2006D-0044

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

FDA Comment Number :

EC10

Submitter :

Dr. David Cella

Date & Time:

04/14/2006 12:04:55

Organization :

Evanston Northwestern Healthcare and Northwestern

Category :

Academia

Issue Areas/Comments

GENERAL

Dear Colleagues,

I read your draft guidance with great interest. After working in this field for 20 years, I am very happy to see that attention to what the patient has to say about health care is getting its due. Thank you for contributing to the science of getting the patient's voice into the evaluation of drug and device efficacy. I have participated in writing some longer and more detailed responses to the draft guidance. Rather than repeat many of the same themes in those responses you will get from organizations with which i am affiliated (e.g., ISOQOL, ISPOR, FACIT.org), I will be VERY brief and make two requests in this comment.

1) Please harmonize Section IV with Sections V and VI. Section VI is twice as long as Sections V and VI combined, and yet Sections V and VI cover what most HRQL experts consider to be the more substantive issues in the field today. More important than page length itself, Section IV is often worded rather harshly and unilaterally, as if there is a "right" way to develop and evaluate an instrument. Most instrument developers and evaluators do not view the field this way, and recognize that there is precious little evidence base for any approach over another. In such a climate, tolerance of well-defended and justified methods seems more appropriate than directives espousing unproven methods. From this perspective, we can collectively encourage better measurement science going forward while accepting the best available today. We owe patients nothing less.

2) Please consider that the more you require that the label claim be the center AND STARTING POINT of the evaluation, the more you will be encouraging us to listen to patients only when they think like a label. I can appreciate that it is important to feel comfortable about the label being represented by the questions being asked, but would urge you to please allow patient input to point us toward what is imortant to them, and THEN attempt to give it a name. Or, at least, allow this to be an equally acceptable approach to justifying a given label claim. Sometimes flexibility in labelng is what it takes to be "all about the patient."

I hope I can be helpful in stimulating and promoting even better measurement science going forward, so that although we may be inclined to accept today's measures today, we will also build better ones for tomorrow (such as PROMIS and other second generation approaches).

sincerely yours,

david cella