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The Use of External Controls in FDA Regulatory Decision Making.

Mahta Jahanshahi1, Keith Gregg2, Gillian Davis2

  • 1BioMarin Pharmaceutical Inc, 105 Digital Drive, Novato, CA, 94949, USA. mjahanshahi@bmrn.com.

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|May 20, 2021
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Summary

The U.S. Food and Drug Administration (FDA) accepted external controls in 45 drug approvals between 2000-2019, especially for rare diseases. Retrospective natural history data was the most common external control source.

Keywords:
Baseline controlsExternal controlsHistorical controlsRare diseaseRetrospective natural history

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Area of Science:

  • Regulatory Science
  • Clinical Trial Design
  • Drug Development

Background:

  • U.S. Food and Drug Administration (FDA) typically requires internal controls for drug approval.
  • External controls are permissible when internal controls are infeasible or unethical, particularly in rare disease populations.
  • Understanding FDA's acceptance of external controls is crucial for drug development.

Purpose of the Study:

  • To review FDA regulatory approval decisions from 2000-2019.
  • To identify pivotal studies that utilized external controls for drug and biologic product approvals.
  • To analyze the reasons and methods for accepting external control data.

Main Methods:

  • Review of FDA regulatory approval decisions for drugs and biologics from 2000-2019.
  • Identification of approvals that leveraged external controls in pivotal studies.
  • Focus on select therapeutic areas and analysis of external control types.

Main Results:

  • Forty-five approvals were identified where FDA accepted external control data.
  • Common reasons for acceptance include rare disease, ethical concerns, serious conditions, and unmet medical need.
  • Retrospective natural history data was the most frequent external control source (44%).

Conclusions:

  • External controls, particularly retrospective natural history data, are valuable for drug development in rare diseases.
  • FDA's acceptance of external controls supports innovative clinical trial designs and avoids delays.
  • Updating regulatory guidance is recommended to acknowledge the utility of external controls.