Association of American, Physicians and Surgeons, Inc. v. FDA
U.S. District Court, DC
17 October 2002

Briefed by: R. Longwell, Esq.


The FDA seeks to address the issue of drug testing for children and promulgates the Pediatric Rule. This Rule requires all new drugs to be tested on a pediatric population. The FDA reasons that not testing all new drugs for a pediatric population unreasonably endangers children’s health.

The plaintiffs challenge the FDA’s authority to promulgate the Pediatric Rule as being outside that granted by Congress. FDA justified its authority under its statutory grant of power to enforce labeling on drugs. The court sides with the plaintiff and finds the FDA has overstepped its authority in making the Pediatric Rule.


The FDCA grants the FDA authority to approve the marketing of new drugs products. However, the FDCA does not regulate how a drug is prescribed. This decision is left to the physician in each individual case. There is nothing in the law to prevent a physician from prescribing a drug to children that has only been tested on adults.

Many new drugs are not tested on children. The reasons for this include the expense and difficulty of finding test subjects, as well as ethical considerations. However, physicians often prescribe such adult tested drugs for children (“off-label”), in contradiction to the manufacturer’s label. The FDA sees this as an unreasonable danger to children’s health and acts.

FDA chose to address this problem with regulations commencing in 1994 and ending with the Regulations Requiring Manufacturers to Assess the Safety and Effectiveness of New Drugs and Biological Products in Pediatric Patients (“Pediatric Rule”) in 1998. The Pediatric Rule sought to require pediatric testing of all new drugs, except those that obtained a waiver to the Rule.

FDA used its authority in the area of product labeling (21 U.S.C.S 321 et seq.) as a basis for asserting it had authority to enact the Pediatric Rule. FDA reasons that because manufacturers reasonably know that their products are being used “off-label” that the use by children is foreseeable and known and as such gives FDA the authority to make the Rule.

Congress also recognized this problem and enacted laws to address it. In 1997, Congress enacted the Food and Drug Administration Modernization Act (FDAMA). Part of the FDAMA provided for an experimental pediatric drug testing program. Subsequent to the FDA promulgating the Pediatric Rule, Congress enacted the Best Pharmaceuticals for Children Act (“BPCA”) in 2002. The BPCA also provided for a voluntary pediatric drug testing program.


  1. FDCA statute is not sufficient basis for authority to enact the Pediatric Rule

    The FDCA permits FDA to regulate the labeling of a drug. To determine whether a label is misleading under 21 U.S.C. ß ß 321(n), the agency should look to whether the “labeling fails to reveal [material] facts…under such conditions of use as are customary or usual.” The FDA cited extensive evidence that some drugs are “commonly” or “usually” used by children, despite the absence of pediatric labeling.

    The FDA also relies upon 21 U.S.C. ß ß 321(p) which defined new drugs as those not recognized to be safe and effective under conditions “prescribed, recommended, or suggested” in product labeling. The FDA argued that this language gives them the authority to regulate drugs for pediatric uses even when such uses are not explicitly claimed by a drug manufacturer in a product’s labeling.

    The court, however, was most bothered by provisions that require manufacturers to (1) conduct studies of drugs for unclaimed uses or (2) devise formulations of the drug tailored to those uses. The court found the relationship between these provisions and labeling to be tenuous. The court reasoned that if Congress had intended such broad power, it would have been more specific. It also noted that the Pediatric Rule impacts new drugs primarily and that they have not established any “customary or usual” use.

  2. Congress, in enacting the BPCA, imposed a “distinct regulatory scheme” to address the issue of pediatric drug testing, thereby demonstrating its intent to occupy the field.
  3. The BPCA and the Pediatric Rule are incompatible and at odds with one another (incentive scheme intended by Congress vs. command and control scheme imposed by FDA). The law controls FDA.



The Pediatric Rule exceeds FDA’s statutory authority and is invalid.


The FDA’s authority to make rules is limited to the authority granted by Congress. The court noted that FDA’s regulatory scheme may indeed be the best way to address the problem, but FDA must have a basis in statute to do so.

The FDA can only regulate the claimed uses of drugs, not all foreseeable or actual uses. Furthermore, off label use of a medical device by a physician applying her medical judgment for a particular patient is not in the province of the FDA.