PhRMA / EFPIA

Research in Your Backyard: Japan
Contribution of PhRMA and EFPIA Member Companies to Japan’s Society & Economy
Through Clinical Trials of Innovative Medicines

May 2014

AgendaIntroduction: Who We Are

3

Pharmaceutical Industry and Society

5

Collaborating on Clinical Trials

7

R&D Spending, Trends, and Results

14Introduction: Who We Are
We are devoted to advancing public policies that support innovative medical research,yield progress for patients today, and provide hope for the treatments and cures of tomorrow

Introduction

Introduction

PhRMA Japan represents U.S. based
pharmaceutical companies. Ouractivities vary from promoting healthcare
system reform to providing medical
information to patients

EFPIA Japan represents R&D-based European
pharmaceutical companies. We focus on patientneeds in Japan, engage in discussions with key
stakeholders regarding public health, and work
to improve the R&D environment forpharmaceutical companies

MissionMission

PhRMA Japan’s mission
is to promote the healthof patients and contributeto the healthier lives ofall people

EFPIA Japan’s mission isto enhance the health ofJapanese patients byintroducing innovative
drugs and vaccines

PhRMAEFPIA3PhRMA and EFPIA member companies

PhRMA member companies

EFPIA member companies

AbbVie GK

Abbott Japan Co.,Ltd.

Guerbet Japan K.K.

Amgen Astellas BioPharma K.K.

Actelion Pharmaceuticals Japan Ltd.

Janssen Pharmaceutical K.K.

Biogen Idec Japan Ltd.

AstraZeneca K.K.

LEO Pharma K.K.

Bristol-Myers K.K.

Baxter Limited

Lundbeck Japan K. K.

Celgene K.K.

Bayer Yakuhin, Ltd

Merck Serono Co.,Ltd.

Eli Lilly Japan K.K.

Bracco-Eisai Co., Ltd.

Nihon Servier Co.,Ltd.

Janssen Pharmaceutical K.K.

Chugai Pharmaceutical Co., Ltd.

Nippon Boehringer Ingelheim Co ., Ltd.

MSD K.K.

CSL Behring K.K.

Novartis Pharma K.K.

Mundipharma K.K.

Ferring Pharmaceuticals Co., Ltd.

Novo Nordisk Pharma Ltd.

Pfizer Japan Inc.

Galderma (Japan) K.K.

Sanofi K.K.

GE Healthcare Japan Corporation

Shire Japan K.K.

GlaxoSmithKline K.K.

UCB Japan Co. Ltd.

4

Pharmaceutical Industry and Society

5Pharmaceutical companies contribute to the society andeconomy in multiple ways
Pharmaceutical companies collaborate with local clinics and hospitals to develop new drugs.
This in turn provides economic stimulus and leads to innovative new medicines with better efficacy and safety profiles

DrugDevelopment

Economic
Stimulation

CreatingJobs

Increasing
Productivity

BasicResearch

Clinical
TrialsToday’s presentation

Innovative Drugs toPromote Healthier andLonger Lives
BetterEfficacy

Improved
Safety6Collaborating on Clinical Trials

7

Discovering and developing new medicines is a long,complex, and costly process
Developing innovative drugs takes an average of 10-15 years and 1-1.5B USD. On average only one medicine is finallyapproved and launched for every 10,000 or more compounds initially discovered. Many promising compounds failafter pharmaceutical companies have spent substantial amounts of money on testing and clinical trials
ClinicalTrials

10,000+COMPOUNDS

250

5PHASE1

3-6 YEARSRegulatory
Review

Apply for new drug approval

Preclinical

Apply to conduct clinical trial

PRE-DISCOVERY

Drug
Discovery

PHASE2

6-7 YEARS

Source: PhRMA
The majority of investment for the development of new drugs is used in conducting clinical trials

Scale-up
to MFG.

PostMarketing
Surveillance

One Approved Drug

PHASE3

0.5-2 YEARS・ ・ ・8What is a clinical trial?
Computer simulations and animal testing are not enough to determine the efficacy and safety of new drugs for people.
Clinical trials are conducted in five phases with the aim of clarifying the impact of experimental drugs on humans

Phase 0First-in-human trial with a small number of healthy adults (10 to 15)
to gather data on what the drug does to the body and what the body
does to the drug

Phase 1

Trial to test a drug with a small number of healthy adults (20 to 80) to
test safety, determine a safe dosage range and identify side effects

Phase 2First trial using actual patients (100 to 300) to test efficacy of a newdrug and further evaluate its safety

Phase 3

Trial with an increased number of patients (1,000 to 3,000) in order toconfirm its effectiveness, monitor side effects, and to compare it toexisting treatments

Phase 4After a new drug has been approved, Post Marketing Surveillance ofactual usage is conducted to confirm that the drug acts as expected9Working with institutions in conducting and supporting clinical trials
Conducting clinical trials involves collaboration between pharmaceutical companies and other institutions,such as hospitals, local clinics, and contract research organizations (CRO)
CLINICHOSPITALPEER REVIEWEDJOURNALDATA

SMOPATIENTSDATAREGULATORYAGENCYPHARMACEUTICALCOMPANIES

Pharmaceutical Companies

CROCRO

Hospitals / Clinics

SMO (Site Management Organizations)

Decide to conduct clinical trials

Support pharmaceutical companies

Conduct trials performed by physicians

Provide additional support to hospitals

and requests support from CRO

with the coordination of clinical trials

and trained research professionals

and clinics during trials

10Conducting clinical trials with local medical institutions
benefits the hosting area
Conducting clinical trials for new drug development contributes to local economies
and improves the quality of life for local citizens

Providing advancedtreatment

Medical facilities conducting clinical trials are able to provide
advanced medical treatments to their patients in these locales

Contributing tolocal economy

The spending on clinical trials in Japan goes to hospitals and clinics
nationwide and supports local economies

Creating jobs

Related stakeholders (local medical facilities and pharmaceuticalcompanies) employ local staff to support clinical trials

11

PhRMA and EFPIA member companies conduct many clinical trials
yearly across all prefectures of Japan682

CLINICAL TRIALS

11,653
SITES**# of clinical trial conducted
201 ~16+*THERAPEUTIC AREAS
Allergy
Cardiology
CNSDermatology
Endocrine Metabolic Disease
Gastroenterology
Hematology
Immunology

Infectious Disease
Musculoskeletal Disease
Oncology
Ophthalmology
Respiratory Disease
Urology
Rare Diseases
Others101 ~ 200
31 ~ 100
Source: www.clinicaltrial.gov (2013 data)*Figures do not include post markeing surveillance **Individual sites participating in multiple trials are counted multiple times

12How many of the 682 clinical trials conducted by PhRMA and EFPIA
member companies in Japan are conducted in each prefecture?
Prefecture not specified

Tottori
Shimane
Okayama
Hiroshima
Yamaguchi

3139164151
72

Hokkaido

19
Osaka
Hyogo
Kyoto
Shiga
Nara
Wakayama

268

Hokkaido
371
255
161
49
63
49

Aomori
Iwate
Miyagi
Akita
Yamagata
Fukushima

Toyama 66
Ishikawa 93
Fukui
43

Hokuriku

Tohoku

Kinki

Kanto Koshinetsu

Chugoku
Tokai

KyushuFukuoka
Saga
Nagasaki
Kumamoto
Oita
MiyazakiKagoshima
Okinawa

4256
175
434361355
59102150765610359

Source: www.clinicaltrial.gov (2013 data)

Aichi
Gifu
Shizuoka
MieShikoku
Tokushima
KagawaEhimeKochi

7284
117
48

320
75
198
77

Tokyo
Kanagawa
Saitama
Chiba
IbarakiTochigi
Gunma
Yamanashi
NaganoNiigata

541
315
182
300
131
94
116
44102
95

13

R&D Spending, Trends, and Results

14

PhRMA and EFPIA companies represent a significant shareof the research and development spending in Japan

2012 All Pharmaceutical R&D Spending in Japan*

1306B JPY

2012 Clinical Trial Related
Spending in Japan Total**

247B JPY

2012 Clinical Trial Related
Spending in Japan PhRMA and EFPIA Subtotal***

88B JPYSource: JPMA data based on company disclosures for fiscal year 2012
*Report on the Survey of Research and Development 2013, Ministry of Internal Affairs and Communications ** 72 JPMA companies *** 19 PhRMA and EFPIA companies participating in JPMA
Note: The 1,306 billion yen figure includes all pharmaceutical R&D expenditures made during fiscal year 2012 by all companies in Japan. The 247 billion yen and 88 billion yen figures refer only toclinical research related spending during fiscal year 2012 disclosed by JPMA member companies

15

To deliver innovative drugs in a timely manner,PhRMA and EFPIA members are increasing simultaneous global
development including Japan to reduce “drug lag” *The trend to perform global development simultaneously has been increasingin countries around the world including Japan
Trends in Global Simultaneous Development
for PhRMA / EFPIA Companies

186
75

2010

96

2012

2017
〔Forecast〕

Number of Global Simultaneous Development Clinical Trials Including Japan
Source: PhRMA research
Number of phase 3 clinical trials under development conducted by PhRMA / EFPIA companies (The study had a total of 19 companies – 10 of which are PhRMA / EFPIA related)* “Drug lag” refers to the delay to approve a new drug such as cases in Japan where approval is delayed compared to other countries, such as the U.S. and Europe.
Conducting more global simultaneous clinical trials shortens the drug lag

16

PhRMA and EFPIA member companies obtained approvalfor 132 new drugs* in Japan over the last 7 years

Number of Drugs Approved in Japan

3940

43

1820

221615

2009

2010

2011

2012

2013

37

33

27

2120

2007

2008

Japan Total

28

PhRMA and EFPIA Companies

Source: PMDA web site

*New drugs refer to New Molecular Entities only approved between April 2007 and February 2014 (Other approvals, such as new indications, are excluded).
Besides the approvals obtained by PhRMA and EFPIA companies, other approvals include products licensed from global companies

17What are Therapeutic Areas?
Therapeutic areas are general categories of illnesses, such as the examples below

Therapeutic Area

ExampleAllergy

Allergic rhinitis, Asthma, Atopic dermatitis, etc.

Infectious disease

Influenza, Hepatitis C/B, Herpes, etc.

Hematology

Anemia, Hemophilia, etc.

Respiratory disease

COPD, Bronchiectasis, etc.

Oncology

Gastric cancer, Lung cancer, Breast cancer, etc.

Cardiology

Heart failure, Brain ischemia, Hypertension, etc.

Gastroenterology

Gastric ulcer, Reflux esophagitis, etc.

CNS

Alzheimer’s disease, Depression, Epilepsy, etc.

Urology

Nocturia, Lupus nephritis, etc.

Dermatology

Acne, etc.

Ophthalmology

Age-related macular degeneration, Uveitis, etc.

Immunology

Rheumatoid arthritis, Crohn’s disease, Psoriasis, etc.

Musculoskeletal disease

Osteoporosis, Lower back pain, etc.

Endocrine metabolic disease

Diabetes mellitus, Hypercholesterolemia, Hyperphosphatemia, etc.

Rare disease

Gaucher disease, Myelofibrosis, Behcet’s disease, etc.

18

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