Patient-reported outcome instrument selection: designing a measurement strategy

Area of Science:

• Clinical Research
• Health Outcomes Research
• Regulatory Science

Background:

• The US Food and Drug Administration (FDA) released draft guidance on using patient-reported outcomes (PROs) for labeling claims.
• A consensus writing group (Mayo/FDA) was formed to address issues in PRO measurement strategy design.
• This paper focuses on the critical aspects of designing a PRO measurement strategy.

Purpose of the Study:

• To outline the essential components for designing a PRO measurement strategy.
• To provide guidance on selecting and adapting instruments for PRO data collection.
• To discuss the validation requirements for modified or newly developed PRO instruments.

Main Methods:

• Defining a clear label claim that PRO data will support.
• Identifying relevant domains for measurement and developing a conceptual framework.
• Evaluating existing instruments and considering modifications or new instrument development.

Main Results:

• PRO measurement strategy development involves clear claim statements, domain identification, and framework creation.
• Existing instruments may require supplementation, modification, or the development of new tools.
• The extent of revalidation for PRO instruments depends on the degree of modification, ranging from cognitive testing to full validation.

Conclusions:

• A "reasonable pragmatism" approach is recommended for developing PRO measurement strategies.
• The best available measurement strategy should be utilized as evidence for labeling claims.
• This ensures the robust and appropriate use of PRO data in regulatory submissions.