Establishing value in managed care: cost-effectiveness or budgetary impact?

Abstract

In the managed care setting, pharmacy benefit managers face considerable challenges when making decisions regarding drug coverage. Although scientifically valid data may be available from randomized, double-blind, placebo-controlled trials, this type of information does not shed light on potential differences between medications in the same class. Furthermore, properly designed, comparative trials are rarely conducted. Randomized naturalistic or pragmatic pharmacoeconomic studies can provide valuable information but are likewise infrequently performed. Economic modeling is an alternative but is not widely accepted because of the potential for bias. Retrospective analyses of information from patient surveys or existing databases are emerging as more popular methods of supporting informed formulary decisions. In the absence of a large-scale, prospective, naturalistic, pharmacoeconomic study, an alternative indication of product superiority can be derived from a continuum of different types of studies (from basic science to clinical trials to retrospective pharmacoeconomic analysis) conducted in a variety of patient care settings. The consistency with which a product demonstrates superiority across all types of studies is critical and is a crucial determinant of whether a health care provider would be likely to use the information for the purpose of decision making.