HISTORY:

Medications prescribed for children that do not fall within the United States Food and Drug Administration (FDA) guidelines for pediatric use and/or dosing may fall into two categories: off-label medications and experimental medications.

For children, the absence of testing and labeling poses risks, such as adverse reactions, ineffective treatment, and restricted access to some important medications. The Food and Drug Administration Modernization Act (FDAMA) of 1997 directed the FDA to identify drugs that should be required to have pediatric labeling, offered incentives to manufacturers to study new and existing products for use in children, and authorized the FDA to require manufacturers to conduct certain studies.

DESCRIPTION OF ISSUE:

Off-label medications are those FDA approved medications prescribed for non-approved indications in children. It is, in other words, the unapproved use of a legal medication. Examples include medications that are prescribed in doses or routes outside the FDA guidelines, medications known to be safe in adults and prescribed without long-term studies demonstrating safety in the pediatric population, or medications approved to treat one type of medical condition, but being prescribed for a different medical condition.

Three-fourths of the drugs prescribed for children have not been tested in children. In these instances, practitioners rely on a variety of other sources when products have not been tested for safety in children and effectiveness for the intended use. The professional standard for off-label prescription is that the unapproved use of a legal drug must be based on reasonable medical evidence with the same judgment as exercised in medical practice in general.

Pediatric experimental, or investigational drugs are those medications currently involved in clinical trials. These medications are undergoing formal study to determine the efficacy and safety for pediatric dosing, but they do not have FDA approval. An example of an experimental medication would be the new cancer medication taken by a student participating in a clinical trial. A research organization is responsible for the protocols for administration and monitoring of these medications.

School nurses receive requests from parents and health care practitioners to administer off-label and new or experimental medications during school hours. School nurses are asked to administer or observe children for response to common medications, e.g., albuterol inhalation solution, that lack published pediatric dosing, to new medications or to those using prescribers’ anecdotal reports, without published information on indications or long-term effects. Courts have upheld the school nurse’s refusal to administer a prescribed medication in a dose exceeding the amount documented in a standard reference such as the Physician’s Desk Reference.

RATIONALE:

School nurses support children’s need for safe and effective drugs. School nurses and other health care providers need age-appropriate drug information obtained through clinical trials of currently available and experimental products. Clinical trials are designed to protect participant safety and rights while gathering important information about a product’s safe and effective use. Responsible research strikes a balance between burden and benefit.

CONCLUSION:

It is the position of the National Association of School Nurses that school districts develop policies and procedures to address staff handling and administration of experimental or off-label medications, if medically necessary during school hours, with student safety as the primary consideration. The school nurse should have all necessary information about the product to support safe administration at school.

Requests to administer experimental medications at school should be evaluated on a case-by-case basis by the school nurse and the prescribing health care provider. For clinical trial medications, the request to administer medication at school should be accompanied by a copy of the written protocol or study summary from the research organization and/or a copy of the detailed consent form signed by the parent/guardian which describes the study (including the potential benefits and risks), the signs and symptoms of adverse reactions to be reported, and the name and telephone numbers of the investigator or research team.

For off-label use medications, the school nurse should have resources with adequate information to support the safe administration at school, i.e., published anecdotal reports of use in children for the indication the prescriber names, reports from the manufacturer or a reliable pharmacy or college of pharmacy, current medical journals, or information from a pediatric medical or mental health facility.

References/Resources:

American Academy of Pediatrics. (1995) Guidelines for the ethical conduct of studies to evaluate drugs in pediatric populations. Pediatrics 95(2):286-294

American Academy of Pediatrics. (1996) Unapproved uses of approved drugs: The physician, the package insert, and the Food and Drug Administration: Subject review. Pediatrics 98(1):143-145.

Anonymous. (August 9, 2000) Pediatric clinical trials are on the rise as new law goes into effect. (http://pediatrics.medscape.com).

Buck M. L. (2000) The FDA Modernization Act of 1997: Impact on Pediatric Medicine. Pediatric Pharmacotherapy 6 (12):1-4.

Schwab N. C. (October, 2000) Legal Issues for School Nurses. School Health Alert supplement.

Schwab N. C. and Gelfman M. H., Eds. (2000) Legal Issues in School Health Services. North Branch MN. Sunrise River Press.

US Food and Drug Administration. (January, 2001) The Pediatric Exclusivity Provision. Report to the US Congress.

Adopted: June 2001