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0:00
Hello. My name is Seoan Huh,
the lead author of Chapter 13,
Regulatory Considerations
in the Development
of Gene Therapy Products.
0:12
As shown in this second slide,
I will walk you through
a brief introduction of
regulatory landscape
in gene therapy,
a quick overview of
regulatory definitions
of gene therapy,
designations, and expedited
regulatory pathways
to get these lifesaving
medicines to patients faster,
several unique regulatory
considerations for
gene therapy and close
with emerging trends.
0:41
Here's a brief introduction of
the gene therapy
regulatory landscape.
Since the first gene
therapy trial in the 1970s,
the regulatory landscape
for development of
gene therapy products has
evolved considerably,
and the field has grown
exponentially since then,
exceeding 500 clinical trials
involving gene therapy
by the time this chapter
was written in 2022.
Here is a quick
historical overview of
the five decades since the
first gene therapy trial.
In the US, the NIH formed
a Recombinant DNA
Advisory Committee,
or the RAC, in the
1970s to provide
a platform for discussion of
emerging techniques in
the field of gene therapy
and to reduce the risks
associated with gene
therapy transfer protocols.
In the 1980s, this
committee first approved
a clinical trial protocol
for gene therapies.
In the 1990s, FDA and
NIH assumed control of
clinical trial protocols,
and the FD&C Act introduced
many amendments and related laws
to modernize the regulations,
including breakthrough
therapy designation, RMAT,
and comprehensive guidelines for
cell and gene therapies in 2010.
In the EU, which you
can see on the bottom,
the concept of ATMPs was
introduced in the early
2000s and in 2008,
established mandatory
requirements
for ATMPs to obtain
marketing authorization.
In 2010, we saw the
establishment of
PRIME scheme to facilitate
innovative medicines
to be developed.
As the field grows, regulatory
science is constantly
adapting to new
technologies to ensure
regulatory oversight to
meet the complexities and
challenges of these technologies
without stifling innovation.
We will look into some of
the key considerations
in gene therapy regulations
in this lecture.
