Court Decision Invalidating FDA's Pediatric Rule

On October 17, 2002, Judge Henry H. Kennedy of the U.S. District Court for District of Columbia struck down FDA's "pediatric rule." Among other things, the rule allowed FDA to require that drug and biologics applications "contain adequate evidence to support dosage and administration in some or all pediatric subpopulations . . ." It also allowed FDA to request of already-marketed products additional evidence to support pediatric dosage and administration and, in some circumstances, to require an applicant to develop a pediatric formulation. ¹


As its primary defense, FDA relied on several labeling provisions in the Federal Food, Drug and Cosmetic Act to argue that it has authority to require manufacturers to make certain statements about a drug. FDA pointed to a provision stating that a drug's label must reflect its "customary or usual" use. FDA also relied on provisions whose applicability turn on what is "prescribed, recommended, or suggested" in a product's labeling. Because many drugs officially indicated for adults are routinely used by pediatric patients, the agency asserted that pediatric use is "suggested" by the labeling - even when the labeling expressly disclaims effectiveness for pediatric populations. But the court concluded that "use of a drug nowhere indicated by the label and, in fact, specifically disclaimed by the label is, quite simply, not 'suggested' by that label."

Judge Kennedy also addressed the fact that Congress recently extended, for five years, the program under which companies may receive an additional six months of patent term extension or marketing exclusivity for a product if they conduct pediatric studies on the product (The Better Pharmaceuticals for Children Act, or BPCA). He concluded that Congress had adopted "an incentive scheme" that conflicted with the regulation's "command and control approach" to pediatric labeling. Because Congress had considered FDA's approach and had opted for a different tack, both the court and the agency were required to respect its decision.


Judge Kennedy concluded that the agency needs more statutory support for wide-ranging regulatory initiatives than a few phrases in the labeling provisions. Because FDA frequently relies on its labeling authority to construct fairly rigorous requirements for manufacturers, the agency may have to look beyond those provisions - or seek new statutory authority - to support major regulatory initiatives.

For example, the decision can be read to call into question the legality of Phase IV study requirements and PDUFA III's post-approval risk management initiative that includes "post-approval surveillance" and "restricted distribution" requirements. In addition, the opinion may undercut the agency's ability to suggest certain study designs or request changes to an application in order for the sponsor to gain approval. These requests, however, do not usually come as "formal" requests by the agency. Rather, they are "informally" suggested by review officers.

In addition, Judge Kennedy's opinion will almost certainly generate legislative interest from several prominent members of Congress. When it enacted BPCA last winter, Congress debated codifying the pediatric rule, which enjoys broad support from children's advocates. It is highly likely that proponents of codification, particularly Senators Kennedy and Clinton, will attempt to do so again when Congress returns for a lame-duck session next month.

¹ Association of American Physicians and Surgeons v. U.S. Food and Drug Administration, --- F.Supp.2d ---, 2002 WL 31323411 (D.D.C. 2002)