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The Real World Is Not Enough: Comparability Is Common Obstacle For RWE In US FDA Decisions

Executive Summary

Unsuccessful attempts to use real-world evidence for approvals highlight the need to demonstrate relationship between real-world data and clinical trial patients. The Pink Sheet examines what went wrong for Bluebird/Bristol’s Abecma, Ultragenyx’s Dojolvi, and Karyopharm’s Xpovio.

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