Federal Court Invalidates the FDA Pediatric Rule: AAPS v. FDA

By Paul Arshagouni
Health Law & Policy Institute

On October 17, 2002, federal district court Judge Henry H. Kennedy, Jr. struck down the Food and Drug Administration’s (FDA) Pediatric Rule, holding that the agency had overstepped its authority.  See Association of American Physicians and Surgeons, Inc. v. U.S. Food and Drug Administration, 2002 U.S. Dist. LEXIS 19689 (D.D.C., October 17, 2002).

The FDA promulgated the Pediatric Rule in 1998 as an effort to ensure that drugs commonly used for the treatment of children are actually tested for pediatric use.  The Pediatric Rule requires drug manufacturers to study the efficacy and safety of their products in children or risk denial of FDA approval.  The Rule also requires manufacturers to devise formulations of drugs for use in children.  The regulations apply to all drugs that may be used in children even if the drug manufacturer does not request approval for use in children.

The Food Drug and Cosmetic Act (FDCA) prevents drug manufacturers from marketing new medications without first obtaining approval from the FDA.  Once a drug receives approval, however, the FDA does not regulate how doctors may prescribe it.  Prior to enactment of the Pediatric Rule, drug manufacturers routinely tested new medications only on adults.  Pediatricians were often left to extrapolate proper dosing for children.  As children often absorb and metabolize drugs differently than adults, this carried potential risks.

Congress recognized this problem and, as part of the Food and Drug Administration Modernization Act in 1997 (FDAMA), provided for voluntary incentives to encourage pediatric drug testing.  When this voluntary scheme failed to produce significant results, the FDA promulgated the Pediatric Rule requiring pediatric testing unless the manufacturer can show that it is highly unlikely for the drug to be used in the pediatric population.  See 21 CFR § 201.23.

While the pharmaceutical industry has generally accepted the Pediatric Rule, several groups including the Association of American Physicians and Surgeons (AAPS) and the Competitive Enterprise Institute (CEI) remained opposed.  In December 2000, AAPS, CEI, and Consumer Alert filed a lawsuit asking the federal court to overturn the Pediatric Rule, claiming that the FDA overstepped its authority.  Proponents of the Pediatric Rule include the American Academy of Pediatrics (AAP), which filed an amicus brief supporting the rule.

Judge Kennedy agreed with the plaintiffs and ordered the FDA not to enforce the Pediatric Rule.  In reaching his decision, Judge Kennedy looked both at the FDCA and the Best Pharmaceuticals for Children Act (BPCA) to determine whether Congress intended to give the FDA authority to enact the rule.

Looking first at the FDCA, Judge Kennedy found that Congress granted the FDA the authority to ensure that drugs are safe and effective and that they bear adequate labeling for all of their claimed uses.  See AAPS at 8.  Citing to prior case law, the FDA claimed that labeling could be misleading if it fails to include information concerning a drugs customary or usual uses, even if the manufacturer does not initially claim those uses.  The FDA relied on extensive evidence that many drugs are commonly or usually used by children despite an absence of pediatric labeling.  Judge Kennedy was unconvinced.  He reasoned that “the Pediatric Rule primarily impacts new drugs, which do not yet have any ‘customary or usual’ use.” Id.

Judge Kennedy held that, based on its reading of the FDCA, the FDA does not have the authority to require testing for drug indications not claimed by the manufacturer.  Ironically, the court found support for this view in public statements made in 1992 by David Kessler, then head of the FDA, and a proponent of the Pediatric Rule.  In a speech before the AAP, Dr. Kessler indicated that the FDA did not have authority to go beyond the indications contained in a drug’s approval application.

The court was also persuaded by its reading of BPCA.  Like the FDAMA, the BPCA, signed into law in January 2002, provided economic incentives to drug manufactures to voluntarily conduct pediatric testing.  See id. at 13.  Judge Kennedy reasoned that Congress was well aware of the Pediatric Rule and failed to expressly endorse it.  The court found that the mandatory aspect of the Pediatric Rule was in conflict with the voluntary aspect of the BPCA.  As Congress was clearly aware of the Pediatric Rule when it passed the BPCA, Judge Kennedy concluded that Congress had considered a mandatory approach but opted instead for a voluntary system, adding, “[t]he FDA, as well as this court, must respect this judgment.”   Id. at 14.

In April and May 2002, legislation was introduced into both the Senate and the House to formally codify the Pediatric Rule.  See 107th Cong., S2394 and HR4730.  While Senate Bill S2394 has passed the Senate health committee with bipartisan support, no further action has occurred.

Interestingly, Judge Kennedy expressly stated that he was not ruling on the merits of the FDA’s regulatory scheme.  He indicated that, while the Pediatric Rule may well be a better tool and more thoughtfully reasoned than what will remain, the issue before him was only the Rule’s statutory authority, not its wisdom.  Given this ruling, proponents of the Rule will need to go back to Congress and try and convince them of its wisdom.

The full text of Association of American Physicians and Surgeons, et al. v. U.S. Food and Drug Administration, can be found at http://www.dcd.uscourts.gov/00-02898.pdf.