Owing to the recent senatorial election results in Massachusetts, anticipated health care reform agendas for the United States are undergoing change again as the electorate and Congress struggle with a burgeoning health care system. Among those areas still being defined in the context of health care reform is comparative effectiveness research (CER). Conflicted economic priorities and desired levels of clinical outcomes in the marketplace along with increasingly complicated science have created a potentially unique opportunity for CER.
To understand the growing interest in CER, it is important to appreciate the evolution for payment of health care services in the US health care system as well as the shift in those who determine the value of a product or service. Before the availability of health care insurance, patients determined the who, what, and when of health care services to be provided. They also determined the payment or relative value of those services.
The advent of health insurance for hospital and some physician care services began in the mid-20th century. By the latter portion of the century, health insurance had evolved rapidly to cover additional aspects of health care, such as prescription drugs.
This expansion in coverage was accompanied by a shift in decision making from the patient to the insurance provider. First, decisions around payment and service discounts transitioned from the patient or employer. Eventually, insurers began influencing patient access to services through the use of contracted networks.
Hospital formularies in the 1950s had focused on product safety and an adequate supply of medication, but the role of the P&T committees changed as enrollment in MCOs rose along with spiraling increases in medication costs. This led to the use of formularies to leverage discounts and rebates from manufacturers. As a result, P&T committees began using a variety of management tactics to control costs and promote the rational use of medications; this also led to benefit management outsourcing.1 All of these efforts continued the shift of health care decision making away from the patient and into the hands of a third party.
The use of health outcomes data for therapeutics and formulary decisions has resulted from the expanding role of P&T committees along with increasing economically driven decision making in managed care. MCOs as well as other insurers rely on pharmacoeconomic data and models to control rising costs and increased use. This information helps determine the value of a product when making formulary decisions.2 This strategy also allows third-party payers of patient care services to ensure spending on proven medical treatments as part of their fiduciary responsibilities under their insurance or management services contract.
Roots of CER
The typical goal for CER is to target health care spending on proven medical treatments that are effective in defined, real-world populations. Comparative effectiveness can be determined through the use of many research methods, including randomized trials, systematic reviews, database analyses, and prospective observational studies.3 The origins for these methods emerged from organized health care systems that lie in health-related economic analyses, pharmacoeconomics, and health technology assessment.