Pediatric Drug Testing Update: Pediatric Exclusivity

In: Drug

16 Jun 2010

Pediatric ExclusivityPL. 107-109 of the BPCA reauthorizes the pediatric studies provision of the 1997 Modernization Act to improve the safety and efficacy of pharmaceuticals for children. It continues to encourage pharmaceutical companies to conduct studies of on-patent drugs that are used in pediatric populations (but that are not labeled for such use) by extending their market exclusivity. In addition, P.L. 107-109 authorizes studies for off-patent drugs by the federal government or other entities with the expertise to conduct pediatric clinical trials.

PL. 107-109 contains some provisions pertaining to NIH:

1. Pediatric Drug List and Research Fund. The Director of NIH, in consultation with the Commissioner of the FDA and experts in pediatric research, are required to develop, prioritize, and publish a list of drugs for which pediatric studies are needed and for which the DHHS Secretary must award contracts for the conduct of pedi-atric clinical trials. The FDA Commissioner is also required to consult with the NIH Director to issue written requests for studies to holders of approved applications for drugs lacking marketing exclusivity. To carry out these studies when drug companies refuse to do so, the law authorized $200 million in fiscal year 2000, and such sums as are necessary for each of the five succeeding fiscal years.
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2. Foundation for the NIH. This provision allows the DHHS Secretary to refer a drug to the Foundation for the NIH when he or she determines that there is a need for information relating to the use of the drug and the drug manufacturer does not agree to conduct a study.

3. Institute of Medicine Study. The DHHS Secretary is required to contract with the Institute of Medicine to conduct a study on best practices that relates to research involving children.

4. National Cancer Institute Directives. At least two pediatric oncology specialists from the National Cancer Institute (NCI) are required to participate on the Pediatric Subcommittee of the Oncologic Drugs Advisory Committee to review and evaluate data concerning the safety and effectiveness of drugs for use in the treatment of pediatric cancers.

In addition to requiring the NICHHD and FDA to compile the list of drugs, the BPCA reauthorized an existing economic incentive (extended protection from market competition) for pharmaceutical companies that conduct pediatric studies requested by the FDA.

P.L. 107-109 also amends the Food, Drug,and Cosmetic Act to:

  • eliminate the user fee waiver for pediatric supplements to a human drug application.
  • provide priority status for pediatric supplements.
  • include neonates within age groups for pediatric studies.
  • provide for the dissemination of pediatric supplement information.
  • set forth requirements for the additional six-month exclusivity period for new pediatric drugs or those already marketed.

In return for doing pediatric testing, the drug companies were allowed to ask for pediatric exclusivity—an extra six months of patent protection and freedom from generic competition for all formulations of the drug, not just the one being tested.


The recognition of the rights of children to have access to safe and effective drugs—and of the needs of health care providers to have access to age-appropriate drug information—is increasingly influencing biomedical research. Numerous drugs have now been studied for pediatric doses. The support of the FDA in mandating pediatric labeling will play an important role in motivating manufacturers to work with NIH pharmacology pediatric research units and with other academic clinicians and researchers to study both new and established drug therapies in children.

The result is likely to be an increase in both the quantity and quality of pediatric drug research conducted in the U.S. As a final example, the FDA has classified these drugs as important new pediatric therapies:

  • Colfosceril palmitate/cetyl alcohol/tyloxapol (Exosurf®, GlaxoSmithKline) and beractant (Survanta®, Ross) are pulmonary surfactants for respiratory distress syndrome in newborn babies.
  • Pegademase bovine (Adagen® Injection, Enzon) is an enzyme, adenosine deaminase, that is used to replace an enzyme missing in children with severe immunodeficiency disease.
  • Succimer (Chemet®, Sanofi-Synthelabo) is an oral drug for the treatment of lead poisoning in children.
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Pediatric drug testing, as authorized by Congress and the FDA, has turned the corner and seems to be heading in the direction of obtaining clinically acceptable efficacy and safety data for use in children.

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