Daily Archives: December 21, 2016

Call to Action for the Australian CF Community

Please support the Australian CF Community by writing to our Health Minister to ask for access to the lifesaving medications Kalydeco and Orkambi via the PBS using the guidelines below. This letter has been copied from an email distributed to the Victorian CF Community from the CEO of CF Victoria. Your letter will help save lives.


(This letter has been copied from an email received from the CEO of CF Victoria)

Dear Friends Rest assured that we are as equally devastated by last Friday’s PBAC announcement on Kalydeco and Orkambi, as you are. Right now it is important that we stand together as a community and push for these decisions to change. So much evidence exists supporting the effectiveness of these medications and so many people are dependent on their provision. We need to know why PBAC made this judgement call. I am asking you to contact the Hon Sussan Ley MP, Minister for Health and Aged Care. Tell her why the decision to defer Kalydeco and reject Orkambi is flawed and why access to these drugs is needed immediately. I invite you to include the following statements in your correspondence: Orkambi

  1. Clinical trials here in Australia and overseas have proven Orkambi’s clinical effectiveness and safety. The drug was seen to not only improve health outcomes, but also quality of life, it reduced exacerbations and subsequent hospital admissions.
  2. We must move swiftly as every day that a person is denied access to Orkambi is another day of potential life limiting lung damage.
  3. There are 1,000+ Australians with cystic fibrosis (CF) over the age of 12 years waiting for Orkambi.


  1. Kalydeco, Vertex’s first modulator drug, is proven to be a ‘game changer’ for people with the G551D cystic fibrosis gene change.
  2. The unique mechanism of action of Kalydeco revolutionised CF treatment, as it became the first to address the underlying causes of the disease.
  3. The PBAC deferred the decision on whether Kalydeco would be available for approximately 30 young Australians aged 2-5 years with the G551D gene change.  These children are depending on access to this life saving medication.  Without it they risk further lung damage.


  1.  In Australia we pride ourselves on a fair and supportive health system. People with CF have now found themselves caught in the middle of a battle between the Government and Vertex over price. Things need to change.
  2. The CF community should not be penalised because of a breakdown in a commercial agreement.

The Minister can be contacted through the following means: The Hon Sussan Ley MP P.O. Box 6022 CANBERRA  2600 Phone: (02) 6277 7220 Email: minister.ley@health.gov.au  We will not stop advocating and seeking answers until every person with CF has access to these life changing drugs.

Karin Knoester CEO

Cystic Fibrosis Victoria