Off-label use facts for kids

Off-label use happens when a medicine is used in a way that hasn't been officially approved. Imagine a medicine was approved to treat headaches. If a doctor uses it to treat stomach aches, that's off-label use. It also counts as off-label if the medicine is given to a different age group than approved, or if the dose is different from what's officially recommended.

Using medicines off-label is quite common. Before a medicine can be officially approved for a specific use, it has to go through many tests called clinical trials. These trials show if the medicine is safe and works well for that particular use. Until these tests are done and approved, the medicine can't legally be advertised or sold for other uses.

What is Off-Label Use?

Off-label use means a doctor prescribes a medicine for a purpose, age group, or dose that isn't listed on its official approval. This approval is usually given by government health agencies, like the Food and Drug Administration (FDA) in the United States. They check if the medicine is safe and effective for its intended uses.

Why Do Doctors Use Medicines Off-Label?

Doctors might use medicines off-label for several reasons:

• New Discoveries: Sometimes, doctors find that an existing medicine helps with a condition it wasn't originally made for.
• Rare Conditions: For very rare diseases, there might not be enough patients to do large clinical trials. Doctors might use an existing medicine if they believe it can help.
• Children's Medicine: Many medicines are first tested and approved for adults. It can take a long time to test them specifically for children. Doctors often need to use these medicines off-label for kids, adjusting the dose carefully.
• Urgent Needs: In some serious situations, if no approved medicine works, a doctor might try an off-label option as a last resort.

Is Off-Label Use Legal?

Yes, in many countries, it is legal for doctors to prescribe medicines off-label. While drug companies can't promote or advertise a medicine for off-label uses, doctors have the freedom to decide what's best for their patients. This is part of their medical judgment. However, they usually need to explain the risks and benefits to the patient or their parents.

Examples of Off-Label Use

A common example is using certain antidepressants to treat nerve pain, even though they were originally approved for depression. Another example is using a medicine approved for adults with a certain condition for children who have the same condition, but for whom the medicine hasn't been specifically tested and approved.

Safety and Off-Label Use

Even though off-label use is legal, it comes with some things to consider. Since the medicine hasn't been fully tested for the off-label use, there might be less information about its safety or how well it works for that specific purpose. Doctors must carefully weigh the possible benefits against any risks. They rely on their experience, medical research, and discussions with other experts.

How Doctors Decide

When considering off-label use, doctors look at:

• Scientific Evidence: They check if there are any studies or research papers suggesting the medicine might work for the new use.
• Patient's Needs: They think about the patient's specific health situation and if other approved treatments have failed.
• Potential Risks: They consider any possible side effects or dangers of using the medicine in a new way.
• Informed Consent: They talk to the patient (or their parents) about why they are suggesting an off-label medicine, what the known risks are, and what other options exist.

See also

In Spanish: Uso fuera de indicación para niños