Optimizing Real-World Evidence for Maternal and Neonatal Outcomes

Optimizing Real-World
Evidence for Maternal and
Neonatal Outcomes
Deb Discenza, MA
PreemieWorld
Founder/Publisher/CEO
Jennifer Degl, MS
Speaking for Moms &
Babies, Inc.
Founder, Author/Speaker
Eric Stanek, PharmD
Scientific Lead
Anthem, Inc.
Michael Grabner, PhD
Principal Scientist
HealthCore, Inc.
Thomas Miller, PhD, MBA
Vice President & Global Head
Bayer US LLC

Agenda
• RWE Preterm Birth Case Study & Payer Perspectives
o Eric Stanek, PharmD
o Michael Grabner, PhD
• NICU Parent Perspectives & Why They Matter
o Deb Discenza, MA
o Jennifer Degl, MS
• Use of RWD in Neonatal Clinical Trials
o Thomas Miller, PhD, MBA

RWE Preterm Birth Case Study &
Payer Perspectives
Eric Stanek, PharmD
Scientific Lead, Enterprise Analytics Hub
Anthem, Inc.
eric.stanek@anthem.com
Michael Grabner, PhD
Principal Scientist, HEOR
HealthCore, Inc.
mgrabner@healthcore.com
healthcore.com @HealthCoreRWE

Speaker Disclosures
Michael Grabner is an employee of HealthCore, Inc., which conducts research
sponsored by a wide variety of private and public entities, and a shareholder of
Anthem, Inc.
Eric Stanek is an employee and shareholder of Anthem, Inc.
Case study funding was provided by Sera Prognostics Inc., the maker of the
PreTRM® test, to HealthCore, Inc.

HealthCore: A Subsidiary of Anthem
Clients & Collaborators
Anthem Subsidiaries
Parent Company
45+M total active medical members
1 in 8 Americans in affiliated health plans across all 50 states
Government Agencies
FDA, PCORI, NIH, CDC
Academia
Including Wharton
Industry
& Associations

Real-World Data and Evidence
Real-World Data (RWD)
The data relating to patient health status and/or the
delivery of healthcare routinely collected from a variety
of sources. RWD can come from a number of sources,
i.e.:
• Electronic health records (EHRs)
• Claims and billing activities
• Product and disease registries
• Patient-generated data including in home-use
settings
• Data gathered from other sources that can inform on
health status, such as mobile devices
Real-World Evidence (RWE)
The clinical evidence regarding the usage and
potential benefits or risks of a medical product
derived from analysis of RWD.
RWE can be generated by different study designs or
analyses, including but not limited to:
• Randomized trials, including large simple trials
• Pragmatic trials
• Observational studies (prospective and/or
retrospective)
Source: fda.gov/science-research/science-and-research-special-topics/real-world-evidence

Preterm Birth (PTB) US Statistics
380,000
babies born prematurely each
year in the US (9.8% overall
premature birth rate)
12x
higher preterm direct
healthcare costs vs. that of a
full-term delivery
21,000
annual infant mortality in US
before first birthday
17 days
hospitalization length of stay
following preterm birth, vs. 3
days for full term
17%
infant deaths attributed to
preterm birth and low birth
weight
55%
NICU admission rate for
preterm birth, compared to
9% for full term

PTB Burden
• The five leading causes of infant death are birth
defects, preterm birth & low birth
weight, injuries, sudden infant death syndrome, and
maternal pregnancy complications
• Premature babies are at greater risk for cerebral
palsy, respiratory and cardiovascular complications,
and delays in development – the effects of which
are both acute and long-term
~410k
Anthem births per year
11%
US births covered by
Anthem
56%
Anthem births covered
by Commercial
44%
Anthem births covered
by Medicaid
One of the biggest
challenges in addressing
preterm birth is the
majority are born to
mothers with no known
risk factors, making
targeting interventions very
difficult

• PTB risk prognostic test
• Sensitivity (75%) and specificity (74%)
• Target population: asymptomatic, singleton
pregnancies without evident PTB risk
• Allows identification and quantification of PTB risk
in those 4 out of 5 moms
Potential of a New Risk-Screen-and-Treat Approach
Today, we cannot
predict
4 out of 5
preterm births

PreTRM Evidence Base
Analytic Validity Clinical Validity
Economic Value
(vs usual care)
Clinical Utility (clinical
trials of test + treat
strategy
vs. usual care)
• Established
• Multiple publications
• Established
• Multiple publications
(Saade 2016, Boggess 2017,
Markenson 2020)
• Established
• Budget Impact Model
(published, Caughey 2016)
• Anthem Budget Impact
Model replication (internal
2019)
• Anthem Commercial cost-
effectiveness model
(published, Grabner 2021)
• Anthem Medicaid cost-
effectiveness model (internal
2020)
• Anthem financial model
(internal 2021)
• Developing
• PREVENT study (published,
Branch 2021)
§ Single center, randomized,
controlled, Sera-sponsored
• AVERT study (results 2022)
§ Single center, non-
randomized cohort study
with historical control,
Sera-sponsored
• PRIME study (ongoing)
• Real-world data (opportunity)

PreTRM Cost-Effectiveness Analysis (CEA)
Use of a novel prognostic test
during pregnancy to identify
women at risk of PTB
combined with evidence-based
treatment is estimated to reduce
total costs
while preventing PTBs and their
consequences.
“
Reference: Grabner M, Burchard J, Nguyen C, Chung H, Gangan N, Boniface JJ, Zupancic JAF, Stanek E. Cost-Effectiveness of a Proteomic Test
for Preterm Birth Prediction. Clinicoecon Outcomes Res. 2021;13:809-820; https://doi.org/10.2147/CEOR.S325094

PreTRM CEA – Key Methodological Challenges
• Defining model structure that is as complex as necessary and as simple as
possible
• Conducting de novo, customized meta-analysis of treatment effects given the
need to combine a range of existing low- to high-intensity interventions
• Using claims to identify moms that fit the intended use population of the PreTRM
test (e.g., singleton births only, without preterm labor or progesterone therapy)
• Aligning on reasonable base-case input parameters and plausible ranges for
sensitivity analyses (e.g., % of moms that will be screened, % adherent to the
treatment)

PreTRM CEA – PTB Burden from Anthem Claims
• Shown is the
distribution of infant
and maternal costs by
gestational age at birth
(2016, HIRD*)
• These costs mirror the
distribution of costs in
US data as a whole and
therefore offer
validation of the use of
Anthem-affiliated
commercial claims data
*HIRD: HealthCore Integrated Research Database

PreTRM CEA – Reducing PTBs & Saving Costs
• Intervention: multi-component high-
intensity case management (HICM)
and vaginal progesterone for the
remainder of the pregnancy for
women assessed as high-risk
• PTB rates and costs were based on
real-world cohorts of >40,000
mothers and infants (identified
separately) with birth events in 2016
• Risk-screening-and-treat strategy
dominated usual care with 870 fewer
PTBs (20% reduction) and $54 million
less in total cost ($863 net savings per
pregnant woman)
Each blue dot in the ICER scatter plot represents one of the 10,000 PSA simulation outcomes. The scatter plot is wedge-shaped with all mass
in the second quadrant. All dots below the x-axis represent cost savings. In the base case, all simulations are associated with cost savings
and PTB reductions.
PTB = preterm birth; PSA = probabilistic sensitivity analysis
Incremental Effectiveness (PTBs averted)
Incremental
Costs,
USD
2018
in
millions

Broad Maternity RWE Portfolio
• Maternal-Child Health is a Key Focus Domain for the Anthem
Enterprise Analytics Hub
• Cost, maternal and neonatal/infant outcomes, and quality of care across
geographies, providers, disparities/SDoH, comorbidities, and procedures
• Output advises policy, clinical programs, innovative payment programs,
and contributes to scientific literature – to improve maternal child
outcomes
• HealthCore has over a dozen on-going pregnancy-related studies
with government and life-science partners, including the FDA and
EMA
• Pregnant women often excluded from trials, so real-world safety and
effectiveness evidence is essential
Psoriasis
Anti-
epileptics
Migraine
Atopic
dermatitis
Multiple
Sclerosis
Vaccines
Preterm
birth
(PreTRM®
test)
Ischemic
placental
disease
Areas of HealthCore’s
Current Maternal Health Studies

Thank You
Contact us at rwe@healthcore.com to find out more information.
healthcore.com @HealthCoreRWE

NICU Parent
Perspectives &
Why They Matter
Deb Discenza, MA
PreemieWorld
Jennifer Degl, MS
Speaking for Moms & Babies,
Inc.

Hello!
I am Deb Discenza
M.O.M. of Becky, Founder/Author
PreemieWorld™ LLC
Alliance for Black NICU Families™
Crystal Ball Health, LLC

Hello!
I am Becky
My Mom gave birth to me 10 weeks early.
This is a picture of her holding me for the first
time. What you don’t see are trauma and her
hands shaking.
19

26 Weeks - Ignored
▷ I was ignored when I told my OB that I had been early, too.
▷ “It’s your first pregnancy, you will be late.”
▷ The NICU left more scars than the ones from Becky’s IV lines.
OB: Obstetrician
NICU: Neonatal Intensive Care Unit

30-weeks: “She’s doing great!”
▷ Becky was 2 lbs. 15.5 oz at birth at 30 weeks.
▷ Lots of ups and downs, avoided surgeries, almost lost her 2x with stopping
breathing and in another instance, sepsis.
▷ Becky went home 38 days later, with oxygen and a monitor.
▷ She was hospitalized again for feeding and breathing issues.

NICU Babies: High Care, Little Research
▷ We parents often discuss our children’s hospital bills and how the NICU
revenue funds the rest of the hospital.
▷ So where are the stacks of high-end research for this high-end care
comparable to Cancer, Diabetes, and now COVID?
▷ Jon Davis was on the team for Surfactant. Decades ago.

The Reminder: The Patients
▷ This is not just a stay in the NICU, it’s a lifetime of challenges: behavioral,
mental, physical and emotional. Then add racism.
▷ We are not M.D.s but we are M.O.M.s and we have strong influence this
patient/provider community. We remind you.
▷ At INC, I “joke” with our global stakeholders about “taking roll call” in doing
their part to pool the data. But it has worked.
INC: International Neonatal Consortium

Real World Evidence & Collaboration
▷ Jen and I sit on the Leadership Team of INC.
▷ We are valued members, giving speeches, attending meetings, providing
valuable input, collaborating on articles, being asked to contribute our own
parent commentary in the Journal of Pediatrics.
▷ I also started Preemie Crystal Ball to create a data portal for families that
will dramatically benefit everyone involved.

Why do I care so much?
▷ Becky has Autism Spectrum Disorder.
▷ Becky has ADHD.
▷ Becky has Cerebral Palsy that was diagnosed at 13.5 years old along with
Scoliosis because I knew something was wrong.
She did, too…
▷ I was a preemie, too. As old as neonatology.
▷ I see so many families struggling in and out of the NICU. We have to do
better. These are not just babies. They are society’s legacies.
ADHD: Attention Deficit Hyperactivity Disorder

Why do I care so much?
▷ PPD, PTSD and a special needs child can rip a family apart. Trauma is lifelong
and is ultimately society’s burden.
▷ Post-NICU coordination and benefits are highly inadequate. Families are
isolated and left to figure it out themselves.
▷ Is it any wonder that my daily news feed regularly includes horror stories
about families with premature babies?
PPD: post-partum depression
PTSD: post-traumatic stress disorder

▷ Maternal Near Miss Survivor
▷ Micro Preemie Mom
▷ Author
▷ Speaker
▷ Teacher
▷ Maternal & Neonatal Health Advocate
Hello!
I am Jennifer Degl

• Born at 23 weeks gestation.
• Weighed 575 grams (1 lb, 4 oz).
• Spent 121 days in the NICU.
• Lives with advanced BPD and more.
• Experienced more medical interventions
and pain in my first 4 months of life than
most healthy adults will ever experience.
Hello!
I am Joy
BPD: Bronchopulmonary Dysplasia

Parents of Premature
& Medically Fragile Babies
▷ Have had very little resources available to them to prepare them for their
NICU stay and to guide them in navigating the complex medical decisions
that need to be made.
▷ Have been reluctant to consent for their babies to participate in clinical
research.
▷ Have not had a voice at the table during conversations on clinical research
planning and execution, and therapies that impact the survival rates of their
babies and future babies.

How INC Takes Action
▷ NICU parent leaders were invited to join them since inception, as members of
workgroups.
▷ NICU parents have served as co-chairs and had other integral roles in INC
projects.
▷ Currently, two NICU parents are on the Leadership Team, providing weekly
insight from our lived experiences.
▷ There is no more need for guessing.
▷ Our experiences and opinions matter and we are included as key
stakeholders, along with physicians, nurses and researchers.

What’s Next for NICU
Parent Stakeholders?
▷ We recently published an editorial in the Journal of Perinatology regarding
our parent perceptions from INC multistakeholder surveys on how research
is communicated in the NICU. (We previously published one in J. Pediatrics).
▷ The culture of research communication in neonatal intensive care units: key
stakeholder perspectives (Click HERE)
▷ I (parent) was a lead author on the primary paper. (Click HERE)
▷ I (parent) am presenting this research at the Pediatric Academic Societies
(PAS) meeting in Denver on April 22.

How Can You Get Involved?
▷ Visit the INC website to see what workgroups you may want to join and
share our Communications Toolkits.
▷ Read our recent paper and editorial companion to see if our research and
conclusions resonate with you/your work.
▷ Reach out to the INC leadership to get access to the remaining data that
was not presented in the papers to see if you can dive more into the
perceptions of the stakeholder groups.
▷ Reach out to us (parents) if you want us to contribute our unique
perspectives to your work or research.

Additional Resources
▷ International Neonatal Consortium
▷ Critical Path
▷ NICU Parent Network
▷ EFCNI
▷ iCAN

Connect With Us
Deb Discenza, MA
PreemieWorld
Jennifer Degl, MS
Speaking for Moms &
Babies, Inc.
PreemieWorld.com
BlackNICUFamilies.org
CrystalBallHealth.com
JenniferDegl.com

Use of RWD in Neonatal
Clinical Trials
Dr. Thomas F. Miller
Vice President & Global Head, Pediatric Development
Bayer
Thomas.Miller@bayer.com
http://www.bayer.us/

Use of RWD in Neonatal Clinical Trials
• Key challenges with clinical trials that enroll infants:
• Children can’t advocate for themselves – parents’ perspective regarding placebo-
controlled trials
• Long enrollment periods – many trials do not achieve enrollment goals
• Sponsor may fail to secure Health Authority incentives if trial doesn’t complete
• Sources of RWD for use in trials
• EMRs
• Registries
• Natural History Studies
• Peer-reviewed publications
• Potential use of RWD in clinical trials, including those focused on neonates
• Information standardization – the INC RWD project
• Using large (RW) datasets and AI/machine learning to identify ‘patient endotypes’
• Synthetic control arms

Thank You
Dr. Thomas F. Miller
Vice President & Global Head, Pediatric Development
Bayer
Connect with me:
E-mail: thomas.miller@bayer.com
International Neonatal Consortium - Critical Path Institute

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Optimizing Real-World Evidence for Maternal and Neonatal Outcomes

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