Abstract
Aims
Patient-reported outcomes (PROs) have recently gained greater credibility with regulatory
bodies aiming to standardise their use and interpretation in RCTs, thereby supporting
medicinal product submissions. For this reason, the United States (US) Food and Drug
Administration (FDA) and the European Medicines Agency (EMEA) have released guidelines.
This review paper provides an overview of the current perspectives and views on these
guidelines.
Method
To evaluate the FDA and EMEA PRO guidelines, 47 expert responses to the FDA guidance
were qualitatively reviewed. Two reviewers independently extracted data from these
letters and checked these responses to warrant consistency and agreement in the evaluation
process. A PubMed literature review was systematically examined to obtain supporting
evidence or related articles for both the guidance documents.
Results
Generally, there is agreement between regulatory authorities and the research community
on the contents of the FDA and EMEA PRO draft guidance. However, disagreements exist
on significant philosophical topics (e.g. the FDA focuses more on conceptual models
and symptoms than the EMEA) and design topics (e.g. the FDA is more restrictive on
issues of recall bias, blinding of oncology trials and degrees of psychometric validation
than researchers and the EMEA). This could influence the approval of PRO claims.
Conclusion
PRO guidance from the EMEA and FDA has been valuable, and has raised the profile and
active debate of PROs in oncology. However, our review of the current opinion shows
that there are controversial aspects of the guidance. Consequently, greater latitude
should be given to how the guidance is interpreted and applied.
Keywords
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Article info
Publication history
Published online: November 17, 2008
Accepted:
September 30,
2008
Received:
September 10,
2008
Identification
Copyright
© 2008 Elsevier Ltd. Published by Elsevier Inc. All rights reserved.
