Everyone hopes that their baby is born healthy and never gets sick. But even healthy infants may need medicine sometimes. Babies who are born prematurely and need to be in the hospital will almost certainly receive medicine while in the hospital and they may need some medicines even after they go home. So it is important to understand some things about medicines for infants and children.
Individual countries create their own laws to govern drug development and establish regulatory bodies to oversee the process. In the United States, the regulatory body is the Food and Drug Administration (FDA). Many countries choose to coordinate the regulatory process on a regional basis. Examples include the European Medicine Agency (EMA) and the Association of South-East Asian Nation (ASEAN).
Laws and regulations evolve over time. Now it is standard to evaluate medicines for safety and how well they work but that wasn’t always true. In the United States, it wasn’t until 1938 that manufacturers had to demonstrate a medicine was safe and 1962 before they had to demonstrate it actually worked for the purpose that it was promoted for (effectiveness). Safety and effectiveness are usually determined through different types of rigorous research starting with experiments in a laboratory then experiments in animal models and finally clinical trials in humans. The results of these experiments are then submitted to the regulatory bodies as part of an application to approve the medicine to be marketed.
A medicine is approved or “labeled” for a specific purpose (also called an indication) in a specific group of people. For instance, a drug might be approved to treat high blood pressure in adults or to treat ear infections in children age 2 and above. The company who owns the drug decides what purpose(s) and group(s) to study. The drugs are made in forms that are appropriate for the population in which they are approved. So the high blood pressure medicine for an adult may be a tablet or capsule and the antibiotic for the child is more likely to be in liquid form or possibly a chewable tablet. To read more about the FDA approval process click here.
Once a medicine is approved and available on the market, a person who is licensed to prescribe medicine is not required by law to prescribe the medicine only for the purpose or population in which it was approved. This practice is called “off-label use”. Because most drug research is performed in adults, most drugs are approved for adults, not infants or children, at least initially. This has resulted in the need to prescribe medicines off- label for infants and children much of the time. How often it is necessary to prescribe off-label varies depending on what group of patients (hospital vs. doctor’s office) and what types of medicines are being examined but estimates as high as 80% (8 out of 10 times) have been reported in recent medical literature. Healthcare professionals use published dosing references which are developed from the medical literature to guide them in prescribing these medicines. The American Academy of Pediatrics addresses off-label medication use in children in their March 2014 policy statement.
Because medicines approved for adults often come in a form or strength that can’t be taken by young children, the medicine may need to be reformulated by a pharmacist. For example, tablets may be finely crushed and suspended in a liquid. This process is called compounding. Compounded medications may have different storage requirements and expiration dates than the original drug product. Follow any directions such as “shake well” or “refrigerate” that are provided on the medication bottle or by the pharmacist.
The limited drug research in infants and children and the resulting need to prescribe medicines off-label has been identified as a shortcoming of the current medication development system and efforts to improve it have been undertaken. Several laws were passed in the 1990s and early 2000s to promote more drug development specifically for infants and children. This has resulted in new information and labeling changes over the last two decades (see here).
So although the need to prescribe medicines off-label for infants and children remains common, it is slowly becoming less so. Off-label use is legal and is not research. If your child requires medicine, here are somethings to consider discussing with your doctor and pharmacist:
- What is the medicine used for?
- How does the medicine work?
- Has the medicine been studied in and approved for infants or children?
- What is the dose for my child?
- What are the possible side effects?
- What can I do to decrease the risk of side effects?
- Is the medicine commercially available or does it need to be compounded?
- How should I store and administer the medicine?
- When should the medicine be discarded?