Analytic difficulties in applying quality of life outcomes to clinical trials of therapy for HIV disease

by Samuel A. Bozzette, Naihua Duan, Sandra H. Berry, David E. Kanouse

Purchase Print Copy

 FormatList Price
Add to Cart Paperback14 pages Free

Despite considerable enthusiasm for the concept, quality-of-life or health status outcomes are seldom employed in clinical trials. The failure of common analytic procedures to meet key requirements may have contributed to this limited acceptance. In particular, (1) results of clinical trials must be expressed in terms that clinicians, regulators, administrators, and patients find useful, (2) reasonable means of summarizing outcomes should be available a priori, (3) scale units should have real world meaning, (4) unwarranted assumptions regarding scale properties should be avoided, and (5) adequate methods for handling attrition due to death or drop-out are necessary. The authors have developed approaches to these problems. They summarize overall outcome using a weighted sum of scores for scales covering several domains and weights derived from regressions of scale scores on a reference variable. The authors' multistate survival analysis, an extension of standard survival analysis, attempts to circumvent some of the difficulties with attrition and poor scale properties of health status/quality of life measures. Multistate survival analysis has two components, a description of survival-in- state, and significance test based on transitions-from-state. In survival-in-state analysis, the authors generalize the usual survival analysis to consider the proportions of participants with a health or quality status which is at least equal to that indicated by specified ordered states. In transition-from-states analysis, they generalize the Mantel-Haenszel procedure to simultaneously consider upward and downward transitions from previous health or quality state to improved or worsened states. These approaches incorporate mortality without requiring specification of a score value for death, allow reasonable handling of attrition, give results denominated in proportions or time units rather than score values, and allow assessment of net changes in status for individual participants between evaluations.

Originally published in: Psychology and Health, v. 9, 1994, pp. 143-156.

This report is part of the RAND Corporation Reprint series. The Reprint was a product of the RAND Corporation from 1992 to 2011 that represented previously published journal articles, book chapters, and reports with the permission of the publisher. RAND reprints were formally reviewed in accordance with the publisher's editorial policy and compliant with RAND's rigorous quality assurance standards for quality and objectivity. For select current RAND journal articles, see External Publications.

Our mission to help improve policy and decisionmaking through research and analysis is enabled through our core values of quality and objectivity and our unwavering commitment to the highest level of integrity and ethical behavior. To help ensure our research and analysis are rigorous, objective, and nonpartisan, we subject our research publications to a robust and exacting quality-assurance process; avoid both the appearance and reality of financial and other conflicts of interest through staff training, project screening, and a policy of mandatory disclosure; and pursue transparency in our research engagements through our commitment to the open publication of our research findings and recommendations, disclosure of the source of funding of published research, and policies to ensure intellectual independence. For more information, visit

The RAND Corporation is a nonprofit institution that helps improve policy and decisionmaking through research and analysis. RAND's publications do not necessarily reflect the opinions of its research clients and sponsors.