When should alternative outcome measures be explored?

• Cannot extrapolate efficacy from adults

• Studies with clinical outcomes necessary

• Impractical to measure the same clinical endpoint(s) as in trials in adults, older pediatric patients;

  • requires cooperation:FEV1
  • requires understanding and verbal:VAS for pain

• Consider:

  • modification of adult efficacy measures, new efficacy measures, identify and validate potential surrogate markers, PK/PD relationships

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