July 1, 2009
Submitted via Docket No. FDA-2009-N-0247 at www.regulations.gov.
While we understand and support the incentives that are necessary to attract private, commercial investment capital to the development of important new medical therapies, we have grave concerns that confidentiality measures intended to protect trade secrets may be used also to hide unfavorable information.
If, for example, the FDA halts cancer research on patients for whatever reason, current regulations permit the FDA to communicate only with the drug sponsor, usually a company or academic institution. This means that trial participants and patients making clinical trial choices can get their information only from the sponsor. The FDA cannot correct misinformation or offer their perspective on the problems in the clinical trial to anyone without the sponsor’s permission.
Similarly, there are occasions when an unapproved drug is being tested in a clinical trial design which the FDA sees as questionable. The public, including patients and investors, are not usually aware of these concerns unless the FDA discusses them at an FDA public advisory committee meeting. If questionable trial design leads to FDA disapproval, the public may never know why the drug was not approved. A drug company might disclose vague information about the trouble their drug has encountered, but if this information is misleading, the FDA is prohibited from refuting the drug company’s account.
These situations are not unusual due to the proprietary information regulations as defined and enacted by the FDA. The FDA regulations prohibiting disclosure of proprietary information were intended to protect corporate trade secrets and financial information. This prohibition has created an impenetrable shield behind which the FDA and drug companies hold their discussions.
In 2001 and 2002, the so-called Martha Stewart insider trading scandal provided an example how transparency can change the public perception of drug development. Because of wide-spread publicity in 2001 about ImClone’s anti-cancer agent cetuximab, thousands of cancer patients were encouraged and anxiously awaited its approval as repeatedly implied by ImClone press releases and statements. Their optimism proved unwarranted on December 28, 2001 when ImClone’s new drug application for cetuximab was rejected for review by the FDA. A patient reading ImClone’s press release about the FDA’s action would have thought that the FDA’s refusal to file action was just a minor bump in the drug approval road. However, the “Refusal to File” letter was leaked to The Cancer Letter, and the public was able to learn that FDA had serious concerns with deficiencies in the application. These deficiencies took over two years to correct.
The public had a unique view into this situation because FDA’s “Refusal to File” letter was leaked. Subsequent disclosures not only resulted in the conviction of both Martha Stewart and Samuel Waksal (CEO of the company) for insider trading, but also resulted in Congressional hearings and articles that illustrated how the lack of transparency during drug development can mislead and potentially harm patients and investors.
Another example where the lack of transparency misled the public is with erythropoietin-stimulating agents (ESAs). ESAs were first approved by FDA for use in oncology in 1994. Snippets of research results between 1994 and 2007 indicated that off-label use of ESAs might be linked with an increase in mortality; however, until FDA put the issue into the public eye at the May 2007 ODAC meeting, the public was not privy to the gravity of FDA’s concerns. Ultimately, a new label which restricted use of ESAs was issued by FDA in July 2008, 14 years after initial approval. If FDA had been able to take its concerns public earlier, could the timeline have been shortened?
The FDA is charged with protecting the public. We believe fulfillment of this charge should allow the FDA to permit appropriate public disclosure of necessary information to patients without compromising proprietary interests. We believe that it is time to review disclosure regulations, with an eye to increasing public availability of information at certain critical times in the drug review process, especially in the rare event that accurate and complete information is not provided by the sponsors.
Please do not hesitate to contact us directly if we can be of help in this important effort.
Carlea Bauman, President
C3: Colorectal Cancer Coalition
These comments are submitted on behalf of C3: Colorectal Cancer Coalition (C3), a non-profit, nonpartisan advocacy organization that is committed to the fight against colon and rectal cancer. We appreciate the opportunity to comment on the Food and Drug Administration’s (FDA) consideration of appropriate regulatory transparency.
C3 pushes for research to improve screening, diagnosis, and treatment of colorectal cancer; for policy decisions that make the most effective colorectal cancer prevention and treatment available to all; and for increased awareness that colorectal cancer is preventable, treatable, and beatable. C3 believes in fully disclosing sources of financial support, per our disclosure policy which can be viewed at www.FightColorectalCancer.org/funding.htm. C3 has received funding from Amgen, Bristol-Myers Squibb Oncology and ImClone Systems, in the form of unrestricted educational grants. Neither these companies nor any of our other corporate supporters has influenced our comments on this issue.