Tuesday, 26 August 2014

The Australian Kalydeco Affair

  1. 1.
    the unjust or prejudicial treatment of different categories of people.

On Thursday 31st July 2014 the Department of Health (DOH) convened with Australian CF Clinicians and Cystic Fibrosis Australia, they met as stakeholders regarding the listing of Kalydeco on the PBS...
Patients "specifically excluded" by the proposed criteria:
"Whilst patients with FEV1 of less than 40% of predicted may also benefit from treatment with  ivacaftor, the extent of the benefit in comparison with standard care is unknown because the main randomised clinical trials did not enrol patients in this category of severity. All stakeholders agreed that data about these patients would become available *over time and therefore, they could be considered for subsidy in the future – that is they do not currently qualify for subsidy based on the evidence presented by the company to the PBAC in seeking the listing of ivacaftor on the PBS. Patients previously treated with ivacaftor would be grandfathered on to the PBS-subsidised treatment, subject to meeting the PBS eligibility criteria described above." - Excerpt of the proposed PBAC/DOH  prescribing criteria.

"Over time" . . . 

Over time . . . these words (among others) linger weeks after I first laid eyes on this report. I have waited to write about the situation to give myself 'time' to come down from a boil to a simmer. The concept of time for people with lung functions below 40% is vastly different from those above 40%. These patients count the months, the days, the nights, the hours, the minutes ... time is not a 'luxury' they have. What time are the DOH exactly referring to?. Even more disturbingly "all stakeholders agreed" ... ?!!

For the record: I am a stakeholder and I object in the strongest possible way to this inhumane criteria.

To add to this inhumane proposal is the DOH's hair splitting rationale for excluding advanced CF patients; "no clinical data" - an excuse I find utterly and completely unacceptable! True, there have not been any clinical trials conducted with patients in this category, however, there is PLENTY of clinical data if the officials bothered to gather and look at it. Vertex's compassionate program which was rolled out after the FDA first approved kalydeco years ago, has all the evidence required to make compelling clinical proof. To not officially acknowledge this is unethical.

Give me a break, call a spade a spade: It's all about the money - not the lack of clinical data!!

Whilst the majority of the report will benefit most eligible Australians who are waiting for kalydeco, it blatantly discriminates against the most vulnerable of the CF community. As officials try to massage the message to our community that overall the proposed criteria is positive and there aren't many people that will be affected right now by this particular (brutal) part of it. Cystic Fibrosis Australia described this meeting as a "success" in their latest kalydeco update. To which I beg to differ and am aghast that the CF advocates are not up in arms defending the rights of the patients who will be "specifically excluded". Maybe in some kind of parallel universe neglecting the very vulnerable is a "success", but not in the world I live in. 

The obvious truth is this preposterous criteria is based on money saving measures, not medicine. Certainly not putting these patients interest/health first! I believe it's wrong, cruel, brutal, callous, unethical, unjust, inhumane ... you get the gist.
Anyone who thinks this is 'okay' or who tries to rationalise that in this instance that there is 'not much hope' for these patients, in my opinion are falling into a mindset of a society being marinated by the eugenics mentality ...and there is nothing more terrifying than being the patient on the receiving end of that particular mentality.

This debacle calls into question the very value of life, or more importantly how Australia values the lives of the most vulnerable in our community. Why is the life of the patient whose FEV1 at 35% on kalydeco worth less than the patient with 130% FEV1? Or 10- 15% for that matter? Just because they have not climbed over the magical '40%' doesn't warrant them to be denied the right to kalydeco.

Some may say, 'well in Australia we have double lung transplants for advanced CF patients'... In terms of a patient waiting for a double lung transplant, kalydeco can enable them to be as strong as possible for this major operation, help their body to absorb as much nutrition from their food as they can, to clear their lungs as much as possible, which could have a very positive effect on mortality rates on the waiting list for transplants. And to state the blatantly obvious, if I had the choice, I would rather take two pills a day than have my organs transplanted + all the other complications that go along with transplants. I know people overseas who have come off the transplant list thanks to kalydeco, indeed, kalydeco saved myself from signing up.
For those who are not eligible for a transplant due to superbug infections for example (i.e. Mycobacterium abscessus), kalydeco gives them the gift of time. By extending their life and bring a quality of life better than they have known in recent years, precious time with their loved ones... There are many other ways to weigh up whether a treatment is helping a patient; weight, energy, appetite, sleep, pain, mental disposition, bone density, blood levels, sweat chloride test, sputum production, cough, infection levels, oxygen saturation levels etc etc etc. These patients deserve a chance, just like their peers in 18 other developed countries. As Birdy sings in her song "we know full well there's just time" ... this is true for every living person - these patients lives, their time, is just as important as anyone else's!

~ A note to the patients who may be affected by this criteria ~
 I truly hope that this proposed criteria doesn't make you lose heart for your own situation ... I remember when I was very unwell this sort of news would have tipped me over the edge! So I hope that you are staying hopeful and strong because you have every reason to be. Please don't give up fighting for your life and fighting for access to kalydeco. I will continue to fight this criteria and I hope you will fight it with me! Whether you carry G551D or any of the other gating mutations or if you carry DDF508 (because the PBAC have stated this criteria is a precedent for pipeline meds, referring to the Lumacaftor + Kalydeco combo), we can change proposal this if enough people object, we need to fight this together!! I have been in the under 40% category many times before in my life, I know how hard life is - I also know with the right support things can get better, even more so how important kalydeco is when your very unwell! It is worth fighting for ... Please please, write to the officials or ask your loved ones to. Let them know that this is not okay! I mean this is crazy, no other country has such a ridiculous criteria - Australia prides itself on a so called "world class" health care system, well they've dropped the ball on this, but lets just call this proposal a bit of a blip that needs ironing out! We're stakeholders in this, potentially all CF patients in Australia could be affected by this criteria in the long run, let's make a stand and say NO! 
We're not ready to be angels yet and I will never give you up. 

"Don't give me up
Don't give...
Me up" 

Birdy - Not About Angels 

Links to reports:
1) Update on the PBAC review of KALYDECO™(ivacaftor) in Australi

2) CFA Secures A Meeting With Vertex