OBJECTIVES: To identify United States (US) decision maker perceptions of data from observational studies and other health economics and outcomes research in the evaluation of emerging health technologies. METHODS:  We conducted qualitative one-on-one interviews with payers (5), clinicians (6), and hospital administrators (4) in the US to determine perceptions of data collected outside of randomized controlled trials in the evaluation of emerging health technologies.

RESULTS: Clinical efficacy and safety postmarketing assessments have more of an impact on decision making than other types of study data collected outside clinical trials. Compared with other stakeholders, clinicians placed a particularly high value on patient registries, whereas, hospital administrators placed a high value on budget-impact analysis. Annual/semiannual review of drug classes by health plans and hospital formulary committees may include patient registry data; however, these data typically are not available when a new health technology is first introduced into the market. Real-world outcomes and head-to-head comparisons are highly desirable, but were also the most commonly mentioned data gaps in evaluating new health technologies. Clinicians more commonly sought data that affect patient treatment decisions, confidence in prescribing, and subpopulation data. Payers and hospital administrators were interested in real-world outcomes data in their specific patient populations, and data that could have an impact on costs, cost offsets, resource utilization, and readmissions. CONCLUSIONS: Stakeholders in the US are seeking more comparative effectiveness and economics information to better inform decision making in the evaluation of new health technologies. The impact of comparative effectiveness research and economics on formulary decision making will likely have more impact in the future. If study data are to be considered  valuable in supporting health care decision-making,  the rigor, transparency, and the customer perspective needs to drive the study methods and designs.