Comments of Members of the Patient, Consumer and Public Health Coalition on “Manufacturer Communications Regarding Unapproved Uses of Approved or Cleared Medical Products”

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Comments of Members of the Patient, Consumer and Public Health Coalition on “Manufacturer Communications Regarding Unapproved Uses of Approved or Cleared Medical Products”

(Docket No. FDA-2016-1149)

Members of the Patient, Consumer and Public Health Coalition strongly oppose the off-label promotion of approved or cleared medical products.  There are valid reasons for doctors to consider off-label treatments, and some off-label use is common practice. However, doctors and patients should discuss the use of these off-label treatments with a clear understanding of the lack of clinical evidence, the doctor’s reasoning for choosing off-label treatment, and the risks.  Patients should have informed consent, which includes signing a piece of paper that explains the product is not approved by the FDA for the indication that the product is being prescribed for, and it also should include a discussion of what that means regarding the lack of objective evidence that the benefits outweigh the risks for most patients. 

We are very concerned that the FDA does not have the resources to adequately monitor such promotion and patients will be harmed.   Off-label promotion has one overriding goal:  to increase the use of medical products for patients and for conditions for which the product is not approved by the FDA.  It is not approved because the sponsor has not proven to the FDA that the product is safe and effective for that indication.  Without that evidence, the product can harm patients in several ways. First, patients will be more likely to be prescribed treatments that are not effective for their specific condition.  Second, patients are more likely to be prescribed treatments that are not safe for their specific condition.  And third, patients are more likely to be prescribed treatments that are more likely to harm than to help them.

It is impossible for FDA or any other agency to ensure that off-label communications are truthful and scientifically sound.  That would be a very resource-intensive task far beyond the resource capabilities that FDA possesses.     

Limiting what a company can say to only published clinical trials is not sufficient.  It would not guarantee that the information is truthful and non-misleading.  Clinical trials can be biased by design, execution, or selective reporting.  They can be published in journals without peer review or minimal standards.  Unfortunately, some journals will publish almost anything submitted.  Numerous Coalition members serve as peer reviewers, and have reported that even publication in a peer-reviewed journal does not guarantee that the clinical trial results and interpretation are unbiased and accurate.

Some medical products which look promising initially later appear less so when FDA reviewers identify major concerns with the study or data.  That critical check and balance process is why we believe in the importance of FDA and why we oppose the promotion of medical products for off-label uses.

Increasingly, FDA is approving drugs and devices on small, preliminary studies.  In the case of devices, more than 95% are not required to do any clinical trials.  Frequently, the apparently promising results of small exploratory studies or clinical experiences fail to be confirmed when tested with a larger, controlled trial.  That means that even the use approved by FDA may be less safe or less effective than was thought, making off-label promotion even riskier.

To make the situation worse, companies have few, if any, incentives to robustly test their product once it is on the market, either for its approved indication or other indications.  The companies and the lawyers they hired to speak at the FDA public meeting want to promote their products for unapproved uses so that they can sell more products. Moreover, the companies have said that they believe promotion of their products is protected as free speech.  If the medical product sponsors value free speech so dearly, why do firms that settle with patients who have been harmed by such products insist on non-disclosure agreements that make it impossible for those patients to exercise their own free speech and share information with other patients and their physicians? 

There is little incentive for medical product sponsors to disseminate research results which show that their products do not work, so that information rarely sees the light of day.  It’s not difficult to find cases on the Internet where a product worked for at least one patient. However, conversely, it’s not easy to find where patients were harmed, because that information is rarely published or disclosed.

Medical product manufacturers have a history of using misleading information to promote off-label uses.  These practices are rampant in the pharmaceutical industry. Almost all of the major drug manufacturers have been involved in lawsuits for improper off-label marketing tactics.  Collectively, they’ve been fined hundreds of millions of dollars. These hefty fines and legal settlements have apparently not discouraged off-label promotional violations, and neither the FDA nor the Justice Department have been able to curtail these abusive practices.  Legitimizing off-label promotion in the medical device arena will make these abuses even worse.

Some argue that FDA should allow off-label promotion, as long as the agency ensures that the information is “scientifically sound, responsibly presented, and provides as full an understanding as possible about the limitations of available evidence.”  That sounds good, but apparently assumes that the research results that the sponsor is distributing are truthful and accurate, and not distorted by selectively reporting information, or has serious flaws that may not be made clear to readers. That is not a safe assumption to make.  

In summary, we strongly oppose allowing companies to promote medical device products for off-label uses.  There are not adequate safeguards to allow such promotion to be limited to only scientifically sound, complete and unbiased data.  While some patients are helped by off-label uses, many also are harmed by the same poorly-regulated practices. Our concern is that expanding off-label promotion to medical devices will increase the number of those patients harmed.

When companies submit data to the FDA, agency reviewers have the expertise and responsibility to review those data.  That is an essential part of the process. Allowing off-label promotion conflicts with that process at the same time it removes an important incentive for companies to complete high quality clinical trials for new indications.

Thank you for the opportunity to present the views of the Coalition.  The under-signed member organizations of the Coalition represent many thousands of physicians, medical experts, patients, and advocates.

American Medical Student Association

American Medical Women’s Association

ASHES (Advocating Safety in Healthcare E-Sisters)

Association for Medical Ethics (AME)

Association for Pelvic Prolapse Support

Breast Cancer Action

Center for Medical Consumers

Community  Catalyst (MA)

Connecticut  Center for Patient Safety

Consumer Federation of America

Consumer Reports

Consumers Union of Texas

Consumers Union of Washington, DC


Essure Issues

Institute for Ethics and Emerging Technologies

Jacobs Institute of Women’s Health

Kids in Danger

Medshadow Foundation

MISSD (Medication-Induced Suicide Prevention & Eduction Fund in Memory of Stewart Dolin)

Mothers Against Medical Error

MRSA Survivors’ Network

National Alliance for Hispanic Health

National Breast Cancer Coalition

National Center for Health Research (NCHR)

National Committee to Preserve Social Security and Medicare

National Consumers’ League

National Organization for Women

National Physicians Alliance (NPA)

National Women’s Health Network

Our Bodies Ourselves

Project on Government Oversight (POGO)

Quinolone Vigilance Foundation

The TMJ Association

Union of Concerned Scientists

Washington Advocates for Patient Safety (WASPS)

Women Heart


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