August 2024 SOCRA Source Journal - Journal - Page 33
JOURNAL ARTICLES
Clinical Trial Operations:
From Mouse Models
to the Market
Tia L. Patterson, Ph.D., CCRP
Regional Clinical Study Manager,
BeiGene, USA
Tia L. Patterson, Ph.D., CCRP
Abstract: Clinical research is comprised of the pre-clinical process, the drug development process, the clinical trial
process, and commercial and production processes. This article provides a high level overview of these processes,
with a focus on the point when a sponsor has a compound that is ready for clinical trials in humans. Key components
include selection of a clinical research organization, kick-off meetings, regulatory affairs (using North America as an
example), feasibility and site selection, budgets and contracts, and enrollment of research participants.
Introduction
This article will provide a highlevel overview of the process
of drug development through
approval. Preclinical through
all clinical trial phases will be
addressed with highlights of
key information that must be
part of each step in the process.
From inception of an idea to
a pharmacist being able to
昀椀ll a prescription written by a
clinician, the process takes a lot
of money and time to ensure a
drug is safe and effective.
The Pre-Clinical Process
Drug development usually starts
with an animal model, most
often in mice. Researchers begin
the pre-clinical process when
they have an idea that they want
to research in the laboratory to
move their research further. Preclinical research tests the idea in
animals using in vivo studies.
If researchers develop a process
or procedure that seems to
be effective, they would then
want to demonstrate the same
ef昀椀cacy in a human being by
evaluating the safety and dosing
of the drug. Researchers would
alter the animal re昀氀ect the
disease that is being studied to
ascertain whether the therapeutic
treatment has the potential to
work in animals. Once the dosing
and safety pro昀椀le is identi昀椀ed in
the animal, a starting dosage is
determined for a phase 1 (昀椀rst in
human) clinical trial to determine
whether the therapeutic design
will also have the potential to
work in human beings.
To determine the effect of the
drug, the animal model needs
to use the same disease that will
be studied in human beings.
Researchers are looking at drug
responses in the animal model
to determine whether the drug
or biologic treatment will be
effective against the disease.
Assuming that researchers do
昀椀nd ef昀椀cacy, they must then look
at safety before transitioning to
clinical trials in human beings
(Figure 1 on next page).
There are four steps in the safety
exploration: Target selection,
series selection, pre-lead
selection, and lead selection.
Target selection evaluates
the safety of the target by
identifying toxicities related to
pharmacology and toxicities
associated with the intended
use of the drug and potential
combinations. Series selection
looks at in silico tools, in vitro
screens, and selectivity pro昀椀ling.
Pre-lead selection evaluates the
safety of the pre-leads in in vitro
cytotoxicity and genotoxicity
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