New FDA Regulations for Compassionate Use of Investigational Drugs
(From the August 2016 issue of Research Now)
With an increasing number of requests for compassionate use (also known as expanded access use) of Investigational Drugs (not FDA-approved) for treatment of patients with serious or immediately life-threatening diseases or conditions, the U.S. Food and Drug Administration (FDA) has provided a more streamlined approach to gaining access to these potentially life-saving experimental drugs. The information below highlights some of the changes that the FDA has made:
The FDA has issued a new form (Form FDA 3926) specific to expanded access requests. The new form replaces the previous Forms FDA 1571 and 1752.
The Form FDA 1571 and 1572 will continue to be used for all other types of Investigational New Drug (IND) submissions.
Form FDA 3926 can also be used for certain follow-up submissions to the FDA for an individual patient expanded access IND including annual reports, safety reports and change in treatment plans.
A new guidance document has been issued in a question and answer format, which includes the definition of expanded access, categories of expanded access, and other information on how to gain expanded use access. This guidance describes criteria that must be met to authorize expanded access use, requirements for expanded access submissions, and safeguards that are intended to protect patients and preserve the ability to develop meaningful data about the safety and effectiveness of the drug through clinical trials or drug development.
If you have any questions regarding these changes or plan on seeking compassionate use or expanded use for a patient, please contact our FDA regulatory support group, Drug and Device Development Services, at Regulatory@NationwideChildrens.org, or call (614) 355-1827. Our regulatory specialists, Sue Marting, or Chris Shilling (director) will be able to assist you with gathering appropriate information, completing all forms and submitting the appropriate documentation to the FDA.