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August 2017

Regulatory Science in Neonates: A Framework That Supports Evidence-Based Drug Therapy

Author Affiliations
  • 1International Neonatal Consortium, Tucson, Arizona
  • 2Institute of Translational Medicine, University of Liverpool, Liverpool, England
  • 3Pediatric Therapeutic Area, Novartis Pharmaceuticals, East Hanover, New Jersey
  • 4Tufts Medical Center, Boston, Massachusetts
JAMA Pediatr. 2017;171(8):721-722. doi:10.1001/jamapediatrics.2017.1360

The International Neonatal Consortium (INC) integrates global stakeholders to promote clinical drug development for neonates.1 The INC focuses on generalizable methods for using data to support claims that a drug is safe and efficacious when used to treat a specific indication (regulatory science).

The framework provided by regulatory science is similar to evidence-based medicine, including a “gold standard” approach of drawing on more than 1 adequately powered randomized clinical trial (RCT) when possible. Regulatory science includes several steps that are not usually emphasized in evidence-based medicine, including preclinical studies of toxicology and effects, optimized drug formulations, a transparent justification of dosage levels and trial designs, and a series of studies (including phase 1 and 2 studies, known as therapeutic exploratory studies) that are used to design the pivotal RCTs (known as therapeutic confirmatory studies).2 These steps promote the success of large RCTs while decreasing risks within the large RCTs for patients, funders, and investigators. Regulatory science includes a framework for circumstances in which conducting large RCTs is not feasible.3 The information generated by applying regulatory science supports judgments about drugs and biologics, typically allows a product to be placed on the market, and supports the rational use of the marketed drug. An adapted regulatory science framework can also support the use of drugs if they need to be used off-label.

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