Happy 65 Roses Day everyone!
I plan to write an update about my health soon, but in the meantime I thought I would write about the Kalydeco situation in Australia.
Kalydeco is still not available in Australia, due to lengthy price negotiations between Vertex and the Australian Government. These negotiations first started over a year ago. This means Australia is now over two years behind the US and over one year behind the UK and Europe in terms of Kalydeco access.
We need Vertex to lower the price and we need the Australian Government to compromise on price. We need this to happen now.
For the last two years I’ve been very involved with #YesToKalydeco Campaign on both facebook and twitter. Many of us behind the campaign met for the first time in person in Canberra last Wednesday. Cystic Fibrosis Australia organised two events, a morning tea and evening cocktail event, aiming to raise awareness about Cystic Fibrosis & the urgent need for Kalydeco.
So after a very rushed birthday (I think I will celebrate this later when Keith is back from his trip to the middle of nowhere), I flew to Canberra for these events. It was an action packed day, with a morning tea, where we all met each other for the first time, spoke to several MPs about Kalydeco & heard from Peter Dutton the Health Minister, followed by a meeting with the senior advisor to the shadow Health Minister, then finding out that I was giving a speech that night, quickly writing my speech in about an hour, getting to the cocktail event and almost collapsing as I had a hypo pre speech (skulled some soft drink and luckily made it through!). The speech went really well, I was glad that I could let Peter Dutton know exactly how I feel regarding the Kalydeco situation, for a full 3 minutes!! It was also great talking to several other MPs that night, especially Senator Anne Urquhart & MP Ken Wyatt.
I’m sure the MPs that were present came away from these events understanding the urgency behind the PBS listing of Kalydeco. I’m very hopeful that we will hear the outcomes from recent meetings between Vertex and the health department in the near future & that soon they will agree on a price.
A real highlight for me was meeting everyone for the first time in person, after working together with the campaign for over 2 years!! I hope the next time we see each other is at a celebratory Kalydeco party!
I have added a copy of my speech that I presented in Canberra below. I spoke at Parliament House to the Minister of Health, Peter Dutton, several other MPs and members of the CF community:
Hi everyone, my name is Genevieve Handley and I have been extremely fortunate to have been on Kalydeco for almost 2 years, through the Vertex compassionate program.
Before I started Kalydeco my health was hugely unstable and my lung function was 41%. To put this in perspective 30% is commonly when transplant is discussed.
I struggled to keep up with work and uni, and had to spend up to 2 hours a day on exhausting, never ending, treatments.
For 20 years I had about 3 hospital admissions a year. This meant I had over 60 hospital admissions, equalling more than 3 years of my life in hospital.
Amazingly, Kalydeco has allowed me to stay out of hospital for almost two years now. This is a 20 year record for me. For the first time in my life my health is stable and my lung function is 70%.
I now spend less than 5 minutes on treatment per day, saving many hours per week. I am back at uni and planning to start working soon.
Kalydeco has allowed me to do things that I never thought I would be able to do, like visiting Machu Picchu, at a very high altitude, with no breathing issues whatsoever. Even recently, I have had some non CF health issues, but thanks to Kalydeco, my lungs have been stable. Previously, I would be in hospital, probably on oxygen, potentially not recovering.
Before Kalydeco I rarely thought further ahead than 5 years, in fact I rarely even committed to events for the weekend, as I did not know if I would be well.
Kalydeco has changed my life expectancy significantly. I can now stop worrying that a transplant, or a life threatening chest infection is around the corner and instead think about having children, my career, I even need to think about superannuation!
Having experienced what Kalydeco can do first hand, I know exactly how important it is that the 200 patients in Australia access Kalydeco now. Every day the decision is delayed, people are getting sicker. This is urgent, I have lost too many friends to CF, most in their 20s.
Peter Dutton and Members of Parliament, our lives are in your hands. Without Kalydeco we will die prematurely. Currently 90% of eligible patients in the world outside Australia have access to Kalydeco. Kalydeco is available in the US, UK and majority of Europe. Yet, Australia, a country which prides itself in being one of the most developed in the world, has not approved Kalydeco. We need you to make a difference, please do everything you can to help the 200 Australians to access Kalydeco.
Some photos from Wednesday:
Naomi, Faye, Rachelle, myself and Leah, five of the many passionate people behind #YesToKalydeco
Great turn out from the Cystic Fibrosis community at the morning tea event in Canberra
Presenting my speech at Parliament house. To my left is the Health Minister, MP Peter Dutton and Senator Anne Urquhart, to my right are MP Ken Wyatt, Mitch Messer, CFA President & Michelle Skinner, CFA CEO. I would like to sincerely thank Senator Urquhart & MP Ken Wyatt for hosting the events, as well as Senator Urquhart for organising for me to give a speech at the cocktail event. It is great to see politicians supporting Cystic Fibrosis and #YesToKalydeco.
There are 200 people in Australia waiting for Kalydeco to be approved. Right now Kalydeco is going through the approval process in Australia, and we have until June 12 to show our support for this medication. We need as many people as possible to submit comments to the PBAC, explaining what Cystic Fibrosis means to you and how a medication like Kalydeco could make a significant difference to people with CF.
Anyone can submit comments – people who are on Kalydeco, people who are waiting for Kalydeco or other medications (anyone in the CF community can submit comments), families, friends, work colleagues, medical professionals, even people from overseas. Please forward this to anyone and everyone, we need as many comments as possible!
There is an online form with five questions, where you can discuss:
- What medicines CF patients take and how well they work
- The benefits of Kalydeco for you or others with CF
- How you think this medicine will affect the lives of patients and their families or carers
- Other benefits from having Kalydeco reimbursed (eg fewer hospital visits, less time off work)
Examples of comments:
- You can discuss specifics about your health, such as I get puffed out walking up a flight of stairs or spend 2 hours on treatment per day. If you are on Kalydeco you can mention any changes.
- You can also mention how much time you spend in hospital and how much you can attend school, uni or work, such as I spend 12 weeks in hospital a year and can only work 1 day a week, but I hope to work more once I’m on Kalydeco. If you are on Kalydeco you can mention any changes.
- You can also mention your future goals and how these would change with Kalydeco, such as having a family, a career, travelling or growing old with grey hair! Given that it may be going through the life saving drugs program, it is important to mention the realities of CF and what Kalydeco means in terms of life expectancy.
You must fill in the form by 12 June.
The ‘Medicine to which your submission relates’ is: KALYDECO
The ‘Date of the PBAC meeting’ is: JULY 2013
Personal details will remain confidential.
Background information about Kalydeco:
For those that do not have G551D, Kalydeco / VX809 trials will be occurring in Australia soon, so the approval of this medication is paving the way for future medications. I have added a list of mutations that are involved in current trials here:
This time last year all I wanted for my birthday was Kalydeco.
Each year my birthday wish has been for a CF cure. Last year was the first time that it felt close, frustratingly close as I knew it would be a while before it was approved in Australia. Luckily the named patient program meant I was able to start Kalydeco in September. The change has been astonishing, my lung function has improved 30%, now my highest in 3 years, I am at my highest weight ever, and my sweat test is now below the diagnostic point for Cystic Fibrosis.
So what could I possibly want this birthday?
1. Fast approval/listing in Australia so the 200 people with G551D can access Kalydeco.
2. Fast research for everyone with F508del and other rare mutations, so we can all have a medication like Kalydeco.
3. Someone that can do my uni assignments for me so I can relax on my birthday 😉
Photo from my first day of Kalydeco. It was a beautiful sunny day in Adelaide, sitting outside a cafe at the RAH. I dont think my smile could get much bigger actually!
I’ve decided to write a blog so that anyone who is interested can follow my progress with Kalydeco. I was approved on to the Named Patient Program in Australia last week, and hopefully will start Kalydeco later this week.
2012 has not been a great year for me in terms of my health. In December 2011 I was travelling in Vietnam when I reacted to pantoprazole and developed acute kidney failure, with a creatinine of 460. Whilst my creatinine and kidneys are now normal, my reflux has been much worse as I cannot take that class of reflux medications (proton pump inhibitors). I already had a nissen fundoplication at the age of 12, so it looks like there are limited current surgical options.
My lung function has been lower this year, so I have been having IV antibiotics more frequently, about every 3 months. I have had much less energy this year, I cant remember the last time I woke up and didnt feel really tired. I have been struggling to keep up with part time uni and work and have had to decrease my commitments recently. I have been coughing overnight and in the morning much more than usual. So I am looking forward to any possible improvement with Kalydeco!
Hopefully it will arrive this week, I dont think it will seem real until it is here and probably not until I hopefully see an improvement!
Genes: G551D and F508del, diagnosed at age 3.5
Sweat Test: 102 (110 when diagnosed in 1989)
FEV1: 41% (90-100 as a child, 85% in 2003, 75% 2005-2010, 65% 2011)
Chest: Extensive bronchiectasis according to CT, pseudomonas since age 3 (nothing besides pseudomonas cultured over the last 15 years)
Weight: 55.6kg (55-60 over the last 10 years)
Height: 166.7cm (my BMI of 20.0 is reasonable for someone with CF. I had overnight feeds from age 12-17 so that helped me get to a normal height & weight)
Current Meds: Creon Forte, Azithromycin, Abdeck (vitamin), Ranitidine (reflux), Pulmozyme, Hypertonic Saline, inhaled TOBI as needed, intravenous antibiotics every 3-4 months.