“Off-Label” Drug Use: Pursuing Profits at the Expense of Safety?

Let me state the obvious: companies sell you stuff with one purpose in mind, to make money. McDonald’s doesn’t sell you quarter pounders because the company believes what you need to live a better life is to eat more quarter pounders. The NRA doesn’t advocate gun ownership because it believes you need to own five Glock 9s (you don’t), but rather so the gun makers can sell more guns. Mercedes doesn’t make expensive cars because its board of directors hope to improve the world by selling you cars with heated leather seats. Every company sets out first to last to make money. And the more money the better.

So it goes with pharmaceutical companies. The general public may sleepwalk through the concept and lazily presume that the primary motivation for drug companies is to develop medications which cure diseases or which minimize the suffering from diseases. But in fact the motivation for pharmaceutical companies is to make money, and a lot of it. This is rather obvious and not a controversial point, and I’d like to believe that every “BigPharma” corporate board would agree with me. But it helps to keep this profit motive in mind when doing research on drugs you have been prescribed or which you are currently taking. And to be hyper-vigilant about assessing any new “wonder drugs” which hit the market.

“Off-Label” Drug Promotion

Recently, the pharmaceutical industry strenuously objected to new regulations implemented by the U.S. Food and Drug Administration (FDA) on off-label drug uses. Essentially, several advocacy groups supporting the pharmaceutical industry filed petitions opposing heightened restrictions on the marketing and sale of drugs for unapproved or “off-label” uses.

Let’s back up.

There are approved and unapproved uses for prescription drugs. For approved uses, the drug has been thoroughly tested and evaluated. The FDA has determined that the benefits and risks are acceptable and that this determination is backed by “strong scientific data.” Finally, for approved uses, the drugs have labels which set out clearly how to use the drug and for what specific purposes.

Then there are unapproved uses. Once the FDA approves any drug, your doctor is then allowed to prescribe the drug to treat another condition which is not listed as an approved use. Basically, once the FDA green-lights a drug, it relies on competent, unbiased physicians to make the best decisions for their patients in prescribing the drug, for whatever purpose.

Doctors may prescribe a drug for an unapproved use when the doctor believes the drug will help the patient in some way and there is no other drug available to help the patient. The FDA uses the example of a cancer drug approved for one type of cancer, but the oncologist prescribes it to treat a different form of cancer that does not have a similar medication available for treatment.

This sounds reasonably benign, even helpful, but drug companies can be aggressive in promoting off-label use. I have written about troubling off-label uses on this site. One off-label drug use nightmare occurred when doctors prescribed Risperdal for adolescent boys with certain behavior issues. Risperdal was originally approved solely to treat adult patients with schizophrenia. But Johnson & Johnson pressed for FDA permission to market the drug to treat other conditions, such as bipolar disorder and autism, and eventually to permit use in children. It was then expanded further to treat adults and children suffering from attention deficit disorder, anxiety and depression. As it turned out, Risperdal can promote the growth of female breast tissue (“gynecomastia”). Female breasts on adolescent boys is a terrible and traumatic disfigurement. And thousands of lawsuits have resulted from these injuries.

Thus, it is critically important that off-label drug use and off-label drug promotion are monitored carefully. You would think that the pharmaceutical industry would wholeheartedly agree, but  . . .

BigPharma Objects to New FDA Off-Label Regulations

The FDA recently published its latest guidelines on regulating off-label drug use. The guidelines give the FDA new authority to police the selling of prescription drugs for unapproved uses. Specifically, the FDA focused on “intended use,” which is “the objective intent of the persons legally responsible for the labeling of drugs.” The goal is to make sure the companies promoting a drug are not pushing for it to be used in an unintended way. Intended use analysis is helpful to the FDA as it “helps the FDA gauge whether companies are intentionally marketing products for unapproved uses.” See FDA Website. The new rule was supposed to give more power to the FDA to hold manufacturers liable for promotion of off-label, unapproved uses of drugs.

The drug industry has objected, calling the new rule a “vague standard” with “no support in existing law.” At the end of the day, the drug companies simply do not want to be held liable for getting caught promoting drugs for off-label use. The industry’s position seems to be “get out of our way and let us make as much money as possible.”

They may get their wish. Recently, President Donald Trump has issued a “regulatory freeze” on all FDA rules and regulations. From what I’ve seen coming out of Washington lately, regulations on BigPharma are likely to get weakened, not strengthened, over the next four years. If you do not do your own research, you may be vulnerable to prescription drugs with unwelcome and harmful side effects.

What to Ask Your Doctor:

If your doctor prescribes a drug for an unapproved use, you should ask:

• What was the drug originally approved for?
• Why am I getting it for my condition?
• Are other drugs available that have been approved to treat my medical condition?
• Do studies support the use of this drug to treat my condition?
• Will this drug work better to treat my medical condition than using an approved treatment?
• What are the benefits and risks of treating my medical condition with this drug?

As always, be careful out there.