Email from Robert “Skip” Nelson of the FDA to Alice Dreger, cc Ellen Feder and Anne Tamar-Mattis, dated September 8, 2010, in response to this: follow_up_to_FDA_090310.pdf

Dear Dr. Dreger,


FDA normally does not discuss an investigator's activities with those not involved with their work. In our memorandum to OHRP, our office provided a summary of our factual findings and a brief review of the safety and efficacy of the use of Dexamethasone for prevention of virilization in female fetuses with congenital adrenal hyperplasia (CAH). Other than some additional clarification of these factual findings, we will not be answering any further queries.


An IND exemption is issued for the specific clinical investigation contained in the IND application. As the available evidence indicates that Dr. New is no longer conducting an FDA-regulated clinical investigation using Dexamethasone for the prevention of virilization in female fetuses with CAH, the IND exemption is no longer in effect. The fact that an IND exemption was issued in 1996 would have little bearing on how FDA would assess a future IND application for the use of Dexamethasone for preventing fetal virilization due to CAH. We will not speculate on how FDA would respond to a future IND application.


There are other circumstances (besides being employed by a sponsor) in which a clinical investigator could be in violation of FDA regulations governing the promotion of an unapproved use of an investigational drug. However, Dr. New is not conducting an FDA-regulated clinical investigation and thus these regulations do not apply. FDA regulations do not forbid physicians from using (and recommending) medications for an "off label" indication, presumably based on their professional judgment that the use of that medication is safe and effective. Our memorandum to OHRP clearly states that the use of Dexamethasone for preventing fetal virilization from CAH has not been approved by FDA as safe and effective. Concerning the on-going clinical use of Dexamethasone, FDA does not regulate the practice of medicine. As for your concerns about the promotion of this "off label" use, FDA has an office staffed with appropriate legal and regulatory experts who review advertisements and make determinations as to their appropriateness. An important consideration is whether the advertisement is or is not protected "speech" under the First Amendment to the U.S. Constitution. Our office does not have expertise in this area.


We are aware of the controversy about the continued use of Dexamethasone for the prevention of virilization in female fetuses with CAH. You raise some of the issues that must be taken into account in addressing the appropriateness of this on-going clinical practice. Our memorandum clearly states that further prospective studies are required to determine whether prenatal treatment with dexamethasone should be routinely recommended. The use of Dexamethasone in a prospective clinical investigation would require an IND application.


Robert "Skip" Nelson, MD PhD

Pediatric Ethicist, Office of Pediatric Therapeutics

Tele: (301) 796-8665; Mobile: (240) 328-7146; Fax: (301) 847-8619

E-mail: Robert.Nelson@fda.hhs.gov

Mailing Address:
WO/Building 32, Room 5126
10903 New Hampshire Avenue, Silver Spring, MD 20993-0002

This communication does not constitute a written advisory opinion under 21 CFR 10.85, but rather is an informal communication under 21 CFR 10.85(k) which represents the best judgment of the employee providing it.  This information does not necessarily represent the formal position of FDA, and does not bind or otherwise obligate or commit the agency to the views expressed.


See our letter to the “Bad Ad” division here. See relevant FOIA responses here.