Abstract
BACKGROUND:
Comparative effectiveness research (CER) has been proposedin the United States as a way to compare new drugs and technologies with established alternatives and determine not just whether a therapyworks, but how well it works compared to other options.
OBJECTIVES:
To define the current use of CER in the development of newdrugs and technologies and explore what is needed for this research approach to reduce or stabilize health care costs in the United States.
SUMMARY:
In 2010, the Patient-Centered Outcomes Research Institute (PCORI) was established by the Patient Protection and Affordable CareAct (PPACA) to coordinate federally funded CER and recommend research priorities. Hochman and McCormick’s (2010) evaluation of 328 randomized trials, observational studies, and meta-analyses involving medications published between June 2008 and September 2009 in 6 key journals show that most published research did not fulfill the criteria of CER (defined ascomparison to active treatment) and that most study design is driven by FDA requirements rather than the need to develop evidence to facilitate selection of the most effective therapy. Since PPACA provides alternative funding for CER, it could encourage funding more studies to help determinewhich treatment delivers the best value per unit of investment from clinical, humanistic, and economic perspectives. Manufacturers may avoid CER because it increases product development costs, but a drug proven more effective is more likely to be accepted by formulary committees, increasing the drug’s market share, whereas payers may reject or limit use of a newdrug that performs less effectively in comparative studies.
CONCLUSIONS:
CER may not directly reduce expenditures for drugs andmedical technologies. The results may vary widely from case to case; however, despite often significantly higher prices for new drugs, it is important to look beyond product costs to the overall impact on health carecosts, including medical cost offsets that may occur through improved health or decreased morbidity. To truly decrease cost and improve quality,cost-effectiveness will have to be integrated into CER with the objective of prioritizing efficient therapies in the real-world health care system. If the methods and output of CER improve, the resulting cost-effectiveness ratios will also be more useful to the payer. CER should ultimately, therefore, be a useful tool to help patients, providers, and decision makers provide themost effective and most cost-effective interventions.