OBJECTIVES: The Institute for Clinical and Economic Review’s (ICER) assessments of value are intended to inform pricing and coverage decisions of healthcare interventions in the US. Besides assessments of value, ICER routinely publishes payer and policy recommendations on their website, despite the lack of publicly available guidelines for these recommendations. Historically, US payers tend to manage utilization of newly approved drugs through implementation of strategies such as prior authorization (PA), step-therapy (ST) or quantity limits (QL). Our goal is to examine the utilization management strategies suggested by ICER to US payers and policy-makers when areas of uncertainty are raised during ICER assessments of gene therapies.

METHODS: We reviewed ICER’s policy recommendations published for N=3 gene therapies (Luxturna, Zolgensma [and Spinraza, joint spinal muscular atrophy (SMA) assessment], and Hemgenix). We mapped the areas of uncertainty highlighted during the assessment considering key words such as ‘uncertainty’, ‘remaining uncertainty’, ‘lack of’, ‘long-term safety and efficacy data’ to the key ICER recommendations for payers and policy‑makers.

RESULTS: If a standard of care (SOC) for the indication is not available, PA is a strategy that ICER suggests for drug management; if an SOC is available, PA in combination with ST is recommended instead. If the implementation of outcomes-based contracts (OBCs) is feasible (e.g., treatment outcome that correlates to the drug’s efficacy is easily measurable in clinical practice), ICER highlights this as a possibility to address uncertainty with long-term treatment effect as well.

CONCLUSIONS: Although there are no guidelines on ICER’s payer and policy recommendations, for gene therapies, these seem to be driven by high costs coped with uncertainty with long-term treatment effect. PA, ST (alone or in combination), and OBCs are strategies suggested by ICER to US payers and policy-makers to manage uncertainty when deciding on the coverage of gene therapies.