New treatment Trikafta
SUBSIDY from 01/04/2022 (12 year old +)
There was much excitement and joy for the CF community with the news that the long awaited modulator drug Trikafta was listed on the PBS for those aged 12+ with CF who have at least one F508del mutation of the CFTR gene. The government funded it on the PBS so that it was available for access at an affordable cost. After Trikafta became available we have seen its transformative effects, with many happy stories about its life-saving and life-changing effects.
SUBSIDY from 01/05/2023 (6 to 11 year old)
This was the best news for the 500+ families who had dreamt of a new future thanks to access to this amazing medication! Previous reports about Trikafta trials in children have been very positive, they evaluated safety and efficacy and it was shown to be generally well tolerated and safe, and participants showed improvements in their lung function, their sweat chloride and their weight.
While it was a great day for many of our CF community, I would also like to acknowledge that not everyone with CF will benefit from Trikafta, however it is great to know that the pharmaceutical company Vertex is still working on other modulators for rarer CF genes that affect about 10% of the CF population.
Thank you to everyone who lobbied, wrote submissions, signed a petition, shared their story on social media, shared and liked posts about Trikafta, and helped to keep the campaign going to get Trikafta funded.
The earlier these medications are started, the greater the predicted benefits - more years are gained, more time with a higher lung function and a significantly lower chance of transplant.
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.