Last month the FDA’s Office of Prescription Drug Promotion (OPDP) issued a warning letter to the Burzynski Research Institute Inc. and the Burzynski Clinic regarding claims made for its antineoplastons A10 and AS2-1 injections that violate the Federal Food, Drug, and Cosmetic Act and FDA regulations 21 CFR 312.7(a). That regulation prohibits a sponsor or an investigator from representing an investigational drug in a promotional context as safe and effective while it is still under investigation.
While the OPDP’s move is a welcome step in shutting down Burzynski’s clinical trial pyramid scheme, in which, according to some patient fundraising blogs, trial participants pay more than $100,000 to become guinea pigs for antineoplaston therapy, the letter provides little clarity regarding what is or is not a promotional claim.
The letter specifies, “This provision is not intended to restrict the full exchange of scientific information concerning the drug, including dissemination of scientific findings in scientific or lay media. Rather, its intent is to restrict promotional claims of safety or effectiveness of the drug for a use for which it is under investigation and to preclude commercialization of the drug before it is approved for commercial distribution.”
However, some of the so-called violations strongly resemble claims made in countless biotech and pharma press releases posted on company websites.
For example, “ANP was well tolerated with easy manageable side effects of fatigue, skin rash, and electrolyte abnormalities and no chronic toxicities . . . These results compared favorably to radiation therapy and chemotherapy (Mandell, et al. 1999, 7 percent overall survival at 2 years and 0 percent at 5 years), but should be confirmed in phase III trials scheduled to begin in 2009.”
On the face of it, that statement seems identical to hundreds of statements made every day by other companies, so what distinguishes Burzynski’s regulation-violating promotion from permitted “exchange of scientific information?”
Clarity is on Hold
Crystal clarity on that question may be a long time coming. The biopharma community has been clamoring for the FDA’s guidance on a related question – how to use social media – for nearly two years, and the agency has answered with silence – a silence set to continue for another two-year period, under the new FDA Safety and Innovation Act.
That silence has led to a “gotcha mentality” that uses enforcement, rather than guidance, to communicate policy.
For companies that want to avoid running into trouble with their public communications, warning letters such as the Untitled Letter issued to the Burzynski Clinic are some of the only clues available as to what the regulations allow and do not allow.
Stephen King, a public affairs specialist for the FDA’s Center for Drug Evaluation and Research, offered a statement that provided a few more hints on the Burzynski case.
“The FDA takes seriously its role in assuring Americans the drugs they use are safe and effective and manufactured according to current good manufacturing practices (cGMP). The agency also seeks to ensure patients who are participating in clinical trials and expanded access programs are appropriately protected,” King said. “To date, no randomized, controlled trials showing the effectiveness of antineoplastons have been published in peer-reviewed scientific journals nor have all of the trials needed to approve antineoplastons as a treatment for cancer been conducted.”
So far, so good. But again the FDA’s communication falls short of total clarity as to why certain claims violate regulations and others don’t. In the Burzynski case, the clinic’s lack of evidence to back up its claims, and track record of plundering its patients’ retirement funds may contribute to the OPDP’s actions, but nothing in the OPDP letter actually cites false claims or misunderstanding by patients. “The totality of these claims suggest that Antineoplastons, investigational new drugs, are safe and/or effective for the treatment of various types of brain tumors indicated above, when they have not been approved for these uses,” the letter states, suggesting that the only and entire violation by the Burzynski clinic is that it said the words “safe and effective” before the drugs had been approved ‑ words which are ubiquitous in drug company statements regarding clinical trials of unapproved drugs.
“The promotion of investigational new drugs is prohibited by law and the agency is concerned BRI’s promotional claims will mislead patients about the safety and efficacy of unapproved antineoplastons,” said King, providing few hints as to how other companies should modify their language regarding safety and efficacy of their own unapproved drugs.
OPDP instructed the Burzynski Clinic to immediately cease dissemination of “violative promotional materials” for antineoplastons and to submit a written response by Nov. 1.
A company spokesman for the Burzynski Clinic, Azad Rastegar, told BioWorld, “Yes, we have received a letter from the FDA. While we don’t think we have done anything wrong, we have complied with their requests.”