Orkambi – Better progress than we could expect!

Orkambi – Better progress than we could expect!

Update #1: Day 3, 28 June 2018 Oh, Ohh, Ohhhh #ORKAMBI!!!!! Today is a good day. Matthew’s tolerance for Orkambi has been very positive so far. He started his full dose yesterday after his first day on a half dose was ‘unremarkable’ in terms of adverse side effects. It has been a busy time in here with many, many sets of observations, four lung function tests per day (only one today) and three lots of intensive physiotherapy to support the treatment and movement of sputum from the lungs. On admission, Matthew’s lung function was 56.2% (1.8 litres). It fluctuated slightly on day one and two between 55.3% (1.77 litres) and up to 58.76% (1.88 litres) for his last one yesterday at 1.30 pm. Today’s lung function has climbed to 60.78% or 1.94 litres. It is clear to me that when Matthew is doing his lung function tests that it is getting easier to get a bigger breath in. Something I have seen diminish over the last 6 or so months. Matthew is clearing gunk well and some patterns are forming that will help with establishing treatment plans at home. The doctors are delighted as are the physiotherapists. There are expectations of severe initial side effects including problems with his breathing and airways being constricted, nausea, cold-like symptoms and headache, yet these are present so far. Doctors believe, based on the research of others who are on Orkambi, that we could expect an initial and significant drop in his lung function too. This has not happened obviously with an approximate 5% improvement in just a few days, this gives us so...
The Big ‘O’ Brings New Hope

The Big ‘O’ Brings New Hope

Background The 26th of June 2018 marks a monumental day for our family.  It marks the first day of access to a drug that is otherwise unattainable for our son Matthew.  A drug that potentially can stop the progression of an insidious disease he lives with called Cystic Fibrosis. At the time of writing this article, the below message was written to our loved ones to announce our news. We have been waiting three years from the time Orkambi was approved for safe use here in Australia.  This is a drug that has a price tag of around $260,000 per person per year.  The Australian Government and Vertex (the manufacturers of Orkambi) have been at a stalemate, unable to reach agreement on a ‘fair price’ for people living with Cystic Fibrosis to access. In the same week, Matthew started access, a new application was presented to the PBAC – Pharmaceutical Benefits Advisory Committee to have Orkambi included for access on our Pharmaceutical Benefits Scheme. To date, we do not know the answer but know that in the United Kingdom, access was again denied and Vertex is now likely to pull out of the country leaving thousands without access.  Hundreds have died waiting, hundreds more will die wondering ‘what could have been’. This is our story. The Big 'O' Brings New Hope! Day 1 – 26 June 2018 Matthew Started His Orkambi Journey today. He is the first paediatric patient in South Australia to be granted access! Today marked a new day in the history of our family. Matthew started his journey on Orkambi. Yes, that elusive drug that promises so...