YSM Issue 93.2

FEATURE
Genomics / Clinical Research
LETTING
EXPERIENCE
GUIDE THE WAY
The ultimate goal of clinical and
translational research is to leverage
scientific discovery and innovation
to drive improved treatments and patient
outcomes. In light of this goal, it seems
reasonable that patients and the public
should be involved in the research process.
Yet, there is a disconnect between researchers
and the public, even in translational settings.
There is increasing awareness of the
necessity of patient and public involvement
in both clinical and preclinical research.
There are several disadvantages at play
when these partnerships are absent.
For one, scientists often lack personal
experience with the diseases they study.
As a result, they may not be attuned to the
most pressing treatment needs in disease
communities, potentially limiting their
ability to translate knowledge production
into knowledge use. In other words, lack of
patient input on the research process can
result in research waste––in which scientific
communities produce research findings
that have minimal real-world application.
Moreover, given that a great deal of research
is publicly funded, scientists have a duty to
be accountable and transparent with the
public. This is greatly facilitated by public
involvement in research, which therefore
has inherent democratizing value.
What is patient and public involvement?
The patient and public involvement (PPI)
model is an approach that acknowledges
the need to include patients and the
public in research. PPI is flexible and can
consist of varying degrees of participation
BY ZOE POSNER
in the research process. Involvement often
focuses on including patients in research
design and in disseminating results. Less
commonly, it encompasses participation
in data analysis and methodological
design. Significantly, PPI methods can be
implemented in a broad range of research
areas, from clinical studies and cancer
research to groundbreaking research in
the basic life sciences.
PPI offers several benefits over traditional
research, which is executed solely by the
investigating team of scientists. As implied
by its name, PPI democratizes research by
increasing the number of voices included
in the research process. Researchers are
optimistic that including public voices
can produce studies that are more ethical
and practical in nature. In health studies
especially, patients can offer critical and
overlooked perspectives in the research
process. They can highlight aspects
of the disease and treatment that are
deprioritized in the academic community
(such as drug-induced cytotoxicity), and
also help researchers identify the most
pressing pathobiological questions in the
patient community.
Recently, PPI is gaining traction for
its potential to address representational
inequality in research. There is an extreme
lack of representation in biomedical
sciences and health professions. As a
result, the unique health perspectives and
grievances of minority populations are
easily overlooked. Additionally, those who
participate in clinical trials or experimental
drug treatments are typically those with the
best access to healthcare––most frequently,
individuals who are affluent and white.
These factors contribute to persistent
disparities in health outcomes. PPI, by
including a diverse patient population, can
help foreground the health experiences
of marginalized populations. Researchers
who conduct PPI studies are aware of the
need to increase diversity in research.
While this awareness is encouraging, many
of these researchers recognize that thus
far, a majority of patient involvement in
research is by wealthier white patients.
Groups like the Community and Patient
Partnered Research (CPPRN), which
focuses on improving mental and
behavioral health outcomes for Black and
Hispanic populations, are working towards
ensuring greater diversity in patient
networks to overcome this challenge.
Finally, patient-centered research holds
promise for improving the study of rare
pathologies, including rare cancers.
Rare diseases are difficult to study due to
restricted availability of patient samples.
Patient-centered approaches to rare
diseases, by generating patient networks,
can facilitate the collection of patient
samples and data over a broad geographic
range. This can compensate for the low
frequency of disease incidence. The Count
Me In Initiative has spearheaded five
projects involving patient-partnered cancer
research. Most recently, the initiative has
launched The Angiosarcoma Project,
which focuses on angiosarcoma—a rare,
notoriously aggressive cancer that develops
in the lining of blood and lymph vessels.
The Angiosarcoma Project: A model for PPI
The Angiosarcoma Project, with 338
patients, is the largest angiosarcoma
project to date and has produced several
novel and high-impact findings. The
project was led by Corrie Painter of the
Broad Institute. In the initial stages of
the project, Painter ensured that patients
were involved in the development
of the online platform that would
then be accessible to them and other
patients. After receiving guidance from
angiosarcoma patients, Painter and
her team built out the project, received
direct patient feedback, and synthesized
that feedback in an iterative process.
This serial generation-feedback-revision
IMAGE COURTESY OF FLICKR
24 Yale Scientific Magazine September 2020 www.yalescientific.org