Current pediatric research limits and promises
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Description of current pediatric research agenda. Introduction to translational research.
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Current pediatric research limits and promises
- 1. Pediatric Research Current state, difficulties and promises F. Cachat 1,2 1 Pediatric Department, Samaritain Regional Hospital, Vevey, 2 Départment of pediatrics, Pediatric Nephrology Unit, University Hospital, Lausanne, Switzerland
- 3. Research question Clinical Answer Research staff Research Staff recruitment / retention / mentoring Clinical question formulation Targeted population Population study generalization / minorities Study type Limitations of studies / RCT Generalization of findings Implementations Guidelines formulation Clinical research methodology
- 5. Where and how is pediatric research performed / current state Increase in RCT in the USA but also in developing countries (India, China…)
- 6. Where and how is pediatric research performed / current state RCT in major vs. small research centers
- 9. Clinical question Answer Research staff Research Staff recruitment / retention / mentoring Clinical question formulation Targeted population Population study generalization / minorities Study type Limitations of studies / RCT Generalization of findings Implementations Guidelines formulation Clinical research methodology Determinant of research Potential difficulties
- 11. Current limitations: research faculty
- 13. Solution: training in research / better mentoring Mentee or Protégé Mentor
- 14. Clinical question Answer Research staff Research Staff recruitment / retention / mentoring Clinical question formulation Targeted population Population study generalization / minorities Study type Limitations of studies / RCT Generalization of findings Implementations Guidelines formulation Clinical research methodology
- 15. Research settings / targeted population Population with condition X Study population Conclusions 1 2 1. Is the study population representative of the general population? Internal and external validity of the study 2. Are the conclusions of the study applicable to the general population? Implementation of the findings to the general population / translational research
- 18. Research settings / targeted population
- 19. Is the population representative of my patients? Is the research standart adequate?
- 22. Clinical question Answer Research staff Research Staff recruitment / retention / mentoring Clinical question formulation Targeted population Population study generalization / minorities Study types Limitations of studies / RCT Clinical research methodology Generalization of findings Implementations Guidelines formulation
- 26. Current limitation: long-term studies Treatment Placebo Treatment vs. placebo Surveillance Years (/decades) Outcome Years (/decades) RCT long-term results
- 27. Solution: cohort studies systematic reviews / meta-analysis RCT Cohort studies RCT long-term results
- 32. Solution: cohort studies systematic reviews / meta-analysis RCT conflicting results / meta-analysis
- 33. New treatment against what? Rejection of H0 One tailed test 10% Response rate 30% Response rate Small number of subjects RCT against placebo
- 34. RCT best available treatment
- 35. New treatment against what? Rejection of H0 One tailed test Non inferiority trials Equivalence trials 30% Response rate 35% Response rate Sample size considerable larger than placebo-controlled trials RCT best available treatment
- 36. Equivalence, non-inferiority and superiority trials Superiority trial Non inferiority trial
- 37. Equivalence, non-inferiority and superiority trials
- 38. Clinical question Answer Research staff Research Staff recruitment / retention / mentoring Clinical question formulation Targeted population Population study generalization / minorities Study types Limitations of studies / RCT Clinical research methodology Generalization of findings Implementations Guidelines formulation
- 39. Basic research Biomarkers (PSA, CRP…) Individual patient Community Clinical trials Evidence (Guidelines…) Translational Research Type I Translational Research Type II