Testimony to FDA panel on consideration of off-label promotion of medical devices

November 10, 2016

Thank you for the opportunity to speak today.  My name is Jack Mitchell, and I’m the director of government relations for the non-profit National Center for Health Research, which performs public health research and conducts patient advocacy.  This morning I’m speaking on behalf of the Patient, Consumer and Public Health Coalition, to which NCHR belongs and helps to coordinate.

The Coalition includes both large and small nonprofit organizations across the country that are united to ensure that medical treatments are safe and effective, and to enhance the scientific and public health focus of the FDA.  We represent millions of patients, consumers, researchers, and medical professionals.

Our coalition has opposed previous efforts to allow the promotion of off-label use, and we still continue to strongly oppose it.

There are valid reasons for doctors to prescribe treatments off-label.  Many drugs are prescribed off-label as common, accepted practice. However, doctors and patients should discuss the use of these treatments with a clear understanding of the level of evidence, the doctor’s reasoning, and the risks.

Patients should have informed consent, which includes signing a piece of paper that explains that the product is not approved by the FDA for the indication that the product is being prescribed for.  It should also include a discussion of what that means regarding the lack of objective evidence that the benefits outweigh the risks for most patients.  That is the critical calculus that the FDA must always balance.

We are concerned that the FDA does not have the resources to monitor such increased off-label promotion and patients will be harmed.

In our view, the off label promotion for medical devices has one primary goal: To increases the use of medical products for conditions and indications for which the product is not approved by the FDA.  It is not approved because the sponsor has not adequately proven to the FDA that the product is safe and effective for that particular indication.  The result can be that:

  1. Patients will be more likely to be prescribed treatments that are not effective or safe for their specific condition.
  2. Patients also may be more likely to be prescribed treatments that are may be more likely to harm than to help them.

It is virtually impossible for FDA or any other agency to ensure that off-label communications are uniformly truthful and scientifically sound.  That would be an extremely resource-intensive task at an agency where resources are often stretched to the very limit.

Representatives from our collation have testified at FDA advisory committees regarding medical products.  Some of these products have looked promising in the companies’ presentations trials, but then FDA reviewers identified key problems that raised major concerns about the study or the underlying data.  In other words, the entire FDA process is based on the assumption that a sponsor’s analysis is not always unbiased and of sufficient scientific quality to merit FDA approval.  That is why we believe in the critical importance of FDA’s role and why we oppose the promotion of medical products for off-label uses.

As I mentioned, off-label uses are already widespread and commonplace.  For many drugs and devices, in fact, a significant proportion of use is off-label.  Unfortunately, research shows that off-label uses increase the number of adverse events.  If promotion of off-label usage is allowed, we believe, more patients will inevitably be harmed.

Increasingly, FDA is approving drugs and devices based on smaller, preliminary studies.  In the case of medical devices, the vast majority 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 further tested with a larger, controlled trial.  Sponsors already have few, if any, incentives to robustly test their product once it is on the market, either for its approved indication or for other indications.

Some observers have suggested that increased transparency or data sharing about clinical trials could allow practitioners and patients to evaluate clinical trials themselves.  Supposing that there is sufficient data for us to evaluate the data and that it is not selectively released, we believe that many medical professionals and nonprofit organizations have neither the time nor the expertise to do so.

Those advocating for off-label promotion of their products contend that such claims are protected as free speech and that restriction off-label promotion is an unfair abridgement of that free speech.  As a lapsed and recovering journalist, I’m a big fan of the First Amendment.

However, if it is true that not allowing promotion of off-label uses of medical devices is a restriction of free speech, then why do companies which settle lawsuits with patients who have been harmed insist on non-disclosure agreements that make it impossible for those patients to exercise their own free speech?  Why are patients who settle lawsuits so frequently not allowed to speak publicly about what happened to them?  We believe such restrictions are also another barrier or impediment to identifying patterns of adverse events involving medical devices.

Companies don’t have an incentive to disseminate clinical trials that show that their product does not work.  It’s relatively easy to find cases where a product worked for at least one or a few patient with a simple internet search.  Such information is more difficult to find where patients were harmed, because it is either not published or not promoted.

The history of promoting off-label use in the pharmaceutical world has been dismal.  This list of major violations includes almost all of the major drug firms.  Each has been successfully sued for improper off-label marketing tactics.  Collectively, they’ve been fined hundreds of millions, if not billions, of dollars.

As former chief of oversight and investigations for the Senate Special Committee on Aging. I’ve seen the devastating human impact of the over-use and massive off-label prescribing of antipsychotic drugs in nursing homes, for which multiple pharmaceutical companies have been fined hundreds of millions of dollars.

Nevertheless, those hefty fines and legal settlements have not discouraged off-label promotional practices, and many violations have been made by repeat offenders.  Neither FDA nor the Justice Department has been able to curb these abusive off-label practices with lawsuits and huge fines.  It is hard to believe that legitimizing off-label promotion for devices will not make these type of abuses even more widespread.

Proponents of device of-label promotion have argued that FDA should allow promotion of off-label uses, but that the agency must insure that it is “scientifically sound, responsibly presented, and provides as full an understanding as possible about the limitations of the available evidence.”  That sounds reasonable but it apparently assumes that the clinical trial results that the company is distributing are truthful and aren’t distorted by selectively reporting information, or have serious flaws that may not be apparent.

In summary, we strongly oppose allowing the promotion of device products for off-label uses. We believe that there are not adequate safeguards to allow such promotion to be limited to only scientifically sound, complete, and unbiased data.

While admittedly some off-label use is accepted and has positive outcomes, as you are hearing from patients’ testimonies at this meeting, many have been harmed by off-label use.  The patient Jeremy Lew related yesterday the painful details of coming very close to death or complete paralysis because a device was surgically inserted into his neck which was not approved by FDA for that purpose, but was advertised as being approved.  As you’ve heard, this is not an academic or legal debate only—there are huge human costs and immense suffering.

Our concern, simply stated, is that allowing off-label promotion will cause more patients to be harmed than helped.  When companies submit data to the FDA, agency reviewers have the expertise and responsibility to thoroughly review such data.  That careful process is what has kept Americans more safe from medical harm since the creation of FDA more than seventy-five years ago.

Allowing off-label promotion conflicts with that delicate balance and tends to remove an important incentive for companies to complete high-quality clinical trials for new indications; which would produce reliable data of both efficacy and safety.

I worked in the FDA Commissioner’s office for seven years, and I know the dedication that agency employees have.  They will get the job done when they have access to the appropriate resources and information necessary to do perform their life-critical responsibilities.   Thank you.