Developing a Collaborative Study Protocol for Combining Payer-Specific Data and Clinical Trials for CER

Abstract

T he demand for comparative effectiveness research (CER) by health care providers and payers represents new opportunities for the U.S. government, research organizations, and pharmaceutical companies to generate "meaningful evidence" for use in medical decision making.1 CER studies conducted with a payer perspective should develop questions, select outcomes, and utilize data that are applicable to the payers themselves for use with their formulary and reimbursement decision-making processes. CER studies for prescribers should be designed and implemented to inform evidence-based therapeutic guidelines, providing actionable information from their everyday practice use. The challenge is how to conduct CER studies that satisfy the simultaneous requirements of scientific rigor and applicability to the respective decision makers. One solution is to address the demand for "real-world" data (RWD) by involving decision makers and other key stakeholders early on in the development of the research designs and implementation of study protocols when conducting CER studies. RWD have been defined "as data used for decision-making that are not collected in conventional RCTs" (randomized controlled trials); 2 therefore, the ability to gather input from the payer is essential to ensure collected endpoints are applicable to the decision makers themselves.RCTs are considered the "gold standard" for providing evidence about a product's efficacy and are the basis for supporting formulary decision making. While the internal validity of RCTs is well known and established, the controlled protocols of RCTs may not have the desired level of external validity for a managed care organization's (MCO) population. Consequently, health care decision makers are examining other sources of data to supplement RCTs for their health care coverage policies. Health care providers and payers use available evidence from both RCTs and RWD sources to decide whether a particular drug product offers tangible clinical benefits and value compared with existing therapies. Improving medical outcomes and providing positive impact on health care expenditures are shared goals of providers, payers, and the pharmaceutical industry. 3Developing CER Studies to Inform Payer Decision Making Payers are interested in CER results and evidence-based value assessments of comparator therapies to use in their coverage decision-making processes. Some have proposed that CER involving systematic reviews of effectiveness evidence could improve the coverage and reimbursement processes. 4 However, now more than ever, there is a need for better evidence generation rather than just better synthesis of existing evidence, which raises the question of how more meaningful evidence could be generated and how the decision makers could be involved in the identification of evidence gaps, design of study protocols, and implementation of CER studies, particularly those that propose to use RWD. It also is important to determine when additional studies, and related designs, are needed; value of in...