Compassionate Use

Since 1987, the FDA has had rules in place that have enabled patients, under specific circumstances, to access drugs or biologics that are still in development for treatment purposes. These expanded access or compassionate use program rules were amended in 2009 by the FDA to ensure “broad and equitable access to investigational drugs for treatment.”

The regulations include the following:

  • Criteria that must be met in order to authorize the expanded access use
  • Requirements for expanded access submissions
  • Safeguards to protect patients and the clinical trial process

The regulations also include general criteria for granting expanded access:

  • The patient must have a serious condition or disease for which there is no comparable alternative therapy available
  • The patient must be unable to participate in a clinical trial
  • The potential benefit must outweigh the potential risk of using the treatment
  • There should be no impact on the completion of the clinical trial or the drug’s approval

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