Major Project 2020


Our charity is proud to have provided home spirometers to 50 CF adults living in regional NQ and far NQ.  This technology has helped CF adults perform lung function testing at home while in self isolation, avoiding the need to attend hospital for testing.  The challenges of the COVID-19 pandemic were acutely felt by our CF community.  Our CF group are vulnerable and the monitoring of lung function is very important for early detection of deterioration in pulmonary health over time.  The spirometer is able to sync to the patient’s mobile phone and the data stored.  The data can also be emailed to their CF team for assessment and discussed at their telehealth appointments.  The cost of this project was $18,696-99.

New treatment

A new drug called Trikafta is a triple combination therapy of ElexacaftorTezacaftor, and Ivacaftor that is capable of preventing or slowing down lung disease and reducing other typical symptoms of CF.  It has been designed for those who have at least one DeltaF508 mutation, the most common CF gene, seen in about 90% of all people with CF.  Some people in Australia have already been taking Trikafta on a clinical trial, and some have been granted access for compassionate use.  Those taking Trikafta have experienced general improvement of their CF symptoms such as an increase in their lung function, reduced lung infections, fewer hospitalisations, and weight gain.  This is not a cure, but it will extend and save lives.  There is approx 10% of the CF population that have 2 rare genes, and the drug is not suitable for them.  We are hopeful that the pharmaceutical company Vertex will be able to develop additional drugs so that no one is left behind.

In February 2020 Vertex submitted Trikafta to the TGA (Therapeutics Goods Administration) for approval for use in Australia for people with CF who are 12yo and older.  Generally, the TGA’s safety and efficacy process will take about 12 months.  Once approved the drug can proceed to the PBAC (Pharmaceutical Benefits Advisory Committee).  The PBAC is an independent body of doctors, health professionals, health economists and consumer representatives.  The PBAC’s main role is to recommend new medicines for listing on the PBS (Pharmaceutical Benefits Scheme) so that they are affordable. No new medicine can be listed unless the Committee makes a positive recommendation.  PBAC will have their next meeting 10th-12th March 2021 and we are hopeful Trikafta will be on the agenda.  The PBAC agenda will be available on their website on 24th December, when it will be open for consumer comments.  Comments will close on 10th Feb.  You can be part of the lobbying campaign by contacting your local member of parliament and senator or by sharing the fight for Trikafta over social media.  The PBAC will announce their decision on 23rd April.

Vertex announced in September 2020 positive results from a Phase 3 trial of Trikafta in CF children aged 6-11.  They investigated, safety, tolerability, and efficacy.  Results showed that the children tolerated Trikafta well.  Vertex plans to file a supplemental new drug application to expand the age of approval for Trikafta.