Advantages and Limitations of Utility Assessment Methods in Rheumatoid Arthritis
ARIEL BERESNIAK, ANTHONY S. RUSSELL, BOULOS HARAOUI, LOUIS BESSETTE, CLAIRE BOMBARDIER, and GERARD DURU
ABSTRACT.
Utility assessment and cost-utility analyses such as costs/quality-adjusted life-years (QALY) are frequently presented to demonstrate the value of new treatment options in rheumatoid arthritis (RA). However, utility indicators require various methods that introduce significant methodological challenges, which directly influence the results and ensuing reimbursement decisions. Our objective was to review and discuss these challenges and the validity of frequently used utility assessment techniques in the context of RA. Coding the intensity of preferences or variations in patient satisfaction in order to assess utility implies extreme mathematical assumptions about a patient's rationality regarding his/her preferences towards different given health states. The construction and assumptions of commonly used "direct approaches" (standard gamble, time tradeoff, visual analog scale) and indirect approaches (EQ5D, HUI, SF6D) are presented. Other approaches such as transformation in utility of data from clinical (Health Assessment Questionnaire) or quality of life instruments ("mapping technique") are analyzed as they appear to generate uncertainty and a wide variation in estimated utility values in the context of RA. Utility assessment and cost-utility analyses in RA, which form the basis of the QALY, are frequently published and often requested by health technology assessment agencies to assist reimbursement decisions. However, when interpreting the results, the medical community must take into consideration the limitations and significant uncertainty of these approaches.
In light of these findings, real cost-effectiveness analyses based on observed clinical outcomes appear to be more robust and reliable to assist decision-making, particularly in the context of RA. (First Release Oct 15 2007; J Rheumatol 2007;34:2193-200)
Key Indexing Terms:
UTILITY
QUALITY-ADJUSTED LIFE-YEARS
RHEUMATOID ARTHRITIS
From LIRAES, University Paris-Descartes, Paris; CNRS, National Center of Scientific Research, Lyon, France; Data Mining International, Geneva, Switzerland; University of Alberta Hospital, Edmonton, Alberta; University of Montreal, Montreal Rheumatology Institute, Montreal, Quebec; Centre Hospitalier Universitaire de Québec-CHUL, Quebec, Quebec; and University of Toronto, Mount Sinai Hospital, Toronto, Ontario, Canada.
A. Beresniak, MD, PhD, LIRAES, University Paris-Descartes and Data Mining International; A.S. Russell, MA, MB, BChir, University of Alberta Hospital; B. Haraoui, MD, University of Montreal, Montreal Rheumatology Institute; L. Bessette, MD, Centre Hospitalier Universitaire de Québec–CHUL; C. Bombardier, MD, University of Toronto, Mount Sinai Hospital; G. Duru, PhD, CNRS, National Center of Scientific Research.
Address reprint requests to Dr. A.S. Russell, Rheumatic Disease Unit, University of Alberta, 562 Heritage Medical Research Centre, Edmonton, Alberta T6G 2S2, Canada. E-mail: as.russell@ualberta.ca
Accepted for publication June 21, 2007.