OBJECTIVES: An increasing number of treatments for rare diseases have been approved or are in development, contributing to rising costs for health systems. As a result, a shift in the payer decision-making process to set drug prices within these health systems has occurred. We sought to recognize and understand trends in international reference pricing, including formal and informal price referencing, as well as evaluate the potential differential impact of reference pricing methods and practices on innovative high-cost and rare disease therapies relative to traditional pharmaceutical products.

METHODS: We conducted qualitative research with (ex-)payers and payer advisors to evaluate current and emerging price setting methods and innovative access approaches for traditional treatments compared with innovative, high-cost, rare disease therapies.

RESULTS: Differences in decision-making processes for price setting existed between mature and emerging health systems. For mature health systems, the pricing approach for both list and net price was generally similar and followed a standardized process. Changes in pricing assessments usually relied on new policies and/or legislation to drive evolution in approach. Emerging health systems provided more flexibility in pricing assessments than high-income markets, including informal price referencing to markets in which details of net price were more visible.

CONCLUSIONS: Reference price benchmarking was generally important for both mature and less mature health systems. Payers in more mature health care systems demonstrated limited flexibility in approaches to setting list and net prices and relied more on HEOR principles. Payers in markets with less mature health care systems were more likely to leverage informal price setting approaches to ensure price was optimized relative to other markets and reflected relative clinical value versus competitors.