Research

Research and Latest News
If you are a doctor or professional who knows of any new developments or research we would be grateful to hear about it. Please email use the Contact Us page or lynne@breathtakers.co.uk

Bronchitol

Pharmaxis (ASX:PXS) announced that an advisory committee of the U.S. Food and Drug Administration (FDA) has voted to recommend the approval of Aridol™ (mannitol bronchial challenge test) for use as a bronchial test to assess bronchial hyperresponsiveness to aid in diagnosing patients who have symptoms of asthma or symptoms that are suggestive of asthma.

The recommendation was made by the FDA’s Pulmonary-Allergy Drugs Advisory Committee following a meeting on 20 November 2009.
The FDA often seeks the advice of its advisory committees when evaluating potential new products but is not required to follow its recommendation. If approved, the FDA will determine final prescribing information. Pharmaxis filed an NDA for Aridol in March 2009 and the FDA is scheduled to advise the result of its review by 27 December 2009.

The full release is available on the Pharmaxis website.

Pharmaxis today announced that it has filed an application with the European Medicines Agency (EMeA) to market Bronchitol in Europe for the treatment of cystic fibrosis. The media release is available on the Pharmaxis website.
OB Research in Australia
The Queensland Centre for Pulmonary Transplantation are carrying out research, the main focus of which is on obliterative bronchiolitis, particularly after lung transplantation and the condition following bone marrow transplantation with the obvious links to post infectious OB. Most of the research is laboratory based as yet.

You can follow progress at the Prince Charles Hospital site:
www.tpchfoundation.org.au/Latest-News/Media-Releases/Lung-road-to-Paris.aspx
Bronchitol trials news
Pharmaxis today announced positive results from its Phase 3 cystic fibrosis trial. The media release is available on the Pharmaxis website at http://www.pharmaxis.com.au/library/2009_05_04_CF301_Top_Line.pdf.

Dr Alan Robertson will host a conference call at 8.00am Sydney time on Tuesday 5 May to discuss the results. Details of the call are included in the announcement.

Look out for 'Health and Well being' questionnaire
The British Market Research Bureaux will be sending out thousands of 'Health and Well being' questionnaires to families in January on behalf of the Department for Children, Schools and Families (DCSF). Information from the questionnaire will be used to find families to take part in a further survey, in spring, about disabled children's services. Parents' answers will be used to develop a National Indicator for disabled children, which will measure the performance of each local authority's disabled children's services. The 'Health and Well being' questionnaire is being sent out to make sure families who are currently not receiving any services are included in the survey as well as those that are. Families chosen to complete the survey will be spread across each local area and have children with a range of disabilities.
- Why is the Disabled Children's Services National Indicator important?
This is the first time there has been a specific indicator for disabled children's services. Local areas will be able see how they are performing and compare with others. Areas that perform badly will be expected to improve their services and are likely to channel money and resources into meeting National Indicators where they are underperforming.
- What is a National Indicator?
In April 2008, a new system was introduced to measure and compare the performance of each local authority in England. Every local authority will now be measured against 198 National Indicators, including the Disabled Children's Services National Indicator to see how well they are serving their local community.
- What difference have other National Indicators made?
One National Indicator says that patients should be referred to a health specialist within 18 weeks of being referred by a GP - the introduction of this National Indicator has significantly reduced patient waiting times.
- If I receive a survey, why should I complete it?
The Disabled Children's Services National Indicator is a key tool to ensure services improve in your local area. It will focus education, health and social care's attention on services for disabled children in a way that hasn't been seen before. Your experiences are crucial in measuring local services for families of disabled children. The survey is a unique chance to have your say - it you get the opportunity to take part, please do, and encourage other parents to do the same. It will be initially sent to some parents in the Spring and then to a larger sample in the summer. After that it will be sent out on a regular basis once a year.

New Drug Trial- Bronchitol
Latest news from Pharmaxis, an Australian drug company is that they are trialing 'Bronchitol'. Bronchitol hydrates the lungs, helps restore normal lung clearance and allows patients to clear mucas more effectivley. At this stage Parmixis are conducting trials with cystic fibrosis patients over the age of six and bronchiectasis patients over the age of eighteen. As they progress with the development of Bronchitol they will be investigating other disease areas where excess mucus impacts on patients quality of life. This may well have a positive impact for OB sufferers and Breathtakers will be following the progress.
You can visit the Pharmaxis web below.
www.pharmaxis.com.au
28,000 families missing out on extra tax credits
Contact a Family and the Every Disabled Child Matters campaign have been working with HM Revenue and Customs (The Revenue), and identified that around 28,000 families in the UK may be missing out on much needed extra income through additional tax credits. This is mainly families who have not informed HMRC that they receive Disability Living Allowance for a child, and therefore have not had their tax credits adjusted accordingly. The Revenue has agreed to carry out work to identify these families - but if you think this applies to your circumstances getting advice now may ensure you don't miss out in the meantime.
Note: Now all new successful Disability Living Allowance claims for children will trigger an orange card sent off to HMRC. This will act as an alert for HMRC to adjust any tax credits claim in respect of that child.
For advice about DLA and tax credits telephone the Contact a Family
helpline -               0808 808 3555         0808 808 3555.

Contact-a-family Archived News
                     General Archived News

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Planning the journey for life-limited children

ACT is holding a conference focusing on what can be done at each step of a life-limited child's life to ensure they always have access to the best possible care and support. They will also be celebrating their 21st birthday. The conference is being held at the National Motorcycle Museum, Birmingham on Wednesday 18 November 2009

For further details of the programme and to book a place email emma@act.org.uk or visit
http://www.act.org.uk/index.php/act-news/act-conference-2009.html

News from Rare Disease UK

Rare Disease UK recently announced that the European Council recommendation calling on EU Member States to take action to tackle rare disease, was formally adopted by EU Health Ministers.

Rare Disease UK is undertaking a programme of meetings with officials in all of the UK nations to discuss how they can work in partnership to take this important initiative forward. They are also in the process of setting up working groups, which will be instrumental in developing a draft national strategy to present to government partners.

We look forward to hearing more about this over the coming months.

Discussions for a national plan for rare diseases in the UK get off to a promising start

Following the European Commission’s Communication last November defining a strategy for EU countries to create and adopt their own plan for rare diseases, a Parliamentary dinner-debate was held in the United Kingdom in mid-February, gathering a range of experts in order to discuss a national rare disease plan for the UK. Co-hosted by Dr. Ian Gibson, a Member of Parliament from the House of Commons, and Genzyme Therapeutics UK & Ireland, the event was attended by patient representatives, clinical experts, members of industry, and Parliamentarians. Key themes included variations in access to quality service; ensuring “a complete package” of care – from diagnosis to treatment; increasing communication between experts and between countries; and the need for a mechanism that ensures sustainability. In the UK, as elsewhere, scarce expertise leads to a “patchy” provision of services. The lack of a national structure engenders late, missed, or incorrect diagnoses – often with severe health consequences. Many rare diseases fall outside the National Commissioning Group’s prevalence threshold, and thus are lost between the two main health bodies (the NCG and NICE). This means that patients must lobby for their treatment. Although some groups have been successful in obtaining the medicinal products they seek, it was agreed that rare disease patients should not have to resort to this practice. National centres of excellence were brought forward as a strategic means to reduce variations in care by grouping together expertise for particular disorders. However, the point was made that such centres would not diminish the need for improved knowledge and communication at the local level. The UK plan would ideally ensure a “complete treatment pathway” in addition to ascertaining that drugs are approved and available. Home nursing and resources for travel were cited as examples of care that many patients need in addition to medicines. Sharing knowledge and improving communication on all levels would need to be addressed in the plan. Finally, ensuring sustainability arose as a factor to be built into a national plan. With funding often dispensed on a short-term basis, the means are needed to make rare disease support “a permanent feature” of the UK healthcare system. The evening was viewed as a promising starting point, and a scheduled steering group meeting of the Rare Disease UK alliance and the Genetic Interest Group is expected to continue the momentum to develop a national plan.

OBsurvey - epetition:
“We the undersigned petition the Prime Minister to make a commitment to establish funding for a national monitored survey of Obliterative Bronchiolitis (OB), an orphan lung disease which children can die from and require life long treatment, including lung transplant. There is currently no treatment or published results of research yet OB has a very major impact on healthcare and social resource issues.”
Details of Petition:
“OB is a rare, untreatable and irreversible lung disease that occurs after injury to the lower respiratory tract. OB can be caused by infection, pneumonia (particularly with adenovirus), breathing in toxic fumes or fluid. Though limited to few children, diagnostic criteria are not fixed therefore potentially there are many undiagnosed cases. OB is not recognised in the public mind. A national survey would provide the critical information needed to establish diagnostic recommendations to clinicians as well as consolidating knowledge of outcomes which are currently poorly outlined. Such knowledge would allow discussion as to whether children reaching agreed criteria could be included in future clinical trials of medicines and the re-examination of the viability of traditional medicines such as steroids and immune suppressives, as well as raising awareness of OB and opening possible future opportunities for further research.
Read the Government’s response
Thank you for taking the time and trouble to sign this e-petition requesting that the Government provides the funding for a national monitored survey of Obliterative Bronchiolitis.
Obliterative Bronchiolitis (OB) is a very serious and debilitating condition, and the Government is committed to doing everything it can - alongside the NHS and local GPs - to help people suffering from the disease. We fully understand why parents and carers of children affected by OB want to see more research conducted into the condition to seek better treatments and an eventual cure. However, it may be helpful to explain that the Government itself does not undertake research such as this directly. This type of research is something that would be funded by the British Paediatric Surveillance Unit (BPSU), a body jointly set up by the College of Paediatrics and Child Health, the Health Protection Agency and the Institute of Child Health, London in 1986 to undertake active surveillance of rare paediatric disorders. Details of the work that they do can be found on their webiste at http://bpsu.inopsu.com/
The BPSU is operationally intependent of Government, although the Department of Health provides their funding, and it is for them to decide what surveys to undertake. At the present time, the Government is not aware of any planned survey into Obliterative Bronchiolitis. If you wish to contact the BPSU directly, you can contact them in writing at 5-11 Theobalds Road, London WC1X 8SH or via telephone on 020 7092 6000 020 7092 6000.

Breathtakers is currently pursuing the petition request with the BPSU as suggested by the government.
Latest Government Plans
Tax credits
Some good news! Up to 30,000 families will be better off thanks to changes to the tax credit system after campaigning by EDCM and Contact a Family. Find out if you or families you know can benefit at http://www.edcm.org.uk
http://www.edcm.org.uk/transformingservices
British Paediatric Orphan Lung Diseases (BPOLD)
Last year a nationwide survey began to try and establish the numbers of families affected by OB in the UK. The research intended to look at characterisation of the disease in each individual patient, and to determine aetiology, severity and the impact of the disease on the patients and their family. The initial call for patients identified 307 patients across the UK with a provisional diagnosis of OB. Unfortunately the study has been terminated because of lack of funds or support from any local research governance committee. For more information go to:
British Paediatric Orphan Lung Diseases (BPOLD) www.bpold.co.uk
BPOLD aims to establish a registry of nine rare lung diseases in children in the UK. (OB is one) The registry will provide data on the prevalence and incidence of individual rare lung diseases and inform research projects which will increase our understanding of these diseases and ultimately improve treatment strategies for these children.

To date there has been no other publicised research about OB in the UK, however we will endeavour to include any new information if it becomes available.

Every-Disabled-Child-Matters Archived News

Breathtakers

Supporting sufferers of Obliterative Bronchiolitis and other Rare Lung Disease

Read the Government’s response