Pediatric Research Current state, difficulties and promises F. Cachat 1,2 1 Pediatric Department, Samaritain Regional Hosp...
Aims <ul><li>How research is conducted around the world </li></ul><ul><li>Current limitations to research </li></ul><ul><l...
Research question Clinical Answer Research staff Research Staff recruitment / retention / mentoring Clinical question form...
Where do we come from / current state <ul><li>Numbers of pediatric trials </li></ul><ul><li>Medline:  </li></ul><ul><li>- ...
Where and how is pediatric research performed / current state Increase in RCT in the USA but also  in developing  countrie...
Where and how is pediatric research performed / current state RCT in major   vs. small research centers
Introduction summary 1 <ul><li>Dramatic increase in the number of RCT performed children over the last 50 years, mainly ov...
Introduction summary 2 <ul><li>Increasing importance of clinical research originating and/or taking place in developing co...
Clinical question Answer Research staff Research Staff recruitment / retention / mentoring Clinical question formulation T...
Current limitations: research faculty <ul><li>Research faculty recruitment and retention </li></ul>
Current limitations: research faculty
<ul><li>Mentoring </li></ul><ul><li>Career planning </li></ul><ul><li>Performance feedback </li></ul><ul><li>Professional ...
Solution: training in research / better mentoring Mentee or Protégé Mentor
Clinical question Answer Research staff Research Staff recruitment / retention / mentoring Clinical question formulation T...
Research settings / targeted population Population with  condition X Study population Conclusions 1 2 1. Is the study popu...
Research settings / targeted population <ul><li>Biais of recruitment </li></ul>Sexe Age Race
Research settings / targeted population
Is the population representative of my patients? Is the research standart adequate?
Clinical question Answer Research staff Research Staff recruitment / retention / mentoring Clinical question formulation T...
Current limitations of RCT <ul><li>( Extremely ) long-term outcomes for most of the chronic diseases  (heart failure, chro...
RCT long-term results
RCT long-term results
Current limitation: long-term studies Treatment Placebo Treatment vs. placebo Surveillance Years (/decades) Outcome Years ...
Solution:  cohort studies systematic reviews / meta-analysis RCT Cohort studies RCT long-term results
RCT long-term results
RCT long-term results
RCT conflicting results
RCT conflicting results
Solution: cohort studies systematic reviews / meta-analysis RCT conflicting results / meta-analysis
New treatment against what? Rejection of H0 One tailed test 10% Response rate 30% Response rate Small number of subjects R...
RCT best available treatment
New treatment against what? Rejection of H0 One tailed test Non inferiority trials Equivalence trials 30% Response rate 35...
Equivalence, non-inferiority   and superiority trials Superiority trial Non inferiority trial
Equivalence, non-inferiority   and superiority trials
Clinical question Answer Research staff Research Staff recruitment / retention / mentoring Clinical question formulation T...
Basic research Biomarkers (PSA, CRP…) Individual patient Community Clinical trials Evidence (Guidelines…) Translational Re...
Translational research <ul><li>The integration of advancements in molecular biology or other basic science to the individu...
Translational research type II <ul><li>Implementation of RCT findings into the community </li></ul><ul><li>There is a mass...
Barriers to implementation <ul><li>Unaware of the guidelines </li></ul><ul><li>Mistrust of guidelines </li></ul><ul><li>Gu...
Conclusions (1) <ul><li>Clinical research in pediatrics has made substantial progress over the last decades </li></ul><ul>...
Conclusions (2) <ul><li>Agenda  </li></ul><ul><ul><ul><li>Better and earlier training for medical students, residents and ...
Outline <ul><li>Where do we stand now? </li></ul><ul><ul><li>Progression over the last decade </li></ul></ul><ul><ul><li>W...
Outline <ul><li>Where are the limitations? </li></ul><ul><ul><li>People :  </li></ul></ul><ul><ul><ul><li>Faculty recruitm...
Outline <ul><li>How to improve </li></ul><ul><ul><li>People :  </li></ul></ul><ul><ul><ul><li>Early student / resident / f...
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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. 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
  2. 2. Aims <ul><li>How research is conducted around the world </li></ul><ul><li>Current limitations to research </li></ul><ul><li>Solutions to improve research </li></ul>The challenges of research The rewards of research Finding cures / improving health delivery to the patient
  3. 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
  4. 4. Where do we come from / current state <ul><li>Numbers of pediatric trials </li></ul><ul><li>Medline: </li></ul><ul><li>- Child and Hypertension </li></ul><ul><li>- Child and RCT </li></ul>Steadily increase in the number of clinical studies involving children over the last two decades, especially RCT
  5. 5. Where and how is pediatric research performed / current state Increase in RCT in the USA but also in developing countries (India, China…)
  6. 6. Where and how is pediatric research performed / current state RCT in major vs. small research centers
  7. 7. Introduction summary 1 <ul><li>Dramatic increase in the number of RCT performed children over the last 50 years, mainly over the last 10-20 years all over the world </li></ul><ul><li>Research concentrates in the USA and Europe </li></ul>
  8. 8. Introduction summary 2 <ul><li>Increasing importance of clinical research originating and/or taking place in developing countries </li></ul><ul><li>Research still concentrates in major academic centers </li></ul>
  9. 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
  10. 10. Current limitations: research faculty <ul><li>Research faculty recruitment and retention </li></ul>
  11. 11. Current limitations: research faculty
  12. 12. <ul><li>Mentoring </li></ul><ul><li>Career planning </li></ul><ul><li>Performance feedback </li></ul><ul><li>Professional network </li></ul><ul><li>Connectedness and acculturation to medical school </li></ul><ul><li>To improve faculty satisfaction, productivity, institutional loyalty and retention </li></ul>Solution: Faculty Development Program
  13. 13. Solution: training in research / better mentoring Mentee or Protégé Mentor
  14. 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. 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
  16. 16. Research settings / targeted population <ul><li>Biais of recruitment </li></ul>Sexe Age Race
  17. 17.
  18. 18. Research settings / targeted population
  19. 19. Is the population representative of my patients? Is the research standart adequate?
  20. 20.
  21. 21.
  22. 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
  23. 23. Current limitations of RCT <ul><li>( Extremely ) long-term outcomes for most of the chronic diseases (heart failure, chronic kidney diseases, dementia, psychosis, asthma…) </li></ul><ul><li>( Major ) discrepancies in major studies findings and ( prohibitive ) cost of major clinical studies </li></ul><ul><li>( Extremely ) limited benefit of new drugs </li></ul><ul><li>(Poor) generalizability / implementation of RCT findings </li></ul>
  24. 24. RCT long-term results
  25. 25. RCT long-term results
  26. 26. Current limitation: long-term studies Treatment Placebo Treatment vs. placebo Surveillance Years (/decades) Outcome Years (/decades) RCT long-term results
  27. 27. Solution: cohort studies systematic reviews / meta-analysis RCT Cohort studies RCT long-term results
  28. 28. RCT long-term results
  29. 29. RCT long-term results
  30. 30. RCT conflicting results
  31. 31. RCT conflicting results
  32. 32. Solution: cohort studies systematic reviews / meta-analysis RCT conflicting results / meta-analysis
  33. 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. 34. RCT best available treatment
  35. 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. 36. Equivalence, non-inferiority and superiority trials Superiority trial Non inferiority trial
  37. 37. Equivalence, non-inferiority and superiority trials
  38. 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. 39. Basic research Biomarkers (PSA, CRP…) Individual patient Community Clinical trials Evidence (Guidelines…) Translational Research Type I Translational Research Type II
  40. 40. Translational research <ul><li>The integration of advancements in molecular biology or other basic science to the individual (type I), and from the advancement of clinical trials to the community (type II) </li></ul>
  41. 41. Translational research type II <ul><li>Implementation of RCT findings into the community </li></ul><ul><li>There is a massive lack of type II translational research </li></ul><ul><li>State of the affair: </li></ul>
  42. 42.
  43. 43. Barriers to implementation <ul><li>Unaware of the guidelines </li></ul><ul><li>Mistrust of guidelines </li></ul><ul><li>Guidelines too complicated </li></ul><ul><li>Lack of time, resources </li></ul><ul><li>Peer pressure </li></ul><ul><li>Patient’s preference </li></ul>
  44. 44. Conclusions (1) <ul><li>Clinical research in pediatrics has made substantial progress over the last decades </li></ul><ul><ul><ul><li>Better regulation </li></ul></ul></ul><ul><ul><ul><li>Better participant protection </li></ul></ul></ul><ul><ul><ul><li>Better research methodologies / collaboration </li></ul></ul></ul><ul><li>Major challanges for the next years </li></ul><ul><ul><ul><li>Long-term studies </li></ul></ul></ul><ul><ul><ul><li>Minorities inclusions </li></ul></ul></ul><ul><ul><ul><li>Funding issues </li></ul></ul></ul>
  45. 45. Conclusions (2) <ul><li>Agenda </li></ul><ul><ul><ul><li>Better and earlier training for medical students, residents and fellow </li></ul></ul></ul><ul><ul><ul><li>Better career plan for clinician-scientists </li></ul></ul></ul><ul><ul><ul><li>Better collaboration between researchers and clinicians, between pediatric and adult specialists </li></ul></ul></ul><ul><ul><ul><li>Develop and implement translational research type I and II </li></ul></ul></ul>
  46. 46. Outline <ul><li>Where do we stand now? </li></ul><ul><ul><li>Progression over the last decade </li></ul></ul><ul><ul><li>Where is pediatric research currently performed? </li></ul></ul>
  47. 47. Outline <ul><li>Where are the limitations? </li></ul><ul><ul><li>People : </li></ul></ul><ul><ul><ul><li>Faculty recruitment / motivation / training </li></ul></ul></ul><ul><ul><li>Location : </li></ul></ul><ul><ul><ul><li>University-based research vs. office-based and potential biais </li></ul></ul></ul><ul><ul><ul><li>Developing countries research and potential risks </li></ul></ul></ul><ul><ul><li>Research subject protection : </li></ul></ul><ul><ul><ul><li>Research involving minorities (pregnancy, newborn, ethnic minorities, orphan diseases) and potential risks </li></ul></ul></ul><ul><ul><li>Research design : </li></ul></ul><ul><ul><ul><li>Long-term studies: inherent difficulties (time lag) </li></ul></ul></ul><ul><ul><ul><li>RCT: inherent difficuties (placebo-controlled studies (best treatment vs placebo / non inferiority trials / power) </li></ul></ul></ul><ul><ul><ul><li>Implementation of basic or clinical findings (translational research) </li></ul></ul></ul><ul><ul><ul><li>Funding issues </li></ul></ul></ul>
  48. 48. Outline <ul><li>How to improve </li></ul><ul><ul><li>People : </li></ul></ul><ul><ul><ul><li>Early student / resident / fellow involvment </li></ul></ul></ul><ul><ul><ul><li>Mentoring </li></ul></ul></ul><ul><ul><li>Location : </li></ul></ul><ul><ul><ul><li>Office-based research / community research / network studies </li></ul></ul></ul><ul><ul><ul><li>Top-bottom and bottom-up research </li></ul></ul></ul><ul><ul><ul><li>Regulations / subject protection </li></ul></ul></ul><ul><ul><li>Research subject protection : </li></ul></ul><ul><ul><ul><li>Enrollement of minorities </li></ul></ul></ul><ul><ul><ul><li>Regulations </li></ul></ul></ul><ul><ul><ul><li>Clinical research centers </li></ul></ul></ul><ul><ul><li>Research design : </li></ul></ul><ul><ul><ul><li>Cohort studies / systematic reviews – meta-analysis </li></ul></ul></ul><ul><ul><ul><li>Translational research type I and II </li></ul></ul></ul>

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