Page 13 - HFA Dateline 2020-2021 Special Issue

released as a draft in 2013 and guidance in 2017 and updated in gene therapies. This framework

finalized in 2015. It discussed 2020 to help industry apply these contained several new guidance
many recommendations for new regulations that together documents, including hemophilia
manufacturing, testing and are referred to as the FDA CGT. This brought the total
patient safety and follow- regenerative medicine number of guidance documents
up. framework. Both human in the framework to 27. In 2021,
gene editing and transfer, we can see how the evolution
In 2016, Congress passed as well as genetically of hemophilia gene and cell
the 21st Century Cures Act modified cells that therapy has been affected by the
or Cures Act, which includes lead to a sustained factor development of this framework.
provisions designed to expedite production in hemophilia, are
and streamline development of considered to be regenerative EARLY 2020: FDA LAUNCHES
innovative new medicines. This medicine therapies by FDA. CELL AND GENE THERAPY
law builds on FDA’s existing GUIDANCE FRAMEWORK
On Jan. 28, 2020, FDA launched
responsibilities and established Guidance for developing its landmark guidance framework
regenerative medicine therapies, CGT products specifically for for CGT products. Updates
including gene therapy, cell hemophilia was published by included improvements to the
therapy, products made from FDA in 2018. Then in 2020, it was guidance for hemophilia and
tissues and combinations of updated when FDA launched its
these products. FDA published expanded framework for cell and CONTINUED ON PAGE 17

GUIDANCE, REGULATION AND LAWS THAT MAKE UP
THE U.S. FDA FRAMEWORK FOR DEVELOPING NEW CELL
AND GENE THERAPY (CGT) PRODUCTS FOR HEMOPHILIA

Federal Food, Drug, & Cosmetic Act (FD&C)
The key law giving FDA responsibility to oversee safety of drugs, A look at the
medical devices, and other products like food and cosmetics. U.S. US FDA Framework
Laws for Developing New
Public Health Service Act (PHS) made by Congress Cell & Gene Therapy
Law that gives FDA more pathways and manufacturing information for
biologic drugs, or drugs made from living organisms or their parts (e.g. Products for
genetic materials like DNA)
Regulations Hemophilia
21st Century Cures Act (CURES) of 2016 (CFR Title 21)
Law that creates regenerative medicine advanced therapy (RMAT) are made by FDA based on laws
framework, to speed development of innovative cell, gene, and tissue
products.
Coronavirus Aid, Relief, & Economic FDA Guidance
Security Act (CARES) of 2020 are made by FDA with help from the public
In response to COVID-19 outbreak, gives FDA greater responsibility to help industry and the public interpret regulations
for managing interruptions & disruptions in drug manufacturing.

2015 2017 2018 2020 GCT Framework:
Design of Early Regenerative Medicine Framework: CGT Therapy for Updated CGT Therapy for
Hemophilia Guidance
Phase CGT Trials Human Cell & Tissue Products CGT in Rare Disease Guidance 2021
Guidance Same Surgery Exception Hemophilia Guidance Long-term CGT Trial Follow-up CGT
RMAT Expedited Programs CGT Manufacturing Guidance Manufacture
Medical Device Use with Products During COVID
CGT “Sameness” for Orphan Drugs
Image Source: Adapted for Sigilon Therapeutics, Inc. with permission of the author. © 2021 Angela N. Johnson. All rights reserved.
SPECIAL ISSUE 2021 13