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Prices for new US drugs doubled in 4 years as focus on rare disease grows

  • According to a Reuters survey of 45 newly launched medicines, the typical yearly list price in the U.S. More than doubled, rising from $180,000 in 2021 to over $370,000 in 2024.
  • This price surge relates mainly to advances in therapies for rare diseases, which now represent 72% of new drug launches and typically command higher prices due to small patient populations.
  • The most expensive new drug in 2024 was Lenmeldy, priced at $4.25 million for a one-time gene therapy treating a rare inherited brain and nervous system disorder.
  • Patients and insurers often manage high costs through savings programs and rebates, yet experts like Tinglong Dai emphasize the need for streamlined pre-authorization and alternative pricing models similar to Europe's.
  • The rising prices suggest continued industry focus and investment in rare disease therapies, with no clear sign of slowing absent significant reductions in development costs.
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U.S. News broke the news in New York, United States on Thursday, May 22, 2025.
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