TY - JOUR
T1 - Interpreting and reporting results based on patient-reported outcomes
AU - Revicki, Dennis A.
AU - Erickson, Pennifer A.
AU - Sloan, Jeff A.
AU - Dueck, Amylou
AU - Guess, Harry
AU - Santanello, Nancy C.
N1 - Funding Information:
Source of financial support: Funding for the meeting was provided by the Mayo Foundation in the form of unrestricted educational grants; North Central Cancer Treatment Group (NCCTG) (CA25224-27) and Cancer Center grants (CA15083-32).
PY - 2007
Y1 - 2007
N2 - This article deals with the incorporation of patient-reported outcomes (PROs) into clinical trials and focuses on issues associated with the interpretation and reporting of PRO data. The primary focus and context of this information relates to the evidentiary support and reporting for a labeling or advertising claim of a PRO benefit for a new or approved pharmaceutical product. This manuscript focuses on issues associated with assessing clinical significance and common pitfalls to avoid in presenting results related to PROs. Specifically, the questions addressed by this manuscript involve: What are the best methods to assess clinical significance for PROs? How should investigators present PRO data most effectively in a Food and Drug Administration (FDA) application? In labeling or in a scientific publication? Guidelinesfor interpreting clinical significance of PROs and for comprehensively reporting on the methods, measures and results of clinical trials that incorporate PROs are important for clinicians, regulatory agencies, and most of all to patients. Clear specifications for considering a finding on a PRO measure, as clinically meaningful, need to be determined by instrument developers and psychometricians; they need to be reported for all clinical trials involving PRO end points. Clinical trial reports need to be comprehensive, clear, and sufficient to enable any reader to understand the methods, PRO measures, statistical analysis, and results.
AB - This article deals with the incorporation of patient-reported outcomes (PROs) into clinical trials and focuses on issues associated with the interpretation and reporting of PRO data. The primary focus and context of this information relates to the evidentiary support and reporting for a labeling or advertising claim of a PRO benefit for a new or approved pharmaceutical product. This manuscript focuses on issues associated with assessing clinical significance and common pitfalls to avoid in presenting results related to PROs. Specifically, the questions addressed by this manuscript involve: What are the best methods to assess clinical significance for PROs? How should investigators present PRO data most effectively in a Food and Drug Administration (FDA) application? In labeling or in a scientific publication? Guidelinesfor interpreting clinical significance of PROs and for comprehensively reporting on the methods, measures and results of clinical trials that incorporate PROs are important for clinicians, regulatory agencies, and most of all to patients. Clear specifications for considering a finding on a PRO measure, as clinically meaningful, need to be determined by instrument developers and psychometricians; they need to be reported for all clinical trials involving PRO end points. Clinical trial reports need to be comprehensive, clear, and sufficient to enable any reader to understand the methods, PRO measures, statistical analysis, and results.
KW - Clinical significance
KW - Clinical trials
KW - Health-related quality of life
KW - Minimal important differences
KW - Patient-reported outcomes
KW - Statistical analysis
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U2 - 10.1111/j.1524-4733.2007.00274.x
DO - 10.1111/j.1524-4733.2007.00274.x
M3 - Article
C2 - 17995470
AN - SCOPUS:36049043190
SN - 1098-3015
VL - 10
SP - S116-S124
JO - Value in Health
JF - Value in Health
IS - SUPPL. 2
ER -