December 20, 2016

The PBAC again rejected Vertex’s Orkambi submission and it was not been recommended for reimbursement. A third major PBAC submission is on the cards for 2017.

Read the official statement by Vertex here

Cystic Fibrosis Community Care has responded with an open letter to the community

August 24, 2016

The agenda for the November 2016 PBAC meeting is now available and Kalydeco (2-5 years) and Orkambi (12+) are both listed.

Your comments and support are needed as a patient, carer, member of the public, health professional or member of a consumer interest group.

The Consumer Comments Submission form and further information can be found on the PBS Website.

CFA will be distributing materials to support the reimbursement of both drugs shortly.

6 June 2016

Now is the Winter of our Discontent … but is our dissatisfaction about to end?

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.

Download the social media assets.

23 May 2016

In April, disappointing news that the PBAC rejected Vertex’s proposal for the reimbursement of the cost of Orkambi rocked the CF community in Australia.

On the other hand, CFA and CFCC have had great support from the community, our clinicians, CF CAN and the media in our campaign for Orkambi. Thank you!

And now, it's time to contact the politicians. 

CFA has developed a Politicians Support campaign to inform local members of the issues that affect the CF community. CEO Nettie Burke's Communiqué has everything you need to lend your voice to the campaign, including a few simple questions to ask your local leaders, as well as, their contact details.

17 May 2016

The PBAC ruling rejecting the reimbursement of Orkambi and the lack of support and interest in healthcare in the Federal Government’s budget have caused a great deal of disappointment and dissatisfaction in the cystic fibrosis community. We need your help to figure out our next steps.

Click here to read the CFA CEO's Communiqué on how you can help

12 May 2016

"They're little pink tablets of hope. That hope is just sitting there for all these patients with CF..."

In case you missed the full story on Orkambi by Today Tonight Adelaide featuring the fabulous Morgan and Bec, as well as CFA CEO Nettie Burke and Professor Peter Wark, you can watch it here now.

27 April 2016

Our health system is ailing and placing Australians at risk. Cystic Fibrosis Australia is calling on the community to find their voice and share their stories...

Click HERE for CFA communique

Find out how you can get involved

22 April 2016

Orkambi, the latest new generation drug for cystic fibrosis, will not be recommended for reimbursement by the Pharmaceutical Benefits Advisory Committee (PBAC) for Australians with cystic fibrosis (CF)…

Click HERE for CFA communique

March 2016


The Therapeutic Goods Administration (TGA) approved Orkambi for people aged 12 years or older, based on research involving over 1,100 patients.

The Orkambi reimbursement process to have the Pharmaceutical Benefits Advisory Committee (PBAC) list the drug on the Pharmaceutical Benefits Scheme was set in motion.

Click HERE for CFA communique

January 2016

Canadians over the age of 12 with two copies of the F508del mutation are celebrating this week the approval of Orkambi by Health Canada.

Click HERE for CFA communique

11 November

Cystic Fibrosis Australia CEO Nettie Burke's comments after attending the North Amercian CF Conference in October 2015.

Click HERE for CFA communique

29 September

Cystic Fibrosis Australia respond to European recommendations regarding Orkambi and the the extension of Kalydeco for children under six years of age.

Click HERE for CFA communique on latest European recommendations regarding Orkambi and Kalydeco

7 July 2015

Click HERE for CFA article on Orkambi FDA approval  

3 July 2015

Cystic Fibrosis Australia can confirm the U.S. Food and Drug Administration (FDA) has approved Orkambi, the combination cystic fibrosis (CF) drug containing 400mg of lumacaftor and 250mg of ivacaftor (daily dose) for people who are 12 years of age and living with the F508del homozygous gene mutation. 

Click HERE for the detailed Cystic Fibrosis Australia Message

May 2015

Clinical Trial results of Orkambi with 2 copies of F508del were published and reviewed in New England Journal of Medicine in May 2015.  

Click HERE for more details.


Kalydeco PBS Listing Campaign

Kalydeco Update - December 20, 2016

In a perplexing turn of events, approval of the Kalydeco submission was deferred by the PBAC. 190 submissions supporting Kalydeco were received through the PBAC portal and CFA has been told that the PBAC and Vertex will continue to work on a solution.

Read the official statement by CFA here

Kalydeco Update - September 28, 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. The KALYDECO KIWI study resulted in a drop in sweat chloride and most importantly, little lungs were kept healthier for longer.

CFA is asking the cystic fibrosis community to support the approval of KALYDECO for 2-5 year olds by going to the PBAC website and completing a consumer comment. Comments will not be accepted after 5pm (AEST) on 5 October 2016.

The key points that need to be made are:

1. There is nothing like Kalydeco… it is a drug that treats the disease, not the symptoms

2. Kalydeco slows down lung damage resulting in better health outcomes across a child’s life

3. Overseas studies show 50 mmol/litre reduction in sweat chloride

4. Every day of delay, young lungs are experiencing irreparable damage

5. 200 Australians over the age of 6 years are currently are benefitting from KALYDECO. Let’s expand it by 30 people and support little Australians between the ages of 2-5 years.

Please call CFA on 02 98895171, if you have any questions. If you would like to create a My Story video, please send it to [email protected] All My Story videos will be used to make our case with the PBAC. You can view some of the wonderful creative films here


1 December 2014

In addition to G551D, the PBS subsidy for Kalydeco has now been expanded to include people aged 6 years and older who have gating (class III) mutations including: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G970R and G1349D. 

Click HERE to download the Cystic Fibrosis Australia Media Release

Click HERE to download the Vertex Media Release


26 October 2014

Kalydeco PBS Listing from December 1, 2014.

After over a year of waiting for affordable access to the life changing drug Kalydeco, Federal Health Minister, Peter Dutton MP, has announced that this revolutionary medication will be listed on the PBS from December 1.   Mr Dutton has said "that all cystic fibrosis patients six years and older who have a G551D mutation in the CFTR gene will be treated with ivacaftor for as long as needed."

Click HERE to download Health Department Media Release

Click HERE to download Cystic Fibrosis Victoria Media Release

Click HERE to download Cystic Fibrosis Australia Media Release

28 August 2014

Criteria for access to Kalydeco through the PBS explained

Cystic Fibrosis Australia (CFA) has released the following statement to help clarify the prescribing criteria as outlined by PBAC following the Stakeholder meeting.  CF clinicians from around Australia and representatives from CFA agreed with the PBAC on these points, given the data currently available.

Click HERE to download copy of CFA Statement

Further discussions on this will take place on 3 September when CFA hosts a meeting with Vertex Pharmaceuticals. Read more on proposed meeting


11 August 2014

Prescribing Criteria for PBS listing of Kalydeco

As an outcome of the Stakeholder Meeting held on 31 July, the Pharmaceutical Benefits Advisory Committee (PBAC) released a statement which reflects the pay for performance framework recommended by the Pharmaceutical Benefits Advisory Committee (PBAC) in March 2014, under which all eligible patients could be treated with PBS-subsidised ivacaftor but the price paid by the Government varies depending on the magnitude of the benefit gained by patients.  

Click HERE to download a copy of this statement.

Vertex responded that they remain committed to reaching a resolution to this situation and reiterated that it firmly believes that all Australians with the G551D mutation ages 6 and older should have the opportunity to receive Kalydeco, just as patients have in 18 other countries around the world where they have funding agreements in place.Read full statement


31 July 2014

Stakeholder meeting

A stakeholder meeting was held on 31 July 2014 to discuss the Pharmaceutical Benefits Scheme (PBS) listing of ivacaftor (Kalydeco) for the treatment of cystic fibrosis in patients aged six years and older who have a G551D mutation in the CFTR (cystic fibrosis transmembrane conductance regulator) gene.  Invited participants included members of the Pharmaceutical Benefits Advisory Committee (PBAC), representatives from Cystic Fibrosis Australia and cystic fibrosis specialists from Centres in New South Wales, Victoria, Queensland and South Australia.

The focus of discussions was on finalising the  prescribing criteria for listing the product on the PBS, which were released to the public on 11 August.


18 July 2014


Kate and Steve MacPherson have started a petition which they would like to present to the House of Representatives. Parliament's Standing Committee on Petitions has strict guidelines for submission of petitions including the need for them to be signed by hand.  It will not accept electronic petitions.  Therefore we encourage you to download the petition sheet from the link below and physically capture the details of your friends, families, neighbours etc including their signatures and post or deliver to our office at 80 Dodds Street, Southbank 3006.

Click HERE to download the petition page.

14 July 2014

Block on Social Media comment

Peter Dutton has taken the unusual step of blocking members of the CF community from putting messages about YesToKalydeco on his Facebook and Twitter sites. We are upset that he is not wanting to listen to our valid concerns about his government's failure to move quickly to help those in our community whose lives could be dramatically improved on Kalydeco.

CFA CEO Michelle Skinner has contacted Minister Dutton's office and is awaiting a response. Updates and additional information will be posted as it becomes available.

CF organisations are urging everyone who is affected by the Kalydeco decision, and those of you who might be one day, to begin writing letters to your Federal Member or Senator asking them to support the introduction of Kalydeco onto the PBS and raise this matter in Question Time.  Click HERE to download a sample letter which can be used as a guide.

25 June 2014

Positive results from latest clinical trial combining Kalydeco and new drug lumacaftor in people with most common CF mutation

Yesterday pharmaceutical company Vertex announced that its latest trials involvingivacaftor (Kalydeco™) in combination with a new drug lumacaftor (VX-809) for people with two copies of the F508del mutation of cystic fibrosis showed significant improvements in lung function and other key measures of the disease.

The potential treatment is the first to combine two pills to address the underlying genetic cause of CF in people with the F508del mutation. The Phase 3 trials were conducted at approximately 200 clinical trial sites in North America, Europe and Australia. In total, more than 1,100 people with two copies of the F508del mutation, ages 12 and older, participated in the studies.

The trials tested two different doses of the ivacaftor and lumacaftor combination therapy. Compared with those on placebo, participants who took the combination treatment showed significant and consistent improvement in lung function and in other important health measures, including weight gain, and a reduction in the rate of pulmonary exacerbations. 

Repairing the defective protein in people with the F508del mutation is a particularly challenging and intricate process. In this mutation, a series of problems prevents the protein from reaching the surface of the cell. Lumacaftor is designed to move the F508del CFTR protein to the cell surface where ivacaftor can improve its function and help increase the normal flow of salt and fluids in and out of the cell.

Source: http://www.cff.org/aboutCFFoundation/NewsEvents/2014NewsArchive/6-24-Vertex-Phase-3-Results-Lumacaftor-Ivacaftor.cfm

Click here to view a full copy of the Vertex Announcement 

Click here to view the media release issued by CFV on this development.

11 June 2014

Kalydeco Changes Lives

Record your 30 second message on how  Kalydeco has changed or could change your life on this dedicated YouTube channel - www.youtube.com/KalydecoChangesLives

Click here for suggested guidelines and instructions for recording and uploading your message or download as a PDF.

2 June 2014

Kalydeco Raised at Senate Estimates

On Monday 2 June at the Hearing of the Community Affairs Legislation Committee which comprises the Health Portfolio, Senator Nick Xenophon raised the issue of Kalydeco.  He pressed Ms Felicity McNeill, First Assistant Secretary,  Pharmaceutical Benefits Division, Department of Health on why Australia was one of the last developed nations to approve subsidizing this drug.  
Estimates of government expenditure are referred to Senate committees as part of the annual budget cycle. This opportunity to examine the operations of government plays a key role in the parliamentary scrutiny of the executive.

A transcript of this particular discussion is can be downloaded HERE.  For the full record of this meeting go to http://bit.ly/SenateEst2Jun2014.

28 May 2014

Kalydeco listing on the Agenda at Parliament House 65 Roses events

Representatives of the cystic fibrosis community from around Australia will be gathering in Canberra today at Parliment House as part of this year's 65 Roses Celebrations.  Faye Upston, Kate McPherson and Rachelle Haikalis, three ladies with a thorough and passionate understanding of the need for Kalydeco, form the Victorian contingent. 

Here Faye, Kate and Rachelle explain their Hopes and Intentions prior to the event .

Here Faye and Kate describe their Meeting with the Minister

20 May 2014

Media Release - Vertex submits new criteria for Kalydeco PBS listing to Government.

The manufacturer of Kalydeco has submitted to the Department of Health what it believes is a new and more equitable criterion for the provision of the breakthrough therapy on the Pharmaceutical Benefits Scheme (PBS).  “According to Vertex, the alternate criteria are aligned with advice from Australian Cystic Fibrosis clinicians and allows for ongoing treatment for all people with cystic fibrosis and the G551D gene aged 6 years and older," said Michelle Skinner, CEO of Cystic Fibrosis Australia.  “We look forward to accessing and reviewing the amended criteria as soon as possible and will continue to pressure Vertex and the Department to put the health and wellbeing of Australians ahead of cost savings and politics.”

5 May 2014 

Media Release - Vertex developing an alternative PBS listing criteria 

Vertex, the manufacturer of Kalydeco, today shared with Cystic Fibrosis Australia its rationale for rejecting Pharmaceutical Benefits Scheme (PBS) listing criteria developed by the Pharmaceutical Benefits Advisory Committee.  Vertex has committed to working with the Department of Health on a compromise that is based on the feedback of Australian clinicians and trial data. Vertex will present an amended set of criteria to the Department in the coming weeks.   Read more

1 May 2014 

CEO update on Kalydeco Funding

 WE have now had a few days to digest the PBAC minutes and what they might mean. I think it is fair to say that there are still many unanswered questions and areas where we need clarity. CFA have taken a lead in meeting with Minister Dutton on Tuesday this week, and also in speaking to our CF Centre Directors to get their views. Next Monday there is a meeting scheduled with Vertex and a teleconference for CEO’s to hear what Vertex have said. I would still like to hold a meeting with interested members of the Victorian community, but would prefer to wait until next Monday to set the date, when we will have a more comprehensive view of where all parties sit in the negotiations. I will be in touch early next week. Warm regards, Helen


29 April 2014 

CFV Response to Latest Developments in Kalydeco Funding 

CFV, together with the CF community across Australia, has found today’s announcement by the Pharmaceutical Benefits Advisory Committee (PBAC) regarding Kalydeco, disappointingly unclear. While the PBAC has indicated that it is a positive recommendation a number of conditions have been attached and the implications of these conditions need further clarification. Please be assured that CFV is determined to ensure appropriate access to this medication for those who need it. We will be seeking further advice over the next few days, and will be organising a community meeting at a date to be confirmed. Read more


29 April 2014    

Statement from Michelle Skinner, CEO, Cystic Fibrosis Australia - Kalydeco and PBAC Update

Update on CFA activity in response to PBAC minutes and Vertex media statement. Following publication of the Pharmaceutical Benefits Advisory Committee (PBAC) minutes on Kalydeco, Cystic Fibrosis Australia (CFA)  met with the Minister for Health, the Hon Peter Dutton MP.  CFA will shortly meet with representatives from Vertex to better understand the company's decision to reject the conditions outlined yesterday by the PBAC. Like the CF community, CFA is keen to understand exactly why the conditions are unacceptable and to what extent Vertex is willing to work with the PBAC to find a solution to this impasse. Read more


13 March 2014   

Pharmadispatch - Patients Want Intervention on Kalydeco 

CFA’s outgoing CEO David Jack described the process for funding Vertex’s breakthrough drug forcystic fibrosis patients with the G551D gene mutation as “curious” and called for interventionfrom Health Minister Peter Dutton, arguing that “there is no justification for further delayingaccess to treatment in my opinion.”Jack and incoming CFA CEO Michelle Skinner were speaking to PharmaDispatch as thePharmaceutical Benefits Advisory Committee (PBAC) meets to consider another submissionfrom sponsor company Vertex.  Read More


11 February 2014 

Media Release -  Give the Breath of Life

An urgent plea for compassion has been floated over Parliament House in Canberra in an attempt to expedite the reimbursement of a lifesaving therapy for a rare form of cystic fibrosis.  As parliamentarians sit for the first time this year, a billboard affixed to an eight story high hot air balloon has called on the Federal Government and the manufacturer of Kalydeco to agree on a price for the medicine and “Give The Breath of Life” to 200 Australians living with cystic fibrosis caused by a gene mutation known as G551D. Read more


20 December 2013 

CFA Letter  to CF Community - PBAC Recommends Subsidy for Kalydeco

The Pharmaceutical Benefits Advisory Committee (PBAC) has recommended the inclusion of Kalydeco on the Pharmaceutical Benefits Scheme (PBS), however, to use a sporting analogy, while we have triumphed during the first half there is still another half to play before victory is celebrated.  A positive PBAC recommendation now allows Vertex and the Department of Health to negotiate a price agreement.  Once a price agreement is reached, the Minister for Health, the Hon Peter Dutton, must seek Cabinet approval to subsidise Kalydeco on the PBS. Read more