1. Center-specific Outcomes 2010 Current Status Future Directions SUM07_1.ppt

2. Under the Contract, SCTOD will-  Collect data (and specimens)  ALL allogeneic HCTs with a U.S. recipient or donor  Related donor-recipient repository  Other cellular therapies  Quality of life data  Secure, efficient electronic data capture system  Analyze data  Center-specific outcomes for U.S. centers: related and unrelated donor transplants  Perform analyses of optimal size for the adult donor registry and cord blood unit inventory  Conduct and support other research using the data collected under the contract  Disseminate data  Within the Program  To the scientific and medical community  To patients, families and the public

3. Center-Specific Outcomes Analysis  Performed periodically by NMDP since 1994  Annually from 2002 - CIBMTR PhD support  Risk-adjusted analysis of one-year survival for unrelated donor transplants  Includes all transplant centers  Includes all transplant recipients over a five year interval  Adjusts for multiple known risk factors  Presents 95% confidence interval for predicted survivals at each center

4. What is the REAL goal?!  Provide an equitable, balanced, scientific performance measure and tool(s) that can be used by the profession to define and improve quality. While:  Acknowledging limitations  Avoiding misuse  Striving for continuous improvement

5. HOW TO DO IT BEST ?  Engage transplant community  ASBMT Quality Outcomes Committee  Forum on Assessing Center-specific outcomes – September 2008  Engage the public  CIBMTR Consumer Advocacy Committee  Active research program into the processes and resources that determine performance  Quality Improvement  Continue annual assessment of center- specific outcomes  Unrelated donor transplants  Add related donor transplants in 2010

6. TOWARDS 2010  Developed report of recommendations from Forum, draft plan for reports  Presented draft plan at Tandem 2009  Incorporate feedback (April 2009)  Presented draft plan to HRSA (May 2009)  Published final plan (Sept 2009)  Conduct analyses and report (July 2010)  Repeat Forum every 2 years (Sept 2010)

7. Timeline 2010  January 2010 – Begin center notification  Jan-April – Follow center submission  May – July – Build center outcomes data file  July – Close data set, finalize  Aug – Analyze data, notify centers with inadequate data for analysis  Sep 2010  Discuss analysis, report, tools for centers and others, future planning

8. Timeline 2010  Sep 2010 – Finalize analysis  Submit final report to HRSA  Oct - Nov –  Provide individual center outcomes to centers  Provide center characteristics and univariate outcome data (tools)  Collect centers’ “public” commentary  Dec or later – Post updated data on HRSA website  New framework and appearance?

9. Changes for 2011 Lessons Learned  Remind centers earlier  Copy all communication on this topic to center director  Better integration with overlapping components  Center Volume Reports  CPI trimester requirements  Proactive communication regarding forms based errors  Review data completeness and notify earlier

10. Timeline 2011  Sep/Oct 2010 – Center Volume Report 2009  Confirm HCT for 2009  Sep 2010 – CPI reports for all allogeneic HCT now active  2009 HCT data included  Jan 2011 – CPI report issued  First reminder to centers (re data 2005- 2009) for center outcomes reporting  Jan – Mar 2011 – Monthly reminders and reports on forms due

11. Timeline 2011  Apr 2011 – CPI report closes out HCT for 2009 and earlier for one year outcomes  Apr/May 2011 – Dataset assembly  Notification of center director re exclusions  Jun 2011 – Analysis  Jul 2011 – Reports and tools to centers  Aug 2011 – Collect center commentary  Sep 2011 – Report to HRSA  Nov - Dec 2011 – Post/Publish on HRSA (.gov) website  Depends upon review cycle, effort to post

12. What’s New in 2010  Inclusion of HCT data 2004-2008  UNR 2004-2008, REL 2008  New data collection instruments  TED, CRF or both  New data elements  Comorbidity – HCT-CI  Cytogenetics in AML  Distance from center  Income surrogate

13. What’s New in 2010  New modeling considerations:  Combined related vs. unrelated modeling  Larger overall numbers  Time window for analysis (3 or 5 yr)  How and when to incorporate new variables and new techniques?

14. Objectives for Forum  Review current year outcomes report  Open, transparent DISCUSSION  Process  Analysis  Reporting  Solicit input and new ideas  Risk Adjustment  Quality Improvement

15. TOPICS FOR TODAY

16. Review of Analyses  Criteria for center and recipient inclusion in the analysis  Analytic methodology  Differentiation of variables for analysis versus investigatory consideration  Overview of adjustment model and significant factors  Overview of results for centers

17. Statistical & Methodologic Issues  Do the models work similarly for related and unrelated HCT?  Can the time window of analysis be narrowed?  3 year vs. 5 year  Model building versus outcomes reporting  What is the right p-value for risk adjustment model building?  Large dataset, is 0.05 too permissive?

18. Statistical & Methodologic Issues  Are small centers and large centers equally at risk for an adverse rating?  How and when should we introduce new variables?  Sorror, Cytogenetics

19. Hot topics for discussion  Are there pediatric risk factors that should be considered?  Are the criteria for inclusion of centers appropriate  How should they be described?  Are there other variables that should be considered for investigational data collection?  Can we measure and use a proxy for “late referrals”?

20. Is one year survival still the best outcome?  Review rationale for 1 year survival  Discuss uses and limitations for 100 day mortality  Descriptive versus multivariate modeling  Resources

21. Can we adequately characterize “investigatory HCT”?  Roy Jones and Helen Heslop on behalf of the ASBMT Quality Outcome committee  Can we develop reproducible criteria?  How should the information be used?  Descriptive for each center?  Introduction into risk adjustment models?

22. Striving for performance improvement – what data can help?  Review CIBMTR plans for additional data to provide to centers  What is the value of the ‘risk score’ in the current report?  Can and should a “risk budget” equation or tool be made available (Stiff)  Will there be unintended consequences?

23. Going Public?!?  How are the data currently displayed (Murphy)  Review ideas for change in 2010  Discussion  What data is useful to the public, and is there data that may be unintentionally misleading?  How can payers use the data, and is there additional data that CIBMTR can provide?  Is there a minimum HCT number to be included on public website, as opposed to report to HRSA and center?  What should it look like?

25. Distribution of over and underperforming centers  157 Centers in analysis  14 centers under performing  1-204  21-501  Over 509  11 centers over performing  1-200  21-502  Over 509