Comments on 'Guidance for Industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support labeling Claims'.

Abstract

I have a particular interest in the FDA's guidance on patient-reported outcome (PRO measures as I specialize in the design, development and use of such measures and license them to pharmaceutical companies, research organizations, academics and clinicians for use in clinical trials, other research and routine clinical practice. My measures include: the Diabetes Treatment Satisfaction Questionnaire (DTSQ) in it's status (DTSQs) and change (DTSQc) forms and related measures for other conditions including the HIVTSQ, RTSQ, RetTSQ, GHerpTSQ, ThyTSQ, and the newly designed DTSQ-Teen and DTSQ-Parent. The DTSQs and c are fully linguistically validated in more than 60 language versions.
- the Well-being Questionnaire (e.g. W-BQ12) generic measure of well-being is psychometrically validated for a range of populations including those who have diabetes (type 1 and type 2) macular disease and growth hormone deficiency and fully linguistically validated in 25 language versions.
- the ADDQoL measure of the impact of diabetes on quality o life with related measures for other conditions including RDQoL, RETQol, MacDQoL, HDQoL, ARHDQoL, ThyDQoL, ADDQoL Teen and recently designed ADDToL Jnr (for 5-8 year olds) and ADDQoL Jnr+ for (9-12 year olds). The ADDQoL, MacQoL and RetQoL are linguistically validated in 16-25 language versions.
I welcome the FDA guidance as a much needed source of information about the standards required on PRO design, linguistic validation, psychometric validation and use and recognise that the guidance may be very useful in encouraging good practice.

I comment on issues in the order in which they first appear in the guidance and thereafter identify omissions that I ask be considered for inclusion.