3for1 Expo 02November2021

1. Understanding DTx:
Journey into Imagination
Emily Kunka, MS
Digital Business Transformation

3. Digital Therapeutics (DTx)
deliver medical interventions directly to patients
to prevent, improve, manage, or treat disease.
They are evidence-based, clinically evaluated
software.

4. Digital Health
Data capture, storage, and display for care
provision and management
Digital Medicine
The use of digital technologies as tools for
measurement and intervention in the
service of human health
Digital
Therapeutics
Deliver intervention to prevent,
improve, manage, or treat disease

5. A Closer Look
Digital Solutions
Digital Medicine
Adherence programs
Patient journey / experience
Software IN a medical
device (SiMD)
Software AS a medical
device (SaMD)
Software within hardware which
powers or controls the mechanics
of the physical device.
Direct refill services, fitness and
lifestyle trackers
Companion labeling app for a
marketed drug
Standalone software intended to
prevent, modify, or treat disease
DTx

6. Software as a Medical Device (SaMD)
is software intended to be used for one or more
medical purposes that can perform without
being part of the device hardware

7. Therapy Solution
3 Requires FDA approval or
clearance as SaMD
Digital Therapeutics vs. Digital Solutions
4 Generally reimbursable by
payers
High(er)
than
solution
Low
5 Cost to Develop
1 Generally requires an Rx to use
2 Evidence-based, supported by
clinical trial data

8. Regulatory Pathway Under Construction
Although there are guidelines (e.g. the Guidance on Mobile
Medical Applications), a formal SaMD pathway does not
currently exist and is in development

9. In the Meantime...
We are currently borrowing the existing medical device pathway
under the Center for Devices and Radiological Health (CDRH)

10. Premarket Submission Pathways
De Novo
○ Granted to novel devices of low to
moderate risk
○ Aims to prove superiority or non-inferiority of the product
to the standard of care
○ Clinical data is required to support reasonable assurance
of safety and effectiveness

11. Premarket Submission Pathways
○ Aims to demonstrate
substantial equivalence to
another approved “predicate”
product which is legally
marketed in the US.
510K Clearance

12. Breakthrough Designation
Devices with 510(k) clearance that also
● Are for serious, life-threatening, or
debilitating diseases or conditions
● Have preliminary clinical evidence of
substantial improvement over available
therapy
● Offer a distinct advantage over existing
devices
● Allow for expedited review
● Can make reimbursement easier

13. Trends in Existing DTx
● Mainly used in chronic disease
● Focus on real world data (RWD)
● Focus on outcomes allowing value-based payment
models
● Largely based on Cognitive
Behavioral Therapy (CBT)
○ Focus on changing patient’s
behavior or thinking

14. DTx
Development

15. Development Process
● Shorter development
lifecycle
● Flexible development
process
○ Able to update and
enhance the product in
between each dev
phase

16. Key Differences: DTx vs. Drug Development
It is an iterative and agile process from prototype to the
minimum viable product (MVP)
*only pivotal phase III product and its data are submitted
for approval

17. Key Differences: DTx vs. Drug Development
Implement
design controls;
a formal
methodology of
product
development

18. Key Differences: DTx vs. Drug Development
Independent (3rd party)
cybersecurity risk
assessments are performed
annually which consider how
new risks are mitigated and
design decisions are made

19. $45-55 MM
Cost to bring a concept through FDA submission is significantly lower
than for molecular drug development ($350 MM)

20. Clin Dev
2-5 years
As opposed to the 10-15 years clinical development
takes for molecular drugs

21. Mechanism of Action (MOA)
● How the product works e.g.
how it affects the outcome(s)
of interest
● Similar to an “active
ingredient” in a drug

22. Target Product Profile (TPP)
● Patient population(s) of interest
● Product concept and prototypes
○ Hypotheses testing product
usability and engagement
levels with each user (i.e.
HCPs, patients, caregivers)
○ Hypotheses testing
endpoints/potential impact

23. Development Standards
The precise path a DTx must
take and the level of clinical
evidence it must provide is
dependent on
● the novelty of the product
● how great a risk it poses
should it malfunction

24. RWD STUDIES
Post-Market Health
Economics and Outcomes
Research (HEOR) studies
to assess ability to lower
costs and improve
outcomes.
At least 1 year
$1MM - onward
1,000s of patients
DISCOVERY
Target Product Profile
(TPP) defines the desired
characteristics, intended
use, and target
population(s). Feasibility,
usability.
6 months to 1 year
$1-5MM
0 patients
PIVOTAL STUDY
Patients of a wider
demographic to show
efficacy. Almost
always an RCT with
control arm.
1-2 years
$7-20MM
200-600 patients
POC & PILOTS
Proof of concept
and pilot studies to
show mechanism of
action working and
safety. Explores
patient engagement.
6 mo - 1 yr
$1-5MM
150-300 patients
Development Lifecycle
APPROVAL

25. PDT Pilot Studies
● Gather data on optimizing
usability, engagement, and
efficacy
● Improve the user experience
while not affecting the
mechanism of action
● Explore a range of “dosing”
regimens

26. Developing Treatment Regimens
based upon physician optimization from observation
and experience, rather than dose-response theory or
logic.
Methodology is empirical

27. PDT Validation Studies (RCTs)
● Trials go direct-to-patient using a
decentralized clinical trial (DCT) model
○ Quicker speed of recruitment
● Endpoints are largely digital versions of
traditional clinical measures, but can
also be novel digital biomarkers
(dBMs)
● dBMs are compared to existing gold
standard endpoints accepted by
providers and payers

28. Control Arm / Sham Product
● Use of a control group may be
unethical or not feasible (unable to
mask) in some cases
● Sham app mimics the digital
intervention software without
including the MOA
○ Need to ensure sham product
does not create a clinical
response through placebo effect
Sham Real

29. Unique Challenges
● Hitting “okay” button ≠ sufficient consent
● MOA can be unclear
● Poorly defined exposure (using time,
frequency, duration) makes response
relationship and therapeutic target
(optimum exposure) difficult to measure
● Inter-individual variability in response

30. Treatment Paradigms
1. Monotherapy - First line therapy on its own
2. Concert therapy - Designed to be used with
other (oftentimes traditional) interventions
* Some products can be used as both monotherapies and concert
therapies, depending on design and evidence

31. Concert Therapies
Companion - Designed be used to treat a condition alongside
pharmacological product and/or HCP treatment. Usually not
FDA approved.
Combination - Designed to be used in conjunction with a
specific pharmacological product (single national drug code
unique device identifier [NDC-UDI])
Adjunct - Designed to be used with a category of
pharmacological interventions or treatments

32. Advantages to Pursuing FDA Approval
● Increased scientific and clinical credibility with HCPs
● Creates higher barrier of entry to competitors
● Allows companies to make a claim regarding a
specific condition
● Brings product legitimacy/acceptance and ability to
be reimbursable from payers
● Generally allows for a higher price point

33. Drawbacks of Pursuing FDA Approval
● Slower, more costly
development process
● Less flexibility to
continuously improve the
product
● Incurs commercial risk as it
defers ROI

34. Benefits
● FDA approval does not
guarantee payor uptake or
patient engagement
● Iteration of the product
after FDA approval can
require additional
approvals
Risks
● Collect usability data
○ Give visibility into
personalized care
delivery
● Reduced safety concerns
● Dev timeline is shorter
● Cost of goods decreases
with volume
● Requires less human
capital

35. Post Market

36. Post Market SaMD Surveillance
● Must demonstrate continued safety,
effectiveness, and performance in the
real world
● Data from several sources:
○ SaMD product itself
○ Registries
○ Electronic health information
sources
■ National Evaluation System for
health Technology (NEST)

37. Real World Performance Data (RWPD)
Real World
Health Data
(RWHD)
User
Experience
Data (UXD)
Product
Performance
Data (PPD)

38. Real World Health Data (RWHD)
Outputs and outcomes related to the SaMD
definition statement. Used to monitor product’s
continued safety and effectiveness.
Can inform changes to the intended use of a
product, and support expanded functionalities
and use in broader target populations.

39. User Experience Data (UXD)
Outputs derived from user experiences related to
the real-world use of a product.
Facilitates timely identification and correction
of user issues, and improves the utilization and
effectiveness of the software.

40. Product Performance Data (PPD)
Outputs and outcomes demonstrating the
accuracy, reliability, and security of a product.
PPD monitoring allows for timely patches and
updates to correct software bugs and security
vulnerabilities. Can support modification of
claims.

41. Regulatory Oversight and Policing
Responsibility may depend on the
regulatory designation
○ Food and Drug Administration
○ Federal Trade Commission
○ Federal Communications
Commission
○ National Institute of Standards and
Technology
○ Office of the National Coordinator
for Health Information Technology

42. Post Approval Obligations
Failure to comply with
post-clearance or approval
regulatory requirements could
subject the company to
○ Substantial penalties
○ Recall/withdrawal of
product from the market

43. Commercialization

44. Launch Strategy
● Marketing must also educate and drive
awareness
○ Utilize medical science liaisons, key
opinion leaders
○ Attend industry events which speak
to the data and answer questions
○ Publications to peer reviewed
journals

45. Scale and Commercialization
● Pathways to scale and
commercialize are still
largely uncharted
● The market is rapidly
evolving, getting
increasingly competitive
○ Difficult to forecast
demand

46. Customer Economic Model Contract Terms
Providers and
Employers
Risk-sharing Per member per
month (PMPM)
Payors % Savings Recurring
Pharma Licensing, royalty,
milestone
Recurring or term
Patients Free with
reimbursement
Over-the-counter
Example
Payment
Model
by
Customer

47. Business Model - B2C
Advantages
Early revenue opportunities
Demonstrate product traction
Disadvantages
High costs to commercialize
Not covered by insurance

48. Business Model - B2B Self-Insured Employers
Advantages
Faster sales cycle
Disadvantages
Requires an additional sales step
to employee to capture usage fee
Labor intensive, deal by deal

49. Business Model - Health Systems, Payers, PBMs
Advantages
Set rate for DTx usage
Use of digital formulary
Disadvantages
Long contracting process
May require pilot or testing period
Fee-For-Service

50. PBM Formularies - Examples
● CVS Caremark
○ Sleepio
○ Daylight
○ Hinge Health
○ Hello Heart
○ Torchlight
○ Whil
○ Vida
○ Naturally Slim
○ Weight Watchers
○ Kurbo
● Express Scripts
○ Livongo
○ Propeller Health
○ Omada Health
○ LifeScan
○ Learn to Live
○ SilverCloud Health
○ Wildflower
○ Quit Genius
○ Prevail Health
○ Back with Care
○ Hinge Health
○ RecoveryOne

51. Business Model - Health Systems and Payers
Advantages
Based on cost savings
Disadvantages
Difficult to quantify cost savings
Value Based Reimbursement
Advantages
Uses CPT codes
Disadvantages
Requires FDA approval and path
to FDA approval is still undefined
Device-Like Reimbursement
[Alternatives under consideration]

52. PDT Pricing in the Market
Price of DTx is significantly less than conventional therapies
*Rx is typically for a 3-6 month treatment period

53. Pricing Strategies
Moving from a direct-to-consumer (DTC) offering to
a medical benefit, to a pharmacy benefit can 10x
the price each time
$20/month
$200/month $2,000/month
DTC Medical
Benefit
Pharmacy
Benefit

54. Perspectives

55. Patient Perspective
If an FDA-approved
app or online tool was
available to treat your
medical condition,
how likely would you
be to try it?
Very likely 21%
Somewhat likely 33%
Somewhat unlikely 15%
Very unlikely 14%
I don’t know 17%
Source: PwC Health Research Institute’s Annual Report

56. HCP Perspective - Rx Anecdote
A doctor has 500 patients… but only 100 are prescribed the PDT. Why?
100 patients “Do everything I ask them to do. They don’t need a PDT.”
100 patients “Never do anything I ask them to do. They would never use a PDT.”
100 patients “Don’t have a smartphone, and can’t use the PDT.”
100 patients “Don’t have a health plan that would pay for a PDT.”
“After this triage, there are only 100 left who can use the PDT.”

57. Summary

58. Paradigm Shifts
Hardware Software
Deterministic Risks of
Physical Products
Evolving Risks of Digital
Products
Stable and Predictable
Volume
Potential for
Exponential Volume

59. Reality Check
● Patient, provider, and payer appetite for DTx is
still uncertain
○ We still need to show long term patient
engagement with DTx products
● PDTs considered software as a medical device
(SaMD) do not currently have a benefit category
recognized by the Centers for Medicare and
Medicaid Services (CMS)

60. Unmet Needs / The Road Ahead
● Integrate DTx into electronic prescribing, dispensing, and
medical record platforms and HCP workflows
● Expand outside of chronic diseases
● Build payer reimbursement models (dedicated national codes!)
● Develop better reporting metrics on DTx utilization
● Grow more comprehensive DTx formularies
● Develop regulatory evaluation and commercialization
pathways for other major health authorities (EMA, TGA, etc.)

61. UCB DBT’s Vision in DTx
● Partner externally to co-develop and
co-commercialize new DTx products
● Add DTx into a more comprehensive portfolio of
products for which UCB can synchronize our
products and services for more a holistic,
continuous patient care continuum

62. Imagination, imagination.
A dream, can be, a dream come true.
With just that spark, from me and you.
One little spark, of inspiration
Is at the heart, of all creation.
Right at the start, of everything that's new.
One little spark, lights up for you.

63. Thank You!

64. ● https://www.darkdaily.com/fda-approves-digital-therapeutics-technologies-to-treat-pati
ent-behavioral-conditions-that-interfere-with-positive-healthcare-outcomes/#:~:text=D
igital%20therapeutics%20combine%20apps%20and,focus%20on%20specific%20clinical%
20outcomes.
● https://dtxalliance.org/understanding-dtx/#:~:text=Digital%20therapeutics%20(DTx)%20
deliver%20medical,spectrum%20of%20diseases%20and%20disorders.
● https://www.nature.com/articles/d41586-019-02873-1
● https://www.fda.gov/media/112680/download
● https://www.fda.gov/media/112901/download
● https://www.pwc.com/us/en/industries/health-industries/library/digital-health-needs-evi
dence-and-buy-in.html
● https://www.nature.com/articles/s41746-020-00370-8
● https://torreya.com/publications/digital-therapeutics-and-future-of-pharma_torreya-feb
2020.pdf
● https://medrhythms.medium.com/looking-back-ahead-at-the-digital-therapeutics-indus
try-2e8454d342c5
● https://www.fda.gov/media/106331/download
● https://www.fda.gov/media/80958/download
● Digital Therapeutics, Dawn of a New Treatment Class, Ahead of the Curve, Cowen and
Company
Sources