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Pediatric Drug Development and The Orphan Drug Act Incentives

The Office of Orphan Products Development (OOPD) of the Food and Drug Administration (FDA) has long recognized pediatric patients as "therapeutic orphans" due to the lack of adequate pediatric dosing information among drugs that are on the market. This issue has been addressed, in part, by the pediatric exclusivity provision of the Food and Drug Administration Modernization Act (FDAMA) of 1997,1 and more recently by the mandatory Final Rule promulgated by the FDA on December 2, 1998.2 The pediatric exclusivity was reauthorized in 2002, and is scheduled to sunset in 2007.3 Therefore, OOPD encourages drug sponsors to give attention to the multiple economic incentive provisions of the Orphan Drug Act4 including tax credits for clinical research and waiver of Prescription Drug User Fee Act (PDUFA) application fee, and to obtain orphan-drug designation of a drug or biological product intended for pediatric use. The designated orphan drug or biological product is entitled to seven years of orphan-drug marketing exclusivity upon its approval for pediatric use. Additionally, the sponsor should also consider Orphan Drug Grants Program, which provides research grants to defray the cost of pediatric clinical testing expenses.

The Orphan Drug Regulations5 stipulate that a drug sponsor may request orphan-drug designation of a drug or a biological product under development for only a subset of persons with a particular disease or condition if the subset is "medically plausible." In the pediatric population, growth and developmental changes can influence the way drugs are absorbed, distributed, metabolized, and excreted, which are vastly differently from the adult. Based on these unique pharmacokinetic properties, OOPD has determined that pediatric patients constitute a "medically plausible" subset of patient population. Therefore, a sponsor of a new drug or biological product may seek orphan-drug designation for treatment of a disease or condition in the relevant pediatric subset of patient population. With regard to currently marketed drugs with no approved pediatric indication, OOPD will consider a pediatric indication a new "orphan" indication, for which the sponsor may request orphan-drug designation. The number of pediatric patients affected by the disease or condition in the United States for which the drug is intended must meet the statutory prevalence limit of 200,000, or - if the number exceeds 200,000 - the costs of drug development cannot be reasonably recovered by drug sales in the United States. It should be emphasized that the economic incentives apply only to the clinical pediatric drug development, and the orphan-drug marketing exclusivity applies to the designated pediatric indication of the drug or biological product.

For additional information on how to obtain orphan-drug designation, please contact OOPD at (301) 827 3666, or visit our website at How to apply for Orphan Product Designation


1See FDAMA Section 111 (1997) codified at the Food, Drug, and Cosmetic Act Section 505A (21 U.S.C. 355a). The pediatric exclusivity provision provides an economic incentive of additional six months of marketing exclusivity for certain drugs if the drug sponsors submit requested information to the FDA related to their use in pediatric population. This provision does not apply to most biological products and antibiotics since they are regulated differently.

2See Regulations Requiring Manufacturers to Assess the Safety and Effectiveness of New Drugs and Biological products in Pediatric Patients: Final Rule, 63 Fed. Reg. 66,632 (1998). The Final Rule compels the sponsors to conduct relevant pediatric studies of new drugs and biological products to support directions for pediatric use.

3See The Food, Drug, and Cosmetic Act, Section 505A (21 U.S.C. 355a), as amended (2002).

4See Orphan Drug Act, P.L. 97-414 as amended.

5See Orphan Drug Regulation: Final Rule, 57 Fed. Reg. 62076 (1992).

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