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0:00
This is a talk on
the overview of
regulatory development for
gene and cell-therapies
in the United States,
delivered by Carolina
Iglesias-Lopez
from the University
Autónoma of Barcelona.
0:15
What the talk covers is
about the US regulation of
cell and gene therapies
and which products are
defined and classified as
cell and gene
therapies in the US.
The speaker then
goes into depth into
the regulatory development
and will give us an overview
of the regulatory road map
for cell/gene therapies and
discusses the
regulatory procedures
available during
that development,
in particular, focusing
on scientific advice,
expedited programs, and
BLA review and approval.
Finally, the speak
gives an overview of
the regulatory
milestones achieved for
the current approved
ATMP products.
0:56
In the US, cell and
gene therapies,
also known as advanced
therapies are classified as
biological products
and are regulated by
the Food and Drug
Administration or the FDA,
which is the regulatory
medicines agency in the US.
The US federal
regulatory framework
consists of two main statutes:
the Federal Food Drug
and Cosmetic Act,
FDCA, and the Public
Health Services Act, PHSA,
which provide the FDA with
the legal authority to regulate
human medicinal products
including drugs, biological
products, and devices.
Biological products are
specifically regulated
under section 351 of
these two main statues.
In turn, FDA regulations are
contained in the Code of
Federal Regulations, CFR,
which provides details on
how the FDA implements the
activities that are defined
in the PHSA and FDCA.
Regulations for biological
and medical devices
are found in Title 21 of the
Code of Federal Regulations.
As we will explain
later, in the US,
regenerative medicine
therapy implicitly
includes advanced therapy
medicinal products.
Finally, it is important
not to confuse
advanced therapy
medicinal products with
other category of products
called "human cells
tissues and cellular
tissue-based products,
abbreviated as HCT/Ps,
which fall under other
legislative sections
and are not considered
biological products.
