2006D-0044
  

  
 Guidance for Industry on Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims

  


  

  

  


  

  


  

  


  

  
FDA Comment Number : 
  

  
EC3
  



  

  
Submitter : 
  

  
Dr. William Riley
  

  
Date & Time: 
  

  
04/07/2006 05:04:46
  



  

  
Organization : 
  

  
NIH
  



  

  
Category : 
  

  
Federal Government
  



  

  
Issue Areas/Comments 
  


  

  


  

  
GENERAL
  


  

  



  

  
GENERAL
  


  

  



  

  
 Lines 302-313 of the FDA draft guidance, provided only as an example of selecting items that measure actual conditions, suggests that performance items (how often did you walk up a flight of stairs in the past week?) are more valid than capability items (can you walk up a flight of stairs?).  Performance items are inherently no more or less valid than capability items, but are instead different constructs.  Capability is a necessary but not sufficient condition for performance.  Performance items can be confounded by motivation (e.g. too depressed to walk up a flight of stairs) or opportunity (e.g. no stairs in the house).  Whether measuring capability or performance, the critical issue is that the items or scales have been validated (e.g. relationship to a test of walking up a flight of stairs for capability; relationship to naturalistic observations of the subject walking up a flight of stairs for performance).  Either construct could be used as the basis for a claim if the measure is valid.



I suggest that this section be deleted from the guidance.  If not, then retain lines 302 and 303. If an example is needed, then I suggest revising the subsequent lines (304-308) as follows:

"For example, asessment of a patient's capability to perform daily activities should be based on items that reflect actual capability and minimize the opportunity to respond based on desired or optimal capability.  Assessment of performance of daily activities should avoid hypothetical scenarios in which the patient has not had the opportunity to perform the activity."



I should note that I am a member of the NIH PROMIS Steering Committee. PROMIS(Patient Reported Outcomes Measurement Information System) has been struggling with capability vs. performance measures of physical functioning and will be evaluating differences between capability and performance responses during this project.  It would quite confusing for researchers if the PROMIS item bank included capability items, but the FDA included guidance language indicating a clear preference for performance items over capability items.  Ultimately, the issue is not capability vs. performance items but whether or not these items are valid measures of their underlying constructs.



Other than this particular concern, I believe that the draft document provides clear and detailed guidance on PROs, and has the potential to greatly improve PROs in future clinical trials.