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U.S. Department of Health and Human Services

Drugs

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Roadmap to Patient-Focused Outcome Measurement in Clinical Trials (text version)

(PDF version - 463KB) 

Step One: Understanding the disease or Condition

A. Natural history of the disease or condition

  • Onset/Duration/Resolution
  • Diagnosis
  • Pathophysiology
  • Range of manifestations

B. Patient subpopulations

  • By severity
  • By onset
  • By comorbidities

C. Healthcare environment

  • Treatment alternatives
  • Clinical care standards
  • Healthcare system perspective

D. Patient/caregiver perspectives

  • Definition of treatment benefit
  • Benefit-risk tradeoffs
  • Impact of disease


Step Two:
Conceptualizing the Treatment Benefit

A. Identify concept(s) of interest (CoI) for meaningful treatment benefit:

  • Survival
  • Feelings
  • Functioning

B. Define the context of use (CoU) for clinical trial:

  • Disease/Condition entry criteria
  • Clinical trial design
  • Statistical analysis plan

C. Select clinical outcome assessment (COA) type:

  • PRO
  • ObsRO
  • ClinRO
  • Performance Outcome (motor, sensory, cognition)


Step Three:
Selecting/Developing the Outcome Measure

A. Search for existing COA measuring COI in COU:

  • Measure exists
  • Measure exists but needs to be modified
  • No measure exists
  • Measure under development

B. Begin COA development

  • Document content validity (qualitative or mixed methods research)
  • Evaluate cross-sectional measurement properties (reliability and construct validity)
  • Create a user manual
  • Consider submitting to FDA for COA qualification for use in exploratory studies

C. Complete COA development:

  • Document longitudinal measurement properties (construct validity, ability to detect change)
  • Document guidelines for interpretation of treatment benefit and relationship to claim
  • Update the user manual
  • Submit to FDA for COA qualification as effectiveness endpoint to support claims