Abstract

Objectives

The Institute for Clinical and Economic Review (ICER) conducted a special assessment of Trelegy Ellipta and Breo Ellipta for chronic obstructive pulmonary disease to inform the Centers for Medicare and Medicaid Services (CMS) drug price negotiation process. In this manuscript, we examine the type, quality, and impact of the observational evidence included.

Methods

We described the type of observational evidence used in ICER’s assessment with a primary focus on adherence and persistence data. Risk of bias was assessed using the ROBINS-I.

Results

Observational data included in ICER’s review primarily informed the assessment of differential adherence and persistence between single and multiple inhaler products. The majority of studies were real-world evidence (RWE), utilizing claims or electronic health record data, and were deemed to be at moderate or severe risk of bias. RWE reported that Trelegy Ellipta and Breo Ellipta were associated with greater adherence and persistence compared with generic multiple-inhaler or multiple-daily-dosing alternatives. Persistence data were critical for differentiating between products in the cost-effectiveness analysis; however, persistence was based on prescription refills which is an imperfect measure of treatment continuation.

Conclusions

RWE played a critical role in evaluating the comparative clinical and cost-effectiveness of Trelegy Ellipta and Breo Ellipta to inform Centers for Medicare and Medicaid Services drug price negotiations. Differences in persistence informed differential pricing to generic multiple-inhaler or multiple-daily-dosing alternatives, demonstrating how data collected outside of clinical trials can inform value assessments. However, RWE limitations and the lack of evidence linking adherence or persistence to clinical outcomes highlight the need for future research.