Transparency: Should CF-care centers”open their books” and

publish treatment outcomes?

PRO

byErik Wendel, Danish Cystic Fibrosis Association

Hamburg 2010 - Cystic Fibrosis Europe Symposium

Ref. Jim Littlewood, CF Trust , UK

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

Why Data Collection and Publishing

Ref. Michael P. Boyle, NACFC 2007

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

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

Open data must come withopen CF Care Centertreatment standards

(What they do, how they do it )

• 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

Open data - Where are we

• 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

Why Open Data - The Danish Story

__________________________________________________________________________________________________

Year 2000-2006 Age ave. Age spread__________________________________________________________________________________________________

2006 (19 patients) 28,21 yrs 15 - 38 yrs2005 (9 patients) 26,44 ysr 11 - 42 yrs 2004 (4 patients) 18,50 yrs 09 - 18 yrs2003 (6 patients) 34,50 yrs 22 - 52 yrs2002 (9 patients) 24,33 yrs 11 - 35 yrs2001 (8 patients) 30,38 yrs 22 - 45 yrs2000 (9 patients) 23,44 yrs 10 - 34 yrs__________________________________________________________________________________________________

64 patientsAge at death with out transplantationAge at transplantation CFF 2007

Danish patients death/TX 2000-2006

2006                    Sex Center Born TX † -TX Age ∆ SKS ∆ RH Notes

1 M SKS 1972 TX   34      2 M SKS 1973 TX   33      3 K SKS 1973 TX   33      4 M SKS 1978 TX   28      5 M SKS 1978 TX   28      6 K SKS 1979   † 27      7 K SKS 1981 TX   25      8 K SKS 1981 TX   25      9 K SKS 1983   † 23      

10 K SKS 1985 TX   21      11 M SKS 1985   † 21      12 K SKS 1986 TX   20      

13 K SKS 1991   † 15 25,61   13/150 = 8,67%                   

1 M RH 1968 TX   38      2 M RH 1971 TX   35      3 M RH 1972 TX   34      4 K RH 1972 TX   34      5 M RH 1974   † 32      

6 M RH 1976 TX   30   33,83 6/300 = 2%7 M RH 1987   † 19     Car accident                   

2005                    Sex Center Born TX † -TX Age ∆ SKS ∆ RH Notes

1 K SKS 1964 TX   41      2 K SKS 1973   † 32      3 K SKS 1980   † 25      4 M SKS 1983 TX   22      5 M SKS 1985 TX   20      6 K SKS 1994   † 11 25,17   6/150=4%                   

1 M RH 1963 TX   42      2 K RH 1979 TX   26      3 K RH 1986 TX   19   29,00 3/300=1%

Investigation of more detailed data needed

Why Open Data - The Danish Story

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

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

Data are not different

Data are different

DK Reactions to 2005 Data Outcomes

Stage 1: The data are wrong

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

Typical reactions to Data Outcomes

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

NACFConference 2008

NACFConference 2008

Christine Rønne Hansen, 2009

Christine Rønne Hansen, 2010

Christine Rønne Hansen, 2010

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

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.