ReviewPatient-reported outcomes: Assessment and current perspectives of the guidelines of the Food and Drug Administration and the reflection paper of the European Medicines Agency
Introduction
In the recent years, the use of patient-reported outcomes (PROs) has increased significantly.1 HRQOL measures involve subjective patient assessment or evaluation of important aspects of well-being2 that are affected by current disease and/or treatment. Prominent examples of cancer-related HRQOL tools are the EORTC QLQ-C30 and the Functional Assessment of Cancer Therapy General (FACT G).3, 4
Recently, the Food and Drug Administration (FDA) introduced the umbrella term PRO and attempted to standardise PRO use to provide a more systematic treatment-review process. For this reason, the FDA released draft guidance on PRO measures in February 2006: Guidance for Industry. Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims.5 Also, the European Medicines Agency (EMEA) produced a reflective paper of regulatory guidance for the use of HRQOL measures in the evaluation of medicinal products in cancer in July, 2005.6
However, despite considerable effort to develop guidelines, it is not entirely clear what evidence the regulatory bodies would require in supporting claims when reporting PRO data. To date, established PRO measures are criticised by the regulatory agencies, leading to rejection of many PRO labelling claims.
The main objective of this paper is to review the current opinion relating to the guidance, and to make recommendations based on our review.
Section snippets
Materials and methods
The aim of the study is to provide an overview of the current opinion in relation to the two major guidance documents: the FDA Patient-Reported Outcomes draft (2006) and the EMEA HRQOL reflection paper (2005). A thematic qualitative approach was used to compare recommendations and requirements from each guidance. Written comments, invited by the FDA and submitted to the FDA web site (Table 1), have been reviewed. These reviewed responses related to the FDA guidance were also considered in
Results
The FDA PRO guidance generated 47 written responses, totalling to 364 pages of comments, which are accessible on the FDA web site. These comments mainly came from professional groups in both academia and the pharmaceutical sector (Table 1). No documents were found on the EMEA web site about the EMEA HRQOL reflection paper, but the literature search identified several studies (discussed later) that looked at these guidelines. The search on PubMed and the search of grey literature generated
Discussion
In oncology phase III RCTs and registration trials, PROs are increasingly used for providing information about HRQOL in patients who undergo new treatments. Both the FDA and EMEA increasingly appear to be willing to accept PROs in support of medicinal labelling claims or in the evaluation of medical products such as cancer drugs.
The views of the FDA on PROs could be described as extensive, with detailed requirements and a restrictive nature. The EMEA has provided more global statements and
Conflict of interest statement
EORTC and Andrew Bottomley have received an unrestrictive academic research grant funding from AstraZeneca, UK. Dave Jones is an employee of AstraZeneca. Lily Claassens’ Fellowship was in part funded by an academic grant from AstraZeneca.
Acknowledgements
We thank Dr. Bhash Parasuraman (AstraZeneca, USA) for her comments on our paper.
This research was funded by an educational academic grant from AstraZeneca, UK. In addition, part of the publication was supported by Grant Numbers 5U10 CA011488-37–5U10 CA011488-38 from the National Cancer Institute (Bethesda, Maryland, USA). Its contents are solely the responsibility of the authors and do not necessarily represent the official views of the National Cancer Institute.
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