Application of Real World Data

to clinical development

Hiroshi Onogi

Senior DirectorClinical Science Division, Research & DevelopmentAstraZeneca K.K.

July 4, 2014

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Disclaimer

Any views or opinions expressed in this presentation

are solely those of the author and do not necessarily

represent those of AstraZeneca.

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Contents

• Real world data

• Benefit to the pharmaceutical companies

• Utility of real world data in drug development

• Examples

• Summary

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What is a real world evidence?

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Why is RWD needed?

Traditional randomized controlled trials (RCT) are not well

suited to address real world outcomes

Effects in populations across different age, sex, race, co-

morbidity and co-intervention composition

Evaluation of rapidly changing interventions or technologies

Comparisons of active treatments or all possible therapeutic

options

Application to health care organization, delivery, and

payment models

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Sources of Real World data

Real World data can be collected in a number of ways, most commonly

from

Patients Registries

Health Databases

Patient and population

surveys

Electronic Patients records

Observational data

from cohort studies

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Japan has some valuable Real world

data

National Database (MHLW) ▪ Claims (In-patient, Out-patient, Pharmacy) from all the payer

▪ Special Checkup results

▪ Claims (In-patient, Out-patient, Pharmacy) from 20 large employee insurance

Japan Medical Data Center

▪ Hospital accounting data▪ DPC data▪ Clinical test result

Medical DataVision

▪ Clinical database integrated in hospital information system – Claims, Electronic medical records,

Drug, Lab result, Health checkup result

University Hospital

SOURCE: Public domain, Web page

Data sources Types of data

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RWD support R&D and Commercial Customers

across the product lifecycle

▪ Identify market unmet needs based on RWE

▪ Provide RWE insight into Disease Target Product Profile

▪ Support the preparation of reimbursement and regulatorydossiers

▪ Provide RWE insight to inform trial design and interpretation of results

▪ Provide RWE insights that support appropriate pricingand optimal access

▪ Provide RWE evidence against competitive threats

▪ Support acquisition of long-term outcomes and safety data

▪ Provide fact base to refine product key messages

▪ Understand patient care pathway in real world

▪ Estimate burden of disease

▪ Analyze local market disease and cost burden to develop tailored value propositions

▪ Evaluate impact of inclusion/exclusion criteria on trial recruitment

▪ Generate evidence in cost-effectiveness against lower-cost substitutes

▪ Proactively monitor drug safety

▪ Compare product compliance and outcome against competitors in real world setting

Po

ten

tial im

pact

Exam

ple

s

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Inclusion criteria

• Diagnosis of type 2 diabetes

within last year

• Age between 18 and 74

• Prescription for pioglitazone

• Prescription for metformin

• HbA1c>=7.0 and <=10.0%

Exclusion criteria

• Any historical diagnosis of type

1 diabetes

• Any history of pancreas injury

• Any history of acromegaly or

Cushing’s Syndrome

Example Electric

Health Record

database of type 2

diabetes patients

1000

Number of eligible

patients

128

Patients filtered

by trial criteria

Example of clinical feasibility

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Linking across different data sources

Data linking required

Unlinked Simple linked data Fully linked

Hig

h

Va

lue

of

RW

E a

na

lys

is

Base

In addition,•Cost effectiveness •Patient adherence •Comparative Effectiveness

research(CER)In addition,•Clinical outcome•Epigenomic information•Treatment effect•Safety data

▪Prevalence, incidence, Demography

▪Description of patient treatment patterns within single care setting

Through innovative and advanced analytical deliverables developed

using claims and electronic health record data

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Data

base

Data

base

Data

base

Data

base

Data

base

Data

base

RWE “back-end” is a hub partner and provide

analytical support to the company

RWE Hub

partner

Health

information

exchange

Integrated

delivery

networks

Community

physicians

Pharmacy

benefit

managers

Hub

partnersAdd-on

partners

RWE front-end RWE back-end

Build jointly

R&D Team

Payer interface

Commercial Brand

teams

Company

Insight Analytic capabilities Data access11

Summary

• Availability of large real world data will enable biopharmaceutical companies to make better use of information to create strategies for development to post-marketing phase.

• Maturity of infrastructure such as service provider, rule is Key to advance RWD utility in Japan.

• Pharmaceutical companies need to build new internal structure to maximize a value of RWD in drug development.

• Efficient and effective use of data is considered to lead to competitive advantage

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remove it from your system and note that you must not copy, distribute or take any action in reliance on it. Any unauthorized use or

disclosure of the contents of this file is not permitted and may be unlawful. AstraZeneca PLC, 2 Kingdom Street, London, W2 6BD, UK,

T: +44(0)20 7604 8000, F: +44 (0)20 7604 8151, www.astrazeneca.com

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