On November 1, 2019, the Food and Drug Administration’s (FDA)Office of Prescription Drug Promotion (OPDP) sent an Untitled Letter to the sponsor of an investigational new drug that is being studied to treat brain cancer. According to OPDP, the sponsor’s corporate website represented in a promotional context that the investigational new drug was safe and effective for the treatment of brain cancer, in violation of 21 C.F.R. Section 312.7(a). OPDP found this website to be concerning because of the seriousness of this disease and the relatively few available treatment options.
We will briefly describe OPDP’s objections and note our observations.
- The letter included three statements from the website that OPDP found to be objectionable:
- “[Investigational new drug] has cured a rare form of brain cancer.”
- “Delivering human antibodies for the treatment of cancer.”
- “After 5 years, patients treated with [investigational drug] have an overall survival rate of 25-30%, compared to 3% standard therapy, demonstrating antibodies are safe and effective.”
OPDP determined that these “conclusory statements” suggest that [investigational new drug] has been established as being safe and effective in treating brain cancer.
- Additionally, OPDP noted that the website did not include information to clearly indicate that the drug was an investigational new drug that has not been approved for commercial distribution in the U.S.
This Untitled Letter reminds us that:
- Sponsors of investigational drugs must clearly indicate on websites and other materials that the products are investigational and have not been approved for commercial distribution.
- Sponsors must not represent or suggest that an investigational new drug is safe or effective. It is appropriate to accurately describe the results of clinical studies (acceptable “scientific exchange”). However, as this sponsor found out, it is the use of conclusory statements or representations regarding the safety and effectiveness of the investigational new drug (e.g., “cured” “for the treatment of cancer” “demonstrating antibodies are safe and effective”) that OPDP considers promotional, and thus violate the investigational new drug regulations. Websites and other communications about investigational new drugs should be reviewed by the sponsor’s internal review committee, which include medical, regulatory, and legal representatives.
- We continue to see the agency focusing on pre-approval promotion. Off-label promotion enforcement remains a thorny legal issue for FDA; pre-approval promotion does not.