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On the 16th December 2016, the Pharmaceutical Advisory Committee (PBAC) announced that they were deferring the decision to allow access to Kalydeco for Cystic Fibrosis sufferers in the 2-5 year old age group.
Cystic Fibrosis (CF) is the most common life threatening genetic condition in Australia. Cystic Fibrosis (CF) primarily affects the lungs and digestive system because of a malfunction in the exocrine system, responsible for producing saliva, sweat, tears and mucus. The current life expectancy for a person living with CF is 38.
There is currently no cure. People with CF develop an abnormal amount of excessively thick and sticky mucus within the lungs, airways and the digestive system. The mucus causes impairment of the digestive functions of the pancreas and traps bacteria in the lungs resulting in recurrent infections which lead to irreversible damage. Lung failure is the major cause of death for someone with CF.
From birth, a person with CF undergoes constant medical treatments and physiotherapy.
Kalydeco was recently included on the PBS for people with the 551D/508 genetic mutation over the age of 6. However, although the Therapeutic Goods Association (TGA) gave their approval for the 2-5 year old age group, based on trials outside of Australia, the PBAC have claimed there is not enough evidence and have deferred their decision. There are 30 children Australia wide who need this medication. Everyday they are without it, is another day irreparable damage is being done to their lungs.
I am a mum of a 2yo girl, Scarlett who has this genetic mutation. I was also fortunate enough to be asked to be the parent advocate in a meeting with the PBAC and other relevant stakeholders prior to this decision being made. I came away feeling confident that our voice had been heard and that our little girl and the 29 other 2-5 yo's in the country would be approved to begin taking Kalydeco as soon as possible. Unfortunately, it is not the case. I have a beautiful, caring, crazy intelligent 2 year old, who had no say in the genetic make up she was given. She has a terrible disease that will end her life prematurely and puts our little family under significant physical, financial and emotional strain constantly. The PBAC has decided that for whatever reason, her quality of life is not worth the endorsement of this life changing drug. Please support our family and the other 29 families in Australia whose little children's lives will be dramatically changed for the better if this drug is endorsed. Every day is making a difference. Thank you.
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Sam Jesney needs your help with “Malcolm Turnbull: Say #YesToKalydeco”. Join Sam and 3,043 supporters today.