• Like
  • Save
Current pediatric research limits and promises
Upcoming SlideShare
Loading in...5
×
 

Current pediatric research limits and promises

on

  • 832 views

Description of current pediatric research agenda. Introduction to translational research.

Description of current pediatric research agenda. Introduction to translational research.

Statistics

Views

Total Views
832
Views on SlideShare
832
Embed Views
0

Actions

Likes
0
Downloads
5
Comments
0

0 Embeds 0

No embeds

Accessibility

Categories

Upload Details

Uploaded via as Microsoft PowerPoint

Usage Rights

© All Rights Reserved

Report content

Flagged as inappropriate Flag as inappropriate
Flag as inappropriate

Select your reason for flagging this presentation as inappropriate.

Cancel
  • Full Name Full Name Comment goes here.
    Are you sure you want to
    Your message goes here
    Processing…
Post Comment
Edit your comment

    Current pediatric research limits and promises Current pediatric research limits and promises Presentation Transcript

    • 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
    • Aims
      • How research is conducted around the world
      • Current limitations to research
      • Solutions to improve research
      The challenges of research The rewards of research Finding cures / improving health delivery to the patient
    • 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
    • Where do we come from / current state
      • Numbers of pediatric trials
      • Medline:
      • - Child and Hypertension
      • - Child and RCT
      Steadily increase in the number of clinical studies involving children over the last two decades, especially RCT
    • Where and how is pediatric research performed / current state Increase in RCT in the USA but also in developing countries (India, China…)
    • Where and how is pediatric research performed / current state RCT in major vs. small research centers
    • Introduction summary 1
      • 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
      • Research concentrates in the USA and Europe
    • Introduction summary 2
      • Increasing importance of clinical research originating and/or taking place in developing countries
      • Research still concentrates in major academic centers
    • 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
    • Current limitations: research faculty
      • Research faculty recruitment and retention
    • Current limitations: research faculty
      • Mentoring
      • Career planning
      • Performance feedback
      • Professional network
      • Connectedness and acculturation to medical school
      • To improve faculty satisfaction, productivity, institutional loyalty and retention
      Solution: Faculty Development Program
    • Solution: training in research / better mentoring Mentee or Protégé Mentor
    • 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
    • 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
    • Research settings / targeted population
      • Biais of recruitment
      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 Targeted population Population study generalization / minorities Study types Limitations of studies / RCT Clinical research methodology Generalization of findings Implementations Guidelines formulation
    • Current limitations of RCT
      • ( Extremely ) long-term outcomes for most of the chronic diseases (heart failure, chronic kidney diseases, dementia, psychosis, asthma…)
      • ( Major ) discrepancies in major studies findings and ( prohibitive ) cost of major clinical studies
      • ( Extremely ) limited benefit of new drugs
      • (Poor) generalizability / implementation of RCT findings
    • RCT long-term results
    • RCT long-term results
    • Current limitation: long-term studies Treatment Placebo Treatment vs. placebo Surveillance Years (/decades) Outcome Years (/decades) RCT long-term results
    • 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 RCT against placebo
    • RCT best available treatment
    • 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
    • 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 Targeted population Population study generalization / minorities Study types Limitations of studies / RCT Clinical research methodology Generalization of findings Implementations Guidelines formulation
    • Basic research Biomarkers (PSA, CRP…) Individual patient Community Clinical trials Evidence (Guidelines…) Translational Research Type I Translational Research Type II
    • Translational research
      • 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)
    • Translational research type II
      • Implementation of RCT findings into the community
      • There is a massive lack of type II translational research
      • State of the affair:
    • Barriers to implementation
      • Unaware of the guidelines
      • Mistrust of guidelines
      • Guidelines too complicated
      • Lack of time, resources
      • Peer pressure
      • Patient’s preference
    • Conclusions (1)
      • Clinical research in pediatrics has made substantial progress over the last decades
          • Better regulation
          • Better participant protection
          • Better research methodologies / collaboration
      • Major challanges for the next years
          • Long-term studies
          • Minorities inclusions
          • Funding issues
    • Conclusions (2)
      • Agenda
          • Better and earlier training for medical students, residents and fellow
          • Better career plan for clinician-scientists
          • Better collaboration between researchers and clinicians, between pediatric and adult specialists
          • Develop and implement translational research type I and II
    • Outline
      • Where do we stand now?
        • Progression over the last decade
        • Where is pediatric research currently performed?
    • Outline
      • Where are the limitations?
        • People :
          • Faculty recruitment / motivation / training
        • Location :
          • University-based research vs. office-based and potential biais
          • Developing countries research and potential risks
        • Research subject protection :
          • Research involving minorities (pregnancy, newborn, ethnic minorities, orphan diseases) and potential risks
        • Research design :
          • Long-term studies: inherent difficulties (time lag)
          • RCT: inherent difficuties (placebo-controlled studies (best treatment vs placebo / non inferiority trials / power)
          • Implementation of basic or clinical findings (translational research)
          • Funding issues
    • Outline
      • How to improve
        • People :
          • Early student / resident / fellow involvment
          • Mentoring
        • Location :
          • Office-based research / community research / network studies
          • Top-bottom and bottom-up research
          • Regulations / subject protection
        • Research subject protection :
          • Enrollement of minorities
          • Regulations
          • Clinical research centers
        • Research design :
          • Cohort studies / systematic reviews – meta-analysis
          • Translational research type I and II