Background: Gene and RNA therapies have potential to transform the treatment of rare inherited diseases, but there are concerns about the evidence supporting their use and high costs.
Objective: We analyze the evidence supporting Food and Drug Administration (FDA) approval of gene and RNA therapies for rare inherited diseases and discuss implications for clinical practice and policy.
Methods: We conducted a qualitative analysis of FDA documents outlining the basis of approval for gene and RNA therapies approved for rare inherited diseases between 2016 and 2023.
Gene and RNA therapies are promising treatments for many rare diseases. Pediatric populations that could benefit from these drugs are overrepresented among state Medicaid programs. Using Medicaid State Drug Utilization Data, we examined Medicaid spending and utilization of rare disease gene and RNA therapies.
View Article and Find Full Text PDFBackground: The 2022 Inflation Reduction Act authorizes Medicare to negotiate the prices of 10 drugs in 2026 and additional drugs thereafter. Understanding the sociodemographic and spending characteristics of beneficiaries taking these specific drugs could be important describing the impact of the legislation.
Objective: To describe sociodemographic and spending characteristics of Medicare beneficiaries who use the 10 prescription drugs ("negotiated drugs") that will face Medicare drug price negotiations in 2026.
Objectives: US Medicare will begin negotiating prices for top-selling drugs in 2023. This study describes and estimates potential savings from a therapeutic reference pricing approach, linking comparative effectiveness with the costs of existing therapeutic alternatives, that Medicare could use to adjust the starting point for price negotiations.
Methods: First, we identified target drugs likely to be selected for Medicare negotiation.