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Accessing Investigational Drugs Outside of a Clinical Trial

MCN Question of the Week

Accessing Investigational Drugs Outside of a Clinical Trial

QUESTION:

Is there a process for requesting access to an investigational drug outside of a clinical trial?

ANSWER:

Yes.  The US Food and Drug Administration has a process for expanded access.  Expanded access is the use of an investigational drug outside of clinical trials to diagnose, monitor or treat patients with serious or life-threatening diseases or conditions for which there are no comparable or satisfactory therapy options available.

When patient enrollment in a clinical trial is not possible (i.e., the patient is not eligible for any ongoing clinical trials or there are none available), use of an investigational product through expanded access may be possible.  Eligibility criteria for expanded access includes, but is not limited to:

  • The patient must have a serious or immediately life-threatening disease or condition.
  • There must be no comparable or satisfactory alternative therapy to diagnose, monitor or treat the disease or condition.
  • They generally must be unable to participate in a clinical trial.
  • The probable risk from the investigational drug is not greater than the probable risk from the disease or condition.

To obtain expanded access for a patient, the requesting healthcare provider should first contact the pharmaceutical company developing the drug.  The company may provide the drug to patients according to a pre-established protocol.  If no pre-established protocol exists, the pharmaceutical company should be asked for a Letter of Authorization (LOA).  Then requesting healthcare provider should submit an application, using Form 3926 to the FDA for expanded access on behalf of the patient.

The expanded access process also includes requesting approval from an Institutional Review Board (IRB) and obtaining informed consent from the patient for the use of the investigational drug. Once the request is authorized by FDA, the requesting healthcare provider is responsible for managing the patient’s medical care.

Should a patient need an investigational drug on an emergency basis before a written request can be submitted, the FDA can grant the request over the phone and the patient can begin treatment.  The healthcare provider must still submit an expanded access application to FDA within 15 days and notify an IRB within five (5) days of initiation of treatment.

Below are links to the FDA Form 3929 as well as additional FDA reference and guidance documents about requesting expanded use of an investigational drug.

References:

U.S. Department of Health and Human Services (DHHS), Food and Drug Administration (FDA), Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER).  (June 2016).  Charging for Investigational Drugs Under an IND— Questions and Answers, Guidance for Industry

U.S. Department of Health and Human Services (DHHS), Food and Drug Administration (FDA), Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER).  (June 2016).  Expanded Access to Investigational Drugs for Treatment Use — Questions and Answers, Guidance for Industry

 U.S. Department of Health and Human Services (DHHS), Food and Drug Administration (FDA), Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER).  (June 2016).  Individual Patient Expanded Access Applications: Form FDA 3926, Guidance for Industry

 Related Products from MCN Healthcare:

Individual Rights and Responsibilities Manual

 

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