Addressing the Barriers to Pediatric Drug Development Workshop Summary (2008) / Chapter Skim
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2 Regulatory Framework
2 Regulatory Framework
Pages 10-19
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From page 10... ...
Dr. Lisa Mathis, Acting Director, Division of Pediatric Drug Development, U.S.
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Then in 1979, the FDA issued a requirement that labels note specifically whether safety and efficacy had been established in pediatric populations. The 1994 Pediatric Labeling Rule, another FDA regulation, requested that the pharmaceutical industry submit literature and other data providing additional information on the use of drugs in pediatric patients.
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From page 12... ...
This 6-month extension is offered not only for a drug that was studied in pediatric populations, but also for any of the company's formulations, dosage forms, and indications that contain the same active part, or moiety, of a molecule and have existing marketing exclusivity or patent life. For example, if a company markets an oral formulation and a topical cream containing the same moiety but submits a pediatric study for only one of the formulations, the 6 months of marketing exclusivity is added to patent protection for both products.
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(The FDA would be unlikely to study a drug in pediatric patients that has resulted in many adverse events or has low efficacy in adults unless the disease treated by the drug is life-threatening. For example, the FDA would be more likely to accept adverse events for an oncology drug than for a treatment for otitis media or some other non-life-threatening disease.)
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Pediatric Research Equity Act The Pediatric Research Equity Act amends the federal Food, Drug, and Cosmetic Act to authorize the FDA to require pediatric studies of drugs or biologics when other approaches are insufficient to ensure that the products are safe and effective for use in children. Under PREA, the FDA can require pediatric studies of a product for which a New Drug Application is submitted if the agency determines the product is likely to be used in a substantial number of pediatric patients, or if it would provide meaningful benefits for children over existing treatments.
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For example, if the chemical properties of a medication prevent its production as a liquid, it may be waived from study in newborns or children under 5 years of age, who would require a liquid formulation. TABLE 2-2 Best Pharmaceuticals for Children Act Versus Pediatric Research Equity Act BPCA PREA Studies are voluntary Studies are required Includes orphan drugs Orphan drugs and indications are designated exempt Covers drugs only Covers biologics and drugs Studies encompass whole moiety Studies limited to drug/indication under (active part)
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underwent major labeling changes after a 19week clinical pediatric trial of its use for major depressive disorder in patients aged 8 to 17 and obsessive-compulsive disorder in patients aged 7 to 17 found that those taking the drug experienced more limited growth than those not taking it. The label now warns physicians to monitor the height and weight of pediatric patients treated with fluoxetine.
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, Pfizer 11/27/2001 1/8/2004 * An extemporaneous formulation is a pharmaceutical product that has been freshly compounded without prior preparation or in an improvised manner.
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Since then, adverse events have been reported for 54 drugs. These reports include suicidal ideation in patients taking selective serotonin reuptake inhibitors (SSRIs)
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Dr. Dianne Murphy, Director, Office of Pediatric Therapeutics, FDA, and several other participants suggested that in the new iterations of BPCA and PREA, a requirement be included that product labels provide information on results of pediatric trials regardless of the product's approval status and the process.
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