Labeling claims for patient- reported outcomes (A regulatory perspective) FDA/Industry Workshop...

Labeling claims for patient-Labeling claims for patient-reported outcomesreported outcomes

(A regulatory perspective)(A regulatory perspective)

Labeling claims for patient-Labeling claims for patient-reported outcomesreported outcomes

(A regulatory perspective)(A regulatory perspective)

FDA/Industry Workshop

Washington, DCSeptember 16, 2005

Lisa A. Kammerman, Ph.D.Division of Biometrics 2

FDA/Industry Workshop

Washington, DCSeptember 16, 2005

Lisa A. Kammerman, Ph.D.Division of Biometrics 2

Center for Drug Evaluation and ResearchCenter for Drug Evaluation and Research

2FDA/Industry WorkshopFDA/Industry WorkshopSeptember 16, 2005September 16, 2005

DisclaimerDisclaimerDisclaimerDisclaimer

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

IntroductionIntroduction

• Start early in development program– Instrument selection –Role of outcome in clinical study

• Issues to address–Development and validation–Clinically meaningful difference– Implementation

• Start early in development program– Instrument selection –Role of outcome in clinical study

• Issues to address–Development and validation–Clinically meaningful difference– Implementation

IntroductionIntroduction

• Trial Design• Conduct of clinical study• FDA review– Instrument–Study results

• And then …. Labeling Claims

• Trial Design• Conduct of clinical study• FDA review– Instrument–Study results

• And then …. Labeling Claims

To support a PRO claimTo support a PRO claimTo support a PRO claimTo support a PRO claim

• Reliable and valid instrument• Instrument measures concepts

claimed• Instrument measures impact of an

intervention• Well-controlled investigations

• Reliable and valid instrument• Instrument measures concepts

claimed• Instrument measures impact of an

intervention• Well-controlled investigations

Early in product developmentEarly in product developmentEarly in product developmentEarly in product development

• Identify endpoints

• Identify desired claims

• Discuss with FDA

• Identify endpoints

• Identify desired claims

• Discuss with FDA

Selection of PRO InstrumentSelection of PRO InstrumentSelection of PRO InstrumentSelection of PRO Instrument

• Measures the claimed treatment benefit from the patient’s perspective

• Measures both the positive and negative benefits of the intervention

• Measures the claimed treatment benefit from the patient’s perspective

• Measures both the positive and negative benefits of the intervention

• Specific to the intended population

• Specific to the characteristics of the conditions or disease treated

• Specific to the intended population

• Specific to the characteristics of the conditions or disease treated

• Three possible choices–Existing instrument–Modification of existing instrument–New instrument

Document, document, document!Document, document, document!Document, document, document!Document, document, document!

• Development of instrument–Patient involvement–Population–Goals

• Validation of instrument–Format, wording–Cultural issues, multinational

studies–Drug intervention studies

• Development of instrument–Patient involvement–Population–Goals

• Validation of instrument–Format, wording–Cultural issues, multinational

studies–Drug intervention studies

• Concepts: what will be measured

Examples:–Pain severity–Psychological function–Physical function

• Concepts: what will be measured

Examples:–Pain severity–Psychological function–Physical function

• Domain: Q concept Examples:–PFSF: Desire, Responsiveness,

Disinterest–Physical function: symptom

improvement, physical abilities, ADL

• Domain: Q concept Examples:–PFSF: Desire, Responsiveness,

Disinterest–Physical function: symptom

improvement, physical abilities, ADL

• Clinically meaningful difference–Prospective plan–Patient involvement

• Planned use in clinical study–Primary evidence for efficacy–“Value-added”

• Intended population

• Clinically meaningful difference–Prospective plan–Patient involvement

• Planned use in clinical study–Primary evidence for efficacy–“Value-added”

• Intended population

• Implementation–Training and instructions–Format

Trial Design IssuesTrial Design IssuesTrial Design IssuesTrial Design Issues

• Prospectively defined• Blinding• Use of instrument–Standardized instructions–Standardized training–Training and blinding of

interviewers–Recall to baseline

• Prospectively defined• Blinding• Use of instrument–Standardized instructions–Standardized training–Training and blinding of

interviewers–Recall to baseline

Trial Design IssuesTrial Design IssuesTrial Design IssuesTrial Design Issues

• Data capture–Paper–Electronic

Role of PRO as an endpointRole of PRO as an endpointRole of PRO as an endpointRole of PRO as an endpoint

• Primary evidence of efficacy• “Value-added”–Pre-specify–Report findings, regardless of

statistical significance and direction

• Primary evidence of efficacy• “Value-added”–Pre-specify–Report findings, regardless of

statistical significance and direction

Analysis PlanAnalysis PlanAnalysis PlanAnalysis Plan

• Missing data–Entire instruments–Domains– Items–Visits

• Multiple endpoints

• Missing data–Entire instruments–Domains– Items–Visits

• Multiple endpoints

And finally … Labeling!And finally … Labeling!And finally … Labeling!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

• Well-controlled studies• Instrument reliably and validly

measures the concepts claimed• Convey both positive and negative

effects• Specific to the concept measured

SummarySummarySummarySummary

• Start early in clinical development program

• Talk to FDA review divisions• Document, document, document–Development of instrument–Validation of instrument–Clinically meaningful difference– Implementation

• Start early in clinical development program

• Talk to FDA review divisions• Document, document, document–Development of instrument–Validation of instrument–Clinically meaningful difference– Implementation

• Study Design–Blinding–Use of instrument–Prospectively defined

• Analysis plan

• Study Design–Blinding–Use of instrument–Prospectively defined

• Analysis plan

• Labeling –Conveys both negative and

positive effects–Specificity to concepts measured

• Labeling –Conveys both negative and

positive effects–Specificity to concepts measured

Labeling claims for patient- reported outcomes (A regulatory perspective) FDA/Industry Workshop...

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