Lives unaffected by cystic fibrosis

New! Orkambi messaging and sample letters for 2018

We are thrilled to hear that Orkambi has been submitted to the PBAC for the next agenda with an outcome expected mid August. Now is the time to join together and make our voices and heard to ensure that people with CF stop falling through the cracks. Below we have listed key facts that you can refer to when talking about Orkambi, sample letters for you to send to your local, state and federal politicians, links to the PBAC portal for consumer comment and important dates surrounding this submission. 

Key facts:

ORKAMBI by Vertex

  • Fourth PBAC Orkambi submission to the PBAC for reimbursement.
  • The PBAC has confirmed the safety and efficacy but rejected Orkambi on cost
  • Lung function 
    • Preservation of lung function … lung health maintained … Halts further decline …
    • Relative fev1 increase 4.8%
    • 96-week trial outcomes clearly showed that lung damage was slowed by an average of 40%.
    • Reduced lung capacity is a major cause of morbidity and mortality.
  • Exacerbations reduced by 39%
  • Hospitalisations reduced by 61%
  • Antibiotic use reduced by 56%
  • Improved nutritional status, increased BMI and less likelihood of diabetes or less severe symptoms.
  • Mental health - less personal and family stress resulting in less depression and anxiety for all.

PBAC Portal Key Points

  1. We’re thrilled that Vertex has resubmitted for the fourth time to the July PBAC meeting and we support both ORKAMBI agenda listings 12+ years and 6-11 years.
  2. ORKAMBI is designed to treat the underlying cause of CF in people with two copies of the F508del mutation, the most common CF mutation. This is a first!
  3. Currently the treatment arsenal for cystic fibrosis is limited to therapies that treat the symptoms and clinical manifestations of the disease.
  4. ORKAMBI is the first treatment available to patients with this mutation that can prevent permanent, irreversible lung damage.
  5. CF patients experience a 1-3% average decline in lung function per year. Maintaining or improving lung function has a significant clinical benefit.
  6. Even when a CF patient does everything right, they will still experience recurrent pulmonary exacerbations.
  7. People with CF have a fundamental medical need for modulating therapies like ORKAMBI to correct the function of the CFTR protein.
  8. For F508del mutation, patients need both a corrector (lumacaftor) and a potentiator (ivacaftor).
  9. FEV1 improvement varied greatly among patients in the clinical trials; some patients experienced dramatic improvement, some maintained their lung function, some improved modestly. All of these circumstances are clinically meaningful in CF.
  10. ORKAMBI can provide significant improvement in pulmonary exacerbations.
  11. Other positive findings included improvements in BMI and patient-reported outcomes.
  12. The long term potential of ORKAMBI is significant for both adults and children with CF. CFA wants the Australian Cystic Fibrosis Data Registry to be used to gather information on efficacy going forward
  13. Patient responses to ORKAMBI will vary and can manifest in many different ways. Clinicians are best suited to determine whether the drug is effective for each patient.
  14. The 96-week trial results clearly showed that lung damage was slowed by an average of 40%. Reduced lung capacity is a major cause of morbidity and mortality.
  15. Clinical trials inclusion parameters such as FEV1 should not preclude access to this lifesaving therapy for patients that fall outside these parameters
  16. We urge the PBAC to evaluate the totality of evidence of patient response, including but not limited to improvements in FEV1, reductions in pulmonary exacerbations, increases in BMI, reductions in hospitalizations, and improvements in patient-reported outcomes.
  17. We must hold our politicians and bureaucrat’s consciences to the fire so they don’t shy away from the tough decisions

PBAC Links:


Life with ORKAMBI

  1. Lung function 
    1. Preservation of lung function
    2. Maintain lung health
    3. Halts further decline
    4. Relative fev1 increase 4.8%    ...    sustained improvement
  2. Exacerbations
    1. Hospitalisations by 61%
    2. Antibiotic use reduced by 56%
  3. Nutrition
    1. Improved nutritional status
    2. Increased BMI
    3. Less likelihood of diabetes or less severe symptoms.
  4. Mental health
    1. Social inclusion, employment, school and reduced’ late effects’
    2. Less personal and family stress resulting in less depression and anxiety for patients, parents and support networks

Important dates:

  • 27 April 2018 - Consumer Portal opens for comment. 
  • 6 June 2018 – Consumer Portal Closes for comment.
  • 25 June 2018 -  Siblings Event in Canberra.
  • 3-5 July 2018 – PBAC Meeting in Canberra.
  • 17 August 2018 – PBAC Announcement.


About CFA's Advocay plan and strategy

Cystic Fibrosis Australia has an advocacy strategy designed to ensure …

  • cystic fibrosis is kept top of mind with our politicians and decision makers
  • researchers are professionally drawn to the CF field and become involved
  • funds are forthcoming from the corporate and community sectors for a worthwhile cause and
  • the broad Australian community understands the inequity that surrounds CF.

CFA is a robust and authoritative voice for people with cystic fibrosis and their families, carers, partners and support networks. We are often speaking for the powerless to the powerful.

Government, business and the health and community sectors need to understand and appreciate the challenges people with CF face. This includes key issues such as drug affordability and availability, infection control, gene testing and access to the best medical and allied health services.

Over the past 18 months CFA has driven the following advocacy campaigns.

Ongoing campaigns

Pills to the Pollies

Lolly Jar awareness - Send us an email to with your name, contact details and location, local newspaper name, favourite radio station announcer, local MP or Senator name and electorate area and we will send them all a ‘lolly jar’ full of sweets.

We will ask these community leaders to guess how many lollies are in the jar. The answer will be ‘a member of your community knows as they take this number of tablets every day’.

Make your voice heard

Community Videos ‘Why I need Orkambi’

CFA are always bringing videos and letters from the community to present to The Government. If you have a message you want to share and are confident to turn it into a video, please send it to

Upcoming campaigns

Siblings Events

The impact of living with CF reaches far greater than just the person with CF. To highlight this, Cystic Fibrosis Australia will be hosting a Siblings afternoon tea in Parliament House, Canberra. 

Join us as we share anecdotes and stories with friends in this truly unique event.

For more details and to regsiter your interest, email

NFP forum

The process of having drugs approved in Australia is a long and arduous task. With this being a common issue amongst those NFPs working in the treatment and accessibility space.

In October, we invite key representitives from various NFPs around Australia to share their experiences and work togther to devise an easier and more efficient process. 

For more information on this event, please contact

Indigenous and Ethnicity Report

Mental Health Roadshow

Previous campaigns

PBAC Consumer Hearing

Oh Oh Oh Orkambi

Canberra Protest

Bronchitol and Pulmozyme

Department of Health and Minister’s Office Stakeholder Meeting

Make an ‘O’ for Orkambi

Christmas Card campaign to Minister for Health and Vertex