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Why the Sudden Hostility Toward Off-Label Medicines?

The US Food and Drug Administration has an exhaustive process, “to evaluate new drugs before they can be sold.” Specifically, this involves the Center for Drug Evaluation and Research (CDER). The rationale is sound and for the general good:

The center’s best-known job is to evaluate new drugs before they can be sold. CDER’s evaluation not only prevents quackery, but also provides doctors and patients the information they need to use medicines wisely. The center ensures that drugs, both brand-name and generic, work correctly and that their health benefits outweigh their known risks.

Note the goal is neither absolute safety nor perfect efficacy. In other words, no drug is completely safe and without side effects, nor will a drug work every time, exactly as it was designed to. The key is the risk-benefit ratio, where the benefits outweigh the risks or costs.

Otherwise, air travel, driving, and walking would be prohibited as these activities all involve some risk — but that is far outweighed by their benefits.

Drugs are approved for a specific indication, at a particular dose, and this is on the product label, which is actually the multiple folded paper, attached to the medication container with print so small no one can or wants to read it. It summarizes indications, clinical trial results, side effects, warnings, and dosages, all determined by the registration trials leading to FDA review and approval.

When drugs are used as described by the label, this is referred to as “on-label” use. Insurance companies typically pay for on-label treatment. Conversely, “Unapproved use of an approved drug is often called ‘off-label’ use.” The FDA does not object to this,

“From the FDA perspective, once the FDA approves a drug, healthcare providers generally may prescribe the drug for an unapproved use when they judge that it is medically appropriate for their patient.”

Except for two particular FDA-approved drugs. Astute readers will know exactly what two drugs I am referring to.

Why do physicians prescribe off-label drugs?

One reason is that there might not be an approved drug to treat your disease or medical condition.  Another is that you may have tried all approved treatments without seeing any benefits. Examples include,

Used for a disease or medical condition that it is not approved to treat, such as when a chemotherapy is approved to treat one type of cancer, but healthcare providers use it to treat a different type of cancer.

Given in a different way, such as when a drug is approved as a capsule, but it is given instead in an oral solution.

Given in a different dose, such as when a drug is approved at a dose of one tablet every day, but a patient is told by their healthcare provider to take two tablets every day.

Many prescription medications are approved for one indication but used for something entirely different, prescribed by a physician, ethically and legally if they believe the use of this medication in an off-label manner is in the best interests of the patient. Here are several examples.

Antihypertensive prazosin is used to also treat PTSD-related nightmares. Another antihypertensive clonidine is used for ADHD. Antipsychotic quetiapine can be prescribed for insomnia. Heart and blood pressure medicine propranolol can be taken for performance anxiety and sildenafil, also known as Viagra, can treat female sexual arousal disorder, in addition to its well-known use in men.

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