Mar 10, 2025

What do the new Medicare negotiations mean for pipeline orphan therapies?

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— Rob Wood, Ema Slapikaite, Katarina Kitarovic & Sarah Forbes

This infographic breaks down a critical, but under-discussed, implication of the Medicare drug price negotiations: the risk to orphan medicines.

While the IRA aims to lower the cost of some therapies, its structure may unintentionally discourage biopharma companies from investing in new treatments for rare diseases. Why?

  1. Many orphan drugs (OMPs) will no longer be exempt from price negotiations if they’re approved for multiple conditions
  2. These negotiations can lead to significant price cuts, reducing the commercial incentive to expand or even develop OMPs
  3. This shift could affect global innovation and access to orphan medicines

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