History

The need for pediatric drug development goes back more than a century. In the early 1900s, elixirs used to help children sleep led to unintended tragedies and death. Later, in the 1950s and 1960s, thalidomide prescribed to pregnant women for nausea resulted in birth defects, including limb deformities.

FDA's Pediatric Rule of 1994 allowed the labeling of drugs for pediatric use based on:

  • Extrapolation of efficacy in adults and
  • Additional pharmacokinetics (how a drug affects the body), pharmacodynamics (how the body affects a drug), and safety studies in pediatric patients, if the course of the disease and the response to the drug are similar in children as in adults.

Only a small number of well-designed and well-conducted studies resulted from this rule, even though it was designed to improve pediatric labeling.

Additional legislation, passed in 1997 as part of the FDA Modernization Act, provided extra incentive to pharmaceutical companies for pediatric testing by granting an additional 6-month exclusivity period for marketing. As a result, many drugs have and continue to receive pediatric labeling under this provision. Visit FDA’s New Pediatric Labeling Information Database for more information.

Evolution of BPCA

The original BPCA legislation directed the HHS Secretary, acting through the NIH Director, to establish a program for pediatric drug development. The NIH Director delegated lead responsibility for the BPCA’s off-patent drug component to the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).

NICHD was chosen to lead NIH's BPCA efforts, in part, because of its success with the Pediatric Pharmacology Research Unit (PPRU) Network. The PPRU comprised a group of experts in pediatric pharmacology who conducted pediatric clinical trials from 1994 to 2009. Their work initiated key pediatric research, including clinical trial designs, and many label changes for drugs used in children. In addition, NICHD's strengths in sponsoring research and conducting safe and effective clinical trials in populations thought to be “fragile,” such as children and pregnant women, made the institute the natural choice to spearhead NIH's BPCA activities.

BPCA 2002

Established FDA Office of Pediatric Therapeutics

Established Pediatric Advisory Committee

NIH Mandate (409I):

  1. Prioritize testing of off-patent pediatric drugs
  2. Sponsor clinical trials and other research to provide data for the above (via contracts)
  3. Submit data to FDA for labeling change

BPCA 2007 (Food and Drug Administration Amendments Act)

Prioritization:

  • Therapeutics focus
  • Additional language regarding developmental pharmacology and infrastructure for trials (including training)

Trials:

  • Flexibility in funding
  • Proposed Pediatric Study Requests
  • Feasibility study
Data Dissemination via Federal Register and Dockets

BPCA 2012 (Food and Drug Administration Safety and Innovation Act)

FDA's BPCA program made permanent

  • Posting of medical reviews
  • Development of pediatric study plans

NIH's BPCA program still requires renewal every 5 years

Neonates considered a priority

BPCA 2017 (FDA Reauthorization Act)

Data Dissemination:

Priorities:

  • Allowing research priorities to include the identification of biomarkers for pediatric diseases and conditions
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