Case	
  Study	
  
Impact	
  of	
  Kalydeco	
  as	
  a	
  new	
  inclusion	
  on	
  the	
  
PBS	
  list	
  and	
  what	
  i...
say you feel healthy again. Your dreams anything I set out to do in life. Whilst I potential. I’d worry that I wouldn’t get
Breakthrough	
  treatment	
  offering	
  
transforma2onal	
  benefits	
  to	
  pa2ent	
  
quality	
  and	
  extension	
  of	...
Campaign	
  Goal	
  
•  To	
  secure	
  unrestricted	
  reimbursement	
  for	
  Kalydeco•  To	
  secure	
  unrestricted	
 ...
Communica;ons	
  Objec;ves	
  
government	
  and	
  departmental	
  level	
  
•  Raise	
  awareness	
  of	
  a	
  new	
  e...
Campaign	
  Strategy	
  
–  New	
  era	
  of	
  treatment	
  op;ons	
  (e.g.	
  
Kalydeco)	
  
•  Ensure	
  messages	
  an...
The	
  Australian	
  Kalydeco	
  Journey	
  
we are
Cystic Fibrosis
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 Parker
My name is Alexandrena Parker, I am 23 years old, I have Cystic Fibrosis and I have been on Kalydeco fo...
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	
  Jul...
Media	
  Summary	
  
•  Campaign	
  commenced	
  March	
  2013	
  
– 12	
  media	
  stories	
  per	
  month	
  (March	
  2...
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	
...
in health. Kalydeco can change this daily battle immediately for those with the gene G551D. It
will target the cause rathe...
CFTR  Modulation
  
Pre-­clinical Phase  1 Phase  2 Phase  3 To  Patients
Kalydeco™  (formerly
known  as  VX-­770)
Atalure...
David Jack - 4Community - Case Study: The impact of Kalydeco as a new inclusion on the PBS list and what it means for cyst...
David Jack - 4Community - Case Study: The impact of Kalydeco as a new inclusion on the PBS list and what it means for cyst...
David Jack - 4Community - Case Study: The impact of Kalydeco as a new inclusion on the PBS list and what it means for cyst...
David Jack - 4Community - Case Study: The impact of Kalydeco as a new inclusion on the PBS list and what it means for cyst...
David Jack - 4Community - Case Study: The impact of Kalydeco as a new inclusion on the PBS list and what it means for cyst...
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David Jack - 4Community - Case Study: The impact of Kalydeco as a new inclusion on the PBS list and what it means for cystic fibrosis suffers

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David Jack delivered the presentation at 2014 Future of the PBS Summit.

The 11th annual Future of the PBS Summit marks a wonderful opportunity to review future frameworks and preferred outcomes for pharmacy regulators, pharmaceutical companies and wholesalers, practitioners, educators and consumers.

For more information about the event, please visit: http://www.informa.com.au/futurepbs14

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David Jack - 4Community - Case Study: The impact of Kalydeco as a new inclusion on the PBS list and what it means for cystic fibrosis suffers

  1. 1. 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  
  2. 2. say you feel healthy again. Your dreams anything I set out to do in life. Whilst I potential. I’d worry that I wouldn’t get
  3. 3. Breakthrough  treatment  offering   transforma2onal  benefits  to  pa2ent   quality  and  extension  of  life  
  4. 4. 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.    
  5. 5. 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  
  6. 6. 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  
  7. 7. The  Australian  Kalydeco  Journey  
  8. 8. we are Cystic Fibrosis 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.
  9. 9. Alexandrena Parker My 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 Piefke Thinking 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 French Targeted 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 Collins Kalydeco, 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 Nicol I 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 Upston Last 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!
  10. 10. Courier  Mail,  page  1,  30  May  2013  
  11. 11. Herald  Sun,  page  3,  30  May  2013  
  12. 12. The  Sydney  News,  30  May  2013   Gold  Coast  Bulle;n,  27  June  2013  
  13. 13. The  Western  Australian,  page  1,  5  July  2013   Adelaide  Adver;ser,  27  June  2013  
  14. 14. St  George  and  Sutherland  Shire   Leader,  12  July  2013   Bendigo  Adver;ser,  7July  2013  
  15. 15. The  Herald  Sun,  page  2,  15  August  2013   Pharma  Dispatch,  15  August  2013  
  16. 16. Channel  7  News,  5  July  2013   Channel  9  News,  5  July  2013   The  Project,  18  July  2013  
  17. 17. 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)  
  18. 18. The  Australian  Kalydeco  Journey  
  19. 19. The  Australian  Kalydeco  Journey  
  20. 20. The  Australian  Kalydeco  Journey  
  21. 21. 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  
  22. 22. 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 Bright I 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 McCarter I 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  
  23. 23. CFTR  Modulation   Pre-­clinical Phase  1 Phase  2 Phase  3 To  Patients Kalydeco™  (formerly known  as  VX-­770) Ataluren  (formerly known  as  PTC124) VX-­809  +  ivacaftor VX-­661  +  ivacaftor N6022 Restore  Airway Surface  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  Inhaled Powder) Levofloxacin (Inhaled) Arikace™ AeroVanc™ Nutrition   Pre-­clinical Phase  1 Phase  2 Phase  3 To  Patients AquADEKs Pancrelipase Enzyme  Products Liprotamase

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