Five Examples of Off-Label Medical Treatments That Work

Off-label medical treatments are medications or therapies often used by doctors to treat conditions despite the treatments not being a traditional course of treatment for that particular problem. It's surprisingly common these days, as many therapies were initially developed to treat a specific issue (such as epilepsy) that have turned out to be effective in treating other problems (such as headaches). Because it may take years of clinical trials and jumping through hoops to get the new application approved by the FDA, doctors prescribe the treatments "off-label" based on their effectiveness. They are secure in knowing that the treatments are safe because they've already been approved for other uses. 

Is It Okay to Use Off-Label Medications?

Off-label prescription use is legal and is quite common. You may be surprised to learn that medical experts estimate that one of every five prescriptions written in the United States is for off-label use. Most commonly, the medication is used for a medical condition that hasn't been approved to treat but using higher dosages than supported also falls under the off-label heading. In some situations, particularly for treating cancer and severe pain, off-label drugs approved for the treatment of adults are used to treat children. 

The FDA recognizes that seeking approval for every medication for every possible treatment option is an expensive, lengthy, and sometimes impractical burden. The FDA allows doctors to practice medicine as they see fit as long as the medication has been approved for use, recognizing that physicians will prescribe the best medicines for their patients. However, they prefer off-label drugs or therapies to be used only after other medicines explicitly designated for the condition being treated have failed or been insufficient. Several classes of off-label treatments and medications have become almost mainstream, while others are still being debated. 

Anti-Seizure Medications

There are numerous seizure medications on the market to treat epilepsy and other disorders that cause seizures. Anti-seizure medications are prescribed to control migraines as they are to prevent seizures. In some situations, a combination of seizure medications and other off-label drugs such as beta-blockers is the only effective way to prevent severe, chronic migraines. There are currently over 50 anti-seizure medications frequently prescribed to treat migraines, depression, nerve pain, and other conditions. While treating patients with anti-seizure drugs, doctors realized interesting. Positive side effects could help patients with other conditions.

Anti-Psychotics

Anti-psychotic medication is approved for the treatment of Schizophrenia, but many doctors also use it to treat or slow down the progression of other neurological conditions. It is increasingly prescribed for children and adults with Attention Deficit Hyperactivity Disorder (ADHD) and autism. In the elderly, anti-psychotics can help slow the progression of dementia and Alzheimer's Disease. 

Antidepressants

This is one of the most commonly used classes of medication that is prescribed frequently for other conditions, often in combination with other medicines. Bipolar disorder is challenging to treat and usually has a feature of depression or mood swings that consistent antidepressants can minimize. Other indications include ADHD and chronic pain. In the case of chronic pain, there may be a reciprocal effect – pain can lead to depression, and depression may manifest as pain. The blurred line between the two is why many doctors choose to incorporate antidepressants into a treatment program for chronic pain syndrome.

Chelation Therapy

Chelation therapy was developed in the 1950s to treat metal poisoning, especially lead poisoning. Chelation therapy flushes heavy metals and other toxins out of the body by removing them from the bloodstream. The intravenous treatment introduces a chelating agent into the body that eliminates the toxins. Although chelation therapy is currently approved for treating metal toxicity, some practitioners use it for patients who believe the chelating agent can aid in re-opening clogged arteries. For patients who have atherosclerosis, chelation therapy is a less invasive treatment than angioplasty or bypass surgery. It also has fewer potential side effects than some medications prescribed for hardening of the arteries. 

The Institute of Natural Health is a leader in the use of chelation therapy for treating clogged arteries. The treatment is also believed to improve overall body and brain function, including improved memory and vision. Typical off-label treatment includes several intravenous treatments over several weeks, although oral medications are sometimes prescribed as an alternative. 

Beta-Blockers

High blood pressure and heart disease are often treated via beta-blockers. Still, many physicians have discovered they are also effective for treating various other problems, including migraine headaches and anxiety. Because beta-blockers prevent adrenalin from reaching the heart, forcing it to pump harder and faster, it also reduces the body's fight or flight response, reducing stress. 

Interestingly, there are several different categories of beta-blockers that have varying levels of effectiveness for patients with anxiety. Some work best for short-term, situational anxiety in individual doses, while others are more effective for long-term, chronic stress. Off-label prescriptions may be for occasional or daily use, depending on the type of anxiety being treated.

Does Insurance Cover Off-Label Therapies?

If you're considering an off-label treatment or preventative medication, be sure to talk to your health insurance carrier. In some cases, the treatment is covered by insurance, but not always. There are several criteria they will consider, including whether there is a clear indication that the treatment, despite not being FDA approved, is commonly accepted by the medical mainstream to treat your condition. In some cases, prior authorization may be required.

Talking to Your Doctor About Off-Label Therapies

Always discuss suggested off-label therapies with your physician, asking about the benefits and the side effects of using the medication. Your doctor should explain why they are proposing an off-label drug or treatment (it's often the most productive one available) and what the track record is for its effectiveness. Work with a doctor who is experienced in providing the treatment and can oversee your treatment effectively, particularly for long-term treatments such as chelation therapy. Once you have all the facts, you can make an informed decision about whether off-label treatment is right for you.