BACKGROUND: The influence of the Institute for Clinical and Economic Review (ICER) value assessment framework (VAF) on payer decisions has been increasing in recent years. There is limited evidence assessing ICER’s influence on payer decision making in therapies for rare diseases.

OBJECTIVES: To understand the impact of ICER reports in rare diseases in context with other therapeutic areas (TAs) and to analyze trends in ICER’s findings for rare diseases.

METHODS: Double-blinded web-based surveys were fielded in September and October 2021 to payers, and ICER assessments in rare diseases were analyzed to assess trends in cost-effectiveness findings.

RESULTS: Of the 50 respondents, 80% identified rare diseases as the TA that an ICER report would be most impactful on (rated as at least somewhat impactful) their organization’s decision-making processes, followed by autoimmune and metabolic diseases (76% for both). Additionally, respondents identified the cost of therapy (74%) and size of the patient population (56%) as primary reasons for variance in ICER’s impact among TAs. Qualitative survey responses indicated that ICER’s assessments for rare diseases are impactful since in-depth drug and disease knowledge for rare disease therapies is otherwise limited. An analysis of ICER reports from 2017 to 2021 in therapies for rare diseases showed that ICER found only 4 of 34 interventions to be cost-effective at a willingness-to-pay threshold of $150,000/quality-adjusted life-year and suggested wholesale acquisition cost discounts ranging from 37% to 97%.

CONCLUSIONS: While payers identify ICER’s reports in therapies for rare diseases to be highly impactful, these reports nearly always conclude that treatments for rare diseases are not cost-effective and do not seem to impact coverage decisions for these treatments. Still, payers noted ICER assessments of therapies for rare diseases to be particularly valuable since there is generally limited information and/or internal data/expertise in rare diseases.