Minister answers Parliamentary Question on A1AD

Minister answers Parliamentary Question on Alpha-1 Antitrypsin Deficiency – In May, the Alpha-1 Alliance met with Peter Aldous, Conservative MP for Waveney, to discuss the unmet medical need of patients affected by Alpha-1 Antitrypsin Deficiency (Alpha-1) and the Alliance’s campaign for the establishment of nationally commissioned highly specialised service for Alpha-1 in England.

At the meeting, the Alliance briefed Mr Aldous about the current level of care for Alpha-1 patients and our policy recommendations for how services for Alpha-1 patients can be improved in the future. Following the meeting, Mr Aldous tabled a Parliamentary Question to help us determine the next steps in the commissioning process for the Alliance’s application for a highly specialised Alpha-1 service, which was submitted to NHS England in April this year.

Please find below the response by Norman Lamb, Minister for Care Services:

Peter Aldous MP: To ask the Secretary of State for Health (1) how often the Prescribed Specialised Services Advisory Group meets per year; and if he will request that it will consider new specialised service applications for Alpha-1 Antitrypsin Deficiency at its next meeting;

(2) what steps NHS England takes to consider (a) all new specialised service applications and (b) new treatments for Alpha-1 Antitrypsin Deficiency; and whether such steps are subject to review by his Department.

Norman Lamb MP, Minister for Care Services The Prescribed Specialised Services Advisory Group (PSSAG) is a Department expert committee which was set up to provide regular advice to Ministers on which services are specialised and should be prescribed in regulations for national commissioning by the NHS Commissioning Board (NHS England). The Group met most recently in May 2014 and may meet up to four times a year.

Evidence, supporting information and activity on those services currently prescribed in legislation for direct commissioning by NHS England and any new services identified as potentially specialised, are made available to PSSAG from a range of sources, which may include Clinical Reference Groups (CRGs), patient groups, clinicians, commissioners and members of the public. The proposals the group considers are in large part generated by NHS England through its CRGs. The PSSAG makes recommendations to Ministers who, before deciding whether to make regulations, consult with NHS England, as required by section 3B of the National Health Service Act 2006.

NHS England advises that where it becomes the responsible commissioner for a service, it considers the funding priority of the service through its clinical priorities advisory group and manages a process for selecting providers. Any highly specialised services that become the commissioning responsibility of NHS England will be discussed at its Rare Disease Advisory Group.

The commissioning of services for people with alpha 1-antitrypsin deficiency is a matter for individual clinical commissioning groups. We understand the Alpha 1 Alliance is working with NHS England and the Specialised Respiratory Clinical Reference Group to develop a proposal on alpha 1-antitrypsin deficiency for a future PSSAG meeting.

Source: Alpha-1 Alliance 12/06/2014

Dr Ravi Mahadeva steps down as Chair of Alliance

Dr Ravi Mahadeva has resigned as Chair of the Alpha-1 Alliance due to a conflict of interest in relation to his work with NHS England’s Specialised Respiratory Disease Clinical Reference Group (CRG) and the ongoing campaign of the Alpha-1 Alliance.

Dr Mahadeva has assured the Alliance of his ongoing support for improving access to health services for patients affected by Alpha-1 Antitrypsin Deficiency (Alpha-1), which he will continue to pursue at CRG level.

The Alpha-1 Alliance would like to take this opportunity to thank Dr Mahadeva for his services and his dedication in supporting the Alliance and the Alpha-1 Community and we look forward to continuing to work with him in his capacity as a CRG member.

Karen North, Trustee of the Alpha-1 UK Support Group said: “The Alpha-1 Alliance campaign to establish a nationally highly specialised service and ensure access for patients to optimal integrated clinical care and associated therapies remains unaffected and will proceed as planned.”

A new Chair to the Alliance will be appointed in due course.

Source: Alpha-1 Alliance 29/05/2014

Alpha-1 Community meets Jeremy Hunt MP

The Secretary of State for Health, Jeremy Hunt MP, met with representatives of the Alpha-1 community last Tuesday (13^th May 2014) at the Department of Health to discuss the unmet medical need of Alpha-1 patients and the associated requirement to establish a highly specialised service for patients affected by the condition.

The meeting was attended by Mark Pawsey MP, Margaret Millar (co-founder of the group Alpha-1 Advocacy & Action) and Karen North (Trustee of the Alpha-1 UK Support Group), both from the Alpha-1 Alliance’s Executive Committee, Dr David Parr (Consultant Respiratory Physician at the University Hospital Coventry) and the Alliance’s Secretariat.

During the meeting, Margaret Millar and Karen North, representatives of the patient community, shared their experience of living with Alpha-1 and the impact the disease has on their lives.

Margaret Millar, an affected patient who lost a sister to the condition, highlighted the burden the disease places on herself and her whole family, describing living with Alpha-1 as a “steady decrease of quality of life”. She said: “I had to give up work because of the disease, I often have chest infections, my social life suffers and I have difficulties walking my dog”.

Karen North, who also suffers from Alpha-1, shared a similar experience to Margaret’s, describing the challenges imposed by the disease: “Everyday tasks many people take for granted such as bathing, shopping and walking on even the smallest incline will leave me breathless and sometimes gasping for air”.

Dr Parr, a respiratory consultant and Alpha-1 expert, briefed the Secretary of state on the clinical aspects of the disease and the current management of patients with Alpha-1 in the NHS. Dr Parr explained that the rarity and complexity of the disease meant that patients often experience a significant delay in receiving a correct diagnosis and in obtaining expert advice on the disease and its clinical management. He advised that patients desire and need access to an integrated multidisciplinary team based in dedicated centres where clinical expertise can be concentrated to facilitate optimum care.

In the meeting, the Alliance also expressed its concerns over the current uncertainty and lack of clarity of the commissioning process for highly specialised services and its application process. The Health Secretary requested to be kept updated on the progress of the campaign.

Source: Alpha-1 Alliance 29/05/2014

Letters from Cabinet Secretary Health on Alpha-1

As part of the campaign in the Scottish Parliament, Members of the Scottish Parliament (MSPs) Malcolm Chisholm and Graeme Dey wrote to the Cabinet Secretary for Health and Wellbeing, Alex Neil MSP, on behalf of their constituents, to raise the issue of the level of care provided to patients with Alpha-1 Antitrypsin Deficiency (Alpha-1) in Scotland.

The key points in the Minister’s response letter are as follows:

The Minister reiterated the Scottish Government’s commitment to raising the profile of rare diseases such as Alpha-1 and referred to the UK Rare Disease Strategy. An Implementation Plan for Rare Diseases in Scotland is currently being developed with the aim of delivering the commitments set out in the Rare Disease Strategy, published in November 2013, to improve services for people living with a rare disease and their families.

The Minister’s letter provided an overview of the current care arrangements for patients with Alpha-1. A Managed Clinical Network (MCN) approach is in place in Scotland that brings together professionals from primary, secondary and tertiary care with patients and carers, to work in a coordinated fashion across geographical, organisational and professional boundaries. The National Advisory Group of Respiratory MCNs (NAG RMCN) was also highlighted as a structure that helps to ensure a coordinated approach with the aim of improving respiratory services, serving as a platform for local MCNs and other key stakeholders to discuss issues, find solutions and share best practice.

The Minister highlighted the Scottish Government’s interest in welcoming study proposals in Alpha-1 from the research community to further research in this area.

To access a copy of the Minister’s letter on Alpha-1, please follow the link.

Source: Alpha-1 Alliance 20/05/2014

Alpha-1 Alliance briefs MSPs Scottish Parliament

As part of the Alliance’s campaign in Scotland, the Alpha-1 Alliance met with the following MSPs on Thursday 8th May in the Scottish Parliament:

Bob Doris MSP, SNP Member for Glasgow Region
Malcolm Chisholm MSP, Labour Member for Edinburgh North and Leith
Collin Beattie MSP, SNP Member for Midlothian North and Musselburgh
Iain Gray MSP, Labour Member for East Lothian

During the meeting, the Alliance briefed Parliamentarians on the current care of patients with Alpha-1 Antitrypsin Deficiency (Alpha-1) and on the need for the establishment of a highly specialised service in Scotland.

The Alliance will continue its meeting programme in the Scottish Parliament, and further meetings with MSPs have been scheduled for June 25th, 2014.

Source: Alpha-1 Alliance 19/05/2014

Kamada Preliminary Results Inhaled AAT

Kamada Announces Preliminary Results from Phase II/III Pivotal Trial in Europe and Canada of Inhaled AAT to Treat Alpha-1 Antitrypsin Deficiency

Kamada Ltd., a plasma-derived protein therapeutics company focused on orphan indications, announces preliminary top-line results from the phase II/III pivotal clinical trial in Europe and Canada of the company’s proprietary inhaled Alpha-1 Antitrypsin (AAT) therapy for the treatment of Alpha-1 Antitrypsin Deficiency (AATD or inherited emphysema).

The endpoints selected for this trial were based on scientific advice from the European Medicines Agency (EMA) and include those deemed to be clinically meaningful, such as frequency, time to first, duration and severity of exacerbation events, among others. A preliminary analysis of the results indicates clinically meaningful signs for inhaled AAT efficacy as well as additional positive signs in specific study populations. In a very important secondary end point, frequency of severe exacerbation was approximately 50% lower in the AAT group versus placebo. With regards to the primary endpoint of “time to first moderate or severe exacerbation,” early data do not show differences between the two treatment groups.

Kamada’s announcement of the results:-

MTB Support Alpha-1 at Belfast Marathon 2014

MTB McCartan Turkington Breen Solicitors collectively have raised an amount for the Alpha-1 UK Support Group that far exceeded expectations.

Ernie Waterworth, Ernie Telford , Dianne Wilson and Katie Campbell completed the 8 mile Belfast Marathon walk yesterday with John McShane completing the full marathon.

On behalf of the Alpha-1 UK Support Group a big thank you to everyone who took part and supported the event raising awareness and funds for Alpha-1. Through your kind support we are able to continue funding our programmes of supporting research, campaigning for better access to treatment, providing information and supporting Alphas and their families. We know there is so much more we can and need to do to promote better knowledge and understanding of Alpha-1 Antitrypsin Deficiency but we are limited by the funds we receive, so your support is valued and very much appreciated.

John L Mugford
Alpha-1 UK Support Group Chairman/Trustee

Minister answers Parliamentary Questions on Alpha-1

Minister Answers Parliamentary Questions on Alpha-1

MPs supporting the campaign of the Alpha-1 Alliance have raised questions with the Minister Norman Lamb MP in Parliament.

Alison Seabeck, Labour MP for Plymouth, and Dr Stella Creasy, Labour MP for Walthamstow, tabled the questions to help the Alpha-1 Alliance to further clarify NHS England’s application process for specialised services within the NHS.

Please find the Minister’s response included below:

Alison Seabeck MP: To ask the Secretary of State for Health when the next meeting of NHS England’s Prescribed Specialised Service Advisory Group will take place; and whether consideration of applications for highly specialised services for rare diseases like Alpha One-Antitrypsin Deficiency will be on the agenda for that meeting.

Stella Creasy MP: To ask the Secretary of State for Health what research his Department has (a) commissioned and (b) undertaken on the services available in the NHS for patients with alpha-1 antitrypsin deficiency.

Norman Lamb MP, Minister for Care Services: The commissioning of services for people with alpha 1-antitrypsin deficiency is a matter for individual clinical commissioning groups. The Department has made no assessment of these services locally.

The Prescribed Specialised Services Advisory Group (PSSAG) is a Department of Health expert committee which was set up to provide regular advice to Ministers on the services that are specialised and should be prescribed in regulations for national commissioning by the NHS Commissioning Board (NHS England).

We understand the Alpha 1 Alliance is working with NHS England and the Specialised Respiratory Clinical Reference Group to develop a proposal on alpha 1-antitrypsin deficiency for the next PSSAG meeting, which will be held on 16 May.

Source: Alpha-1 Alliance 27 March 2014

Alpha-1 Alliance meets Andrew Lansley MP to discuss need for a national service

Alpha-1 Alliance meets Andrew Lansley MP to discuss need for a national service

The Alpha-1 Alliance met with Andrew Lansley MP, former Secretary of State for Health, to discuss the need for the establishment of a highly specialised service for patients with Alpha-1 Antitrypsin Deficiency (Alpha-1).

Mr Lansley visited the Chair of the Alpha-1 Alliance, Dr Ravi Mahadeva, at Addenbrooke’s Hospital in Cambridge to learn more about the current care provided to Alpha-1 patients and how it can be improved in the future.

Mark Bradford, a patient suffering from Alpha-1, also attended the meeting to share his experience of living with the disease with Mr Lansley.

Although Mr Lansley has left his former role of Secretary of State for Health and now holds the position of Leader of the House of Commons, he still retains a strong interest in health policy issues. Mr Lansley expressed his support of the campaign for better services for Alpha-1 patients in England and stated that he will help the Alliance to further clarify NHS England’s process for new service applications.

Source: Alpha-1 Alliance 26/03/2014

Alpha-1 Alliance in discussions with NHS England

Alpha-1 Alliance in discussions with NHS England

As part of the application for a highly specialised Alpha-1 service, the Alpha-1 Alliance is currently in discussions with NHS England and the Clinical Reference Group (CRG) for specialised respiratory disease to further identify the next stages within the application process.

In February, the Alpha-1 Alliance submitted a service proposal to NHS England to outline the current standard of care provided to patients with Alpha-1 under the NHS and how it could be improved in the future.

The service proposal will be considered at NHS England’s next Programme of Care meeting. The Alliance will continue to work on a more detailed scoping document to provide further information about the elements of the proposed service to NHS England. The scoping document will be submitted to the CRG for consideration in the coming weeks.

Source: Alpha-1 Alliance 25/03/2014