FDA News: Pediatric Trial participation, RMAT Designation, FDA-ARGOS, CDRH Experiential Learning Program – Drug and Device Digest

FDA BRIEF: Week of March 20, 2017

nurse and father tending to child in hospital bed (600x400)

Would Your Child Benefit from a Clinical Trial?

Pediatric clinical trials:

  • yield important information on a drug’s safety, dosing, and/or effectiveness
  • forms the basis for FDA approval & guides product label

Only about 50% of FDA approved drugs been labeled for pediatric use 

  • routine use of treatments that have been tested only in adults
  • pediatric responses  may not be predictable from adult data
  • also varies due to metabolism changes across across age groups e.g. newborn vs. adolescent


This is Not a Test: RMAT Designation Goes Live

By: Peter Marks, M.D., Ph.D.,  Director, CBER
Peter Marks

Regenerative Medicine Advanced Therapy (RMAT) Designation

  • new program to foster development and approval of regenerative medicine
  • per provisions included in 21st Century Cures Act

RMAT designation

  • certain cell therapies, therapeutic tissue engineering products
  • preliminary supportive clinical evidence to treat serious/life-threatening diseases
  • eligible for increased, earlier FDA interactions, priority review, accelerated approval
  • fulfillment of post-approval  accelerated approval  requirements: clinical evidence, clinical studies, patient registries, real world evidence,larger confirmatory datasets, post-approval monitoring




FDA – ARGOS (dAtabase for Regulatory Grade micrObial Sequences)

  • to promote advancement of Infectious Disease Next Generation Sequencing (ID-NGS)
  • in collaboration with the Department of Defense, the Institute for Genome Sciences at the University of Maryland and the National Center for Biotechnology Information
  • publicly available public health resource  for quality controlled and curated genomic sequence data
  • could be used as tool for in-silico (computer simulation) performance validation
  • invites additional collaborators from scientific community – biothreat organisms, emerging pathogens, clinically significant bacterial, viral, fungal, and parasitic genomes
  • goal to collect geographically and historically diverse sequence information for a minimum of 5 isolates per species




Innovative learning opportunity for FDA review staff

  • opportunity to understand the policies, laboratory and manufacturing practices, challenges addressing patient perspective/input, quality system management, device development life cycle
  • NOT intended for FDA to inspect, assess, judge, or perform a regulatory function (e.g., compliance inspection)
  • encourages participation from companies, academia, clinical facilities, medical device incubators and accelerators, health insurers, health technology assessment groups
  • Application Process based on CDRH Areas of Interest : Submission Period: March 23, 2017- April 30, 2017



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