Off-Label Prescribing – Danger or Lifesaver ?

2 issues obsess me today: Prescribing off-label use of drugs and marketing off-label use of drugs to doctors. Here’s why.

1. Why can doctors prescribe any drug for any reason? It’s called “off-label prescribing” and it’s very common. Every drug in the US has to receive FDA approval before it can be marketed or prescribed. The FDA has to determine that it is safe and effective for the medical issue as studied and presented to the FDA. (Read about the FDA’s move to loosen some rules in Sarah Smedley’s blog, FDA May Loosen Rules on Off-Label Drug Use.)

Here’s the catch — the FDA does not regulate the practice of medicine. So once the drug passes the FDA hurdle for a limited, specific use, doctors can prescribe it to any patient for whatever condition the doctor decides. If the doctor strays too far afield he/she might run the risk of malpractice, but generally doctors are protected if others have used the drug that way. It’s called “standard of care” — if Dr. A can say, “Dr. B did it first,” then Dr. A is not liable for the damage a drug might do, even if she or he didn’t warn the patient.

I’m concerned on several levels:


When you doctor prescribes a drug ask if the use will be off-label. If the answer is yes, ask questions:
  • Has this drug been tested on people with the same condition just not in my “special population” (age group or complicating factors like diabetes, obesity or pregnancy)?
  • Why the doc considers this the best option?
  • Is this off-label use common, and how long has it been used for this off-label condition?
  • Are there studies for using it for this off-label condition?
  • What are the side effects?
  • What are the other treatment options?

Informed consent
There is no obligation to tell the patient that the drug has never been tested or approved by the FDA for the condition your doctor is treating you. For example, the FDA has approved Zofran for the prevention of nausea and vomiting associated with initial and repeat courses cancer therapy, and for the prevention of postoperative nausea and/or vomiting. The pharmaceutical owner of Zofran, GlaxoSmithKline, has never sought FDA approval of Zofran for treatment of morning sickness in pregnant women. Yet Zofran has been prescribed to many pregnant women — Zofran is presently linked to serious heart defects among some of the children of those women who took the drug in pregnancy and there are several lawsuits pending.

Indication creep

Drug companies can get a drug approved for one very narrow indication and then doctors can prescribe it for others. One could call it “indication creep.” This circumvents the entire FDA process.

Here’s what The Mayo Clinic has to say: “… medications are often prescribed for off-label drug use with poor or absent clinical evidence. Radley et al reported that 73% of medications prescribed for an off-label use had poor or no scientific support. In critical care patients, off-label drug use was without adequate evidence 48.3% of the time. Because off-label drug use is typically less critically evaluated than is on-label drug use, off-label drug use may be associated with an increase in medication errors.”

Crossing specialties

Doctors with no special training in an area of care can prescribe a drug that should only be prescribed by a specialist. Some drugs are incredibly powerful and addicting, like oxycodone. If a patient is in enough pain to consider using oxycodone, then the patient should be under the care of a pain specialist.

2. Should off-label use be marketed to doctors?

One drug company is challenging the FDA on marketing limits. Pharmaceutical company Amarin wants to have its sales reps legally show doctors published studies on a use for their drug that the FDA has disapproved.

The drug is a prescription level omega-3 fatty acid product called Vascepa. It was previously approved for use in patients with “extremely” high triglycerides. When Amarin asked to expand the indication for use with “severely” high levels the FDA said no. The FDA noted that Amarin has not shown that lowering triglycerides by using Vascepa leads to improved cardiovascular outcomes — it hasn’t proved that it saves lives. Amarin does have a long-term study underway to attempt to show improved mortality outcomes, but it will take several more years and the funding will be difficult for such a small company. (Here’s a good article about it in the New York Times.)

However, Amarin wants to “share” with doctors any published study about using Vascepa for “severely” high level use. That’s “free speech,” according to Amarin. Since the FDA just disapproved that indication, of course it is opposed.  Fiercebiotech.com claims it’s all about marketing a broader use than approved for to doctors. As Sarah Smedley points out in her blog, the FDA is considering relaxing the rules for marketing drugs for off-label use to doctors.

Not all off-label prescribing results in harm and often it does a lot of good. A Washington Post article in 2014 reported many problems with off-label prescribing but gave examples of positive uses. In the article, Consumers should be wary when a doctor prescribes a drug for ‘off-label’ treatment, the writer points to Avastin (Bevacizumab) which was approved for various cancers. It is used off-label for wet age-related macular degeneration. “Note: There’s good evidence that it helps, and Medicare usually covers it. Risks may include eye infection, retinal detachment, bleeding and increased eye pressure. More serious side effects have been seen in cancer patients taking a higher dose.” However, if it really works, it should be allowed.

There are also certain population groups that are not often well represented in clinical trials and therefore most drugs are not indicated for use by them — children, the elderly, pregnant women. people with chronic diseases for example. One report from the Mayo Clinic found that “78.9% of children discharged from pediatric hospitals were taking at least 1 off-label medication.” If doctors were restricted from using drugs off label, special groups like children would not have access to needed drugs.

The FDA and the medical profession needs to determine how best to regulate off-label drug use so that doctors can carefully try off-label uses while still protecting the general population from becoming test subjects without their knowledge.

Until that happens people like you and me need to need to know and understand what we are getting into.

When you doctor prescribes a drug ask if the use will be off-label. If the answer is yes, ask the questions suggested in the box above.

THEN, take a deep breath and ask the doc for time to research the off-label use of the drug.


Suzanne B. Robotti

Suzanne Robotti founded MedShadow Foundation in 2012. Learn more about Su and her mission.


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