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6d ago

U.S. Drug Launch Prices Skyrocket, Driven by Rare Disease Treatments

The median price of new drugs exceeded $370,000 in 2024, with single-dose gene therapies reaching as high as $4.25 million.

U.S. dollar banknote and medicines are seen in this illustration taken, June 27, 2024. REUTERS/Dado Ruvic/Illustration/File Photo
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Overview

  • Launch prices for U.S. pharmaceuticals more than doubled between 2021 and 2024, reflecting a shift toward high-cost treatments for rare diseases.
  • In 2024, 72% of new drug approvals targeted rare diseases, up from 51% in 2019, with many addressing oncology and genetic disorders.
  • Orchard Therapeutics' Lenmeldy gene therapy set a record at $4.25 million for a one-time treatment, while Zevra Therapeutics' Miplyffa costs over $1 million annually.
  • The FDA approved 57 novel drugs in 2024, including seven cell and gene therapies, showcasing advancements in genomic science and biotechnology.
  • Policy measures like Medicare price negotiations, slated for 2026, aim to address affordability concerns as drugmakers defend high prices citing innovation and patient benefits.