rare disease treatments
Coverage of rare disease treatments in the Nexus archive.
- STAT+: Exemption of rare disease therapies in drug-pricing pilots would wipe out huge savings, analysis finds
Excluding rare disease treatments from Medicare drug-pricing pilots would eliminate significant savings, according to a Harvard University researcher. Biotech companies are lobbying the Trump administration to exclude these treatments from the Global Benchmark for Efficient Drug Pricing (GLOBE) and Guarding U.S. Medicare Against Rising Drug Costs (GUARD) pilots, which aim to lower drug prices to levels in other wealthy nations.
- STAT+: Pharmalittle: We’re reading about a Lilly threat to 340B hospitals, Ebola vaccine funding, and more
Eli Lilly is threatening to cut off price discounts from 340B hospitals that fail to submit required claims data, aiming to prevent duplicate discounts. The FDA is set to hold a closed-door meeting with rare disease advocates to discuss regulatory challenges for drug approvals.