Transparency: Should CF-care centers ”open their books” and publish treatment outcomes? PRO

1. Hamburg 2010 - Cystic Fibrosis Europe Symposium Transparency: Should CF-care centers ”open their books” and publish treatment outcomes? PRO by Erik Wendel, Danish Cystic Fibrosis Association

2. Ref. Jim Littlewood, CF Trust , UK

3. Why Data Collection and Publishing • A tool for doctors: • Obtain information to keep on record • Evaluate treatment result • Make new, or change present treatment methods/strategies • Pass information to others • Pass ways of good practice to other – avoid pitfalls • Setting treatment goals • Transparency • Stand up for what You do, and what You belive in • A tool for patients: • Quality improvement • Access to information about other treatment strategies • Quicker access to new/other treatments

4. Ref. Michael P. Boyle, NACFC 2007

6. http://www.cff.org/CCNP/CareCenterReporting/index.cfm?CCaction=PerformanceSummary&IDCC=84http://www.cff.org/CCNP/CareCenterReporting/index.cfm?CCaction=PerformanceSummary&IDCC=84

7. http://www.cff.org/CCNP/CareCenterReporting/index.cfm?CCaction=PerformanceSummary&IDCC=6http://www.cff.org/CCNP/CareCenterReporting/index.cfm?CCaction=PerformanceSummary&IDCC=6

9. Open data must come with open CF Care Center treatment standards (What they do, how they do it )

11. Open data - Where are we • The US have opened the data door • EU and US doctors have agreed on methods, data collection and what to publish • Europe will have to follow with open data too • Patient organisation can speed up this process • We have passed the Point of No Return

12. Why Open Data - The Danish Story • 450 patients • 2 CF Care Centers Rigshospitalet (RH - East Denmark) established 1967 • 300 pts (100 children, 200 adults) • Registration of patient data since 1967 • Skejby (SKS - West Denmark) established 1990 • App. 150 pts, (80 children, 70 adults) • No official registration before 2000 • Year 2001: Danish Registry (RH + SKS) • Year 2007: Published data - Country vs. Center

13. Danish patients death/TX 2000-2006 __________________________________________________________________________________________________Year 2000-2006Age ave. Age spread__________________________________________________________________________________________________2006 (19 patients) 28,21 yrs 15 - 38 yrs 2005 (9 patients) 26,44 ysr 11 - 42 yrs 2004 (4 patients) 18,50 yrs 09 - 18 yrs 2003 (6 patients) 34,50 yrs 22 - 52 yrs 2002 (9 patients) 24,33 yrs 11 - 35 yrs 2001 (8 patients) 30,38 yrs 22 - 45 yrs 2000 (9 patients) 23,44 yrs 10 - 34 yrs__________________________________________________________________________________________________64 patients Age at death with out transplantationAge at transplantationCFF 2007

15. Why Open Data - The Danish Story Investigation of more detailed data needed

17. Copenhagen (east) vs Skejby (vest) Z-score BMI, children born after 01.01.1990, patients del508 homozygotes

18. Copenhagen (east) vs Skejby (vest) Lung function FEV1 of predicted, children born after 01.01.1990, patients del508 homozygotes

19. DK Reactions to 2005 Data Outcomes Data are not different Data are different

20. Typical reactions to Data Outcomes Stage 1: The data arewrong Stage 2 The data are right, but it’s not a problem Stage 3 The data are right; It is a problem; But it’s NOT my problem Stage 4 I accept the burden of improvement

21. What data to go for? • Overall data on FEV1 and BMI, children and adults, is a good start. • BUT most CF patients die from chronic lung infection with Pseudomonas, Burkholderia, Achromobacter etc. • THEREFORE data on how our CF Care Centres handle and treat these infections, and the clinical output they get, IS IMPORTANT AND URGENT FOR PATIENTS

22. NACFConference 2008

23. NACFConference 2008

24. Christine Rønne Hansen, 2009

25. Christine Rønne Hansen, 2010

26. Christine Rønne Hansen, 2010

27. Factors which affect the outcome of CF • CF mutations + ’modifier genes’. • all mutations demand treament when lung infection is present • Lung infection (main cause of death in CF) • Infection type (pseudomonas, burkholderia, achromobacter etc.) • Treatment • Age when infection debut- Individuel reaction to infection (immunrespons) • Individuel reaction to treatment (allergy)- Not possible to predict individual treatment effect or output • The individual approch – Living with CF- Acceptance - Understand CF as a disease and it’s development - Capabillty of action • Social factors - country vs city (zip codes), education, family situation

28. PRO - Take Home Messages • Most patients do not wish to change CF Care Center. • Patients just want their doctors to give them access to the treatments with best clinical output. • For patients, it’s not important by whom a treatment has been developed, as long as it works. • Open data and open treatment standards bring the ’best treatments’ to patients faster. • Open data and open treatment standards may expose the practice of some doctors. It may hurt doctors egos and personal feelings, • BUT - The patients always come first. • We pay the ultimate price for bad choices - with our lives.