Hogue S, Scott A, van Amerongen D, Hollis K, McLeod L, Brogan A. Payer and decision maker use of observational studies for health care reimbursement decisions. Poster presented at the 2013 ISPOR 18th Annual International Meeting; April 30, 2013. [abstract] Value Health. 2013 Nov; 16(7):A459-60.

OBJECTIVES: As cost-containment pressures across Europe intensify, evidentiary hurdles to justify new drugs will continue to grow. Data from clinical trials alone are no longer adequate to meet the needs of all health care decision makers (e.g., payers and health technology assessment [HTA] agencies, physicians and patients), driving a need for robust, complementary data. However, perceived need to collect real-world clinical, patient-centered, and/or economic outcomes through observational studies varies across countries, stakeholders and organizations. The objective was to better understand how decision makers use observational studies to inform reimbursement and/or market access decisions for new products.

METHODS: Desktop research identified the types of observational studies most valuable for reimbursement decision making. This project extends a United States (US) payer study by conducting qualitative one-on-one interviews with decision makers across Europe to review the need for real-world evidence. Participants represented national and regional decision makers from several European countries, to identify specific evidence requirements and design attributes most accepted by payers/ HTA advisors and to understand how real-world evidence contributes to the value of a new drug from the decisonmakers’ perspectives.

RESULTS: Data from observational studies are used to describe patient segments, understand treatment patterns, resource utilization, and provide effectiveness data that supplement clinical trial data as well as inform risk sharing schemes. Observational studies help inform payer decision making but the validity and robustness of the results is often scrutinized. Publication of observational data in a Tier 1, peer-reviewed journal lends critical credibility to real-world study results.

CONCLUSIONS: The current study extends the evaluation into Europe to confirm that as reimbursement decision makers continue to rigorously review new drug therapies, accurate, robust, peer-reviewed published and generalisable realworld data will become particularly important for outcomes- or performance-based access schemes and health care budget management both in the US and Europe.