Individualization of Treatment and Comparative Effectiveness Research

Comparative effectiveness research (CER) comprises of the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care. Its purpose is to assist consumers, clinicians, purchasers, and policymakers to make informed decisions that will improve healthcare at both the individual and population levels. The agenda for CER is very ambitious considering its limited resources and the starkly different informational requirements of the various decision-makers. How results emanating from a single or a fewCER studies can inform all levels of decision-making remains the biggest challenge in the designs of CER studies. This chapter discusses the role of CER in generating individualized information on the value of medical products and how such information has the potential for improving decision-making at all levels. In practice, this notion of generating individualized information and using it to deliver care is denoted as personalized medicine (PM), which allows for the possibility of variation in medical quality based on demographics, comorbidities, preferences, genomics, and even environmental contexts within which care is delivered. However, traditionally, CER and PM are thought to be disparate research strategies. Recognizing that this distinction may be artificial and created by silos in research practices, this chapter discusses some of the key behavioral and economic issues that encourage the adoption of PM in practice and how the current infrastructure for CER studies can be leveraged to evaluate PM and also foster innovation in PM. Indeed, the fields of CER and PM appear to be morphing into the single paradigm of patient-centered outcomes research (PCOR) (also denoted as precision medicine). The chapter ends with discussing some of the tools available in order to prioritize PM research in a prospective manner. Introduction: What Is Comparative Effectiveness Research (CER)? The $2.5 trillion spent on healthcare costs in the USA constituted approximately 18 % of GDP in 2009, and at current rates of growth, healthcare will exceed one fourth of GDP by 2025 (Sisko et al. 2009; CBO 2008). As part of a portfolio of innovative public policies that are aimed to address this challenge, public investments in comparative effectiveness research (CER) are viewed as worthwhile endeavors by payers, providers, and patients (ACES Testimony 2009). CER is meant “to assist consumers, clinicians, purchasers and policymakers to make informed decisions” (IOM 2009). Informing decisions in healthcare policy and clinical medicine most often involve evaluation of alternatives: Which treatment will produce the optimal clinical outcome in a given patient? What type of insurance coverage will maximize social welfare? The fundamental problem of ex ante evaluation is to predict outcomes under alternative policies or interventions. A large literature, dating from the early 1920s, focuses on developing methods to address such problems of evaluation (Neyman 1923; *Email: basua@uw.edu Comparative Effectiveness Research in Health Services DOI 10.1007/978-1-4899-7586-7_15-1 # Springer Science+Business Media New York 2015