This document provides an agenda for a workshop titled "Using real-world data to find undiagnosed patients with rare diseases" hosted by IMS Health at the World Orphan Drug Congress from April 20-22 in Washington D.C. The workshop will explore how real-world data can help address underdiagnosis of rare diseases. Speakers will discuss using large-scale real-world data to transform understanding of rare diseases, and present case studies of companies leveraging real-world data. The workshop will also cover using predictive modeling of real-world data to identify undiagnosed rare disease patients and new engagement models with healthcare providers and payers to increase rare disease treatments.

1. April 20-22, Washington, D.C.
Using real-world data to
find undiagnosed patients
with rare diseases
IMS Health Real-World Evidence Solutions
Speakers
Michel Denarié, MBA, Senior Principal, RWE Solutions, IMS Health
Nadea Leavitt, MSC, Engagement Manager, RWE Solutions, IMS Health
Leanne Li, MBA, Engagement Manager, RWE Solutions, IMS Health
Kelly Peters, VP, Customer Segments, RWE Solutions & Commercial Services, IMS Health
Dr John Rigg, PhD, Principal and Head of Predictive Analytics, IMS Health
Diana Wong, MBA, MSC, BSC, Principal, RWE Solutions, IMS Health
April 20, 9:00am - 12:00pm | Pre-Conference Workshop

2. World Orphan Drug Congress (WODC),
April 20-22, Washington D.C.
2
are seen before diagnosis3
before an accurate
diagnosis3
7.3physicians (avg.)
4.8years (avg.)
1. Global GenesTM
, https://globalgenes.org/rare-diseases-facts-statistics/ 2. Eurordis - Rare Diseases Europe. Survey of the delay in diagnosis for 8 rare diseases in Europe ('EURORDISCARE 2'). www.eurordis.org/IMG/pdf/Fact_Sheet_Eurordiscare2a.pdf
3. Engel PA, Bagal S, Broback M, Boice N. Physician and patient perceptions regarding physician training in rare diseases: The need for stronger educational initiatives for physicians. Journal of Rare Disorders, 2013; 1(2): 1-15
million
people suffer from
a rare disease
worldwide1
350
rare diseases have
been identified1
7,000
of rare disease
sufferers are children1
50%
of patients are
misdiagnosed
initially2
40%
Rare diseases are not so rare,
but finding those patients is taking too long
IMS Health
brings more HOPE
to rare diseases sufferers
We use real-world data and advanced analytics
to help diagnose those patients quickly
Under-diagnosis is a major barrier to the successful
treatment of rare diseases. New tools leveraging
real-world data and innovation in advanced analytics
are creating opportunities for dramatic improvements
in identifying hard-to-find patients.
The purpose of this workshop is to explore the ways in
which real-world data (RWD) can be used to address the
problem of under-diagnosis in rare diseases. The session
will illustrate how companies worldwide are breaking new
ground in understanding rare diseases through a variety
of case studies. It will also include innovative ideas on
how to build new engagement models with providers
and payers to increase rare disease treatments.
Expect a highly interactive session with opportunity for
audience members to ask questions and share their experience.
Using real-world data to find undiagnosed patients
with rare diseases

3. World Orphan Drug Congress (WODC),
April 20-22, Washington D.C.
3
Workshop Agenda:
Using real-world data to find undiagnosed patients with rare diseases
April 20, 9:00am – 12:00pm
9:00-9:10
Introduction – Using real-world data (RWD) to address
the problem of under-diagnosis in rare diseases
Kelly Peters, VP, Customer Segments, RWE Solutions & Commercial Services,
IMS Health
9:10-9:40
Rare diseases are not that rare – How the expansion
of large-scale RWD is helping to transform the landscape
of rare diseases
Michel Denarié, MBA, Senior Principal, RWE Solutions, IMS Health
9:40-10:30
How companies worldwide are breaking new
ground in understanding rare diseases
Diana Wong, MBA, MSC, BSC, Principal, RWE Solutions, IMS Health
Leanne Li, MBA, Engagement Manager, RWE Solutions, IMS Health
10:30-10:40
Coffee Break
10:40-11:20
Finding the needle in the haystack – Real-world data
and predictive modelling helping to find patients with
undiagnosed rare diseases
Nadea Leavitt, MSC, Engagement Manager, RWE Solutions, IMS Health
11:20-12:00
New engagement models with providers and payers
to increase rare disease treatments
Dr John Rigg, PhD, Principal and Head of Predictive Analytics, IMS Health
Kelly Peters, VP, Customer Segments, RWE Solutions & Commercial Services,
IMS Health
10
Click here to register your interest for the IMS Health workshop
www.surveymonkey.com/r/7W7F7Y6
Click here to sign-up for the World Orphan Drug Congress

4. World Orphan Drug Congress (WODC),
April 20-22, Washington D.C.
4
9:00-9:10
Introduction – Using real-world data (RWD) to address
the problem of under-diagnosis in rare diseases
Kelly Peters, VP, Customer Segments, RWE Solutions & Commercial Services,
IMS Health
There are 7,000 rare diseases identified with an estimated 350 million
suffers worldwide, collectively the burden is considerable. This workshop
will explore the ways in which real-world data (RWD) can be used to address
the problem of under-diagnosis in rare diseases. The session will discuss
examples of RWD, focusing on key data assets in the U.S. and Europe,
including case studies to illustrate how these rich sources of data coupled
with predictive analytics can be used to find undiagnosed patients. The
workshop will also include novel ideas on how to build new engagement
models with providers and payers to maximize patient-finding strategies.
9:10-9:40
Rare diseases are not that rare – How the expansion
of large-scale RWD is helping to transform the landscape
of rare diseases
Michel Denarié, MBA, Senior Principal, RWE Solutions, IMS Health
Over the last decade, there has been a proliferation real-world data sources
across the world, fueled by the computerization of pharmacies and the increased
use of electronic medical record systems. This new data influx has given
researchers new avenues to better understand incidence, prevalence,
standard of care, and the overall patient journey.
In this section, we will explore what sources are available in the U.S.
and across the world, from data providers as well as health authorities.
Understanding these different types of RWD, their attributes, pitfalls
and analytical challenges is critical in selecting the most appropriate
RWD and applying it to the study of rare diseases.
30 20 10
10
Detailed Agenda:
Using real-world data to find undiagnosed patients with rare diseases
April 20, 9:00am – 12:00pm

5. World Orphan Drug Congress (WODC),
April 20-22, Washington D.C.
5
9:40-10:30
How companies worldwide are breaking new
ground in understanding rare diseases
Diana Wong, MBA, MSC, BSC, Principal, RWE Solutions, IMS Health
Leanne Li, MBA, Engagement Manager, RWE Solutions, IMS Health
As the importance of rare diseases continues to grow, IMS continues
to leverage its longitudinal patient data assets to address manufacturers’
business and outcomes research questions. In this section of the workshop,
we will present a variety of case studies spanning the U.S. and Europe,
addressing different research objectives, notably:
• Sizing the addressable market for a pre-launch orphan drug, including
the current standard of care for the diagnosed and treated population
• Understanding the frequency and distribution of health care costs for
a rare disease, and the differential in total cost of care vs. common
chronic conditions
• Identifying and segmenting the physicians likely to prescribe a
new orphan drug based on historical prescription patterns and early
product adoption
• Analyzing the burden of illness and epidemiological data
• Investigating care pathways and understanding the extent and effect
of late diagnosis, as well as obstacles in the health care system
preventing early diagnosis
10:40-11:20
Finding the needle in the haystack – Real-world data
and predictive modelling helping to find patients with
undiagnosed rare diseases
Nadea Leavitt, MSC, Engagement Manager, RWE Solutions, IMS Health
The path to a rare disease diagnosis can be long and frustrating for the
patient, as well as costly for the healthcare system. With 7,000 known
rare diseases, each inflicting a very small percentage of the U.S. population,
Health Care Practitioners (HCPs) often do not have any experience with the
rare condition of interest to properly diagnose it. This leads to ineffective
symptomatic treatments, unnecessary surgical interventions and delayed
proper diagnosis.
Raising awareness about rare diseases and a proper way to diagnose them
is necessary to combat this challenge. This section will review how using
IMS Health Real-World Data and advanced predictive modeling techniques
can help identify undiagnosed patients, link them to their treating HCPs
and provide a targeted, HIPAA-compliant and cost-effective way to raise
disease awareness and speed up the time to diagnosis.
50 35 15
40 25 15
Detailed Agenda:
Using real-world data to find undiagnosed patients with rare diseases
April 20, 9:00am – 12:00pm

6. World Orphan Drug Congress (WODC),
April 20-22, Washington D.C.
6
11:20-12:00
New engagement models with providers and payers
to increase rare disease treatments
Dr John Rigg, PhD, Principal and Head of Predictive Analytics, IMS Health
Kelly Peters, VP, Customer Segments, RWE Solutions Commercial
Services, IMS Health
While physician target lists may be the most obvious way for pharma
to utilize output from a predictive model, is it necessarily the best way?
This session will explore challenges with physician target lists, particularly
the need for patient anonymity and the potentially low conversion rates
(number of physicians contacted per new patient on treatment). Operating
models, which may be more rewarding, including partnerships with providers
and payers will be discussed as a potential alternative. These partnerships
may involve developing the algorithm with named patients and clinician
out-reach by trained healthcare professionals, resulting in a more direct
and effective way to find undiagnosed patients.
Audience members will be encouraged to share their thoughts and experience.
This final session of the workshop is intended to be especially interactive,
with an emphasis on collaboratively identifying innovative solutions.
40 20 20
Detailed Agenda:
Using real-world data to find undiagnosed patients with rare diseases
April 20, 9:00am – 12:00pm
Click here to register your interest for the IMS Health workshop
www.surveymonkey.com/r/7W7F7Y6
Click here to sign-up for the World Orphan Drug Congress

7. World Orphan Drug Congress (WODC),
April 20-22, Washington D.C.
7
Michel Denarié is a Sr Principal in the Real-
World Evidence Solutions’ team, developing
new offerings for IMS and helping clients solve
an array of business issues by leveraging the full
potential of longitudinal data across the globe.
Specifically, he develops national and sub-
national patient-centric offerings that analyze
patient and treatment trends over time and
improve sales force effectiveness strategies.
Main functional experience: US and global
longitudinal patient data.
Denarié’s experience in the pharmaceutical
industry spans three decades and encompasses
roles in sales, marketing, and product
development. A frequent author and speaker,
Denarié’s work has appeared in several trade
medical publications. Denarié earned an
M.B.A. from the Colgate Darden Graduate
School of Business Administration at the
University of Virginia. He holds a B.S.
in finance from American University
in Washington, DC.
Michel Denarié, MBA,
Senior Principal,
RWE Solutions, IMS Health
Presenters:
Using real-world data to find undiagnosed patients with rare diseases
Nadea Leavitt is an Engagement Manager in
the Real-World Evidence (RWE) department
of IMS Health. Using longitudinal patient level
data, she conducts and manages research
engagements that help pharma clients answer
an array of business questions, spanning
from market opportunity assessment to
launch preparation tracking, to brand
strategies post launch.
Nadea also leads the development of
innovative offerings combining rich
real-world data and predictive analytics
to identify new opportunities and address
challenging issues. This includes developing
novel disease detection algorithms to find
undiagnosed patients with rare diseases.
Nadea is also leading IMS’s thought leadership
on next-generation physician targeting
strategies using advanced machine learning
techniques. Nadea holds a B.A in Economics
and an M.S. in Applied Statistics.
Nadea Leavitt, MSC,
Engagement Manager,
RWE Solutions, IMS Health
Leanne Li is an Engagement Manager in
the Real-World Evidence (RWE) department
of IMS Health.With extensive experience
overseeing highly complex and customized
consulting engagements, she leverages
longitudinal patient level data assets, applies
consultative problem solving skills, formulates
analytical approaches, and presents
conclusions and recommendations to help
pharma clients tackle business challenges.
Her areas of expertise include source
of business, market sizing, patient and
physician segmentation, and adherence
tracking, spanning various therapeutic
markets such as Immunology, women’s
health, and rare disease. Leanne holds a
B.A in Diplomacy, a M.P.A. in Health Policy
and Management and an M.B.A in Finance
and Marketing.
Leanne Li, MBA,
Engagement Manager,
RWE Solutions, IMS Health

8. World Orphan Drug Congress (WODC),
April 20-22, Washington D.C.
8
Presenters:
Using real-world data to find undiagnosed patients with rare diseases
John Rigg, PhD heads-up the Predictive
Analytics team in IMS Health’s Real-World
Evidence Solutions. He develops innovative
solutions to solve challenging problems
using patient-level data using a variety of
advanced statistical and machine learning
methods. This encompasses applications
such as clinical decision-support tools, risk
stratification calculators, rare disease detection
algorithms and physician targeting alerts.
John has over twenty years of experience
developing predictive analytics solutions in
life sciences, financial services and academia.
He is regularly invited to deliver thought
leadership presentations at universities, life
science organizations and leading conferences.
John has a PhD in Economics from Cambridge
University and has held post-doctoral
academic positions at the London School
of Economics and the University of Essex.
Dr John Rigg, PhD,
Principal and Head of
Predictive Analytics,
IMS Health
Diana is currently driving strategy and
innovation initiatives to advance real-world
evidence technologies at IMS Health globally.
Diana also leads the Enriched Real-World Data
Studies initiative and is building a solution
that combines retrospective research with
prospective data collection within a
streamlined technology.
Prior to IMS Health, Diana worked as a
management consultant on health care
reform in the United States and Europe
defining new roles for all health care
stakeholders. Diana has a BScH specializing
in biochemistry from Queen’s University, a
MSc in type 2 diabetes research from the
University of Toronto and MBA majoring in
Health Sector studies from the Richard Ivey
School of Business at the University of
Western Ontario.
Diana Wong, MBA, MSC, BSC,
Principal, RWE Solutions,
IMS Health
Kelly Peters leads IMS Health’s Real-World
Evidence (RWE) principal community, which
guides the application of RWE in the U.S.
operational processes (supporting pharma
companies directly and their agencies of
record, in addition to advocacy groups acting
on behalf of the patients they serve).
With over 20 years of experience in the
healthcare industry, Kelly specializes in
provision of evidence-based operational
guidance to marketing, sales, commercial
operations, and business development client
teams. She has hands-on experience in the
use of RWE for the assessment of product
potential, development of multi-channel
operational plans tied to reaching that
potential, and creation of diagnostic solutions
across several therapeutic markets
(spanning orphan drug categories through
mainstream conditions that impact a broad
base of patients).
Kelly Peters,
VP, Customer Segments,
RWE Solutions Commercial
Services, IMS Health

9. World Orphan Drug Congress (WODC),
April 20-22, Washington D.C.
9
Trial
optimization
Drug utilizationstudies
Retrospective
studies Market access
Patient journey
Brand
performance
Patient
recruitment
Markeing
effectiveness
Hybrid/Mixed
studies
HEOR/
Safety
RD
Commercial
Economic
models
LRx Data
Hospital
ePRO pRCTs
Registries EMR=
eCRF
Social
Media
EMR
Enriched
Datasets
Claims
Survey
TE
CHNOLOGY ADVAN
CEDANALYTICS
SO
LUTIONSLINKAGES
SECURITY
IMS Health Real-World Evidence Solutions
Our Vision of an RWE Ecosystem
An environment for building deeper insights to benefit the entire enterprise
Imagine a world where everyone can use the same fact base to understand what is really happening in healthcare. Add the
ability to leverage advanced analytics, technology, data management approaches, data visualization and other innovations to
bring relevant data together easily and accurately. You would then have the scientific evidence needed to support your products’
value proposition, safety, access and price. And you could use these insights to make better commercial decisions as well.

10. World Orphan Drug Congress (WODC),
April 20-22, Washington D.C.
10
IMS Health Our Distinctive Approach
IMS Health has taken a holistic approach to real-world evidence (RWE) in pursuit of the most robust and
insightful views of patient outcomes. With the breadth and depth of our capabilities, we are uniquely positioned
to generate and use RWE globally. Applying the appropriate scientific and commercial lens and latest techniques
to anonymous real-world data (RWD) is critical to realizing the value of RWE in healthcare decisions.
Real-World Data
Identify and access the most appropriate data sources while
ensuring patient privacy
• The broadest and deepest collection of scientifically-validated,
anonymous patient-level data assets complemented by custom
data sourcing
Technology-Enabled Analytics
Create consistent data models empowering analyses and leveraging
innovative technologies
• Extraction, analysis and visualization of RWD with IMS Health’s
E360™ technology suite
• Sophisticated data linkage and interpretation
Services and Engagement
Develop on RWE strategy and analyze RWD using scientific rigor,
clear governance, and advanced analytic and processing capabilities
• Strategic support
• Outcomes research, pharmacoepidemiology drug safety
• Health economics market access
• Market-level engagement
Technology-
Enabled
Analytics
Services
and
Engagement
Real-World
Data IMS HEALTH
REAL-WORLD
EVIDENCE
SOLUTIONS
Leadership and innovation across the RWE spectrum
IMS Health Real-World Evidence Solutions

11. IMS Health Real-World Evidence Solutions
is based in 20 countries worldwide with regional headquarters in
RWEinfo@imshealth.com
www.imshealth.com/rwe
The Americas
One IMS Drive
Plymouth Meeting,
Pennsylvania 19462
USA
Tel: +1 (484) 567 6461
Europe
210 Pentonville Road
London N1 9JY
United Kingdom
Tel: +44 (0) 20 3075 4800
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Col.t Tizapan
Mexico D.F.-C.P. 01090
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Singapore 048424
Singapore
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Japan
Tel: +81 3 5425 9541
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