Science to services: consumers need "real-world" science.

Abstract

Over the past decade there has been a revolution in pharmacotherapy for schizophrenia and related disorders. The second generation, or atypical, antipsychotic medications have demonstrated efficacy and generally better side-effect profiles. However, from the perspective of policy makers the higher costs associated with these newer medications leads to tough decisions regarding their continued use in light of an escalating fiscal crisis. For consumers, both persons with the illness and their family caregivers, the budgetary cutbacks leave many scrambling for answers to questions that most treatment (efficacy) studies were never designed to answer. "Should we oppose formulary restrictions on principle alone, or is there scientific data that can be relied on to inform our position?" On a more personal note, many are asking whether or not to switch to one of the newer medications and which medication would be best for them. Unlike CATIE, efficacy studies were never designed to answer such questions. In this article, we start by highlighting how CATIE will fill important gaps in translating the results of efficacy studies to effectiveness in the real world. Both the development of the CATIE methodology and the study design itself reflect what we will refer to as "real-world science": i.e., science that sheds light on effectiveness in vivo and can inform decisions consumers, clinicians, and policy makers are faced with day-to-day. We discuss CATIE in the context of the fiscal crisis hitting MEDICAID programs leading many policy makers to take the more expensive, atypical antipsychotics off the list of medications made available to patients. We argue that studies like CATIE will be highly informative and ultimately vital to policy makers wishing to create mental health policies that will succeed. Throughout, we highlight how CATIE, and real-world science more generally, are vital to consumers striving to find the medication(s) that works best for them. Given the organic research design process, which arguably relies on a fuller range of stakeholders than any study of its kind before, we remain hopeful that CATIE can succeed in generating an unprecedented amount of real-world science that consumers can use.