Development of rare disease
treatment drugs requires
collaboration among patients,industry, academia andgovernment
Feb 5th 2012
Jin Shiomura
Nobelpharma Co. Ltd., Japan


Shareholder[ yes/no ] Yes[ If yes, please describe ] Major shareholder of Hisanaga & Company, who is a major shareholder of Nobelpharma.
Very minor shareholder of several pharmaceutical companies.
Grant / Research support[ yes/no ] Yes[ If yes, please describe ] The Japanese Government
Consultant[ yes/no ] No
[ If yes, please describe ]Employee[ yes/no ] No
[ If yes, please describe ]Paid Instructor[ yes/no ] Yes.
[ If yes, please describe ] A visiting lecturer of Nihon University, School of Pharmacy.
Speakers Bureau[ yes/no ] No
[ If yes, please describe ]( ) None declared

7th ICORD Feb 5th 20122

Nobelpharma - Pipeline & Collaboration Compound

Indication

StatusPatients

Big pharma

Academia

Government

Lunabell®

Dysmenorrhea
associated endometriosis

launched○Janssen△×Nobelzin ®

Wilson’s Diseaselaunched○Teva○○

Nobelbar ®

Neonatal seizures
Status epilepticus

launched×

In house

○

○

Talc

Prevention of malignant
pieural effusion recurrencePre-regist

×

Novatech○○

Fosphenytoin

Status epilepticus
Prevention of convulsive
seizure after surgery

launched△

Pfizer

△

×

5-ALA

Diagnosis of malignant
gliomaPre-regist△

SBI &Cosmo○○

Carmustine wafer

GliomaNDA△

MGI(Eisai)△○

N-acetylneuraminic acid

Distal myopathy

P1/2

○

In house○○

Streptozocin

Gastroenteropancreatic
neuroendocrine tumor

P1/2(final)△

Pfizer/Keocyte○○

Caffeine citrate

Apnesia of prematurity

P3×Boehringer
Ingelheim△○○：with strong support △：with some support ○：without support
7th ICORD Feb 5th 20123

Excellent Example of Collaboration - MILES trial

Lymphangioleiomyomatosis (LAM)
Rare, metastatic neoplasm among women in which the lung becomes infiltrated with smooth muscle cells
89 Patients; 12M (DBT)+12M(followed observation)
Sirolimus stabilized lung function

LAM Foundation

1) Organizing and educating patients
2) Funding the basic and clinical research3) Support patient recruitment and trial logistics

Effective partnership between scientific researchers and the patient community⇒ Progress in better understanding of rare diseases and possible treatments.

MILES Investigatorsa network of 10 US sites and 3 international sites in Canada and JapanNIH Rare Diseases Clinical Research Network・・・Infrastructure (network and data center)
The Cincinnati Children's Translational Research Trials Office
・The contract research organization
・Navigate the complex waters of international trial regulations
・Drug import and export,blinded dose adjustments and over 2000 adverse events
Source:Newsletter of the NIH Rare Diseases Clinical Research Network4
7th ICORD Feb 5th 2012Timeline of the MILES Trial
2004

FDA
IND
ProtocolDrafting

2005

2006

DSMB
Revisions

2007

2008

2009

Other sites
MUSC open
open

Cincinnati siteopen

2010

2011

Last
Visit
IntermAnalysis

Enrollement
ClosesMILES
PublishedMILES
FinalAnalysis

DSMB:Data and safety monitoring board

10 US sites & 3 International sites (Japan & Canada)
Of 111 patients consented, 28 were in 2 sites of Japan
Source:Newsletter of the NIH Rare Diseases Clinical Research Network

7th ICORD Feb 5th 20125

Funding sources for the MILES Trial

US$NCRR/NIH RLDC

3,000,000 CIHR(Canada)

200,000

Japanese Ministry of Health2,000,000 Tuberous Sclerosis Assoc.

200,000

FDA

1,200,000 Cincinnati Children’s

100,000

Wyeth/Pfizer(inc. drug)

1,200,000 Adler Foundation

50,000

The LAM Foundation

500,000

NCRR:National Center for Research Resources

RLDC:Rare Lung Diseases Consortium

Source:Newsletter of the NIH Rare Diseases Clinical Research Network

7th ICORD Feb 5th 20126

Japanese clinical research budget is about
2 billion yen / year
US:Several hundred billion
Japan and the U.S. budget for life sciences

10 Bil yen

to one trillion yen or more?

350
300
250
200

2 tril yen

Japan:2 bil yen

150
100

290bil yen

500USJapanApplic at io n r e se ar c hBasic r e se ar c h / Ot h e r

Ministry of Economy, Trade and Industry July 08, 2007
Created from first public-private dialogue venture working group
7th ICORD Feb 5th 2012

7Conclusion: Intractable drug development,in cooperation with stakeholders
Public understanding

Patients

Cooperative clinical trial

Cooperative clinical trial

Information

ProfitAcademia

Industry
Joint development

Research fund

GrantGoverment
7th ICORD Feb 5th 20128