1. MAST Safety (Domain 2) Clinical Effectiveness (Domain 3) Reinhard Prior, HIM, Berlin May 5, 2010

2. Basic principles of MAST Strong consideration of the existing literature when implementing a new trial Use of validated, and generally accepted outcomes ‘Holistic approach’ considering several domains (not only clinical effectiveness)

3. Effectiveness ”The ability to produce a desired result under real life conditions” “Pragmatic trial”

4. Efficacy ”The ability to produce a desired result under strictly controlled conditions” “Explanatory trial”

5. Which are the ‘desired results’ from a clinical point of view?

6. Generic outcomes (Not disease-specific)

7. All-cause mortality All-cause morbidity Generic Health related Qol instruments (SF-36/12, EQ-5D) Health care service utilisation

8. Number of hospitalisations Number of hospital bed days Number of visits to emergency rooms Number of visits by specialists Number of visits by GPs Number of visits by nurses

9. Disease-specific outcomes (not disease-specific) Scores, Questionnaires, HRQol- questionnaires Depression scales Objective measurements Hb A1c, cholesterol FEV1 NYHA-class, LVEF

10. Requirements for primary outcomes Must be suitable to statistical analysis Must be easy to collect When a score, must be available and validated in all local languages

11. What has already been done? Literature overview

14. 50% of studies: non randomization, no control group 8 % of studies: non randomized with control group Paré et al.

15. Paré et al Paré et al.

16. Paré et al Paré et al.

17. Paré et al

26. How can Renewing Health do it better?

27. Careful design considering the missing evidence and missing aspects (not just replication of previous research) Large scale MAST

28. P atient / population ? I ntervention ? C omparator ? O utcomes ?

29. Levels of evidence: RCT with large sample size RCT with small sample size Prospective study with non randomized control group Retrospective study with non randomized control group Cohort study Case control study Descriptive studies Case reports

30. Randomized controlled trial Quasi experimental methods (‘matched comparison’)

35. MAST A basis to facilitate an integrated evaluation of those domains that have not been addressed or have been insufficiently adressed by previous research.

36. Thank you!