Pharmaceutical Outsourcing Q3 2023 - 24

CLINICAL TRIALS
Transitioning to Precision Medicine
Christian Olsen
Associate VP, Industry Principal, Biologics
Dotmatics
While pharma has produced some incredible blockbusters, it has
largely remained unremarkable at the individual patient level, with
only an estimated 30% to 50% of patients fully responding to many
leading conventional drugs.1
treatments just don't work for some patients. And thus, a lengthy
and costly cycle of trial-and-error often ensues, which can have grave
consequences for some patients. On top of this, a one-size-fits-all
approach is thought to waste tens of billions of dollars on ineffective
treatments annually.2
Moving Away from
One-Size-Fits-All Medicine
A gradual shift
One size does not, in fact, fit all. Some
remarkable feat of data collection, analysis, predictive modeling, and
clinical application.
The ultimate goal of precision medicine is to fully leverage complex
multimodal data and advanced technologies so that patients receive
the exact care they need-a marked difference from the historical
paradigm in which it's estimated that only about 60% of care is
deemed warranted, 30% is considered wasteful, and 10% is labeled
harmful.3
Only once we accept and work with the personal expression
of disease, taking into account the unique genotype (gene) and
phenotype (physical expression) of every person, will we able to
effectively treat diseases, such as cancer.
toward stratified medicine has been happening
for years. With this approach, both prophylactic care and disease
treatment are guided by grouping patients into broad categories,
such as by disease subtype, biomarker presence, responders versus
non-responders, clinical features, and the presence of single-gene
mutations linked to monogenic disease. Precision medicine aims to
move patient treatment to the next level.
What is Precision Medicine?
Precision medicine is rooted in the reality that the way patients
respond to treatment is heavily affected by their individual genetic
variability, their complex biology, and external factors. As such,
precision medicine personalizes each patient's treatment using
accurate diagnosis, a thorough understanding of disease mechanisms
and treatment options, and a multitude of individual patient factors,
such as a patient's genetic and metabolic makeup, lab and test
results, and even environmental conditions, lifestyle, and treatment
preferences. In other words, it's about delivering " the right treatment
to the right patient at the right time. " In some instances, this will be
a matter of narrowing down which existing treatments options are
best suited for a patient based on his or her profile and known data
on how certain patients respond to certain drugs. In other cases, it
might mean actually creating an individualized medicine, such as a
personalized cell therapy or replacement tissue created just for that
patient. While these examples are quite different, achieving either is a
Precision Medicine Advances
While there is still a long road ahead for precision medicine, some
promising advances have been made in recent years. We review a few
highlights below.
Biomarkers
We are seeing growing development and use of biomarkers. These
are informative biological measurements that have a proven link to
specific factors, such as patient's disease risk, potential drug response,
or likelihood of experiencing adverse reactions. They include things
like genetic mutation markers, blood pressure and glucose levels,
lab measurements, imaging results, microbial presence, etc. The US
Food and Drug Administration (FDA) categorizes biomarkers into one
of four groups: molecular, histologic, radiographic, or physiological.
A growing list of biomarkers have been developed to help inform
treatment decisions across a range of conditions, such as cancer, HIV,
thromboembolism, psychiatric disorders, neurologic conditions, and
infectious diseases. Biomarkers are also being used to inform early
drug development efforts and clinical trial design in order to reduce
timelines and increase the chances of success by better targeting
treatments to those patients most likely to respond.4-7
Wearables
Wearable devices, such as smart watches, shoe inserts, wrist cuffs, chest
straps, glucose monitors and pulse oxygen meters, are increasingly
Pharmaceutical Outsourcing | 24 | July/August/September 2023
Pharmaceutical Outsourcing Q3 2023

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