managing clinical trials outside of the us john potthoff, phd president & ceo
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MANAGING CLINICAL TRIALS OUTSIDE OF THE US
John Potthoff, PhD
President & CEO
Theorem Clinical Research
Global, full-service CRO• 1000+ employees
Planning and execution of clinical trials and regulatory applications
Focused business units:• Medical Device and Diagnostics• BioPharmaceutical Development• Clinical Analytics
Coverage of 44 countries
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Why conduct trials outside of the US?
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Gain registration in target company
Support a global development effort
Complex or rare diseases; incident rates may not be high enough in the US to enroll a trial
Timelines for registration may be faster outside of the US to allow a company to begin sales while completing registration in the US
Costs may be reduced for registration outside of the US
Competitive landscape may be advantageous outside the US
Where are trials conducted?
Page 4Data as of 02 December 2012
Actively Recruiting Studies
Non-U.S. Only (49%)
U.S. Only (45%)
Both U.S. & Non-U.S. (7%)
LocationNumber of Recruiting Studies and
Percentage of Total
Non-U.S. Only 14,193 (49%)
U.S. Only 13,010 (45%)
Both U.S. & Non-U.S. 1,898 (7%)
Total 29,101
(Data as of December 02, 2012)
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Focus on Asia Pacific
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Aging Population
Increasing Life Expectancy
Increasing Incidence of
Major Disease
Increasing Health
Consciousness
Higher Disposable
Income
Over 30% of new expenditures on healthcare worldwide are attributable to Asia.
Why Asia for Clinical trials?
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Money
• Cost of clinical trials in Asia is much lower than in US/EU (30-50%)
Time
• Start-up timelines have become more favorable (other than China) and are 4-6 months covering all applications, site selection, contract negotiations, etc.
Reso
urces
• Population exceeds 4 billion and is genetically diverse
• High incidence of some indications: Diabetes Mellitus (> 11%), Hepatitis B, etc.
• Ability to recruit patient naïve and advanced case patients
Season
ality
• Southeast Asia: Advantage for seasonal indications (e.g. influenza)
Regulatory Authorities in Asia Pacific
Country Authority
China SFDA
Hong Kong DoH
Australia TGA
India DCGI
Japan MHLW
Korea KFDA
Malaysia MoH
New Zealand Medsafe
Philippines BFAD
Singapore HSA & AVA
Taiwan DoH
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Asia Regulatory and Start-up Timelines
Regulatory Regulatory
Ethics Ethics Regulatory
Weeks Prep. w1 w2 w3 w4 w5 w6 w7 w8 w9 w10 w11 w12 w13 w14 w15 w16 w17 w18 w19 w20 w21 w22 w23 w24 w25+ +
New Zealand 4 w 7 weeks
8-12 weeks 1-2 weeks
Contract sign off w ithin 1-2 weeks after EC approval
2-3 weeks
Australia 12-16 weeks 2 weeks
4 w Contract sign off w ithin 1-2 weeks after EC approval 1-2 weeks 2 weeks
China 8 w 8-10 months "+" 4-8weeks
1-2 weeks
2 weeks
Malaysia 4 w 4-8 weeks
6-8 Weeks 1-2 weeks
Contract sign off w ithin 1-2 weeks after EC approval
4-8 weeks for issue of import license 2 weeks
Philippines 4 w 8-12 weeks
8-12 Weeks 1-2 weeks
Contract sign off w ithin 1-2 weeks after EC approval
6-8 weeks
South Korea 4 w 8-12 weeks
2-8 weeks (Some ECs require CA approval letter) 1-2 weeks Contract sign off w ithin 1-2 weeks after EC approval
8-12 weeks for issue of import license 1 week
Taiwan 4 w 8-12 weeks
8-12 weeks 1-2 weeks
Contract sign off w ithin 1-2 weeks after EC approval
8-12 weeks for issue of import license 2 weeks
Ethics
Ethics followed by Regulatory
Regulatory followed by Ethics
in parallel
Regulatory and Ethics
Import license application and customs clearance
First SIV
Submission preparation time
KEY ITEMS:
Site Contract negotiation and signature
*Note: China not to scale
Complicated and risk of
delays
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Data Collection in Asia Pacific
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Data Collection
Most Trials conducted in accordance with global regulatory
guidelines*
Virtually all sites conduct clinical
research in English
Virtually all sites can work in electronic
data capture (EDC) systems
Most of the leading EDC tools now allow for multi-language
data processing, allowing sites to add comments in their
local language**
*Some local trials conducted according to local minimal guidelines
**Requires ability of CRO to process data in local
languages
Focus on China and India
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*1.3 billion people*537 active trials in oncology (cancer)*102 active medical device trials
*1.2 billion people*124 active trials in oncology (cancer)*27 active medical device trials
China
India
Drug Controller General of India (DCGI)
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Currently politicizing on policy issues relating to compensation for injury to or death of a participant in a
clinical trial
Currently left to the discretion of the Ethics Committee
Indication under investigation/cause of injury or death may not be considered
Example: Oncology trials often have survival as the outcome measure
Conclusions
Conducting clinical trials outside of the US is a necessary part of reaching a global audience
Conducting clinical trials outside of the US can be highly complex without local knowledge and experience
Several advantages to conducting trials outside of US:• Registration in multiple regions• Access to specific patient populations• Strategy to seek registration in cost advantaged
locations
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