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Digital Therapeutics
Development and Regulatory Requirements
Emily Kunka, MS
Digital Business Transformation
Nomenclature
Digital Health Data and information capture, storage, and display. Examples include
software for care administration and management (i.e. telehealth).
Digital Medicine
Measurement products. Examples include digital biomarkers,
digital diagnostics, remote patient monitoring, eCOA, clinical
decision support software (may or may not require human
intervention).
Digital
Therapeutics
Deliver intervention to prevent, improve, manage, or
treat disease (including adjunct treatments).
Solution Therapy
2 Supported by
clinical trial data
3 Require FDA approval
or clearance
Digital Solutions vs. Digital Therapies
4 Generally
reimbursable by
payers
Low
High(er)
than
solution
5
Cost to Develop
1 Generally requires a
prescription to use
Examples of Each
Digital Solutions
Digital Therapies
Adherence programs
Patient journey / experience
Software-enabled hardware
Algorithms intended to
diagnose or predict
Software intended to treat a
condition or modify a behavior
Direct refill services, fitness
trackers
Companion labeling app for a
marketed drug
Course or
program
Video
game
Digital Therapeutics (DTx)
deliver medical interventions directly to patients
using evidence-based, clinically evaluated
software to treat, manage, and prevent a broad
spectrum of diseases and disorders.
Software as a Medical Device (SaMD)
is software intended to be used for one or more
medical purposes that perform these purposes
without being part of a hardware medical device
Broad DTx Buckets
Software for disease
management
03
● Enables informed decision making
about care to control disease
● Typical in chronic disease states
Software as adjunct
to traditional tx
02
● Enhances standard of care
● Used in conjunction to transform or
work with existing treatment
Software as a direct
treatment
01
● Could replace standard of care
● Could be used independent of other
therapy options
Treat A Disease Manage a Disease Improve Health
Function
(includes prevention)
Medical Claims Medium to high risk
claims
Medium to high risk claims Low to medium risk
claims
Clinical
Endpoints
Must use clinical
endpoints to support
product claims
Must use clinical endpoints to
support product claims
Must use clinical
endpoints to support
product claims
Clinical
Evidence
Clinical trials and
ongoing evidence
generation required
Clinical trials and ongoing
evidence generation required
Clinical trials and ongoing
evidence required
Regulatory
Oversight
3rd party validation of
efficacy and safety
claims by regulatory or
equivalent national body
3rd party validation of efficacy
and safety claims by
regulatory or equivalent
national body
Degree of regulatory
oversight depends on
local regulatory body
frameworks
Patient Access Prescription required Prescription required OR
Non-prescription product
Prescription required OR
Non-prescription product
Digital Health Innovation Action Plan
● Issuing guidance to provide clarity on the medical
software provisions of the 21st Century Cures
legislation
● Launching pilot pre-certification program to develop a
new approach to digital health technology oversight
● Building FDA’s bench strength and expertise in
CDRH’s digital health unit
Regulatory Landscape
● Center for Devices and Radiological Health (CDRH)
● Guidance on Mobile Medical Application (MMA)
● Formal SaMD pathway in development
● Prove superiority via de novo or substantial
equivalence via 510K pathways for medical devices
● Can receive breakthrough designation for expedited
review
● Approval granted or “FDA cleared”
Regulatory Approval
● FDA approval brings product legitimacy/acceptance
and ability to be reimbursable from payors
● FDA approval brings price point higher
● FDA designates certain DTx as prescription only or
direct to consumer
● FDA can exercise enforcement discretion waiving
regulatory oversight if it decides a product is low risk
● May require a change control plan
Regulatory Caveats
● DTx that aim to prevent disease by helping patients
change their lifestyle can be marketed in the US
without providing safety and efficacy evidence to
the FDA
● Current approval process is not set up to handle
DTx that are solely a delivery mechanism for
already-proven therapies not delivered via
technology
Freedom of Information Act
● Allows anyone to see
the correspondence
DTx companies have
had with the FDA to
classify their devices
and obtain clearance
https://www.accessdata.fda.gov/cdrh_docs/pdf20/DEN200029.pdf
DTx Classification Components
Product’s purpose or
indication for use
Target product profile for
use and function
FDA’s Use and Function Classification Categories
Address a
medical
condition
CATEGORY 1
Manage or
prevent
disease or
disorder
CATEGORY 2
Optimize
medication
usage
CATEGORY 3
Treat
medical
disease or
disorder
CATEGORY 4
Categorization
Examples Company Product Description TA Category
Pear reSET
reSET-O
Adjunct Tx for Substance Use Disorder
(SUD)
CNS 4
Propeller RESPIMAT Optimize healthcare utilization in
asthma and COPD
Respiratory 3
Akili AKL-T01 Adaptive sensory stimulus software
(video game) for ADHD
CNS 4
Dthera DTHR-ALZ Reminiscence therapy for Alzheimer’s CNS 4
Big
Health
Sleepio Sleep improvement program with CBT CNS 2
Cognoa Various AI based digital diagnostics and
personalized therapeutics
CNS, Peds 2
KAIA Motion coach Behavioral therapy (physical exercises
and education) for chronic back pain
Musculoskel. 2
Product’s Intended Purpose
● To support a certain type of product claim
● To generate clinical evidence
● To give patients access to product
● To redefine existing relationship to concurrent
therapies
→ written in a definition statement, determines
FDA submission requirements
SaMD Definition Statement
Should include:
● A description of the SaMD’s core functionality
● The state of the healthcare situation or condition
the SaMD is intended for (critical, serious,
non-serious)
● Significance of information provided by the SaMD
to the healthcare decision maker (diagnose/treat,
drive clinical management, or inform clinical
management)
SaMD Proposed Review Pathway Framework
State of
Healthcare
situation or
condition
Significance of information provided by SaMD to
healthcare decision
Treat or Diagnose Drive Clinical
Management
Inform Clinical
Management
Critical Type IV, Subtype 9 Type III, Subtype 7 Type II, Subtype 4
Serious Type III, Subtype 8 Type II, Subtype 6 Type I, Subtype 2
Non-Serious Type II, Subtype 5 Type I, Subtype 3 Type I, Subtype 1
SaMD Proposed Review
Pathway Framework
IMDRF Risk Categorization Level of Review for Level 1 and Level 2 Pre-certified
Organizations’ SaMD
Type Subtype Description Initial Product Major Changes Minor Changes
IV 9 Critical x diagnose/treat SR SR No Review
III 8 Critical x drive SR L1 - SR
L2 - No Review
No Review
III 7 Serious x diagnose/treat SR L1- SR
L2 - No Review
No Review
II 6 Serious x drive L1-SR
L2 - No Review
L1 - SR
L2 - No Review
No Review
II 5 Non-serious x diagnose/treat L1 - SR
L2 - No Review
No Review No Review
II 4 Critical x inform L1 - SR
L2 - No Review
No Review No Review
I 3 Non-serious x drive No Review No Review No Review
I 2 Serious x inform No Review No Review No Review
I 1 Non-serious x inform No Review No Review No Review
SR = Streamlined Review; No Review = No Premarket Submission Required
Scope of the SaMD Review
● What information will be reviewed and how
○ Screen sharing of wireframes
○ Access to development environment
○ Testing logs, audit of test results
● How modifications affect marketing authorization
● How to leverage existing SaMD community standards
Approval of SaMD Submission
● FDA will document their decision in a written
summary and provide it to the organization
● FDA is considering a phased market authorization
where some elements are reviewed pre-market and
others are gathered through RWE to support full
market authorization
Rejection of SaMD Submission
● FDA will determine gaps in the evidence and
determine a plan for future submission
● If there are repeated unsuccessful reviews of a
pre-certified organization’s SaMD it will trigger
reassessment of the organization’s pre-certification
to address systemic issues
Tiers of
Cybersecurity Risk
NOTE: FDA relies on device
manufacturers to determine
the level of cybersecurity risk
associated with their product
Tier 2
Standard risk where criteria
for Tier 2 are not met.
FDA does not mandate
completion of premarket
security audits
Tier 1
Capable of connecting,
either wired or wirelessly, to
another medical or
nonmedical product, a
network, or the internet
A breach could result in
harm to patients
Mitigating Cybersecurity Risk
● Implement design controls
● Independent cybersecurity risk assessments annually
which consider how new risks are mitigated and design
decisions are made
Bridging the Information Gap
● Regulators are considering a requirement to have a
public cybersecurity “bill of materials” in pre-market
applications
● Data rights and governance will be outlined by the
manufacturers in an End User License Agreement,
Terms of Service, and Privacy Policies
Post Market SaMD Surveillance
● Use real world data from several sources:
○ SaMD product itself
○ Device registries
○ Electronic health information sources
■ National Evaluation System for health
Technology (NEST)
Real World Performance Data (RWPD)
Real World
Health Data
(RWHD)
User
Experience
Data (UXD)
Product
Performance
Data (PPD)
Real World Health Data (RWHD)
Outputs and outcomes related to the SaMD
definition statement. RWHD can inform changes
to the intended use of a SaMD product, support
expanded functionalities and use in broader
target populations, and monitor the continued
safety and effectiveness of a marketed SaMD
product.
User Experience Data (UXD)
Outputs derived from user experiences related to
the real-world use of a SaMD product. UXD
facilitate timely identification and correction of
user issues, and improve utilization and
effectiveness of the software.
Product Performance Data (PPD)
Outputs and outcomes demonstrating the
accuracy, reliability, and security of a SaMD
product. PPD monitoring allows for timely
patches and updates to correct software bugs
and security vulnerabilities.
Performance Data Key Objectives
1. Monitoring ongoing safety and effectiveness
2. Supporting modifications of claims
3. Providing input to changes in pre-certification
status
4. Providing feedback for FDA to further refine the
program
Regulatory Oversight and Policing
● Who is responsible for looking after unauthorized
data distribution, etc. can be ambiguous and 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
FDA Pre-Cert
Program
FDA Pre-Certification Program
● Pilot program which assesses companies rather
than products
● Company must demonstrate excellence in software
development and validation
○ SaMD developers would be assessed by FDA or
an accredited 3rd party for the quality of their
software design, testing, clinical practices, and
real world performance monitoring
Selected FDA Pre-Cert Companies
● Pear Therapeutics
● Verily
● Phosphorus
● Johnson & Johnson
● Samsung
● Tidepool
● Roche
● Apple
● FitBit
What this Means
Companies must have robust mechanisms to
collect, monitor, and analyze real world performance
of their organization in addition to their products
Benefits of Pre-Cert
● Pre-Certified companies could submit less
information to FDA than is currently required
○ Possibly not even need to submit a pre-market
submission at all
● Risk-based, streamlined approach maximizes
efficiency and engagement
Anticipated
Program Benefits
End User Business FDA Payor Investor
Patients,
Providers,
Caregivers
SaMD
Developer
Agency
Reviewer
Insurance
Provider
Venture
Capitalist
Enhanced trust in organizations
developing SaMD products
X X X X
Improved quality, safety,
proactiveness to address known and
emerging risks
X X X X
Timely availability of solutions to
patients
X X X X X
Enhanced regulatory simplicity and
experience
X X X X
Business simplicity -
faster market access
X X X X
Overview of Proposed Framework
1. Excellence appraisal and certification to
determine pre-cert levels 1 or 2
2. Review determination based on risk and
pre-cert level
3. Streamlined review
4. Monitor real world performance data
Excellence Appraisal & Certification
● Eligibility and application
● Appraisal
○ Evaluation against pre-cert criteria, including
excellence principles
● Status determination
● Maintenance
Excellence Principles
● Product Quality
● Patient Safety
● Clinical Responsibility
● Cybersecurity
Responsibility
● Proactive Culture
Culture of Quality and
Organizational Excellence
(CQOE) Principles
Pre-Cert Levels
LEVEL 1
LEVEL 2
Objectively demonstrates
excellence in 5 principles without
a track record of delivering SaMD
Has objectively demonstrated
excellence in all 5 principles
with a demonstrated track
record of delivering SaMD
Paradigm Shift
Hardware Software
Deterministic Risks of
Physical Products
Evolving Risks of Non-
Physical Products
Stable and Predictable
Volume
Potential for
Exponential Volume
Development Process Overview
● Similar to that of molecular development, but:
○ Shorter development lifecycle
○ Flexible development
■ Able to update and enhance the product throughout the
development process
● Speed of recruitment and access to patients can be quicker,
using the DCT model
○ Able to recruit direct-to-patient without the need for brick
and mortar sites
● Must demonstrate continued safety, effectiveness, and
performance in the real world
Key Differences
● The process is not as linear and “one and done”
● It is an iterative and agile process
● Enhancements follow feedback from patients as
users and bug detection
● Updates and code revisions are ongoing
● Distributed responsibilities
● Concern over cybersecurity
● As PDTs are SaMD, only pivotal PhIII data is
considered for approval
Development Standards
● FDA approval allows company to make a claim
regarding a specific condition
● Regulatory pathway to approval is still in development
at FDA and open for public comment
● The precise path a DTx must take and the level of
clinical evidence it must provide is dependent on the
novelty of the product and how great a risk it poses
should it malfunction
Risks/Benefits of FDA Approval/SaMD
Benefits
● May allow a higher price point
● Increased scientific and clinical credibility with HCPs
● Higher barrier of entry to competitors
Risks
● Slower, more costly development process
● Less flexibility to improve product
● Defers commercial risk and ROI, increasing risk profile
Regulatory Pathways
● De Novo
○ Granted to novel devices of low to moderate risk
○ Clinical data (if applicable) to support reasonable
assurance of safety and effectiveness required
● 510K Premarket Submission
○ Demonstration of substantial equivalence to
another legally US marketed device.
Regulatory Pathways
● Breakthrough
○ Devices having cleared 510(k) premarket
submission that also
■ Provide more effective treatment or diagnosis of
life-threatening or irreversibly debilitating disease
or conditions
■ Are proven to be unique or offer distinct
advantage over existing devices
■ In some cases, makes reimbursement easier
Regulatory Pathway Use Cases
510(k) De Novo Breakthrough
Renovia
Somryst
Pear Therapeutics
Mahana Therapeutics
Akili Interactive
Pear Therapeutics
MedRhythms
Cognoa
AppliedVR
Cognito
Key Concepts
● Mechanism of Action (MOA)
○ How the product works
○ What you link to outcomes of interest
● Target Product Profile (TPP)
○ Target patient populations
○ Product concept and prototypes
■ Test with HCPs, patients, caregivers
● Understand usability and engagement
○ Hypotheses about users, endpoints, and impact
RWD STUDIES
Post-Market Health
Economics and Outcomes
Research (HEOR) studies to
assess ability to lower costs
and improve outcomes.
At least 1 year in duration
$1MM
DISCOVERY
Target Product Profile (TPP)
defines the desired
characteristics, intended use,
and target population(s).
Feasibility, usability.
6 months to 1 year in duration
$1-5MM
PIVOTAL STUDY
Patients of a wider
demographic to show
efficacy. Almost always
an RTC with control arm.
1-2 years in duration
$7-20MM
200-500 patients
POC & PILOTS
Proof of concept and
pilot studies to show
mechanism of action
and safety. Explores
patient engagement.
6 mo - 1 yr in duration
$1-5MM
150-300 patients
Development Lifecycle
Studies often involve digital biomarkers as endpoints to determine therapeutic effects,
but compare them to gold standard endpoints accepted by providers and payers
APPROVAL
PDT Pilot Studies
● Explore a range of “dosing” regimens
● Gather data on optimizing usability, engagement,
and efficacy
● Improve the user experience while not affecting the
mechanism of action
PDT Validation Studies (RCTs)
● The average study length of an RCT for PDT has
been 6 months
● Average size is 600 participants
● Endpoints are largely digital versions of traditional
clinical measures, rather than digital endpoints
themselves
Control Arm Challenges
● Blinding in a digital intervention can be problematic
● Use of a placebo control group may be unethical or
not feasible (unable to mask) in some cases
● Sham app mimics software without including the
“active ingredient”
○ Need to ensure sham does not create a clinical
response through placebo effect
Clin Dev
2-5 years
As opposed to the 10-15 years clinical development takes for
molecular drugs
Unique Development Challenges
● Informed consent process raises privacy concerns
○ Hitting the “okay” button ≠ sufficient consent
● Inter-individual variability in response
● Poorly defined exposure (using time, frequency,
duration) makes response relationship and therapeutic
target (optimum exposure) difficult to measure
● Mechanism of Action (MOA) can be unclear
Developing Treatment Regimens
Methodology is empirical, based upon observation
and experience, rather than dose-response theory
or logic. Treatment regimes are optimized by the
physician.
Note that you can have a great mechanism of
action, but poor patient engagement.
Treatment Paradigms
1. Diagnostic tool
2. Monotherapy - First line therapy
3. Concert therapy - Companion, adjunct, or
combination therapy with traditional
pharmacological interventions
* Some products can be used as both monotherapies and concert
therapies, depending on design and evidence
Combination Products
Companion - Usually not FDA approved; can be used to
treat a condition alongside pharmacological product
and/or HCP treatment
Combination - Application or sensor that is designed to be
used in conjunction with a specific pharmacological
product
Adjunct - Application or sensor that is designed to be used
with a category of pharmacological interventions or
treatments
Post Approval Obligations
● Failure to comply with post-clearance or
approval regulatory requirements could subject
the company to enforcement actions
○ Substantial penalties
○ Recall
○ Withdrawal of product from the market
Adherence
Plays upon the sentinel effect
● the tendency for human performance to
improve when the performance is being
evaluated by a third party (their physician)
Trends
● Largely based on Cognitive Behavioral Therapy (CBT)
○ Focus on changing patient’s behavior or thinking
● Focus is thus on outcomes allowing value-based
payment models
● Easier to collect real world data
● Keen interest in patient experience, product support
and education, adherence/compliance, data sharing
Pharma
Partnerships
Common Therapeutic Areas
Mental Health
● Depression, anxiety
● Body dysmorphic
disorder (BDD)
● Schizophrenia
● Post traumatic Stress
Disorder (PTSD)
Behavior-Modifying
Conditions
● Autism
● Attention deficit
● Substance Use Disorder
(SUD)
● T2DM, heart failure
Commercialization Preface
● Pathways to scale and commercialize are still largely
unchartered
● The market is rapidly evolving, increasingly
competitive
○ Therefore, difficult to forecast demand
Partnership Considerations
● Upfront payments
● Royalty and milestone payments
● Research and development funding
● Profit sharing
● IP rights
Pharma Partnership Examples
● Akilli and Shionogi
○ Shionogi oversees clinical development, sales and
marketing in select markets
○ Shionogi made an upfront payment to Akili of
$20M
○ Akili will be eligible to receive development and
commercial milestone payments of up to $105M
and royalties from sales in the selected markets
Pharma Partnership Examples
● Click and Otsuka
○ Otsuka is fully funding the development of the mobile
application to commercialize world wide
○ Otsuka will pay Click up to $10M in upfront and
regulatory milestone payments along with the $20M in
development funding
○ Click will receive an additional $272M in commercial
milestone payments contingent upon regulatory
approvals
○ Click will receive tiered royalties on global sales
○ Click can use Otsuka’s patient engagement platform
Business Models
Pricing Questions / Considerations
● How well the existing therapies treat the condition?
○ Perceived differentiation to reference products
● How much is the market willing to pay?
○ Type of reimbursement available
○ Target population’s income level
○ Target population’s social determinants of health
● Pricing can be dependent on the type of customer
● Level of invasiveness/burden
● Safety and efficacy profile
Pricing Questions / Considerations
● Innovativeness/novelty of the product
○ Create a pricing matrix which uses a sensitivity
analysis of market acceptance of various
hypothetical prices
■ “First-in-class” (novel product with new
indication) can be priced higher
Pricing Models
● Risk-Based Model:
○ Value can be viewed from the basis of a percent of
savings
● Value-Based Model:
○ Real world evidence is used to track value
proposition
○ Incentivizes positive results
Value-Based Pricing Calculation
V = R ± D
V = Perceived value of therapy being introduced
R = Price of the reference therapeutic, generally the best
existing alternative
D = Perceived differentiation (both positive and
negative) of the new product to relative to reference
therapy
Pricing Practices
Skimming
● Set entry price at maximum level payers are
willing to afford
● Fewer sales at higher price
● Gradually decrease the price to capture the
growing part of the market
Pricing Practices
Penetration
● Set prices below comparable products to
gain a large market share
● Results in greater volume at a lower price
● Raise prices over time
Rx Only Reimbursement Models
● Physician completes and sends patient enrollment
to the DTx manufacturer who then onboards the
patient directly via a mobile app store access code
● Pts can only interact with the DTx per the label with
the physician monitoring compliance, inputs, and
progress via a dashboard
Reimbursement Landscape
● March 2020 Senate bill called for Medicare
reimbursement of PDT
○ Called for the establishment of a fee schedule to
reimburse with 180 days of FDA clearance or
approval
● Breakthrough designation for FDA cleared medical
devices must fit into a pre-existing medicare benefit
category
Reimbursement Landscape
● No major commercial payers in the US have announced
plans to cover PDTs
● PDT reimbursement has largely been from small PBMs and
other small payers
● A set of 5 CPT codes were adopted in October 2020 for
“remote therapeutic monitoring treatment management
services” which could allow a fee-for-service
reimbursement in the future
● Employer coverage has seen the most success
Go To Market Strategies - Rx Only
Rx-Only / Reimbursement:
○ Pricing based on perceived value
■ Demonstrating superiority = ability to set
premium pricing
■ Need to maintain data of many types
which show value proof points
Go To Market Strategies - Rx Only
Rx-Only / Reimbursement:
○ Demonstrate value through data from:
■ Randomized clinical trials
■ Regulatory approval for indications
■ Demonstrating market need through
market analyses and rates of use
■ Demonstrate successful health outcomes
Go To Market Strategies - Rx Only
Rx-Only / Reimbursement:
○ Pursue value-based contracting with public
payers or private insurance
■ Utilize public programs like Medicaid or
Medicare
○ Can utilize rebate agreements to make pricing
more attractive
Go To Market Strategies - OTC
“Over the counter”:
○ Direct to patient or employer
■ May require more marketing
■ Start with a free product or paying
people to join/use the product
● Move from freemium to premium
when a critical mass is achieved
Go To Market Strategies - OTC
“Over the counter”:
○ Employers can negotiate PMPM fees on
■ total employee basis
■ per engaged user basis
Pricing and Reimbursement Insights
● As DTx come in a variety of forms, it is unlikely
that there will be a formulaic approach to
securing reimbursement
Pricing Strategies
Moving from a DTC offering to a medical benefit, to
a pharmacy benefit can 10x the price
$20/month
$200/month $2,000/month
DTC Medical
Benefit
Pharmacy
Benefit
PDT Pricing in the Market
Typically a 3 month or 90 day period
Customer Economic Model Contract Type
Providers Risk-sharing PMPM
Payors % Savings Recurring
Employers Risk-sharing PMPM
Pharma Licensing, royalty,
milestone
Recurring or term
Patients Free with
reimbursement
Over-the-counter
Example
Payment
Model
by
Customer
Reimbursement Challenges
● Centers for Medicaid and Medicaid Services (CMS)
has not yet issued clear reimbursement guidance
● Need to transition away from National Drug Code
(NDC) and National Health Related Items Code
(NHRIC) numbers towards Unique Device
Identification (UDI) numbers, but there is hesitancy
from pharmacies and payers
● Need to develop Healthcare Common Procedural
Coding System Codes
IP Protection
● Unlike traditional pharmaceuticals, software
cannot be protected through a composition of
matter patient
● Need both patents and granted claims
○ Claims expire in various years
● Create interrelated patents to strengthen
protection
Types of Patents
1. Software interface between the various
computer hardware
2. Peripheral devices connecting the patient,
physician, and caregivers
3. Interface of the hardware to the individual
4. Any combination of the above
Software Copyright
● Used to discourage or prevent unauthorized copies
of the proprietary software
● Prohibits “nonliteral elements” which includes the
structure and sequence of the software code
● Can only be granted after registration once the
software is published
● Can be quicker to get as patents require an
examination procedure by a government agency
Governing Laws and Regulations
● Data Privacy and Security - the collection, use,
disclosure, and storage of personally identifiable
information (PII), including protected health
information and payment card data
● Federal and state healthcare fraud and abuse
laws, false claims laws
Launch Strategy
● Marketing must also drive awareness
● Utilize medical science liaisons, key opinion
leaders, and educators
● Industry events which speak to the data and
answer questions
● Publications to peer reviewed journals
Next Frontier
● Expanding into acute conditions
● Need to develop better utilization metrics
● Need to prove patient engagement with DTx long
term
● Facilitating payer reimbursement in the absence of
dedicated NDC codes
● Nail down components of annual reporting required
FDA Approved DTx
[Use Cases]
Pear Therapeutics - reSET
● 12 week DTx for individuals with Substance Use
Disorder
● Uses CBT as an adjunct therapy to standard
outpatient treatment
● 1 Study
○ Comprehensive CBT (Cognitive Behavioral Therapy) Via reSET App
Pear Therapeutics - reSET-O
● 84 day PDT for Opioid Use Disorder
● Approved as prescription only PDTx
● Based on CBT
● Intended as an adjunct to outpatient treatment
● Submitted randomized controlled trial through FDA’s de
novo approval pathway
● 3 Studies
○ reSET-O RCT
○ A Remote, Decentralized Opioid Use Disorder Study to Evaluate Patient Engagement With a
Game-Based Digital Therapeutic
○ Pilot Study of reSET-O to Treatment-as-usual in Acute Care Settings
Akili Interactive -
● EndeavorRX
○ Attention Deficit Hyperactivity Disorder
■ 5 Studies
● Cognitive ADHD Videogame Exploratory Study (CAVES)
● Electroencephalogram (EEG) Study of Inattention Following Treatment With AKL-T01
● Software Treatment for Actively Reducing Severity of ADHD as Adjunctive Treatment
to Stimulant
● Software Treatment for Actively Reducing Severity of ADHD (STARS-ADHD)
● Software Treatment for Actively Reducing Severity of ADHD - Follow Up
(STARS-ADHD2)
WellDoc
● BlueStar
○ Type II DM management tool
○ Similar to existing therapies, so demonstrating
“substantial equivalence” rather than new clinical
evidence
○ 1 Study
■ Evaluate the Value of Telehomecare for Diabetes
Mahana Tx
● Parallel
○ Program for Irritable Bowel Syndrome (IBS)
■ “FDA authorized”
■ 1 Study
● The ACTIB trial
○ RCT of 558 patients
○ Not found on clinicaltrials.gov
Nightware
● Nightware
○ Nightmares related to different conditions
■ 4 Studies
● Traumatic Nightmares Treated by NightWare
● NightWare Open Enrollment Study
● Traumatic Nightmares Treated by NightWare
● Remote Study of NightWare for PTSD With Nightmares
Amalgam
● iSage Rx
○ Diabetes
■ 2 Studies
● A Digital Health Tool for Insulin Titration (DHIT) Individuals With Type 2
Diabetes: A Prospective Outcomes Study With a Retrospective Control Group
(DHIT)
● mHealth Titration and Management
Propeller -
● Platform for COPD and asthma connected with
sensors on inhalers
● Combination product
● Track medication usage and provides feedback
and insights that enable integration of care
● 13 Studies
Other DTx Companies -
● Click Therapeutics
● Cognoa
● Voluntis
● Curio
● Chrono Therapeutics
(shut down 2019)
● Sidekick Health
● Prosper
● Better Therapeutics
● Bold Health
● Ampersand
● Happify Health
● Hello Better
● Oviva
FDA Approved
SaMD
[Use Cases]
Natural Cycles -
● Natural Cycles
○ Birth control app that helps women track their fertility to
prevent unwanted pregnancies via the rhythm method
○ Analyzes data from past menstrual cycles and body
temperature to determine when the woman is most
fertile
○ When the woman is determined to be ovulating she is
warned to use protection
○ FDA Cleared, CE Marked, Class IIb SaMD
○ 1 study of 15,000 women required
Key Milestones
2010
FDA Approval of DTx
[WellDoc’s BlueStar]
2017
FDA Launches
Pre-Cert Program
Pear’s reSET is 1st
software-only DTx
2018
2019
Pear submitted the
1st application for
FDA marketing
authorization using
the pre-cert program
74 DTx
announced
in 2020
Benefits
● FDA approval does not
guarantee payor uptake
● 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 good decreases
with volume
● Requires less human
capital
$45-55 MM
Cost to bring a concept through FDA submission is significantly lower
than for molecular drug development ($350 MM)
Vision
● DTx add functionalities into a more comprehensive
portfolio, synchronizing products and services
● Can be paired with diagnostics, sensors, wearables,
and even molecular therapies as combination
therapy, expanding indication
● Integrated into electronic prescribing, dispensing,
and medical record platforms
● Price of DTx will be significantly less than
conventional therapies
PwC Health Research Institute’s Annual Report
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%
The Road Ahead
● Patient, provider, and payer appetite for DTx is still
uncertain
● Need payer reimbursement models
● Increase stakeholder buy-in
● Integrate into provider workflows
● Need to develop more comprehensive DTx
formularies
● Regulatory evaluation and commercialization pathways
for other health authorities like EMA, TGA
PBM Digital Health Formularies
● 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
● 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

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DTx Development

  • 1. Digital Therapeutics Development and Regulatory Requirements Emily Kunka, MS Digital Business Transformation
  • 2. Nomenclature Digital Health Data and information capture, storage, and display. Examples include software for care administration and management (i.e. telehealth). Digital Medicine Measurement products. Examples include digital biomarkers, digital diagnostics, remote patient monitoring, eCOA, clinical decision support software (may or may not require human intervention). Digital Therapeutics Deliver intervention to prevent, improve, manage, or treat disease (including adjunct treatments).
  • 3. Solution Therapy 2 Supported by clinical trial data 3 Require FDA approval or clearance Digital Solutions vs. Digital Therapies 4 Generally reimbursable by payers Low High(er) than solution 5 Cost to Develop 1 Generally requires a prescription to use
  • 4. Examples of Each Digital Solutions Digital Therapies Adherence programs Patient journey / experience Software-enabled hardware Algorithms intended to diagnose or predict Software intended to treat a condition or modify a behavior Direct refill services, fitness trackers Companion labeling app for a marketed drug Course or program Video game
  • 5. Digital Therapeutics (DTx) deliver medical interventions directly to patients using evidence-based, clinically evaluated software to treat, manage, and prevent a broad spectrum of diseases and disorders.
  • 6. Software as a Medical Device (SaMD) is software intended to be used for one or more medical purposes that perform these purposes without being part of a hardware medical device
  • 7. Broad DTx Buckets Software for disease management 03 ● Enables informed decision making about care to control disease ● Typical in chronic disease states Software as adjunct to traditional tx 02 ● Enhances standard of care ● Used in conjunction to transform or work with existing treatment Software as a direct treatment 01 ● Could replace standard of care ● Could be used independent of other therapy options
  • 8. Treat A Disease Manage a Disease Improve Health Function (includes prevention) Medical Claims Medium to high risk claims Medium to high risk claims Low to medium risk claims Clinical Endpoints Must use clinical endpoints to support product claims Must use clinical endpoints to support product claims Must use clinical endpoints to support product claims Clinical Evidence Clinical trials and ongoing evidence generation required Clinical trials and ongoing evidence generation required Clinical trials and ongoing evidence required Regulatory Oversight 3rd party validation of efficacy and safety claims by regulatory or equivalent national body 3rd party validation of efficacy and safety claims by regulatory or equivalent national body Degree of regulatory oversight depends on local regulatory body frameworks Patient Access Prescription required Prescription required OR Non-prescription product Prescription required OR Non-prescription product
  • 9. Digital Health Innovation Action Plan ● Issuing guidance to provide clarity on the medical software provisions of the 21st Century Cures legislation ● Launching pilot pre-certification program to develop a new approach to digital health technology oversight ● Building FDA’s bench strength and expertise in CDRH’s digital health unit
  • 10. Regulatory Landscape ● Center for Devices and Radiological Health (CDRH) ● Guidance on Mobile Medical Application (MMA) ● Formal SaMD pathway in development ● Prove superiority via de novo or substantial equivalence via 510K pathways for medical devices ● Can receive breakthrough designation for expedited review ● Approval granted or “FDA cleared”
  • 11. Regulatory Approval ● FDA approval brings product legitimacy/acceptance and ability to be reimbursable from payors ● FDA approval brings price point higher ● FDA designates certain DTx as prescription only or direct to consumer ● FDA can exercise enforcement discretion waiving regulatory oversight if it decides a product is low risk ● May require a change control plan
  • 12. Regulatory Caveats ● DTx that aim to prevent disease by helping patients change their lifestyle can be marketed in the US without providing safety and efficacy evidence to the FDA ● Current approval process is not set up to handle DTx that are solely a delivery mechanism for already-proven therapies not delivered via technology
  • 13. Freedom of Information Act ● Allows anyone to see the correspondence DTx companies have had with the FDA to classify their devices and obtain clearance https://www.accessdata.fda.gov/cdrh_docs/pdf20/DEN200029.pdf
  • 14. DTx Classification Components Product’s purpose or indication for use Target product profile for use and function
  • 15. FDA’s Use and Function Classification Categories Address a medical condition CATEGORY 1 Manage or prevent disease or disorder CATEGORY 2 Optimize medication usage CATEGORY 3 Treat medical disease or disorder CATEGORY 4
  • 16. Categorization Examples Company Product Description TA Category Pear reSET reSET-O Adjunct Tx for Substance Use Disorder (SUD) CNS 4 Propeller RESPIMAT Optimize healthcare utilization in asthma and COPD Respiratory 3 Akili AKL-T01 Adaptive sensory stimulus software (video game) for ADHD CNS 4 Dthera DTHR-ALZ Reminiscence therapy for Alzheimer’s CNS 4 Big Health Sleepio Sleep improvement program with CBT CNS 2 Cognoa Various AI based digital diagnostics and personalized therapeutics CNS, Peds 2 KAIA Motion coach Behavioral therapy (physical exercises and education) for chronic back pain Musculoskel. 2
  • 17. Product’s Intended Purpose ● To support a certain type of product claim ● To generate clinical evidence ● To give patients access to product ● To redefine existing relationship to concurrent therapies → written in a definition statement, determines FDA submission requirements
  • 18. SaMD Definition Statement Should include: ● A description of the SaMD’s core functionality ● The state of the healthcare situation or condition the SaMD is intended for (critical, serious, non-serious) ● Significance of information provided by the SaMD to the healthcare decision maker (diagnose/treat, drive clinical management, or inform clinical management)
  • 19. SaMD Proposed Review Pathway Framework State of Healthcare situation or condition Significance of information provided by SaMD to healthcare decision Treat or Diagnose Drive Clinical Management Inform Clinical Management Critical Type IV, Subtype 9 Type III, Subtype 7 Type II, Subtype 4 Serious Type III, Subtype 8 Type II, Subtype 6 Type I, Subtype 2 Non-Serious Type II, Subtype 5 Type I, Subtype 3 Type I, Subtype 1
  • 20. SaMD Proposed Review Pathway Framework IMDRF Risk Categorization Level of Review for Level 1 and Level 2 Pre-certified Organizations’ SaMD Type Subtype Description Initial Product Major Changes Minor Changes IV 9 Critical x diagnose/treat SR SR No Review III 8 Critical x drive SR L1 - SR L2 - No Review No Review III 7 Serious x diagnose/treat SR L1- SR L2 - No Review No Review II 6 Serious x drive L1-SR L2 - No Review L1 - SR L2 - No Review No Review II 5 Non-serious x diagnose/treat L1 - SR L2 - No Review No Review No Review II 4 Critical x inform L1 - SR L2 - No Review No Review No Review I 3 Non-serious x drive No Review No Review No Review I 2 Serious x inform No Review No Review No Review I 1 Non-serious x inform No Review No Review No Review SR = Streamlined Review; No Review = No Premarket Submission Required
  • 21. Scope of the SaMD Review ● What information will be reviewed and how ○ Screen sharing of wireframes ○ Access to development environment ○ Testing logs, audit of test results ● How modifications affect marketing authorization ● How to leverage existing SaMD community standards
  • 22. Approval of SaMD Submission ● FDA will document their decision in a written summary and provide it to the organization ● FDA is considering a phased market authorization where some elements are reviewed pre-market and others are gathered through RWE to support full market authorization
  • 23. Rejection of SaMD Submission ● FDA will determine gaps in the evidence and determine a plan for future submission ● If there are repeated unsuccessful reviews of a pre-certified organization’s SaMD it will trigger reassessment of the organization’s pre-certification to address systemic issues
  • 24. Tiers of Cybersecurity Risk NOTE: FDA relies on device manufacturers to determine the level of cybersecurity risk associated with their product Tier 2 Standard risk where criteria for Tier 2 are not met. FDA does not mandate completion of premarket security audits Tier 1 Capable of connecting, either wired or wirelessly, to another medical or nonmedical product, a network, or the internet A breach could result in harm to patients
  • 25. Mitigating Cybersecurity Risk ● Implement design controls ● Independent cybersecurity risk assessments annually which consider how new risks are mitigated and design decisions are made
  • 26. Bridging the Information Gap ● Regulators are considering a requirement to have a public cybersecurity “bill of materials” in pre-market applications ● Data rights and governance will be outlined by the manufacturers in an End User License Agreement, Terms of Service, and Privacy Policies
  • 27. Post Market SaMD Surveillance ● Use real world data from several sources: ○ SaMD product itself ○ Device registries ○ Electronic health information sources ■ National Evaluation System for health Technology (NEST)
  • 28. Real World Performance Data (RWPD) Real World Health Data (RWHD) User Experience Data (UXD) Product Performance Data (PPD)
  • 29. Real World Health Data (RWHD) Outputs and outcomes related to the SaMD definition statement. RWHD can inform changes to the intended use of a SaMD product, support expanded functionalities and use in broader target populations, and monitor the continued safety and effectiveness of a marketed SaMD product.
  • 30. User Experience Data (UXD) Outputs derived from user experiences related to the real-world use of a SaMD product. UXD facilitate timely identification and correction of user issues, and improve utilization and effectiveness of the software.
  • 31. Product Performance Data (PPD) Outputs and outcomes demonstrating the accuracy, reliability, and security of a SaMD product. PPD monitoring allows for timely patches and updates to correct software bugs and security vulnerabilities.
  • 32. Performance Data Key Objectives 1. Monitoring ongoing safety and effectiveness 2. Supporting modifications of claims 3. Providing input to changes in pre-certification status 4. Providing feedback for FDA to further refine the program
  • 33. Regulatory Oversight and Policing ● Who is responsible for looking after unauthorized data distribution, etc. can be ambiguous and 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
  • 35. FDA Pre-Certification Program ● Pilot program which assesses companies rather than products ● Company must demonstrate excellence in software development and validation ○ SaMD developers would be assessed by FDA or an accredited 3rd party for the quality of their software design, testing, clinical practices, and real world performance monitoring
  • 36. Selected FDA Pre-Cert Companies ● Pear Therapeutics ● Verily ● Phosphorus ● Johnson & Johnson ● Samsung ● Tidepool ● Roche ● Apple ● FitBit
  • 37. What this Means Companies must have robust mechanisms to collect, monitor, and analyze real world performance of their organization in addition to their products
  • 38. Benefits of Pre-Cert ● Pre-Certified companies could submit less information to FDA than is currently required ○ Possibly not even need to submit a pre-market submission at all ● Risk-based, streamlined approach maximizes efficiency and engagement
  • 39. Anticipated Program Benefits End User Business FDA Payor Investor Patients, Providers, Caregivers SaMD Developer Agency Reviewer Insurance Provider Venture Capitalist Enhanced trust in organizations developing SaMD products X X X X Improved quality, safety, proactiveness to address known and emerging risks X X X X Timely availability of solutions to patients X X X X X Enhanced regulatory simplicity and experience X X X X Business simplicity - faster market access X X X X
  • 40. Overview of Proposed Framework 1. Excellence appraisal and certification to determine pre-cert levels 1 or 2 2. Review determination based on risk and pre-cert level 3. Streamlined review 4. Monitor real world performance data
  • 41. Excellence Appraisal & Certification ● Eligibility and application ● Appraisal ○ Evaluation against pre-cert criteria, including excellence principles ● Status determination ● Maintenance
  • 42. Excellence Principles ● Product Quality ● Patient Safety ● Clinical Responsibility ● Cybersecurity Responsibility ● Proactive Culture Culture of Quality and Organizational Excellence (CQOE) Principles
  • 43. Pre-Cert Levels LEVEL 1 LEVEL 2 Objectively demonstrates excellence in 5 principles without a track record of delivering SaMD Has objectively demonstrated excellence in all 5 principles with a demonstrated track record of delivering SaMD
  • 44. Paradigm Shift Hardware Software Deterministic Risks of Physical Products Evolving Risks of Non- Physical Products Stable and Predictable Volume Potential for Exponential Volume
  • 45. Development Process Overview ● Similar to that of molecular development, but: ○ Shorter development lifecycle ○ Flexible development ■ Able to update and enhance the product throughout the development process ● Speed of recruitment and access to patients can be quicker, using the DCT model ○ Able to recruit direct-to-patient without the need for brick and mortar sites ● Must demonstrate continued safety, effectiveness, and performance in the real world
  • 46. Key Differences ● The process is not as linear and “one and done” ● It is an iterative and agile process ● Enhancements follow feedback from patients as users and bug detection ● Updates and code revisions are ongoing ● Distributed responsibilities ● Concern over cybersecurity ● As PDTs are SaMD, only pivotal PhIII data is considered for approval
  • 47. Development Standards ● FDA approval allows company to make a claim regarding a specific condition ● Regulatory pathway to approval is still in development at FDA and open for public comment ● The precise path a DTx must take and the level of clinical evidence it must provide is dependent on the novelty of the product and how great a risk it poses should it malfunction
  • 48. Risks/Benefits of FDA Approval/SaMD Benefits ● May allow a higher price point ● Increased scientific and clinical credibility with HCPs ● Higher barrier of entry to competitors Risks ● Slower, more costly development process ● Less flexibility to improve product ● Defers commercial risk and ROI, increasing risk profile
  • 49. Regulatory Pathways ● De Novo ○ Granted to novel devices of low to moderate risk ○ Clinical data (if applicable) to support reasonable assurance of safety and effectiveness required ● 510K Premarket Submission ○ Demonstration of substantial equivalence to another legally US marketed device.
  • 50. Regulatory Pathways ● Breakthrough ○ Devices having cleared 510(k) premarket submission that also ■ Provide more effective treatment or diagnosis of life-threatening or irreversibly debilitating disease or conditions ■ Are proven to be unique or offer distinct advantage over existing devices ■ In some cases, makes reimbursement easier
  • 51. Regulatory Pathway Use Cases 510(k) De Novo Breakthrough Renovia Somryst Pear Therapeutics Mahana Therapeutics Akili Interactive Pear Therapeutics MedRhythms Cognoa AppliedVR Cognito
  • 52. Key Concepts ● Mechanism of Action (MOA) ○ How the product works ○ What you link to outcomes of interest ● Target Product Profile (TPP) ○ Target patient populations ○ Product concept and prototypes ■ Test with HCPs, patients, caregivers ● Understand usability and engagement ○ Hypotheses about users, endpoints, and impact
  • 53. RWD STUDIES Post-Market Health Economics and Outcomes Research (HEOR) studies to assess ability to lower costs and improve outcomes. At least 1 year in duration $1MM DISCOVERY Target Product Profile (TPP) defines the desired characteristics, intended use, and target population(s). Feasibility, usability. 6 months to 1 year in duration $1-5MM PIVOTAL STUDY Patients of a wider demographic to show efficacy. Almost always an RTC with control arm. 1-2 years in duration $7-20MM 200-500 patients POC & PILOTS Proof of concept and pilot studies to show mechanism of action and safety. Explores patient engagement. 6 mo - 1 yr in duration $1-5MM 150-300 patients Development Lifecycle Studies often involve digital biomarkers as endpoints to determine therapeutic effects, but compare them to gold standard endpoints accepted by providers and payers APPROVAL
  • 54. PDT Pilot Studies ● Explore a range of “dosing” regimens ● Gather data on optimizing usability, engagement, and efficacy ● Improve the user experience while not affecting the mechanism of action
  • 55. PDT Validation Studies (RCTs) ● The average study length of an RCT for PDT has been 6 months ● Average size is 600 participants ● Endpoints are largely digital versions of traditional clinical measures, rather than digital endpoints themselves
  • 56. Control Arm Challenges ● Blinding in a digital intervention can be problematic ● Use of a placebo control group may be unethical or not feasible (unable to mask) in some cases ● Sham app mimics software without including the “active ingredient” ○ Need to ensure sham does not create a clinical response through placebo effect
  • 57. Clin Dev 2-5 years As opposed to the 10-15 years clinical development takes for molecular drugs
  • 58. Unique Development Challenges ● Informed consent process raises privacy concerns ○ Hitting the “okay” button ≠ sufficient consent ● Inter-individual variability in response ● Poorly defined exposure (using time, frequency, duration) makes response relationship and therapeutic target (optimum exposure) difficult to measure ● Mechanism of Action (MOA) can be unclear
  • 59. Developing Treatment Regimens Methodology is empirical, based upon observation and experience, rather than dose-response theory or logic. Treatment regimes are optimized by the physician. Note that you can have a great mechanism of action, but poor patient engagement.
  • 60. Treatment Paradigms 1. Diagnostic tool 2. Monotherapy - First line therapy 3. Concert therapy - Companion, adjunct, or combination therapy with traditional pharmacological interventions * Some products can be used as both monotherapies and concert therapies, depending on design and evidence
  • 61. Combination Products Companion - Usually not FDA approved; can be used to treat a condition alongside pharmacological product and/or HCP treatment Combination - Application or sensor that is designed to be used in conjunction with a specific pharmacological product Adjunct - Application or sensor that is designed to be used with a category of pharmacological interventions or treatments
  • 62. Post Approval Obligations ● Failure to comply with post-clearance or approval regulatory requirements could subject the company to enforcement actions ○ Substantial penalties ○ Recall ○ Withdrawal of product from the market
  • 63. Adherence Plays upon the sentinel effect ● the tendency for human performance to improve when the performance is being evaluated by a third party (their physician)
  • 64. Trends ● Largely based on Cognitive Behavioral Therapy (CBT) ○ Focus on changing patient’s behavior or thinking ● Focus is thus on outcomes allowing value-based payment models ● Easier to collect real world data ● Keen interest in patient experience, product support and education, adherence/compliance, data sharing
  • 66. Common Therapeutic Areas Mental Health ● Depression, anxiety ● Body dysmorphic disorder (BDD) ● Schizophrenia ● Post traumatic Stress Disorder (PTSD) Behavior-Modifying Conditions ● Autism ● Attention deficit ● Substance Use Disorder (SUD) ● T2DM, heart failure
  • 67. Commercialization Preface ● Pathways to scale and commercialize are still largely unchartered ● The market is rapidly evolving, increasingly competitive ○ Therefore, difficult to forecast demand
  • 68. Partnership Considerations ● Upfront payments ● Royalty and milestone payments ● Research and development funding ● Profit sharing ● IP rights
  • 69. Pharma Partnership Examples ● Akilli and Shionogi ○ Shionogi oversees clinical development, sales and marketing in select markets ○ Shionogi made an upfront payment to Akili of $20M ○ Akili will be eligible to receive development and commercial milestone payments of up to $105M and royalties from sales in the selected markets
  • 70. Pharma Partnership Examples ● Click and Otsuka ○ Otsuka is fully funding the development of the mobile application to commercialize world wide ○ Otsuka will pay Click up to $10M in upfront and regulatory milestone payments along with the $20M in development funding ○ Click will receive an additional $272M in commercial milestone payments contingent upon regulatory approvals ○ Click will receive tiered royalties on global sales ○ Click can use Otsuka’s patient engagement platform
  • 72. Pricing Questions / Considerations ● How well the existing therapies treat the condition? ○ Perceived differentiation to reference products ● How much is the market willing to pay? ○ Type of reimbursement available ○ Target population’s income level ○ Target population’s social determinants of health ● Pricing can be dependent on the type of customer ● Level of invasiveness/burden ● Safety and efficacy profile
  • 73. Pricing Questions / Considerations ● Innovativeness/novelty of the product ○ Create a pricing matrix which uses a sensitivity analysis of market acceptance of various hypothetical prices ■ “First-in-class” (novel product with new indication) can be priced higher
  • 74. Pricing Models ● Risk-Based Model: ○ Value can be viewed from the basis of a percent of savings ● Value-Based Model: ○ Real world evidence is used to track value proposition ○ Incentivizes positive results
  • 75. Value-Based Pricing Calculation V = R ± D V = Perceived value of therapy being introduced R = Price of the reference therapeutic, generally the best existing alternative D = Perceived differentiation (both positive and negative) of the new product to relative to reference therapy
  • 76. Pricing Practices Skimming ● Set entry price at maximum level payers are willing to afford ● Fewer sales at higher price ● Gradually decrease the price to capture the growing part of the market
  • 77. Pricing Practices Penetration ● Set prices below comparable products to gain a large market share ● Results in greater volume at a lower price ● Raise prices over time
  • 78. Rx Only Reimbursement Models ● Physician completes and sends patient enrollment to the DTx manufacturer who then onboards the patient directly via a mobile app store access code ● Pts can only interact with the DTx per the label with the physician monitoring compliance, inputs, and progress via a dashboard
  • 79. Reimbursement Landscape ● March 2020 Senate bill called for Medicare reimbursement of PDT ○ Called for the establishment of a fee schedule to reimburse with 180 days of FDA clearance or approval ● Breakthrough designation for FDA cleared medical devices must fit into a pre-existing medicare benefit category
  • 80. Reimbursement Landscape ● No major commercial payers in the US have announced plans to cover PDTs ● PDT reimbursement has largely been from small PBMs and other small payers ● A set of 5 CPT codes were adopted in October 2020 for “remote therapeutic monitoring treatment management services” which could allow a fee-for-service reimbursement in the future ● Employer coverage has seen the most success
  • 81. Go To Market Strategies - Rx Only Rx-Only / Reimbursement: ○ Pricing based on perceived value ■ Demonstrating superiority = ability to set premium pricing ■ Need to maintain data of many types which show value proof points
  • 82. Go To Market Strategies - Rx Only Rx-Only / Reimbursement: ○ Demonstrate value through data from: ■ Randomized clinical trials ■ Regulatory approval for indications ■ Demonstrating market need through market analyses and rates of use ■ Demonstrate successful health outcomes
  • 83. Go To Market Strategies - Rx Only Rx-Only / Reimbursement: ○ Pursue value-based contracting with public payers or private insurance ■ Utilize public programs like Medicaid or Medicare ○ Can utilize rebate agreements to make pricing more attractive
  • 84. Go To Market Strategies - OTC “Over the counter”: ○ Direct to patient or employer ■ May require more marketing ■ Start with a free product or paying people to join/use the product ● Move from freemium to premium when a critical mass is achieved
  • 85. Go To Market Strategies - OTC “Over the counter”: ○ Employers can negotiate PMPM fees on ■ total employee basis ■ per engaged user basis
  • 86. Pricing and Reimbursement Insights ● As DTx come in a variety of forms, it is unlikely that there will be a formulaic approach to securing reimbursement
  • 87. Pricing Strategies Moving from a DTC offering to a medical benefit, to a pharmacy benefit can 10x the price $20/month $200/month $2,000/month DTC Medical Benefit Pharmacy Benefit
  • 88. PDT Pricing in the Market Typically a 3 month or 90 day period
  • 89. Customer Economic Model Contract Type Providers Risk-sharing PMPM Payors % Savings Recurring Employers Risk-sharing PMPM Pharma Licensing, royalty, milestone Recurring or term Patients Free with reimbursement Over-the-counter Example Payment Model by Customer
  • 90. Reimbursement Challenges ● Centers for Medicaid and Medicaid Services (CMS) has not yet issued clear reimbursement guidance ● Need to transition away from National Drug Code (NDC) and National Health Related Items Code (NHRIC) numbers towards Unique Device Identification (UDI) numbers, but there is hesitancy from pharmacies and payers ● Need to develop Healthcare Common Procedural Coding System Codes
  • 91. IP Protection ● Unlike traditional pharmaceuticals, software cannot be protected through a composition of matter patient ● Need both patents and granted claims ○ Claims expire in various years ● Create interrelated patents to strengthen protection
  • 92. Types of Patents 1. Software interface between the various computer hardware 2. Peripheral devices connecting the patient, physician, and caregivers 3. Interface of the hardware to the individual 4. Any combination of the above
  • 93. Software Copyright ● Used to discourage or prevent unauthorized copies of the proprietary software ● Prohibits “nonliteral elements” which includes the structure and sequence of the software code ● Can only be granted after registration once the software is published ● Can be quicker to get as patents require an examination procedure by a government agency
  • 94. Governing Laws and Regulations ● Data Privacy and Security - the collection, use, disclosure, and storage of personally identifiable information (PII), including protected health information and payment card data ● Federal and state healthcare fraud and abuse laws, false claims laws
  • 95. Launch Strategy ● Marketing must also drive awareness ● Utilize medical science liaisons, key opinion leaders, and educators ● Industry events which speak to the data and answer questions ● Publications to peer reviewed journals
  • 96. Next Frontier ● Expanding into acute conditions ● Need to develop better utilization metrics ● Need to prove patient engagement with DTx long term ● Facilitating payer reimbursement in the absence of dedicated NDC codes ● Nail down components of annual reporting required
  • 98. Pear Therapeutics - reSET ● 12 week DTx for individuals with Substance Use Disorder ● Uses CBT as an adjunct therapy to standard outpatient treatment ● 1 Study ○ Comprehensive CBT (Cognitive Behavioral Therapy) Via reSET App
  • 99. Pear Therapeutics - reSET-O ● 84 day PDT for Opioid Use Disorder ● Approved as prescription only PDTx ● Based on CBT ● Intended as an adjunct to outpatient treatment ● Submitted randomized controlled trial through FDA’s de novo approval pathway ● 3 Studies ○ reSET-O RCT ○ A Remote, Decentralized Opioid Use Disorder Study to Evaluate Patient Engagement With a Game-Based Digital Therapeutic ○ Pilot Study of reSET-O to Treatment-as-usual in Acute Care Settings
  • 100. Akili Interactive - ● EndeavorRX ○ Attention Deficit Hyperactivity Disorder ■ 5 Studies ● Cognitive ADHD Videogame Exploratory Study (CAVES) ● Electroencephalogram (EEG) Study of Inattention Following Treatment With AKL-T01 ● Software Treatment for Actively Reducing Severity of ADHD as Adjunctive Treatment to Stimulant ● Software Treatment for Actively Reducing Severity of ADHD (STARS-ADHD) ● Software Treatment for Actively Reducing Severity of ADHD - Follow Up (STARS-ADHD2)
  • 101. WellDoc ● BlueStar ○ Type II DM management tool ○ Similar to existing therapies, so demonstrating “substantial equivalence” rather than new clinical evidence ○ 1 Study ■ Evaluate the Value of Telehomecare for Diabetes
  • 102. Mahana Tx ● Parallel ○ Program for Irritable Bowel Syndrome (IBS) ■ “FDA authorized” ■ 1 Study ● The ACTIB trial ○ RCT of 558 patients ○ Not found on clinicaltrials.gov
  • 103. Nightware ● Nightware ○ Nightmares related to different conditions ■ 4 Studies ● Traumatic Nightmares Treated by NightWare ● NightWare Open Enrollment Study ● Traumatic Nightmares Treated by NightWare ● Remote Study of NightWare for PTSD With Nightmares
  • 104. Amalgam ● iSage Rx ○ Diabetes ■ 2 Studies ● A Digital Health Tool for Insulin Titration (DHIT) Individuals With Type 2 Diabetes: A Prospective Outcomes Study With a Retrospective Control Group (DHIT) ● mHealth Titration and Management
  • 105. Propeller - ● Platform for COPD and asthma connected with sensors on inhalers ● Combination product ● Track medication usage and provides feedback and insights that enable integration of care ● 13 Studies
  • 106. Other DTx Companies - ● Click Therapeutics ● Cognoa ● Voluntis ● Curio ● Chrono Therapeutics (shut down 2019) ● Sidekick Health ● Prosper ● Better Therapeutics ● Bold Health ● Ampersand ● Happify Health ● Hello Better ● Oviva
  • 108. Natural Cycles - ● Natural Cycles ○ Birth control app that helps women track their fertility to prevent unwanted pregnancies via the rhythm method ○ Analyzes data from past menstrual cycles and body temperature to determine when the woman is most fertile ○ When the woman is determined to be ovulating she is warned to use protection ○ FDA Cleared, CE Marked, Class IIb SaMD ○ 1 study of 15,000 women required
  • 109. Key Milestones 2010 FDA Approval of DTx [WellDoc’s BlueStar] 2017 FDA Launches Pre-Cert Program Pear’s reSET is 1st software-only DTx 2018 2019 Pear submitted the 1st application for FDA marketing authorization using the pre-cert program
  • 111. Benefits ● FDA approval does not guarantee payor uptake ● 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 good decreases with volume ● Requires less human capital
  • 112. $45-55 MM Cost to bring a concept through FDA submission is significantly lower than for molecular drug development ($350 MM)
  • 113. Vision ● DTx add functionalities into a more comprehensive portfolio, synchronizing products and services ● Can be paired with diagnostics, sensors, wearables, and even molecular therapies as combination therapy, expanding indication ● Integrated into electronic prescribing, dispensing, and medical record platforms ● Price of DTx will be significantly less than conventional therapies
  • 114. PwC Health Research Institute’s Annual Report 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%
  • 115. The Road Ahead ● Patient, provider, and payer appetite for DTx is still uncertain ● Need payer reimbursement models ● Increase stakeholder buy-in ● Integrate into provider workflows ● Need to develop more comprehensive DTx formularies ● Regulatory evaluation and commercialization pathways for other health authorities like EMA, TGA
  • 116. PBM Digital Health Formularies ● 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
  • 117. ● 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