Digital biomarkers

1. Transforming Clinical Development
through Digital Biomarkers (dBMs)
Emily (Kunka) Lewis, MS, CCRP
Digital Business Transformation, Neurology
UCB Biopharma

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UCB is a global
biopharmaceutical
company focused on
solutions for people
living with severe
diseases.
By putting people at the
heart of everything we do,
we enable people to live
their best lives, as free
as possible from the
challenges of disease.
From Solution
to Patient
We strive for a unique
patient experience,
providing solutions
with the highest
possible impact.
From Patient
to Science
We pursue a deep
understanding of patient
sub-populations to
develop an original
scientific hypothesis.
From Science to Solution
We aim to translate scientific hypotheses into
innovative solutions and engage patients in the journey.

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UCB’s patient value ambition
UCB Team Name [XXXXXXX] - UCB - Approval [XX-XX-XXX] - Approval date [XX Month XXXX] ! GO TO INSERT>HEADER FOOTER to change.
Creating value for patients now and into the future
Unique outcomes that
help specific patients
achieve their goals
Best individual
experience
Ensuring access to all
those who need these
solutions in a way which
is viable for patients,
society, and UCB

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Becoming more customer and
data-centric in our approach Developing digital platforms for
personalized patient care and
disease identification
Digital mindset Using data and technology
with purpose
Partnering externally for
greater impact
Our digital business transformation journey:
a strategic priority

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Digital
Transformation
Data and Real
World Evidence
Open
Innovation
We continuously innovate
References: 1. UCB 2021 Annual Report.
R&D
28%1
UCB invests about a
quarter of its revenue
in R&D annually
Continual Investment in
Research & Development

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The Evolution of Digital Health in Life Sciences
Testing Contemplation Innovation Realization
2010 2015 2020 2023
● Testing and
piloting of
isolated point
solution →
“pilotitis”
● Application of
“blue sky”
thinking
● Opportunistic
lens
● Advanced
experimentation
with increased
risk
● Struggle to
show ROI as
outcomes have
not met lofty
expectations
● Shift to strategic
lens
● Investment hike
● Iterative and
agile
experimentation
● Digital
ecosystem
development
● Increased
adoption due to
regulatory
advancements
and roll backs
● Seen as an
enabler for
achieving
strategic
objectives and
incremental
value creation
● Enterprise level
adoption and
scale with
revenue
streams

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Life Science companies must be guided by
enterprise strategies that prioritize the
delivery of purposeful, multi-pronged, yet
integrated treatment experiences
So, What Have We Learned?

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What are Digital Biomarkers?
Sensors in
Connected
Devices
Computational
Methods
Digital
Biomarkers
Health indicators that are collected through digital devices which
can provide objective, real-time, continuous,
high-resolution data about a person’s physiological and
behavioral status.

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Types of dBMs
Predictive
03
Biomarker used to identify individuals who are more
likely than similar individuals without the biomarker to
experience a favorable or unfavorable effect from
exposure to a medical productor environmental agent
Monitoring
02
Biomarker measured repeatedly for assessing
status of a disease or medical condition or for
evidence of exposure to (or effect of) a medical
product or an environmental agent
Diagnostic
01
Biomarker used to detect or confirm the presence
of a disease or condition of interest or identify
individuals with a subtype of the disease
PD/Response
06
Biomarker used to show that a biological
response, potentially beneficial or harmful, has
occurred in an individual who has been exposed
to a medical product or an environmental agent
Safety
05
Biomarker measured before or after an exposure
to a medical product or an environmental agent to
indicate the likelihood. presence, or extent of
toxicity as an adverse event
Prognostic
04
Biomarker used to identify likelihood of a clinical
event, disease recurrence or progression in
patients who have the disease or medical
condition of interest
Susceptibility/Risk
07
Biomarker that indicates the potential for
developing a disease or medical condition in an
individual who does not currently have clinically
apparent disease or the medical condition
Biomarkers,
EndpointS
and other
Tools
(BEST)
Framework

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Current Applications
Sleep
Speech
Behavioral
Habits
Activity
step count
heart rate
Cognitive
Assessments
Gait
stride velocity
bradykinesia

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Key Questions to Navigate
● What to measure?
● How do we measure it? What
is the right tool?
○ What is needed to deploy
remotely at scale?
○ How do we validate it?
○ How do we analyze it?
● Why is it important?
● What do we want to achieve?
● What is the right regulatory
pathway?
● How will we integrate it into
clinical practice?

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Identifying What to Measure
Determine the MAH
● Determine the
meaningful aspect of
health (MAH)
● Identify the concept
of interest (COI)
● Define the digital
measure (e.g.
outcome/endpoint)
Understand Patient Journey
● A disease is more than
just a collection of
symptoms and issues.
● Understand the
nuances of the
condition that drive
patient decisions and
behaviors.
ID Specific Pain Points
● Identify the wider context
of the patient experience
● In what capacity would
digital measurement fit
into the patient journey?

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What is the Right Tool? How Do we Measure It?
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Why Are dBMs Important in R&D?
Disease
Heterogeneity
Difficult-to-Measure
Disease States
Treatment
Effect

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Why Are dBMs Important in R&D?
● Capture health status in
the real world, passively,
continuously over 365 days
○ Follow symptom
fluctuation longitudinally

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Added Bonuses
● More realistic
measures of patients’
lived experiences
● Identify potential
adverse events
earlier
● Can be used for
protocol
design/informing
future studies

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TRADITIONAL DIGITAL
SOLUTIONS
No
symptoms
First
symptoms
Diagnosis
Treatment
Therapy
cycles & changes
Outcome
See Doctor
Pharma Core Business
Digital Paradigm Status Quo
• Pt Support Programs
• Connected devices
• Proprietary patient
apps
• Branded websites
Narrow window into
patient journey and
limited scope of support

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TRADITIONAL DIGITAL
SOLUTIONS
No
sympto
ms
First
symptoms
Diagnosis
Treatment
Therapy
cycles & changes
Outcome
Booking
Pharma Core Business
Digital Paradigm with a Platform Approach
• PSP
• Connected devices
• Proprietary patient apps
• Branded websites
• Reach a wider population
• Leverage network effects
• Address specific needs at
various time points
PLATFORM-ENABLED OPPORTUNITY

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Challenges of dBMs in Clinical Development
● Existing gold standards
(either not good to begin with
or one does not exist)
● Many trials currently using
dBMs as exploratory
endpoints, need to move to
evolving them towards
primary and secondary
endpoints

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What Do We Want to Achieve?
● Ability to digitally
phenotype/sub-categorize
participants to enrich trial
population at enrollment and
increase the likelihood of detecting
response to treatment
● Reduce the need for participant
travel
● Shorten clinical trial duration

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What Do We Want to Achieve?
● Increased engagement and adoption
● Increased commercial viability of products and
solutions, utilizing new business models to pursue
■ Label expansion
■ Outcomes research used for reimbursement
■ Scale of solutions which reduce healthcare costs

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What is the Right Regulatory Pathway for Products Using dBMs?
Is your
dBM is a
part of a
product?
■ De Novo
■ 510k
■ PMA

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Why Are dBMs Important in Healthcare?
● Earlier disease detection
● More accurate diagnoses
● Monitoring of disease progression
● Delivery of timely interventions
● Personalized treatment plans
● Inform public health decision-making
● Allows patients to participate in their own
healthcare in a more proactive and informed way
● Improve the quality of information available to HCPs

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How Will We Integrate dBMs Into Clinical Practice?
● Ensure validation for clinical use
○ reliable, accurate, sensitive,
and specific for the condition it is
intended to diagnose/monitor
● Integrate dBM into clinical workflow
(into the EHR, etc.)
○ Establish protocols for correct
usage, subsequent course of action
○ Regularly review data & outcomes,
adjust protocols

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How Will We Integrate dBMs Into Clinical Practice?
● Staff training and
education
○ Ensure HCPs know
how to interpret
data/insights, properly
follow product’s
indications for use
● Work with policy experts
to inform policy and
guidance

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Common Missteps
● Slow decision making and
tendency to over-engineer digital
solutions
● Underestimation of the complexity
and many layers of decision making
required
● Over-reliance on external partners
● To move from buy to build we need
more mature internal tools,
processes, and procedures

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Where Do Folks Go Wrong?
Digital health is treated as a separate function of
the business, rather than an intrinsic driver or
enabler of a broader integrated engagement
and experience strategy around patients

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Remaining Work, As An Industry
Continuous refinement of standardized definitions,
validation criteria, and data security protocols
Democratization of digital knowhow and
capabilities across organizations at an enterprise
level
Advancement of nascent regulatory pathways and
reimbursement policies

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Need for Collaboration Among Key Stakeholders
Tech
Companies
Researchers
Healthcare
Providers
Patients
*pre-competitive,
data-sharing
consortia are key
here

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What Does All of This Mean?
A frame shift aligning our corporate
strategic objectives with what patients
and healthcare providers value (and their
pain points)

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UCB
@UCBUSA
facebook.com/UCBbiopharma
www.ucb-usa.com
Thank You!

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