CEO Update, Christmas Message, 20 Dec 2016
What a year 2016 has been! CFA reflects on our high impact moments and achievements. For more...
CEO Update, CFA Patron's Awards, 20 Dec 2016
Recently, Cystic Fibrosis Australia hosted the Governor General's Patron's Awards. The nine winners of the inaugural awards were announced at a ceremony at Admiralty House in Sydney. For a full list of those honoured, click here...
CEO Update, CF Community head to Canberra, 19 Dec 2016
It is now time to launch Cystic Fibrosis Australia's (CFA) next advocacy event and I hope many of the community will be able to be part of it. CFA has planned a public 'protest' on the lawn at Parliament House on 7 February 2017. For all the details...
PBAC Announcement for Kalydeco and Orkambi, 16 Dec 2016
Let your elected representative know why the DEFERMENT of Kalydeco and REJECTION decision about Orkambi are flawed.
- Orkambi meets the PBAC criteria
- Orkambi is an ESSENTIAL medicine… there is no other OPTION
- Trials proved clinical EFFECTIVENESS & SAFETY
- Orkambi CHANGES health outcomes and LIVES
- It cannot be compare to other treatments … ORKAMBI IS THE FIRST OF ITS KIND
Let your elected representative know why access is needed immediately.
1. Clinical trials here in Australia and overseas have proved Orkambi’s clinical effectiveness and safety and the drug was seen to not only improve health outcomes but also quality of life.
2. Kalydeco, Vertex’s first modulator drug, proved to be a ‘game changer’ for people with the G551D cystic fibrosis gene mutation.
3. Orkambi, created to mask the F508del gene mutations, improves lung function and reduces exacerbations, hospitalisation and antibiotic use. Trial participants reported improved nutrition and subsequent BMI increases, both of which have the capacity to stave off diabetes.
4. Most importantly the Orkambi trial had a positive effect on the mental health of people with CF. Research shows that overall wellness leads to social inclusion and an increase in employment and education participation. These factors in turn reduce personal and family stress resulting in less depression and anxiety for patients, parents and support networks.
5. In Australia we pride ourselves on a fair and supportive health system and now people with CF find themselves caught in the middle of a battle between the Government and the drug development company Vertex over price. Things need to change.
6. The CF community should not be penalised because of a breakdown in a commercial agreement
7. We must move swiftly as every day that a person is denied access to Orkambi is another day of potential life limiting lung damage.
Announcement now available
The Pharmaceutical Benefits Advisory Committee (PBAC) has announced two rulings today and there is no good news for the cystic fibrosis community. For the full CFA CEO Communique...
Vertex Statement on Orkambi. For more...
Let your Local Members or Senator, local newspaper, TV and radio stations know why access to these drugs is a must.
· Australians with CF need access to life-changing treatments to extend and improve life
· Kalydeco and Orkambi tackle the disease rather than just CF symptoms
· Clinical trials showed that…
- Three year outcomes for Kalydeco … reduction in lung damage 47% less than general CF cohort
- 96 week trial outcomes … Orkambi showed a reduction in lung damage 40% less than general CF cohort
- LUNG FUNCTION was preserved and further decline was halted, relative FEV1 increased by 4.8% and a sustained improvement was evident…
- EXACERBATIONS reduced, as did hospitalisations and antibiotic use.
- Reduced Exacerbations by 39% Hospitalisations by 61% Antibiotic use reduced by 56%
- NUTRITION improved, as did BMI and diabetes is less likely or less severe symptoms will be experienced.
- MENTAL HEALTH and QUALITY OF LIFE improved. Social inclusion, employment and education participation increase. Personal and family stress was reduced resulting in less depression/anxiety for patients, parents and support networks
Orkambi Update: 15th December 2016
Item 1: Ireland's Minister Harris welcomes positive engagement on Orkambi with Australian Health Minister
Ireland's Minister for Health, Simon Harris today welcomed positive engagement with the Australian Health Minister on the subject of Orkambi, saying:
“I was delighted to speak to Minister Sussan Ley, the Australian Health Minister, this morning following my recent letter to her on the subject of Orkambi. Minister Ley and I discussed the challenges we have in common on the reimbursement of Orkambi and agreed to explore working together in the interests of patients. Our officials will now engage to progress this matter.
In addition to the positive engagement with Australia, the initial feedback from a number of Countries I have written to and engaged with has been positive.”
Notes to Editors:
· The Minister wrote to Health Ministers in England, Scotland, Canada and Australia regarding Orkambi.
· The Minister also participated in the second Round Table meeting for European Health Ministers and CEO’s/Heads of Europe-based pharmaceutical companies, co-organised by Portugal and the Netherlands, in Lisbon on 7th December 2016.Item 2: Final statement on Ireland's Health Service Executive (HSE) Meeting 14th Dec 2016
Item 2: Final Statement on Health Service Executive (HSE) Meeting, 14th December 2016
Vertex and the HSE met on Wednesday to discuss access to Orkambi for eligible patients in Ireland. We are disappointed that we have not yet reached a definitive solution, but are pleased that discussions are ongoing and that the HSE has agreed to meet again next week.
At the Wednesday meeting, Vertex provided the HSE with another proposal that would provide immediate access to Orkambi for eligible patients. We cannot accept a deal that would inappropriately restrict access to Orkambi for eligible patients nor jeopardise our ability to continue to develop life-changing new medicines for the two out of three people with CF who still don’t have a medicine to treat the underlying cause of their disease.
The HSE has the power to provide access to this important new medicine to CF patients in Ireland who have already waited more than a year since Orkambi was licenced. With each passing day, the disease progresses and these patients simply cannot wait longer. We ask that the HSE share our sense of urgency in these negotiations so that together we can provide access to Orkambi for all eligible patients in Ireland as soon as possible.
Item 3: Ireland 14th December 2016
SEVERAL HUNDRED PEOPLE gathered at the Dáil at Wednesday lunchtime to demand that the Government acts to provide access to the Orkambi drug for cystic fibrosis patients.
The state’s medicines watchdog, the National Centre for Pharmacoeconomics, says that Orkambi is too expensive at the price demanded by maker Vertex Pharmaceuticals at €159,000 per patient annually.
Speakers who need the drug challenged Taoiseach Enda Kenny to come out and face them and tell them “face to face we’re not worth it”.
Sinn Féin President Gerry Adams pressed the Taoiseach in the Dáil during Leaders’ Questions to pursue all options to ensure access to the Orkambi drug for CF patients.
He also called on the Fine Gael leader to give a commitment to patients who are candidates for the drug that they will receive it while negotiations with the manufacturer are ongoing.
Teachta Adams said:
“Ireland has the highest number of people suffering from cystic fibrosis in the world and the largest proportion of families with more than one child suffering from the condition.
“According to Cystic Fibrosis Ireland, it affects around 1,200 adults and children in the state and citizens here suffer with some of the most severe strains of the disease. There are also 455 citizens with cystic fibrosis in the North.”
He said that Oireachtas members have heard in the past two weeks two “emotive and heart-breaking” presentations from people living with cystic fibrosis and their family members.
“We have heard from parents who have buried their children, and patients who manage gruelling daily medical regimes and who endure recurrent hospitalisation,” the Sinn Féin leader said.
“Many of them are valiantly battling for the opportunity to access the potentially life-changing drug Orkambi. Only 40 citizens have been able to secure Orkambi on a trial basis.”
Gerry Adams said he has asked Health Minister Simon Harris “numerous questions” about where the negotiations on Orkambi and other drugs are “but we are none the wiser”.
He said that families have asked six times to meet with the Health Minister “and I would ask the Taoiseach to ask him to meet them as a priority”.
The Louth TD said that some of the families were told recently by Vertex that the company was open to a risk-sharing model of payment among other payment schemes “so I would urge the Taoiseach to confirm if all options have been pursued with Vertex”.
Gerry Adams said that the Minister for Health in the North, Sinn Féin MLA Michelle O’Neill, has no direct responsibility in the matter, she has indicated a willingness to work with the Health Minister in the South to assist in the process.
“I urge the Taoiseach to commit to formally approach counterparts in the Assembly and other EU member states as part of a collaborative discussion to secure access to Orkambi.”
“There is a real urgency about the provision of this drug and I would ask the Taoiseach to consider a proposition of giving a commitment to the families who are candidates for the drug that they will receive it while negotiations are ongoing, even on a trial basis.”
Sinn Féin President Gerry Adams met with 14 parents of children with Cystic Fibrosis (CF) in Leinster House on Thursday 3rd November.
Teachta Adams hosted the meeting, along with Sinn Fein’s spokesperson on health Louise O’Reilly TD, following requests from his constituents and other parents, who believe that the new CF treatment, Orkambi, could significantly improve their children’s health and their quality of life.
Gerry Adams said,
“Today I have listened to the personal stories of how CF affects sufferers, the daily regime of drugs, physical therapy and treatments which these children undergo when they are well pales into insignificance in comparison to what they endure when sick.
“Orkambi could change their children’s lives reducing the amount of drugs they need to take and lowering their number of hospital admissions. They believe that it will significantly extend the life expectancy of their children and give the whole family a chance at a normal life.
“Among the group who came to Leinster House was one woman whose son is currently receiving Orkambi as part of a medical trial. In the 3 years since her son has been taking Orkambi his health has vastly improved and he has had no further deterioration in his lung function.
“I am aware that the National Centre for Pharmacoeconomics is currently engaged in the second round of negotiations with Vertex, the manufacturer of Orkambi, to assess the drugs’ value for money status. However the efficacy of Orkambi does not appear to be in question and indeed it has been approved for use in America.
“In other European countries patients are receiving Orkambi pending its approval by the European Medicines Agency and in the North of Ireland Orkambi can be obtained if it is clinically recommended.
CEO Update, NA CF Conference, 15 Dec 2016
For the second year in a row I attended the North American Cystic Fibrosis Conference (NACFC) and again it was an inspiring event. For more...
CEO Update, Kalydeco & Orkambi Alert, 13 Dec 2016
Once again we are holding our breath this week as we wait for a ruling from the Pharmaceutical Benefits Advisory Committee (PBAC) regarding the two life extending cystic fibrosis drugs… Kalydeco and Orkambi. For more...
CEO Update, Innovation Research Grant, 7 Dec
Cystic Fibrosis Australia (CFA) is pleased to announce the 2017 Innovation Research Award for post-doctoral scientists working in cystic fibrosis (CF) research. For more...
CEO Update, Mycobacterium abcessus, 11 Nov 2016
Today a paper was published in Science reporting the findings of a global study of Mycobacterium abscessus infection in CF.
Queensland CF researchers at The Prince Charles Hospital andLady Cilento Hospital participated in the study by contributing M. abscessus samples. These findings are important as they will inform clinical practice. For more...
CEO Update, Medical Research Future Fund, 10 Nov 2016
The Medical Research Future Fund have announced the Australian Medical Research and Innovation Priorities 2016-2018. One of the exciting priorities is to establish a consumer-driven health and medical research agenda. For the full list of priorities...
CEO Update, CF Lives Matters, 8 Nov 2016
Today Cystic Fibrosis Australia (CFA) launched a new information service for the cystic fibrosis community. It’s called CF Lives Matters, a name that rings true on so many levels.
Vertex Update, 7 Nov 2016
Positive Phase 3 Study of ORKAMBI(R) in Children With Cystic Fibrosis Ages 6-11 Who Have Two Copies of the F508del Mutation Supports a Submission to the European Medicines Agency in the First Half of 2017. For the full press release...
CEO Update, Kalydeco & the PBAC, 28 Sept 2016
On 5th November 2016, the PBAC will meet to decide whether KALYDECO is approved for reimbursement for 2-5 year old children in Australia. This label extension will greatly benefit 30 little Australians. For more...
CEO Update, Orkambi & the PBAC, 5 September 2016
There are 1,000+ Australians who would benefit from Orkambi being approved for reimbursement at the PBAC meeting in November. You can help those in the CF community who really need it and register your support for the listing of Orkambi by simply clicking this link. For the full CEO Communique, click here
Studio 10's Robbo tackles The Great Escape
Robbo and the Studio team joined the annual Cystic Fibrosis Great Escape Car Rally. Check out the video's of his adventure.
From the start of the rally in Dubbo, click here
After a few days on the rally, click here
CEO Update, "My Story", 25 August 2017
CFA plans to create a gallery of great “My Story” videos and compelling arguments for our politicians to use when lobbying for greater health resources. We want the CF community to share what it is like to live with CF and why greater support is needed and warranted. For more...
CEO Update, Cystic Fibrosis Conference 2017, 17 August 2016
In 12 months time – from August 5th to the 8th, 2017 - the 12th Australasian Cystic Fibrosis Conference (ACFC) will be held in Melbourne.
The conference is Australasia's largest event dedicated to cystic fibrosis (CF) and will bring together around 200 lay people and over 350 medical, allied health and nursing delegates plus CF researchers from across the region and around the world.
CEO Update, The Kaleidoscope Project, 11 August 2016
Now is your chance to have your say … The Kaleidoscope Project is a national initiative designed to bring together children with chronic disease, their families, clinicians and policy makers to identify important research topics and priorities. For more...
CEO Update on Kalydeco, 6th July 2016.
The TGA is reviewing Vertex's submission on using Kalydeco for the treatment of patients aged 2-5 years with cystic fibrosis with a G551D or other gating mutation. For the full Communique from CEO Nettie Burke, click here.
There is a very informative article by the KIWI Study Group on CFA’s website that will provide you with evidence of Kalydeco’s safety and efficacy. Click here for full article.
Vertex Kalydeco and Orkambi update
Vertex announces Presentations of Data for KALYDECO(R) (ivacaftor) and ORKAMBI(R) (lumacaftor/ivacaftor) at European Cystic Fibrosis Society (ECFS) Conference. For more
CEO Update on Orkambi, 6th June 2016
Now is the Winter of our Discontent … but is our dissatisfaction about to end?
We are now in the Australian Winter and more than a month after the devastating decision by the PBAC not to reimburse Orkambi, the cystic fibrosis community remains outside the ‘stakeholder consultations’ about the funding of costly new generation drugs.
The consumer voice should be acknowledged and Cystic Fibrosis Australia needs the CF community to mobilise. Click here for more information on how you can help.
To download the social media assets, click here.
CEO Update on Orkambi, 23 May 2016
A month ago today we were devastated with the news that the PBAC rejected Vertex’s proposal for the reimbursement of the cost of Orkambi. We have had great support from the community, our clinicians, CF CAN and the media. It's time to contact the Politicians. For more.
Carrier Screening Education Campaign
Almost all children born with cystic fibrosis are born into families where there is no known family history. Cystic Fibrosis Victoria has launched a Community Education Campaign on Carrier Screening to help create awareness of CF and the availability of Carrier Screening. For more information, click here.
Cystic Fibrosis Australia (CFA)
CFA is a national not-for-profit organisation and the national body supporting people with Cystic Fibrosis. CFA comprises member State/Territory Cystic Fibrosis organisations. CFA's mission is to deliver research, advocacy and quality improvement outcomes supporting people with CF and be the peak national body supporting our State/Territory members with their goals to enhance the quality of life of people affected by CF.
We do this by:
- Collecting and distributing information about CF at a national and international level
- Building networks with acclaimed CF physicians, researchers and CF organisations in Australia
- Organising the biennial Australasian CF Conference
- Developing educational publications to support CF State organisations in their work
- Securing funds for the Australian Cystic Fibrosis Research Trust and assembling the professional expertise needed to administer CF research projects throughout Australia
- Developing a roadmap for CF research in Australia in conjunction with the Australian Cystic Fibrosis Research Trust (ACFRT)
- Sourcing funding support for, and administering the Australian Cystic Fibrosis Data Registry
- Assisting with developing national policy and advocating for change to bring benefits to people with cystic fibrosis and their families
Promote quality improvement in health care
- Facilitate the development and review of national guidelines for ‘Standards of Cystic Fibrosis Care’ and ‘Infection Control Guidelines’
- Resource and coordinate the ‘Peer Review of Cystic Fibrosis Clinics in Australia’
- Publish the Annual Australian Cystic Fibrosis Data Registry Report for use by clinics and researchers
- Organising national fundraising campaigns such as ‘65 Roses’ each May to raise awareness of CF and funds for research and education programs and more.
How you can help
Donations and sponsorships are vital to advance research into CF, conduct advocacy and to ensure improvement in health care services available to people living with CF. You can contribute to helping improve the quality of life of those who live with CF by donating today.