Case  Study  

Impact  of  Kalydeco  as  a  new  inclusion  on  the  PBS  list  and  what  it  means  for  people  who  live  

with  cys;c  fibrosis    

Presented  by  David  Jack  CEO,  4Community  

Imagine you just woke up with a cold. It’s not the worst cold you’ve ever had but it’s enough to knock the edge off you. You feel congested, tight in the lungs, your nose is blocked and you feel really lethargic. Imagine you’ve also got a stomachache, you feel bloated, irritated and uncomfortable. You lay in bed feeling sorry for yourself, hoping that it will pass in a few days and you can go back to doing all the things you want to do. Now, imagine someone told you that your cold and stomachache were permanent. That it was never going away. Imagine that they told you that it actually was just the beginning and it would most likely only get worse. Imagine this is now your ‘normal’ state of being from here on in.

It’s not unbearable and you’re not in a huge amount of pain but you’re now

burdened by your body no matter what you do everyday. You wake up every morning struggling to breathe because you have so much mucus in your body and you’re tired cause you haven’t slept much because your coughing kept you up over night. You’re not hungry but you know you have to eat because you’re already underweight from the stress on your body. You feel nauseated after you eat and you’re stomach is even more upset than it was before. You force yourself to exercise in hope that it might just move the mucus from your lungs and make you feel better. Slowly your cold and stomachache gets a little worse each month. You realize that you can’t keep up with your friend’s busy social schedule and work is becoming overwhelming. Image your cold and stomach ache now requires IV antibiotics on a regular basis because

now the oral medications just don’t cut it. You’re falling behind at work because you’re constantly disrupted by the hospital admissions and you begin to loose touch with some friends who can’t cope with your ill health. Your dreams seem to be getting further away as your health begins to consume your every movement and every thought.

Imagine one day someone told you they could give you 2 little blue pills a day and all your suffering would almost instantly stop. Imagine they told you that you could go back to waking up in the morning with energy, that you could stop vomiting from coughing and you could simply breathe easily. The pills would relieve you from your stomachache and your lungs would no longer feel congested. They would make you feel MJLF�B�OFX�QFSTPO�BOE�ZPV�DPVME�mOBMMZ�

kalydeco

say you feel healthy again. Your dreams start to become a reality and you go back to living a normal, energetic and GVO�mMMFE�MJGF��:PVS�GBNJMZ�BOE�GSJFOET�can stop worrying about your declining IFBMUI�BOE�ZPV�DBO�mOBMMZ�TUBSU�MJWJOH�MJGF�the way you were meant to.

This is what happened to me. This is what happened to me the moment I started on Kalydeco. It’s not until now XIFO�*�TJU�BOE�SFnFDU�UIBU�*�BDUVBMMZ�realize how fast my life was sliding on a downward spiral. I can honestly say without a single hesitation that this drug has changed my life. I’m not angry or resentful I was born with Cystic Fibrosis because without it I wouldn’t be the person I am today. I’ve always had big plans and big dreams and I have always been determined to never let Cystic Fibrosis stand in the way of

anything I set out to do in life. Whilst I was determined and I never lost sight of my dreams the reality was that Cystic Fibrosis was pushing them further and further away.

After moving out of home at age 18 to study and pursue a career in photography, I found myself questioning my future at the beginning of 2011. I had dreams of travelling the world and becoming a renowned and respected photographer but after a rapid decline in health these dreams seemed more like a fantasy rather than reality. At age 22 I’d worry on a daily basis about whether I’d actually get to do all the things I wanted to do before I died. I’d worry that I wouldn’t get to see all the places I wanted to see because travel was too straining on my health. I’d worry that I wouldn’t get to live life to my full

potential. I’d worry that I wouldn’t get to raise a family or grow old to see my children grow up.

Since starting on Kalydeco it’s like all of these worries have lifted and I feel as though anything is possible. I’m not even really sure how to put into words the gratitude I have for all the people that have helped develop such an amazing phenomenon. Kalydeco is more than just a drug that allows me to ‘breathe easily’, it gives me the opportunity to live my life to it’s full potential, it gives me back my happiness and it gives me back my future.

ALEXANDRENA PARKER

Breakthrough  treatment  offering  transforma2onal  benefits  to  pa2ent  

quality  and  extension  of  life  

Campaign  Goal  

•  To  secure  unrestricted  reimbursement  for  Kalydeco•  To  secure  unrestricted  reimbursement  for  Kalydeco

 in  the  Cys;c  Fibrosis  Australia  as  a  valued  provider  of  advocacy,  shortest  possible  ;meframe  and  in  a  manner  that  posi;ons  

Cys;c  Fibrosis  Australia  as  a  valued  provider  of  advocacy,  research,  support  services  and  quality  improvement.    

Communica;ons  Objec;ves  

government  and  departmental  level  •  Raise  awareness  of  a  new  era  in  treatment  op;ons  that  offers  

genuine  hope  to  many  Australians  with  CF  •  Establish  stronger  engagement  with  and  advocacy  from  all  

members  of  the  CF  community  •  Establish  and  enact  an  advocacy  plaOorm  and  framework  for  CF  

issues  •  Establish  collabora;on  and  maintain  cohesion  across  CF  groups  

na;onally  •  Engage  the  ALP  and  Coali;on  to  publically  declare  (prior  to  the  

Federal  Elec;on)  support  for  the  funding  of  CF  ini;a;ves  (i.e.  Federal  Elec;on)  support  for  the  funding  of  CF  ini;a;ves  (i.e.  quality  improvement  model,  access  to  new  treatment  op;ons,  etc)  

•  Ensure  key  Federal  poli;cians,  advisers  and  bureaucrats  (plus  key  journalists)  are  aware  of  Kalydeco  and  recognise  its  importance  to  the  CF  community  the  CF  community  

Campaign  Strategy  

–  New  era  of  treatment  op;ons  (e.g.  Kalydeco)  

•  Ensure  messages  and  tone  are  posi2ve,  consistent  and  conducive  to  bipar;san  support    

•  Establish  a  ‘Cys;c  Fibrosis  G551D  /  Kalydeco  Expert  Group’  to  act  as  advisers  to  Government  

•  Harness  the  involvement  of  as  many  G551D  pa2ents  as  possible  in  advocacy  ac;vity,  notably  local,  metro  and  na;onal  media  outreach  

•  Harness  the  window  between  the  PBAC  mee;ng  and  the  Federal  Elec2on  to  secure  a  public  commitment  from  both  the  ALP  and  Coali;on  to  

reimburse  Kalydeco  …  CF  treatment  op;ons  •  Build  and  sustain  a  concerted  media  rela2ons  campaign  calling  for  Government  funding  of  new  CF  treatment  op;ons  •  Build  and  sustain  a  concerted  media  rela2ons  campaign  calling  for  

Government  funding  of  new  CF  treatment  op;ons  

The  Australian  Kalydeco  Journey  

we are Cystic Fibros i s

T R A N S F O R M I N G L I V E S . . . T H E T I M E I S N O W.

Alexandrena ParkerMy name is Alexandrena Parker, I am 23 years old, I have Cystic Fibrosis and I have been on Kalydeco for

almost 1 year. Before Kalydeco I was extremely underweight, my lung function was low 60% and rapidly

dropping, I was constantly lethargic due to the fact I was unable to sleep properly due to gastric reflux, I

suffered from depression due to my ill health, I could only work casually due to the unpredictable nature

of my health and I was constantly in and out of hospital on a monthly basis. Since starting on Kalydeco

my lung function is now 80% and increasing every day. I have gained 8kg, I have more energy than ever

before, I have finished my teaching degree and am now employed part time lecturing photography, and

I run a successful freelance photographic business and travel interstate frequently working 80 plus hours

per week. I am able to keep up with social activities with my friends and family and I have a positive

outlook on my life and future again. Kalydeco is the best thing that ever happened to me.

Scott, Leila and Flynn PiefkeThinking of how to describe what Kalydeco means to us – my mind just spins, how can we put a life

saving drug into words? Twenty months ago our world came crashing down when our gorgeous son

Flynn was diagnosed with cystic fibrosis. After reading and hearing all of the miracles Kalydeco has

done and is doing for other families then learning Flynn’s gene type, G551d, we feel like the most

fortunate family in the word – better than winning lotto!

It feels like our prayers and dreams have been answered, finally we see light at the end of the tunnel.

Kalydeco will mean the WORLD to us! Kalydeco will allow Flynn to live a long healthy happy life (just

like he deserves) and not allow Cystic Fibrosis to stand in his way.

Kate FrenchTargeted gene therapies for cystic fibrosis would greatly improve the quality of life for cystic

fibrosis sufferers such as me. Although Kalydeco would not be applicable for my gene mutation, it

is an amazing and important step forward in treating cystic fibrosis. While my cystic fibrosis is well

controlled at the moment and does not affect my life as significantly as it affects others, it means that

in the future suffers will have the same opportunities to enjoy life as I have had. I believe that all cystic

fibrosis sufferers should be given the best chance to enjoy a normal and fulfilling life and this can be

helped through such treatments as Kalydeco. Better treatments would mean less time off from my

studies from illness, hopefully less daily medications, less worry about what the future will hold and

more confidence in creating long-term plans and goals for myself.

Jodie CollinsKalydeco, Special-K, Blue Lightening or the robotic sounding VX-770 is coming. Luckily I am one that

will benefit when this wonder drug arrives here. What it will mean is being able to run and play on

the beach without coughing, or sleeping through the night without disturbing anyone. Does it mean

I now pursue my life’s dream of becoming a Veterinarian as I will still be here at the end of my degree,

rather than working as just a Veterinary Nurse, or something simple like not taking up a hospital bed

that’s needed for other CF patients? I don’t know, but the future is now and I can’t wait to live it!

Peter NicolI am a parent of a 10 year old boy that has the G551D mutation. He is extremely active, maintaining

good health by participating in soccer, basketball, playing drums and doing his daily PEP. He has only

had one hospital admission for a tune up in 2009 and his current lung function is around 90. The

thought of his lung function getting worse due to infections, causing further admissions is never far

from our minds. Knowing that there is Kalydeco that will greatly arrest this cycle to enable him to

remain healthy provides so much hope. I would much prefer to discuss his achievements in sport and

life as he grows rather than the topic of mortality.

Faye UpstonLast May I began Kalydeco. Words are not enough to describe how Kalydeco has transformed my

life! I feel as though have been liberated from death row. I have no need for IV’s, my FEV has gone

from 40% to 55%, my weight from 41kg to 56kg, and sweat test from 86 to 46. If I did not sustain

damage from 31 years of CF I would feel normal. I am living my dreams!! It means everything to me.

I have four young sons (seven, five, three and two) and a husband whom I love with my whole heart.

Before Kalydeco (despite my complete personal happiness with my family) life with CF was painful,

gruelling, unrelenting, lonely and exhausting. Now it’s peaceful, happy, energetic, and full of hope

and possibilities. I am profoundly thankful for this opportunity of time, not only that, the quality of

the time is priceless!

Courier  Mail,  page  1,  30  May  2013  

Herald  Sun,  page  3,  30  May  2013  

The  Sydney  News,  30  May  2013  

Gold  Coast  Bulle;n,  27  June  2013  

The  Western  Australian,  page  1,  5  July  2013  

Adelaide  Adver;ser,  27  June  2013  

St  George  and  Sutherland  Shire  Leader,  12  July  2013  

Bendigo  Adver;ser,  7July  2013  

The  Herald  Sun,  page  2,  15  August  2013  

Pharma  Dispatch,  15  August  2013  

Channel  7  News,  5  July  2013  

Channel  9  News,  5  July  2013  

The  Project,  18  July  2013  

Media  Summary  

•  Campaign  commenced  March  2013  – 12  media  stories  per  month  (March  2013  -­‐  April  2014)    

– 3  media  stories  per  week  (March  2013  –  April  2014)  

•  159  media  features  –  print,  TV,  radio,  online  •  Featured  in  all  States/Territories  (except  NT)  

The  Australian  Kalydeco  Journey  

The  Australian  Kalydeco  Journey  

The  Australian  Kalydeco  Journey  

Key  Issues  

1.  Pa;ent  organisa;on  role  2.  Give  pa;ent/carer/family  a  voice    3.  Manage  expecta;ons  and  inevitable  

disappointments    4.  Be  clear  about  who  the  client  is    5.  Be  agile,  ;mely  and  responsive  with  

communica;on  6.  Respect  PBAC  processes,  challenge  

appropriately  7.  Acknowledge  the  bigger  picture  

Julie and Dermot RitchieKalydeco means a new life. The daily management of cystic fibrosis is unrelenting. The daily results

are unpredictable and can be heartbreaking. It is the nature of the beast, unpredictable and cruel.

The life of those with cystic fibrosis requires daily management of a chronic illness in the hope of a

longer chance at life – managing it to the best of your ability, trying to slow down a persistent decline

in health. Kalydeco can change this daily battle immediately for those with the gene G551D. It

will target the cause rather than the symptoms. This is an extremely important breakthrough for all

cystic fibrosis sufferers, irrelevant of their gene type. It is the chance to progress towards a cure for

all sufferers of cystic fibrosis – a chance at a new life.

Simone and Max BrightI am mum to my two beautiful boys Billy and Max Bright. Maxi was born with cystic Fibrosis in 2007. Max is attending primary school and he is loving it. He is doing everything every other kid in his class is doing. As a mum it is wonderful to see. To maintain Maxi and keep his lungs healthy we do two treatments a day of nebulised treatments, chest therapy and take many medications. If he gets sick we increase treatments and medications and hope that he gets through a cold without having to end up in hospital. Last winter, we were on Antibiotics for the whole season – actually most of the year if I am truly honest. Max doesn’t complain, he doesn’t ask why. He knows to keep his lungs in good condition and stay out of hospital he has to do treatments. Understanding CF gene mutations are important and they change life expectancy and treatments for CFers. In 2012 a bi-daily tablet was approved for some CFers who have a certain gene type. It isn’t Max’s time for life changing drugs (he is Double Delta508F) but I know it will be soon. Australia has been one of the leading countries with Cystic Fibrosis treatments. We now need to keep up and give our CFers a life. A life full of breaths and 2 tablets twice a day that will allow them to stay healthy. My name is Simone Bright, I am a CF mum and we need to have these drugs today to help our kids.

Shane McCarterI have been taking Kalydeco for just over six months and it has completely changed my life. Before,

I was staring down the barrel of a lung transplant and could barely stay our hospital for more than six

weeks at a time. I had no quality of life, could do no more than walk around wishing I was healthier

than I was. Now, I’m fit, healthy and have not been to hospital since June 2012! I finally have the

confidence in my body to experience life due to knowing I don’t need to rely on hospital admissions

to keep me alive. I am really excited about what the future holds and it’s all due to this fantastic drug!

Thank  you  

CFTR  Modulation  

Pre-­clinical Phase  1 Phase  2 Phase  3 To  Patients

Kalydeco™  (formerlyknown  as  VX-­770)Ataluren  (formerlyknown  as  PTC124)

VX-­809  +  ivacaftor

VX-­661  +  ivacaftor

N6022

Restore  AirwaySurface  Liquid  

Pre-­clinical Phase  1 Phase  2 Phase  3 To  Patients

Hypertonic  Saline

Bronchitol

Mucus  Alteration  

Pre-­clinical Phase  1 Phase  2 Phase  3 To  Patients

Pulmozyme®

Anti-­Inflammatory  

Pre-­clinical Phase  1 Phase  2 Phase  3 To  Patients

Ibuprofen

Alpha  1  Anti-­Trypsin

KB001-­A

Sildenafil

Anti-­Infective  

Pre-­clinical Phase  1 Phase  2 Phase  3 To  Patients

Inhaled  Tobramycin

Azithromycin

Cayston®

TIP  (TOBI  InhaledPowder)Levofloxacin(Inhaled)

Arikace™

AeroVanc™

Nutrition  

Pre-­clinical Phase  1 Phase  2 Phase  3 To  Patients

AquADEKs

PancrelipaseEnzyme  Products

Liprotamase