Unlicensed and off-label use of medication

Manufacturers must invest a lot of money and resources to get approval for a new medication.

Before being released to the public, the chemicals in medicine must be thoroughly tested to ensure they are safe and effective. Drugs are first tested in laboratories and on animals to determine if the drug is safe and to try to understand how it might work in humans.

Next, drugs undergo extensive clinical trials where patients receiving the new medication are compared to a control group taking a placebo. Often, a third group is also given a medication that is already available on the market, if this is an option.

The goal of these trials is to determine how effectively the medication treats the targeted conditions, how well patients tolerate it, and whether there are any previously unknown or potentially harmful side effects.

Here in the United States, pharmaceutical companies must send the Center for Drug Evaluation and Research (CDER) the evidence from these tests to prove that the drug is safe and effective for its intended use.

A team within CDER then reviews the company's data to establish whether the drug's health benefits outweigh its known risks. If so, the drug is approved for sale.

The Food and Drug Administration's (FDA) approval of a drug means that data on the drug’s effects have been reviewed by CDER. The drug approval process follows a structured framework that includes:

• Analysis of the target condition and available treatments
• Assessment of benefits and risks from clinical data
• Strategies for managing risks

The decisions to grant FDA approval are made through a deliberative process.

Once the FDA approves a drug, the post-marketing monitoring stage begins. This is because it’s not possible to predict all of the effects of a drug during clinical trials, therefore safety monitoring is conducted after a drug gets on the market. When the company uncovers new risks, they add them to the drug's labelling and notify the public.

An unlicensed medication is one that has not been approved for use by a regulatory medical body. Often medications that are licensed for a specific condition can have additional effects on the body. Some of these can be positive and are taken into account when prescribing the treatment. For example, most contraceptive pills also alleviate menstrual pain.

Off-label prescribing is when a physician prescribes a drug that the U.S. FDA has approved to treat a condition that is different from your own. This may occur when the usual first-line treatments of a condition are deemed unsuitable for that patient. This could be due to many factors including pre-existing medical conditions, or interactions with other medications.

A doctor can prescribe a medication off-label if it is safe and effective for the patient. The benefits of the medication must outweigh the risks. Off-label prescribing requires careful consideration of whether the treatment is equally as effective as those designed to treat the same condition.

Some examples of ‘off-label’ uses are:

• Using a medicine for a different illness to that stated in the licence. Scientific research may support this use, but the drug manufacturer has not sought to extend the license.
• Using a medicine at a different dose than stated in the licence.
• If medicines are administered differently, for example, when a drug is approved as a capsule, but is given instead in an oral solution.

Yes, off-label use of a medication is legal. The prescribed medications have a license and are not illegal themselves. As long as safety regulations and ethical guidelines are considered, doctors are allowed to prescribe medications off-label as part of their medical practice.

Using a medicine off-label does not mean that the medication is unsuitable. In fact, the medication may already have a valid license in other countries and have a wealth of evidence supporting its validity for effective and safe treatment of the condition.

For prescription drugs, you still need a valid prescription from your doctor, even if it is for off-label use. If a healthcare professional wants to prescribe an unlicensed medicine, or a licensed medicine off-label, they must follow their professional guide.

Your physician also has to inform you that the medicine is unlicensed for the specific condition that you have and is being prescribed off-label. They must give you information about the treatment and discuss the possible benefits and risks so that you have enough information to decide whether or not to have the treatment. This is called informed consent.