how to speed up access for NHS patients to cost-effective new diagnostics, medicines and devices.

The Government is committed to ensuring that patients have access to effective treatments, including those for pancreatic cancer, on terms that represent value to the National Health Service and the taxpayer.

The National Institute for Health and Care Excellence (NICE) is the independent body responsible for providing advice to the NHS on the clinical and cost-effectiveness of health technologies.

NICE has recommended gemcitabine as a treatment option for pancreatic cancer in technology appraisal guidance published in May 2001, subject to certain clinical criteria, and has been asked to appraise a number of other pancreatic cancer drugs. NHS commissioners are legally required to fund treatments recommended by NICE technology appraisal guidance.

Where a pancreatic cancer drug is not routinely available on the NHS, patients may be able to access it through the Cancer Drugs Fund.

NHS England’s Cancer Drugs Fund panel plans to assess, on the basis of the latest evidence, whether certain drugs, including Abraxane (albumin bound paclitaxel) for advanced pancreatic cancer, should continue to be made routinely available to new patients through the Fund and to consider a number of new drugs for potential addition to the Fund.

NHS England has assured the Department that no patient whose treatment is currently being funded through the Fund will have funding withdrawn, as long as it is clinically appropriate that they continue to receive that treatment. In addition, no drug will be removed from the Fund where it is the only therapy for that condition.

We are committed to maintaining the Cancer Drugs Fund until the end of March 2016. We will carefully consider with NHS England what arrangements should be put in place for the long term.

Surgery, radiotherapy and chemotherapy treatments that may be used for pancreatic cancer are commissioned by NHS England. NHS England’s pancreatic cancer service specification clearly defines what it expects to be in place for providers to offer evidence-based, safe and effective pancreatic cancer services.

NHS England has also committed to make up to £6 million available over the next three years to support six trials by Cancer Research UK - one of which will be on pancreatic cancer - into the use of Stereotactic Ablative Radiotherapy (SABR), an innovative radiotherapy treatment. This will allow patients to receive SABR treatment where clinicians think they could benefit. At the same time doctors can fully assess the effectiveness of this treatment so that, if it proves to be effective, it will be available for patients on the NHS where appropriate.

We are also commissioning an external review of the pathways for the development, assessment, and adoption of innovative medicines and medical technology. This review will consider how to speed up access for NHS patients to cost-effective new diagnostics, medicines and devices.

We have had no such discussions. These are matters for NHS England’s Cancer Drugs Fund clinical panel.

The panel plans to meet on 15 and 16 December to assess, on the basis of the latest evidence, whether certain drugs should continue to be made routinely available to new patients through the Fund and to consider a number of new drugs for potential addition to the Fund.

In making such decisions, the panel will take into account a number of factors, including unmet need.

NHS England has assured the Department that no patient whose treatment is currently being funded through the Fund will have funding withdrawn, as long as it is clinically appropriate that they continue to receive that treatment. In addition, no drug will be removed from the Fund where it is the only therapy for that condition.

We have had no such discussions. These are matters for NHS England’s Cancer Drugs Fund clinical panel.

The panel plans to meet on 15 and 16 December to assess, on the basis of the latest evidence, whether certain drugs should continue to be made routinely available to new patients through the Fund and to consider a number of new drugs for potential addition to the Fund.

In making such decisions, the panel will take into account a number of factors, including unmet need.

NHS England has assured the Department that no patient whose treatment is currently being funded through the Fund will have funding withdrawn, as long as it is clinically appropriate that they continue to receive that treatment. In addition, no drug will be removed from the Fund where it is the only therapy for that condition.

Iron deficiency anaemia can be caused by a number of different underlying conditions and it is for health professionals to advise on the appropriate treatment for individual patients. The majority of people should be able to get all the iron their body requires by eating a varied and balanced diet.

The Government recognises the important role that social enterprise organisations play in the provision of healthcare, and is committed to ensuring that patients receive healthcare services from the providers that are most capable of meeting their needs and improving the quality of services that they provide.

The Department engages frequently with social enterprise organisations that provide healthcare in a wide variety of ways. This includes through initiatives such as the Mutuals Support Programme and the Investment and Contract Readiness Fund both run by Cabinet Office and the Technology Spin-out Fund launched earlier this year by the Department, Big Society Capital and local partnerships. As part of a recently published independent report commissioned by the Department on staff engagement and empowerment in the National Health Service, interviews were held with over thirty providers and workshops with over 150 stakeholders.

The Government firmly believes that patients must receive healthcare services from the providers that are most capable of meeting their needs and improving the quality of services that they provide, regardless of what sector that organisation comes from, including public, private, social enterprises and voluntary services.

It is right that these decisions should be taken independently by local commissioners in the best interests the populations for which they are responsible.

While the Public Services (Social Value) Act 2012 is kept under constant review, there are no current plans to amend it. Under the Act commissioners must consider how improvements to the social well-being of their local area can be achieved from the services they are procuring. The Government believes that this is the right approach to improve the quality of public services, whilst creating better value for money for taxpayers.

This Government believes that social enterprise providers make a valuable contribution to the delivery of NHS services.

In October 2013 the Government commissioned an independent review of staff engagement and empowerment in the National Health Service, led by Chris Ham, Chief Executive of the King’s Fund. The report was published in July 2014 sets out the impact of staff owned mutuals and social enterprises.

The publication notes that “a period of ‘accelerated evolution’ and evaluation of existing and alternative models would enable further evidence to be gathered about the impact of different organisational forms on staff engagement and performance.”

The publication by the Kings Fund can be found at:

www.kingsfund.org.uk/publications/articles/improving-nhs-care-engaging-staff-and-devolving-decision-making

In response, the Government has set up a £1million fund to support “pathfinder” organisations in the acute sector explore how mutual models could increase staff engagement across their organisations.

NHS England is currently conducting a review into urgent and emergency care in England. The Review, led by Professor Sir Bruce Keogh, aims to help build an improved, safer and more sustainable system for the future.

The end of first stage Report on the Review, published in November 2013, set out NHS England’s vision for the future delivery of urgent and emergency care, but recognised that this vision will take three to five years to implement the transformational change proposed.

Since November last year, the Review team at NHS England has been working collaboratively with a wide range of stakeholders from across the system to work out the practicalities for delivering the transformational change required. Work on the cost implications and benefits in terms of patient outcomes, for all aspects of the Review (not just in relation to out-of-hospital urgent care), is ongoing.

NHS England will update on progress with the Review later this year.

Information on the various urgent and emergency care services that operate in England is available on the NHS Choices website to help patients and the public make good choices about what type of facility may best suit their needs. This is supported by a “Find Urgent Care services” portal which allows users to enter a postcode and obtain directions to their nearest facility.

More broadly, NHS England is currently conducting a review into urgent and emergency care services in England. The Urgent and Emergency Care Review aims to reduce pressure on accident and emergency (A&E) by delivering a system that enables more patients to be treated outside of hospital.

The end of first stage Report on the Review, published in November 2013, recognised the need to deliver highly responsive urgent care services outside of hospital so people no longer choose to queue in A&E. The Review further recognised that there are a range of urgent care services currently open including ‘walk-in centres’, ‘minor injury units’, ‘urgent care centres’ and other similarly named facilities that all offer slightly different services, at slightly different times, in different places.

In response to this, the Review proposed to support the co-location of community-based urgent care services in coordinated urgent care centres. These will be locally specified to meet local need, but should consistently use the “urgent care centre” name, to replace the multitude of terms that are available at present. Urgent care centres may provide access to walk-in minor illness and minor injury services, and will be part of the wider community primary care service including out-of–hours general practitioner services.

Since November last year, the Review team at NHS England has been working collaboratively with a wide range of stakeholders from across the system to work out

the practicalities for delivering this change, to ensure that all urgent care centres are able to provide access to a broad range of physical and mental illness and injury

care, for both adults and children. Final decisions on how urgent care centres might be organised will rest with local health economies, but a more consistent offer from such facilities will be advantageous in promoting them as an alternative to hospital based urgent care.

NHS England will update on progress with the Review later this year.

The management of people with diabetes is measured and reported on through the Quality and Outcomes Framework (QOF), the CCG Outcomes Indicator Set (OIS) and National Diabetes Audit. These publications and data are used by local commissioners, providers and healthcare professionals to assess the quality of the services provided and to drive improvements.

General practitioners (GPs) are incentivised to ensure completion of the care processes recommended by The National Institute for Health and Care Excellence (NICE) through the QOF. The latest available data (2011-12) shows a 60.5% completion rate of all the eight measurable NICE-recommended care processes for diabetes. However, it is for individual GP practices, working with the CCGs in their area, to assess their own performance on completion of the care processes, and take action where improvement is necessary.

CCGs can use the information reported from the OIS indicator on completion of the care processes for people with diabetes to assess progress in improvement in their area and against their peers. Where completion rates are low, CCGs can then take action jointly with other practices in their area to make improvements.

To support local commissioning, NHS England has also developed a non-mandatory service specification for diabetes, based on the diabetes NICE Quality Standard, which is currently being piloted with some CCGs. The pilot will be evaluated to assess how useful the specification has been to commissioners, and this will inform decisions about its future development.

The Patient Experience of Diabetes Services survey, part of the National Diabetes Audit, is measuring the healthcare experiences of people with diabetes in England and Wales. The survey pilot tested an online-only approach to measure patient experience in GP and specialist diabetes services. Any diabetes service in England and Wales should be able to use the survey to get feedback from their patients. The survey can help to raise standards and drive out variation, and also empower services to understand at a local level how satisfied their patients are with the service being provided.

The management of people with diabetes is measured and reported on through the Quality and Outcomes Framework (QOF), the CCG Outcomes Indicator Set (OIS) and National Diabetes Audit. These publications and data are used by local commissioners, providers and healthcare professionals to assess the quality of the services provided and to drive improvements.

General practitioners (GPs) are incentivised to ensure completion of the care processes recommended by The National Institute for Health and Care Excellence (NICE) through the QOF. The latest available data (2011-12) shows a 60.5% completion rate of all the eight measurable NICE-recommended care processes for diabetes. However, it is for individual GP practices, working with the CCGs in their area, to assess their own performance on completion of the care processes, and take action where improvement is necessary.

CCGs can use the information reported from the OIS indicator on completion of the care processes for people with diabetes to assess progress in improvement in their area and against their peers. Where completion rates are low, CCGs can then take action jointly with other practices in their area to make improvements.

To support local commissioning, NHS England has also developed a non-mandatory service specification for diabetes, based on the diabetes NICE Quality Standard, which is currently being piloted with some CCGs. The pilot will be evaluated to assess how useful the specification has been to commissioners, and this will inform decisions about its future development.

The Patient Experience of Diabetes Services survey, part of the National Diabetes Audit, is measuring the healthcare experiences of people with diabetes in England and Wales. The survey pilot tested an online-only approach to measure patient experience in GP and specialist diabetes services. Any diabetes service in England and Wales should be able to use the survey to get feedback from their patients. The survey can help to raise standards and drive out variation, and also empower services to understand at a local level how satisfied their patients are with the service being provided.