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ART-RIM Workshop National Cancer institute

ART-RIM Workshop National Cancer institute

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ART-RIM Workshop National Cancer institute

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  1. ART-RIM WorkshopNational Cancer institute Ethan Basch, MD, MSc Memorial Sloan-Kettering Cancer Center Patient-reported Outcomes (PROs) January 22, 2010

  2. Definition “A PRO is any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else.” http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM193282.pdf - Final FDA PRO Guidance (December 2009)

  3. Examples • Symptoms • Severity, frequency, interference, bother, etc. • Quality of life • Various domains • Subjective impressions of improvement/change • Treatment preferences • Satisfaction with care • Compliance with treatment

  4. Standards • Rigorous standards for development, administration, analysis, and reporting of patient-reported data have emerged, and are codified in the FDA Guidance • Technically only apply when measuring effects of treatment with the intention of making a labeling claim, but have been widely accepted beyond the regulatory setting • Nonetheless, poorly designed measures are still common in protocols and publications

  5. Scrutiny • Questions for patients should not simply be “made up” and administered at occasional or inconsistent intervals • Concepts that are best known by patients should not be reported by clinicians

  6. Considerations in Developing or Selecting a PRO Measure Measurement properties of instruments • Reliability • Test-retest • Internal consistency • Validity • Content validity (qualitative) • Construct validity (discriminant) • Ability to detect change • Recall period

  7. Considerations when Administeringa PRO Measure in a Trial Population issues • Validity, literacy, language, cognitive abilities, PS Study design issues • What concepts to measure, parsimony • Frequency and duration of administration • Method and location of administration Missing data • Most ill and most well patients? • Backup data collection methods?

  8. PROs for Measuring Adverse Symptoms • Standard approach to measuring AEs in NCI-sponsored clinical trials: CTCAE • CTCAE v4: >800 items; ~10% are “symptoms” • CTCAE items are reported by clinicians • But clinicians underestimate the frequency and severity of patient symptoms • Therefore, with clinician-only reporting, we have an incomplete picture of toxicity

  9. Patient vs. Clinician Reporting Patient-reporting Clinician-reporting

  10. Adverse Events in Current Labels • Almost half are symptoms

  11. Docetaxel Drug Label Data from “Tax 327” pivotal trial

  12. NCI contract to develop a PRO version of the CTCAE • Initiated 10/08 NCI HHSN261200800043C

  13. Mission of PRO-CTCAE Initiative • Employ rigorous scientific methods to create a system for patient self-reporting of adverse symptoms in cancer trials, which is widely accepted and used; generates useful data for investigators, regulators, clinicians and patients; and is compatible with existing adverse event reporting systems

  14. PRO-CTCAE Scope • Create PRO-CTCAE adverse symptom items • Evaluate measurement properties of items • Build electronic administration platform • Assess feasibility Nine interdisciplinary task committees assembled • Including NCI and FDA representatives

  15. 77 CTCAE Symptoms Identified

  16. Possible Attributes of Each Symptom • Frequency • Severity • Interference with usual activities • Present/Not present • Separate items for each attribute • Between 1 and 3 attribute items per symptom • Selected based on attributes included in original CTCAE items, and nature of each symptom • 122 total items representing the 77 symptoms

  17. Methodological Development • Content validity study • Cognitive interviews • Measurement properties study • Validity, reliability, sensitivity, recall • Platform “usability” study • Feasibility study

  18. Platform: Form Builder

  19. Platform: Patient Interface

  20. Platform: Study Calendar

  21. Summary • Patient self-reporting is the gold standard for symptom assessment • Guidance for developing and administering PRO instruments is available in the FDA document • The PRO-CTCAE provides a lexicon of adverse symptom items which are being developed in keeping with rigorous methodological criteria