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Labeling claims for patient-reported outcomes (A regulatory perspective)

This presentation from the FDA/Industry Workshop (Washington, DC, September 16, 2005) outlines essential strategies for integrating patient-reported outcomes (PRO) into clinical development programs. Key topics addressed include the selection and validation of PRO instruments, their role in measuring treatment effects from the patient's perspective, and the significance of clinically meaningful differences. The importance of documentation, trial design, and adherence to regulatory guidelines is emphasized, aiming to ensure that labeling claims are supported by reliable and valid evidence.

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Labeling claims for patient-reported outcomes (A regulatory perspective)

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  1. Labeling claims for patient-reported outcomes(A regulatory perspective) FDA/Industry Workshop Washington, DC September 16, 2005 Lisa A. Kammerman, Ph.D. Division of Biometrics 2 Center for Drug Evaluation and Research

  2. Disclaimer Views expressed in this presentation are those of the speaker and not, necessarily, of the Food and Drug Administration.

  3. Introduction • Start early in development program • Instrument selection • Role of outcome in clinical study • Issues to address • Development and validation • Clinically meaningful difference • Implementation

  4. Introduction • Trial Design • Conduct of clinical study • FDA review • Instrument • Study results • And then …. Labeling Claims

  5. To support a PRO claim • Reliable and valid instrument • Instrument measures concepts claimed • Instrument measures impact of an intervention • Well-controlled investigations

  6. Early in product development • Identify endpoints • Identify desired claims • Discuss with FDA

  7. Selection of PRO Instrument • Measures the claimed treatment benefit from the patient’s perspective • Measures both the positive and negative benefits of the intervention

  8. Selection of PRO Instrument • Specific to the intended population • Specific to the characteristics of the conditions or disease treated

  9. Selection of PRO Instrument • Three possible choices • Existing instrument • Modification of existing instrument • New instrument

  10. Document, document, document! • Development of instrument • Patient involvement • Population • Goals • Validation of instrument • Format, wording • Cultural issues, multinational studies • Drug intervention studies

  11. Document, document, document! • Concepts: what will be measuredExamples: • Pain severity • Psychological function • Physical function

  12. Document, document, document! • Domain: Q conceptExamples: • PFSF: Desire, Responsiveness, Disinterest • Physical function: symptom improvement, physical abilities, ADL

  13. Document, document, document! • Clinically meaningful difference • Prospective plan • Patient involvement • Planned use in clinical study • Primary evidence for efficacy • “Value-added” • Intended population

  14. Document, document, document! • Implementation • Training and instructions • Format

  15. Trial Design Issues • Prospectively defined • Blinding • Use of instrument • Standardized instructions • Standardized training • Training and blinding of interviewers • Recall to baseline

  16. Trial Design Issues • Data capture • Paper • Electronic

  17. Role of PRO as an endpoint • Primary evidence of efficacy • “Value-added” • Pre-specify • Report findings, regardless of statistical significance and direction

  18. Analysis Plan • Missing data • Entire instruments • Domains • Items • Visits • Multiple endpoints

  19. And finally … Labeling! • Well-controlled studies • Instrument reliably and validly measures the concepts claimed • Convey both positive and negative effects • Specific to the concept measured

  20. Summary • Start early in clinical development program • Talk to FDA review divisions • Document, document, document • Development of instrument • Validation of instrument • Clinically meaningful difference • Implementation

  21. Summary • Study Design • Blinding • Use of instrument • Prospectively defined • Analysis plan

  22. Summary • Labeling • Conveys both negative and positive effects • Specificity to concepts measured

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