A couple of weeks ago, the U.S. Food and Drug Administration (FDA) announced that the final version [PDF link] of its February 2006 draft guidance on patient-reported outcome (PRO) measures is now available. The final document explains the design and evaluation of PRO instruments, as well as clinical trial design and data analysis considerations when using PRO measures.
In some aspects, the final guidance tracks the draft. For instance, both versions define the term "claim" ("A statement of treatment benefit."). In addition, both address the development and assessment of PRO instruments, as well as the design of clinical trials and analysis of data involving PRO instruments.
Consistent with FDA's general preference for sponsors to discuss clinical development issues early in the regulatory review period, the final guidance retains the recommendation in the draft that sponsors consider whether the use of PRO instruments may be warranted early in the product development process in order to ensure that all applicable regulatory expectations with respect to these instruments may be addressed.
But, as outlined by Sidley Austin, the final guidance differs from the draft in several ways:
- It emphasizes that sponsors should define the "role a PRO endpoint is intended to play in the clinical trial (i.e., a primary, key secondary, or exploratory endpoint)" before a PRO instrument is developed.
- It encourages sponsors to link potential labeling claims to specific clinical trials designed to assess the planned concepts by using a target product profile.
- It emphasizes to a greater extent the concept of content validity, or "the extent to which the instrument measures the concept of interest," and states that content validity must be established "before other measurement properties are validated."
- It does not mention any specific analytical methods used to account for missing data.
- It includes a description of challenges related to developing PRO instruments for multiple cultures or languages.
For more information on FDA's final guidance, check out FDA Law Blog's good overview and DIA's upcoming webinar.
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