Academic journal article Applied Health Economics and Health Policy

Inclusion of Compliance and Persistence in Economic Models

Academic journal article Applied Health Economics and Health Policy

Inclusion of Compliance and Persistence in Economic Models

Article excerpt

Key points for decision makers

* Appropriate consideration of compliance and persistence within economic models is critical to enable robust predictions of economic outcomes in real-world settings

* Technical modelling methods that enable appropriate integration of real-world compliance and persistence data are receiving more widespread use and should be considered as approaches for future economic models

* Efforts should be made, where possible, to collect real-world compliance and persistence data to facilitate the wider development of economic models that consider these factors

1. Introduction

It is well known that patient compliance with medication and other treatment regimens can have a profound effect on outcomes. Compliance (taking medications or adhering to screening guidelines as directed) and persistence (continuing to take medications for the recommended duration) affect treatment efficacy, treatment costs, adverse event rates, rates and severity of disease-related sequelae, overall health and quality of life. Given these known effects on real-world clinical outcomes, it stands to reason that compliance and persistence are important factors to consider in economic models that evaluate medications and other therapeutic regimens. However, compliance has historically been incorporated into economic modelling infrequently, and has been considered in only a cursory manner in those instances where it is addressed.

The economic importance of compliance and persistence can be demonstrated at different scales. Nasseh et al.[1] estimated the direct medical costs for the US and for each state due to non-compliance in three chronic diseases (diabetes mellitus, hypertension and dyslipidaemia). They estimated a total burden of over $US100 billion for the US as a whole, with burdens to the individual states ranging from $US284-$US634 per adult in the state population (year 2010 values). The effect of compliance and persistence can also be seen in evaluations of individual drugs. Damen et al.[2] evaluated treatments for schizophrenia that differed only in the rate of compliance, reporting that increases in the compliance rate simultaneously increased quality-adjusted life-years (QALYs) and decreased costs.

In this review, we will describe the current state of economic modelling with respect to incorporation of compliance and persistence considerations. First, we will discuss early efforts to integrate compliance and persistence into economic models, drawing heavily on the excellent review by Hughes et al.[3] We then survey more recent efforts through 2012, noting that there has been increased attention paid to this issue in recent years. The survey of prior modelling efforts is followed by a critical discussion of the ways in which compliance and persistence have been modelled, with emphasis on the strengths and weaknesses of the various approaches. In closing, we suggest some possible future directions for the incorporation of compliance and persistence into economic models for the evaluation of medications and other treatment regimens.

2. Definitions of Compliance and Persistence

One of the challenges in reviewing this area is the large number of terms and the wide range of definitions used in discussing this topic. In 2008, the Medication Compliance and Persistence Special Interest Group of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) proposed standardized terminology.[4] 1 Briefly, the terms 'compliance' and 'adherence' are synonymous, meaning "the extent to which a patient acts in accordance with the prescribed interval and dose of a dosing regimen." For the sake of consistency and clarity, we have used the term 'compliance' throughout. 'Persistence' is defined as "the duration of time from initiation to discontinuation of therapy." While the concepts of compliance and persistence are related, they are clearly distinct. One can be compliant but not persistent, taking medication properly day-to-day, but stopping treatment too soon. …

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