Quality of Life Assessment versus Clinical Outcome Measures: An Example

Abstract

The utility of quality of life assessments as a means of determining the cost effectiveness of a new therapeutic intervention cannot be overstated. Nonetheless, under certain circumstances such an approach may be misleading given the conditions under which the analysis was conducted and, more importantly, the intent of the original therapeutic trial. One example of such a situation involving the use of an intravenous immunoglobulin (IGIV) preparation (GAMMAGARD®, Baxter Healthcare, Glendale, CA) for the prophylaxis of infections in patients with either chronic lymphocytic leukemia or non-Hodgkin's lymphoma (B-cell lymphoproliferative disease) is reported here. An initial multicenter, double blind, placebo-controlled trial of monthly injections of IGIV demonstrated a significant reduction in the incidence of bacterial infections, the goal of the study. On the other hand, a subsequent, independent review of the data concluded that IGIV might not result in improved quality or length of life.

Since the original trial was not designed to consider this issue, it was concluded that quality of life/cost-effectiveness methodology, while a significant consideration, may be at cross purposes with clinical efficacy, particularly when the latter has not previously been demonstrated.