FDA Draft Guidance for Pediatric Labeling.
  The US Food and Drug Administration (FDA) has released a new draft guidance document pertaining to the pediatric information that sponsors and applicants must place within the approved product labeling for human drug and biological products.

The Pediatric Research Equity Act (PREA), originally enacted on December 3, 2003 (Public 92 Law 108-155), codified many of the elements of the pediatric rule, and established requirements for studies of certain drugs and biological products used in pediatric patients. PREA (section 94 505B of the FD&C Act, 21 U.S.C. 355c), reauthorized by FDAAA, as Title IV, on September 27, 2007 (21 U.S.C. 355c) and made permanent in 2012 with the passage of FDASIA, requires pediatric studies for certain drugs and biological products. Specifically, PREA requires applications (or supplements to applications) under section 505 of the FD&C Act or section 351 of the Public Health Service Act (PHSA), respectively, for a new active ingredient, new indication, new dosage form, new dosing regimen, or new route of administration to contain a pediatric assessment, unless the applicant has obtained a waiver or deferral (section 505B (a) of the FD&C Act).

PREA also authorizes the FDA to require holders of applications for previously approved marketed drugs approved under section 505 of the FD&C Act and biological products licensed under section 351 of the PHSA, who are not seeking approval for one of the changes specified above, to submit a pediatric assessment under certain circumstances (section 505B (b) of the FD&C Act). Pediatric assessments “shall contain data, gathered using appropriate formulations for each age group for which the assessment is required that are adequate (i) to assess the safety and effectiveness of the drug or the biological product for the claimed indications in all relevant pediatric subpopulations; and (ii) to support dosing and administration for each pediatric subpopulation for which the drug or the biological product is safe and effective” (section 505(B)(a)(2) of the FD&C Act).

In 2006, FDA released a final rule requiring that all pharmaceutical and biological products intended for use in humans be labeled with the pediatric-specific information in accordance with specified formatting.

The goal of both BPCA and PREA is to provide pediatric information in drug labeling to encourage the appropriate use of drugs in treating pediatric patients.

While some of the data may be extrapolated from adult studies if the effects of the drug are "sufficiently similar in adults and pediatric patients," this is generally limited only to efficacy data, and not safety or dosing, both of which much be calculated separately. "Because pediatric patients may be more or less prone to drug toxicities based on immaturity or developmental changes, the safety of a drug generally should be studied in pediatric patients directly," the guidance explains.

In addition, data generated regarding the safety or efficacy may be sufficient for all populations, or only sufficient to support safe and effective prescribing in limited pediatric subpopulations. The guidance contains a decision matrix that explains which data would be sufficient to support certain claims, and which would not.

The bulk of the guidance, however, deals with the placement of that data within the product labeling. Per the aforementioned decision matrix, sponsors are required to indicate whether the pediatric findings about their product are positive, negative or inconclusive, and place those findings within the "Pediatric Use" subsection of the labeling.
Comments on the draft guidance are due by 29 April 2013.

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