1 / 21

ART-RIM Workshop National Cancer institute

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

pilarw
Download Presentation

ART-RIM Workshop National Cancer institute

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript

  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

More Related