Keith Pollard

Keith Pollard is kindly donating the net proceeds of his book to the Alpha-1 UK Support in memory of his son Jason

 

This remarkable new autobiography recalls the days when rugby league could be brutal and was regularly described as ‘The toughest sport in the World.’

It follows Keith from his days at Hull KR in the mid-sixties where he packed down with legends such as Peter Flanagan and Frank Foster, to the climax of his unique career, playing for Maitland in Australia during the seventies, in the highly competitive Newcastle league.

‘We thought he was a god from another planet’ said Maitland team mate and later Hull FC coach, Peter Sharp, after Keith had played and won in two Grand Finals.

But ‘Red & White Phoenix’ is far more than just a sporting memoir. Keith’s playing days provide the centre piece of a finely woven story, containing tales from Hull’s illustrious history, memories of youthful exuberance, anecdotes from a fascinating working life and a heart-breaking account recalling the tragic death of his son Jason.

ISBN: 9781916429420

Price: £9.95 + £2 P&P

http://www.riverheadbooks.co.uk/shop/

 

 

Mark Pawsey Press Release

Mark Pawsey MP raises awareness of rare illness in Parliament

 

Mark led the first ever parliamentary debate on Alpha-1 Antitrypsin Deficiency on 31st October 2018

Rugby MP Mark Pawsey last week led the first ever parliamentary debate on alpha-1 antitrypsin deficiency, a rare hereditary illness which affects the lungs and breathing of those with this condition.

Alpha-1 antitrypsin deficiency is caused by a genetic abnormality which means that the protein alpha-1 antitrypsin, normally released into the blood to protect from inflammation, is instead trapped in the liver. This can not only cause significant damage to a patient’s liver but also leads the lungs becoming vulnerable to pollutants in the air. In particular, those with alpha-1 are particularly susceptible and sensitive to cigarette smoke.

Symptoms of alpha-1 are often mistaken for asthma or chronic obstructive pulmonary disease, leading to the incorrect treatment which can lead to a patient’s health deteriorating. Because of this, Mark has become a supporter of the Alpha-1 UK Support Group, which seeks to raise greater awareness of the condition and a delegation of alpha-1 patients visited Parliament to hear the debate.

Speaking after the debate, Mark said:

“Alpha-1 antitrypsin deficiency was first brought to my attention in 2012 by Sarah Parrin, when she attended my constituency advice surgery with her son, Stephen Leadbetter. Despite having presented symptoms of alpha-1 over eight years before, he had only just been formally diagnosed with the condition. That delay in diagnosis had real consequences for his health as he was growing up, and demonstrates the scale of the challenge facing patients with alpha-1.”

Mark continued:

“After meeting with Stephen and Sarah, I resolved to find out more about this condition and met with Professor David Parr, who is a consultant respiratory physician at University Hospital Coventry and Warwickshire, and an expert in Alpha-1. From there, I have become a supporter of the Alpha-1 UK Support Group, which recently met in Brandon in the constituency. I am especially pleased that a number of those with alpha-1 were able to attend the debate and it was a great pleasure to meet with them and hear directly about how alpha-1 affects their day-to-day lives.”

Mark concluded:

“I would like to thank the Nigel Adams MP, who spoke on behalf of the Government, for his supportive response although we all recognise that there is more work to be done. I particularly hope the Government will now ensure that the specialised NHS alpha-1 service which was due to be in place by April 2017 will be introduced as swiftly as possible. However, one of the most important things which all of us can do is be more aware of this condition and that is why I was delighted to have support from across the House during this important debate.”

Professor David Parr, Clinical Director for Cardio-Thoracic Services and Consultant Respiratory Physician at University Hospitals Coventry and Warwickshire commented:

“I know from my experience of seeing alpha-1 patients from all over the country in our multi-disciplinary AATD service at UHCW that there needs to be greater awareness of this complex condition amongst healthcare professionals. I very much welcome Mark’s continuing support of the alpha-1 community in their quest to obtain better access to optimal care.”

Sandra Nestler-Parr, Trustee of the Alpha-1 UK Support Group added:

“Mark has been a strong supporter of the alpha-1 patient community in the UK for many years, and we are very grateful that Mark has brought the challenges faced by patients with alpha-1 antitrypsin deficiency to the awareness of a wider audience through last week’s parliamentary debate.”

The full transcript of the debate is available at: https://hansard.parliament.uk/Commons/2018-10-31/debates/D79EB4FC-284C-4B5F-8B68-0BFAAB302325/Alpha-1AntitrypsinDeficiency

 

Video of the debate is available at: https://www.markpawsey.org.uk/news/mark-pawsey-mp-raises-awareness-rare-illness-parliament

 

Westminster Hall Debate

Dear Alpha-1 patients in the UK,

WE NEED YOUR SUPPORT! URGENTLY!

We are delighted to inform you that, further to Mark Pawsey MP’s attendance at our recent patient meeting in Coventry, Mark has secured a Parliamentary debate on “Treatment of alpha-1 antitrypsin deficiency”, to be held next Wednesday from 16.00 – 16.30 at Westminster Hall in the Houses of Parliament (https://calendar.parliament.uk/calendar/Commons/All/2018/10/31/Daily). Matt Western MP who was also at our recent meeting will also be in attendance at the debate.

This is fantastic news, as this event will have cross-party support. We have worked closely with Mark Paswey to prepare the debate.

The key aim of the debate is to get as many MPs as possible to attend, to further raise awareness of Alpha-1 and to highlight the challenges the Alpha-1 patient community faces.

Unfortunately we were only informed about this on Friday and haven’t got much time to prepare. This is where we need your help. How can you do this?

1. Find out who your local MP is (if you don’t know just google your constituency and Member of Parliament)

2. Personalise the letter below and email it to your MP as soon as possible!!

3. On Tuesday, call your MP’s office to ensure that they received your email and to reiterate that you would like to encourage your MP to attend the debate.

4. If you receive a response from your MP ahead of the debate on Wednesday, please email us at info@alpha1.org.uk to inform us whether your MP is planning to attend, so that we can plan accordingly.

You can watch the debate on Parliament TV (https://www.parliamentlive.tv/Commons), which will be live streamed.

Thank you for your support.

The Alpha-1 UK Support Group

 

Dear [MPs Name]

Westminster Hall Debate on Treatment for Alpha-1 Antitrypsin Deficiency
16:00 – 16:30, Wednesday 31st October, 2018

I am one of your constituents and am writing to kindly ask you to attend a Westminster Hall debate on Treatment for Alpha-1 Antitrypsin Deficiency, moved by Mark Pawsey MP, to be held on Wednesday, 31st October 2018, at 4pm.

I live in [town/village/borough] and I suffer from Alpha-1 Antitrypsin Deficiency (Alpha-1), a rare and complex genetic disorder that can cause lung and liver disease in adults and children, and occasionally affects other organs and may require organ transplantation. Severely affected individuals experience significantly reduced life expectancy, severe disability, and great limitations on multiple other aspects of their lives. Currently, no specific treatment for Alpha-1 is available in the UK.

Due to its rarity, Alpha-1 is often misdiagnosed, with an average delay of 6-7 years before patients receive the correct diagnosis, frequently resulting in sub-optimal care of Alpha-1 patients for many years. A nationally highly specialised NHS service for Alpha-1, to improve patients’ access to co-ordinated multi-disciplinary clinical expertise, was approved by the Department of Health’s Prescribed Specialised Services Advisory Group in March 2016, to be operational in April 2018. However, the development of this service has still not commenced.

Only one specific treatment for Alpha-1 exists: intravenous alpha-1 augmentation therapy. This treatment has been available to patients in many countries, including the US, Germany, Spain, Italy, Portugal, Austria and others for many years. The only augmentation therapy product with a license in the UK (which was granted in August 2015) is currently undergoing evaluation by the National Institute for Health and Care Excellence under the Highly Specialised Technology (NICE-HST) appraisal process. On 26th September 2018, NICE published its draft guidance not to recommend this treatment for use in the NHS. The evaluation is currently out for consultation, but we are very sceptical whether the treatment will be recommended for reimbursement by the NHS.

I very much hope that you will recognise the life-changing impact that this disease has on patients like myself, and attend the Westminster Hall debate on Wednesday to learn more about the challenges of Alpha-1 patients and what you can do to support patient experience.

Yours sincerely,

[Your name]

Clinical trial update – 19th October 2018

In light of very recent events, we would like to provide some important information about upcoming clinical trials in Alpha-1 in the UK.

Currently, there are three trials in AATD planned to commence by pharmaceutical/biotech companies (Mereo Biopharma, Arrowhead Pharmaceuticals and CSL Behring). These trials will be undertaken in several different centres with Alpha-1 expertise across the UK.

It has come to our attention that Dr Turner from UH Birmingham implied in a recent email to an AATD patient that these trials will be conducted in Birmingham only and that patients should get in touch with her team now if they want to be considered for participation in any of these trials. This is misleading and FACTUALLY INCORRECT. Patients have no advantage for being chosen to participate in a clinical trial whatsoever if they provide their contact details to Birmingham or to any of the prospective trial centres at this point; in fact, there are several reasons why it may be counter-productive to do so.

The general process for patient recruitment is always the same and as follows: Once a trial has been formally approved, the company conducting the trial will publicly announce more detailed information about the trial, the trial centres and contact information at each trial centre. Once this information is released, patients interested in participating in the trial can then contact the trial centre of their choice (or several different centres if they wish). For logistical reasons, most patients tend to choose a trial centre that is geographically close to where they live, as most clinical trials require regular visits to the trial centre – however, the choice of centre lies entirely with the patient.

A few important points to keep in mind when you think about which trial centre to choose:

• Every clinical trial needs to follow a “trial protocol” which sets out how the trial is conducted, how often patients need to visit the centre, which tests are being performed etc. Each trial centre needs to strictly comply with this protocol. Patients will therefore have the exact same experience throughout the clinical trial, irrespective of the trial centre they choose.
• Every centre where the trial is conducted will have undergone a detailed screening process to ensure that the clinical staff have relevant and sufficient expertise and competence in AATD and that the technical, administrative and ethical requirements are met by the centre.
• A clinical study is less likely to be successful if the majority of patients attend a single trial centre. The more evenly distributed the patient numbers are across different centres, the better. There are several reasons why it is advantageous for the trial if patients choose a trial site geographically convenient for them: if patients have to travel a long way to the trial centre, they are more likely to miss study visits or drop out of the trial.
• The better the patient community advertises AATD trials, the more likely it is that the trials are successful, because we reach a wide range of patients, rather than only those who ‘register’ with a specific centre, as Dr Turner misleadingly suggested in her email to the Alpha-1 patient. All trial centres will offer the opportunity to discuss the trial in detail with each patient, once patient recruitment is allowed to start. 
• The AATD patient community has campaigned for many years to increase disease awareness and clinical expertise across the UK. As a result of this campaign, we now have several very experienced AATD centres across England, with two additional satellite centres currently being set up in the South and the North of England. It is crucially important for patients to utilise all centres, in order to help them gain more expertise and to ensure that all centres remain sustainable.
• Please note that patients who participate in clinical trials do not need to move their routine specialist NHS care to this trial site (even if this is suggested by the trial centre). It is entirely up to the patient to choose which doctor’s care they wish to be under.

We are in regular and frequent communication with all companies who are planning to conduct trials in AATD in the UK. As soon as they are in a position to publish specific information on these trials, including details about the trial centres and contact details, this will be available on our website. For current information on upcoming trials:
• Read the update in our recent newsletter (pages 2 and 3, https://alpha1.org.uk/attachmen…/…/51/A1UK_Newsletter2018.pdf),
• Watch the presentations that Mereo Biopharma, Arrowhead Pharmaceuticals and CSL Behring gave on their respective clinical trial programme at our recent annual patient group meeting – fast-forward to minute 9 on video 2 https://www.facebook.com/pg/alpha1uksupportgroup/videos/. This video includes a list of all prospective UK trial centres for the upcoming Mereo Biopharma trial – fast-forward to minute 23 of video 2.
• As soon as we have any update on any trials relevant in the UK, we will post it in this group.

Karen O’Hara, on behalf on the Alpha-1 UK Support Group, supporting all Alphas in the UK

Clinical utility of alpha-1 proteinase inhibitor

Clinical utility of alpha-1 proteinase inhibitor…

Clinical utility of alpha-1 proteinase inhibitor in the management of adult patients with severe alpha-1 antitrypsin deficiency: a review of the current literature – the authors are Professor David Parr and Dr Beatriz Lara who are the Alpha-1 specialists who operate the NHS Alpha-1 Clinic at University Hospitals Coventry & Warwickshir

https://www.dovepress.com/articles.php?=33769

EU ALPHA-1 AWARENESS DAY | 25 APRIL 2018

 EU ALPHA-1 AWARENESS DAY | 25 APRIL 2018

 For the first time in history, the European Alpha-1 Community celebrates an EU Alpha-1 Awareness Day, on 25 April 2018. Among other activities, the main “kick-off” event takes place the day prior (24th) in the European Parliament from 15:00-17:00hrs. MEP Marek Plura (Poland) will host a round table discussion for MEPs, during which time patient representatives will provide updates from the following countries: Austria, Belgium, Germany, Italy, the Netherlands, Italy, Romania, Spain, Switzerland and the United Kingdom. The event is organized by RPP Health Care and sponsored by Alpha-1 Global, a program that facilitates collaborative efforts among Alpha-1 associations around the world. 

The EU Parliament gathering is directly related to the development of the Alpha-1 European Expert Group Recommendations; a document that was created to raise awareness among governments, health systems, health professionals and society in general about the importance of diagnosing, treating and investigating Alpha-1 Antitrypsin Deficiency (AATD). 

Combined action is sought in Europe in support of the early diagnosis and correct treatment of people with AATD, especially considering that there are still European countries that do not reimburse plasma derived augmentation therapy for AATD patients with severe lung disease and skin diseases such as panniculitis.

 Early diagnosis 

The Recommendations address the complications of uncontrolled pulmonary and liver diseases. “The main difficulty of handling Alpha-1”, the document reflects, “is the early diagnosis, which most patients do not obtain until they already have severe symptoms despite the low cost and effectiveness of diagnostic means.”

The challenge is to consolidate approaches for the training of health professionals and patients, and facilitate the exchange of knowledge, in addition to promoting diagnosis and extending the network of centers of excellence for Alpha-1. “The problem of Alpha-1 is not so much the number of affected patients as their quality of life, which can be significantly impacted by the delay in diagnosis and limitations in access to treatment.” 

The document proposes to the member states the development of a plan for the diagnosis of AATD, within the framework of their Rare Diseases programs. It also alerts health professionals to the need to perform the deficit tests for all patients with COPD, bronchial asthma, bronchiectasis and liver diseases of unknown origin, as well as people with a family history of AATD. 

The European Commission is requested to update its CORDIS study (2002) to find the current status of genetic testing for rare diseases and to issue subsequent recommendations for countries to promote an approach based on maintaining a high level of human health and not discriminating against patients taking effective steps to avoiding both human and health system costs.

Pollution and lung patients 

Air quality (interior and exterior), toxicity of chemical products in the workplace and pollution, are other aspects on which the Recommendations reflects, taking into account that “in environments with contaminated air, the organs of patients with a deficit degenerate in a significantly faster way than those of the remaining patients with COPD. ” Poor air quality is a “silent killer that currently affects 90% of residents in EU cities, especially lung patients,” says UK parliamentarian Seb Dance. “One of the measures that policymakers can adopt to improve the health of Alpha-1 patients is to achieve the highest standards of indoor and outdoor air quality.” 

Coordination and patient care 

The Recommendations also express the need for EU countries to coordinate their efforts. The creation of an EU stamp is requested for the accreditation of Centers of Excellence and its registration in the Orphanet database, as is already the case with Reference Centers in Rare Diseases. Implementing the Cross-Border Healthcare Directive, to support the rights of patients to have the best possible care, is another of the demands included in the document.

Translated from the article by Elena Goyanes on the Alpha-1 Spain website:
https://alfa1.org.es/dia-europeo-del-deficit-de-alfa-1-antitripsina/

 

 

PARTY IN THE PARK CHILDREN’S DAY

PARTY IN THE PARK CHILDREN’S DAY
Saturday 19th August 2017

Folly Farm, Kilgetty, Wales

The Alpha-1 UK Support Group are holding our 2nd Party in the Park specifically targeted at our younger members and their families.

Folly Farm has both indoor and outdoor areas.  The zoo has over 750 animals and you can get up close to furry and feathery farmyard friends in their barn  The vintage fairground has 16 different rides and there are eight adventure play areas.  All set in 120 acres so there should be something to entertain everyone.

https://www.folly-farm.co.uk/

For further information or to register to attend please contact Jemma Coad info@alpha1.org.uk

Obituary Linda Cooke

Linda Cooke
30th October 1950 – 21st January 2016
Our dear Linda Cooke sadly passed away peacefully with her son Andrew, daughter Helen and her best friend Bev by her side on Thursday 21st January 2016.There are special people in our lives who never leave us even after they are gone, Linda is without doubt one of those special people who will always be forever in our hearts, we will miss you.


Here is Linda’s Story……….


Linda was Trustee of the Alpha-1 UK Support Group and was always there, in person, on the end of the phone, by email and Facebook, to offer support and friendship to all who came within her sphere, no matter what walk of life they were from and no matter how ill, she herself was feeling…. 

Linda was always one of the first to welcome new members, who often had just been diagnosed, and had found the Alpha-1 UK Support Group whilst researching Alpha 1 on the internet. She would offer words of comfort, support and encouragement and somehow she always knew what to say to alleviate their fears of this little known illness.  When a member was listed for a transplant, she would offer them love and support, and when they were lucky enough to get “the call” she was there to support their family through this difficult time.

But it wasn’t just within the Alpha 1 community that she was like this.  To all her family and friends, she was a tower of strength, always there for them. Your problem was her problem, she would laugh with you, cry with you, celebrate your successes and give support in times of need.

But for all this the centre of her world was her son and daughter, Andrew and Helen.  Her world revolved around them and I know that she was happy in the knowledge that when the time came for her to leave this life, they would have Louise and Michael there to support them as well as family and friends.

Linda’s transplant gave her six extra years, sadly 3 of them with chronic rejection.  Nevertheless, she maintained a strong constitution and was always thankful of a second chance of life and wouldn’t have changed a thing.

She was so very much more than these words that I have taken from emails that she sent to the Alpha-1 UK Support Group in May 2014.  The first one is from a welcoming email she sent to a new member of the group, a brief outline of her Alpha journey, the second one is her celebrating her transplant birthday or tranniversary as the Alpha 1 community call it, remembering the day she got “the call”.  And although she was in chronic rejection, she still remained optimistic with no regrets.

3/5/2014  –  My name is Linda I am 63 years of age and was
diagnosed a
ZZ Alpha when I was 40 after years of being what Doctors called “chesty” and winters upon winters of bronchitis . I was wrongly
diagnosed as asthmatic.
I had a company medical when I was 40 and emphysema was picked up, further investigations showed I had Alpha 1 antitrypsin enzyme deficiency…”

What is alpha-1-antitrypsin? Alpha-1-antitrypsin (AAT) is a protein mainly produced by your liver that is released into in your blood. There are other proteins called enzymes that are found in all parts of your body that help to make sure that chemical reactions in your body take place normally. Enzymes have many important roles in your lung, including removing bacteria and fighting infections. The AAT protein is needed to control enzyme activity to prevent healthy lung tissue being damaged.

What is severe alpha-1-antitrypsin deficiency?  AATD is a rare, inherited condition that affects around one in 3,000 to one in 4,000 people in the UK. The most common reason for severe deficiency is if you carry an abnormal gene known as Z-AT, which affects the level of your AAT
protein.

To inherit AATD, a Z-AT gene must be passed on by both of your parents. If you carry two Z-AT genes, your AAT level will be only 10-20 per cent of what it should be.  The AAT protein
affected by the Z-AT gene builds up in the liver. This means you don’t have enough AAT to control the enzymes in your body, so healthy lung tissue can be damaged. This can cause emphysema, especially if you smoke.  Emphysema is one of the lung conditions known as
chronic obstructive pulmonary disease (COPD).

“Unbeknown to me at the time I had married an
MZ
man ( who
carried one bad gene ) and we had two children,a son
Andrew who is MZ like his Dad ,and a daughter  Helen who is ZZ like me…….my Mum and Dad were tested as MZ as were my two brothers.I was very fortunate to receive a double lung transplant in May 2009 at Papworth hospital in Cambridge, in fact it was 5 years ago tomorrow…my gratitude to my wonderful donor and her family is never ending ….sadly I have chronic rejection now but I’ve had that for the last two years so you can see I’m hanging on in.
My lung function at the time of transplant was 19% and I was very poorly …so I’ve been given another 5 years of life I wouldn’t have had. The criteria for a transplant then was to be 23% or below …that may have changed since then …..certainly the cut-off age has changed since I had my transplant ..it used to be 60 but they do them on older patients than that now.We have had members who have had transplants
last much longer than mine ….in fact at last
year’s annual meeting we met a member who was 21 years post-transplant…isn’t that amazing ? ….and when I go to my check-ups at Papworth I meet many others who are 10 , 15 and more years post-transplant…I was just very unlucky.
You’ll see we don’t always just talk of our health …. we may have lost many things but fortunately we haven’t lost our sense of humour.

I have been a member of this group since 2000 and I’m now a committee member and trustee, I have learnt so much valuable information since I joined and made so many wonderful friends…I hope
you do too.

04/05/2014  Morning all….special day for me today as it is
my 5 year anniversary of my double lung transplant…it only seems like
yesterday…….

It was a bank holiday weekend just like it is now…my mum had been over for Sunday lunch as usual and I had been showing her my case for Papworth with my new pj’s and all the things I needed for after the
op.
It had only been 9 days since I was listed and I had spent that time preparing things at home…getting my affairs in order and moving money into separate bank accounts to cover bills should I be away from home…cleaning the house, bringing all my washing and ironing up to date…I remember John Doyle said to me that I was like a creature nesting …..I finally finished doing all that on the Saturday and I sent a prayer up to God and said to him ” if ever there was a good time for my transplant now was it I was never gonna be more ready ” ..and he
answered because the next day Ann Thompson, my transplant coordinator, rang me around 5.30 in the afternoon…Mum and I had been watching the musical “Oliver ” Ann asked me if I was well and I replied “I was ” ….she then said “they may have something for me ” she asked me when I had last eaten and drunk anything and not to drink or eat anything from then on.

Everything went into action…I called the kids …neither were ready….I called my neighbours who had always agreed to be my transplant buddies at home …( I had always thought the call would come in the middle of the night ) they came over and Geoff took my Mum home for me…Penny ran around upstairs closing all the windows and switching things off…Helen arrived with her father in law…all were in a state of anxiety and panic, but not me ….I was so calm…where that came from I don’t know but there was a bubble of excitement running through me ..the song “something’s coming ” from West side story had been in my head for the past 9 days and it was in my head that day too …” who knows , could be , something’s coming something good , ..if I can wait”
By the time the car from Papworth came to get me, most of my neighbours had heard the news and were standing on their doorsteps waving me off and wishing me well…Helen and I got in the car (we were meeting Andrew at Papworth, as he was working at his Dads in
Surrey that day)
As we turned the corner of my road the driver put the blue light on and the siren went off.

Helen and I looked at each other in amazement, at what had happened in the last half hour…the rest is history …..My thoughts, of course, are always with my Donor…I thank her every day for her precious gift and my thoughts are with the family today, as they remember the sad day they lost her, but I remain eternally grateful to them…

Linda is now at peace, up there in Heaven, in the loving arms of her Mum and Dad, and chattering away ten to the dozen with Jane Hill, Chris Torrance John Doyle and all the Alphas that have been taken too soon.

R.I.P. Linda Cooke ….You can now breathe easy

30th October 1950 – 21st January 2016

Linda’s Funeral is Thursday 18th February at 12.45 at:

Easthampstead Park Cemetery and Crematorium
South Road
Bracknell
Wokingham
Berkshire
RG40 3DW

Afterwards at:

Easthampstead Park
Off Peacock Lane
Wokingham
West Berkshire
RG40 3DF

Linda’s family have requested that instead of flowers they would like a donation to Alpha-1 UK Support Group using the folling JustGiving page:

https://www.justgiving.com/Linda-susan-cooke

Published: 03/02/2016

An Open Letter to the Alpha-1 Community

This open letter is intended to give an update on the recent activities of the Alpha-1 UK Support Group on behalf of the Alpha-1 community in the UK, and to express the strong support of Alpha-1 Global and the Alpha-1 Foundation for those activities.


The Alpha-1 community in the UK is understandably excited about the 2015 publication of a paper on the RAPID trial, which involved the Alpha-1 (AAT) augmentation therapy Respreeza®, in the prestigious medical journal The Lancet, and the European Medicines Agency’s recent approval of Respreeza® for prescription use in Europe.

The National Institute for Health and Care Excellence (NICE) is currently considering a technology appraisal of Respreeza® to develop recommendations on its use in England. The scope of this potential health technology appraisal (HTA) has been under consultation with different stakeholder groups in the UK.

The Alpha-1 UK Support Group has formally responded in detail to the NICE draft scope consultation, and the charity’s Trustees Karen North and Dr Sandra Nestler-Parr attended the NICE scoping meeting on 16th October 2015.  The aim of the meeting was to discuss with all stakeholders – clinicians, patients, industry, the National Health Service (NHS), etc. – the most appropriate scop
e for a potential HTA for Respreeza®.

NICE is fully aware of all relevant evidence necessary to make a well-informed recommendation to Ministers on the most suitable HTA route.  Ministers will decide by February 2016 whether and how Respreeza® should be appraised by NICE.  The actual appraisal process will not commence until spring 2016 at the earliest.  All relevant information and status updates are available on the NICE website.

All signers of this letter are grateful to NICE for its consideration of the detailed response made by the Alpha-1 UK Support Group to NICE’s review of a potential HTA for Respreeza®.

Alphas have made enormous progress in the last few years in the UK.  Most recently, two Specialist NHS AATD Services have been established at the University Hospitals Coventry and Warwickshire and at the University Hospital Birmingham, offering multi-disciplinary, expert care for Alphas from across England.

The Alpha-1 UK Support Group has been able to drive these improvements in the care of Alphas only by building relationships with different stakeholders and engaging in the formal processes that are put in place by the government and the NHS.

At the same time, the charity continues to focus on its mission to support Alphas across the UK by providing education, advice, financial support, equipment hire and a platform for Alphas to connect with each other and share experiences.

The Alpha-1 UK Support Group’s trustees, and all volunteers, take their responsibility very seriously to work solely in the best interest of the charity’s membership and the wider Alpha-1 community.  They work beyond the call of duty to improve access to optimal care and treatment to enhance the quality of life of Alphas.

The Alpha-1 UK Support Group regularly reviews and updates its website, leaflets and brochures, and continues to make those materials available while review or updating is taking place, as a helpful resource to its membership and the wider community.

Fellow participants in Alpha-1 Global have frequently praised the Alpha-1 UK Support Group for its open and energetic collaboration to expand patient choices and improve access to care for Alphas in the UK and around the globe.

Alpha-1 Global and the Alpha-1 Foundation in particular laud the Alpha-1 UK Support Group for ably representing the patient point of view as NICE considers the scope of an HTA for Respreeza®, and we wholeheartedly support the charity’s strategy in actively participating in this essential process.

Alpha-1 Global

Alpha-1 Foundation

Alpha-1 UK Support Group

Published: 29/10/2015