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T-19: Assessing Trends in Clinical Data Packages for Orphan Indications

Poster Presenter

      Dayna Lesueur

      • Clinical Scientist
      • Alnylam Pharmaceuticals, Inc.
        United States


Drug development for orphan diseases faces many challenges, including a lack of precedence in clinical data packages for indications with limited treatments. The aim is to compare phase 1-3 clinical trial profiles of “first-in-indication” orphan drugs to subsequent approvals in the same indication.


Orphan indications in which several therapies were approved by the US FDA between 2011-2016 were identified via the CDER Novel Drug Summaries; the clinical trial profiles of these therapies were compared. Other therapies approved in the identified indications prior to 2011 were also included.


Between 2011 and 2016, 15 orphan indications had more than one drug approved; 9 (60%) of these were oncology-related indications. Based on the clinical trial data collected from multiple orphan drugs, it was often found that a product approved first in an orphan indication had less phase 1, phase 2, and phase 1/2 studies than the subsequent drug approvals for the same indication. It was also found that drugs approved first in these indications generally had less patient exposure and shorter duration of studies in their early phase development program. Further details of these results will be presented.


This data suggests that “first-in-indication” drugs for orphan diseases require less robust early phase clinical data packages to support initiation of phase 3 pivotal studies and marketing approval. This data supports a potential trend that warrants further evaluation.

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