FDA BRIEF: Week of March 20, 2017
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
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