In the United States, potential new medicines are evaluated and tested through rigorous scientific research before they are approved for public use by the U.S. Food and Drug Administration (FDA). This includes pre-clinical research (animal studies) and clinical trials (studies done in humans). Clinical trials are important because they test the safety and efficacy of an investigational medicine. Advancing therapies through the clinical trial process and obtaining the necessary regulatory approval is the best way to provide access to the greatest number of patients. To learn more about the drug development process, visit our patient resources.

What is Expanded Access?

In some cases, the FDA will allow patients to receive investigational medicines outside of a clinical trial if the patient is not eligible for the trial or they have a life-threatening illness. This is called expanded access, pre-approval access, or compassionate use. Regulatory agencies and pharmaceutical companies evaluate a complex set of criteria when deciding whether to grant access to investigational therapies to individuals or a group of patients. Some of these include:

  • Whether a patient has a serious or life-threatening illness with no other satisfactory alternative therapies, including participation in a clinical trial.
  • Whether there is sufficient evidence that the potential benefit of the investigational medicine outweighs the potential risks.
  • Whether allowing access to the investigational medicine would interfere with the execution or completion of clinical trials that could support regulatory approval of the investigational medicine.
  • Whether there is adequate supply of the investigational medicine to support both clinical trials and an expanded access program.

Read more about expanded access or compassionate use from the U.S. FDA here.

Leadiant Expanded Access Policy

At this time, participation in clinical trials is the only way to gain access to investigational therapies in development by Leadiant. Speak to your physician about whether you are eligible for a clinical trial. Information about Leadiant-sponsored clinical trials can be found on www.ClinicalTrials.gov by entering the keyword “Leadiant” into the Other Terms search box.

Contact Information

If you have additional questions, please contact Joseph M. Wiley, MD, FAAP, Vice President, Medical Affairs, Drug Safety, and Pharmacovigilance, at (301) 670-2182 or joesph.wiley@leadiant.com.

You will receive a response to your inquiry within seven calendar days.