Regulatory Science Core

Project Summary/Abstract - Regulatory Science Core
Establishing early a regulatory-enabling research framework is essential for the successful translation of
promising target biomedical discoveries especially targeted to the next “Disease X” and respond to the 100 Days
Mission (100DM) as well as ASPR Next of the US government. For vaccines and therapeutics, such a framework
would accelerate the technology transfer of novel and promising candidates into the manufacturing and
toxicology testing stages and would ensure robust preclinical data packages used in the compilation of regulatory
dossiers to initiate the clinical testing. A Consortium-wide Regulatory Science Core, aligned and integrated with
the other cores, especially the Data Management Core, will be created to prepare, guide, assess and ensure
that all research and development (R&D) efforts of the consortium projects are embedded in a regulatory-
enabling and Total Quality Management System (TQMS) environment supporting the rapid translation and
readiness of new vaccine candidates towards their suitability for technology transfer to GMP pilot manufacturers,
GLP toxicology testing and prepare a smooth adaptation to seek regulatory approval for their entry into clinical
trials. The expectation is that having access to such a core, the grant R&D efforts will result in high-quality vaccine
prototypes with robust preclinical data packages leading to efficient and rapid transitions into manufacturing and
toxicology testing and accelerating regulatory approvals for the entry into the clinic. The Regulatory Science
Core will have the following aims: 1) Establish a consortium-wide regulatory-enabling TQMS adapted to the
needs of the consortium to accelerate the development timelines of the novel vaccine prototypes. Briefly, an
initial landscape assessment will give insights of the levels of regulatory-enabling and quality processes and
tools used by consortium members. Subsequently, a set of standardized and harmonized templates and tools
will be generated for consortium use and lastly a digital cloud-based document control system to centralize and
archive documents will be created; and 2) Monitor and evaluate consortium-wide quality improvements by
establishing a plan for continuous monitoring and evaluation of the TQMS and strengthen the consortium
performance, time management and pre-investigational new drug (pre-IND) Go/NoGo decision points and be
more efficient in achieving the project milestones. A continuous and sustainable quality learning environment will
build self-reliance and self-sufficiency thus become better prepared for any emerging pandemic. Overall,
including a regulatory science core in this proposal is the generation of a comprehensive system that enables
and is aligned with regulatory requirements such that the novel vaccine candidates emerging from this
consortium can be ready to advance into the critical phases of development if an emerging pandemic caused by
one of these viruses were to occur.

Grant Number: 1U19AI181968-01
NIH Institute/Center: NIH
Principal Investigator: Maria Bottazzi