Download RUZICH, Richard T.

DUE DILIGENCE AND RISK
MANAGEMENT
DO NOT FORGET THE SCIENTIFIC
DATA—TRUST, BUT VERIFY
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LEGAL, FINANCIAL, BUT
WHAT ABOUT SCIENCE?
•In the end, you are buying the data
•Without understanding it, you don’t know the
risks/rewards
•Scientific due diligence must be performed
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THE “HOW TO’s”
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Assume information is not being volunteered
Remain unbiased/accept nothing
Overcome biases of “opinions”
Leave nothing to chance
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WHERE TO LOOK
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MANUFACTURING
PRECLINICAL TO CLINICAL
REGULATORY
MARKETING ISSUES
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MANUFACTURING
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CMC
DMF
USP
EP
All of these resources provide insight on
intellectual property issues, trade secrets, and
cost of goods.
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REGULATORY
• International Conference on Harmonisation
(ICH)
• Europe v. USA
• What is an NCE?
• GMP compliant?
• SOPs
• CTX and IND
• MRP
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PRECLINICAL
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Primary Pharmacology
Secondary Pharmacology
Safety Pharmacology
PK-PD/ADME
“S” versus Bell Shaped
Non-Clinical Safety—assess for completeness,
quality.
• Toxicology—NCE, NBE
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CLINICAL
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What are the protocols?
GCP yet again
Independent QC/QA to ensure quality
Permission to conduct studies
Country or continent specific differences in
acceptability
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Oncology
• Protocols, CRFs, and results of all completed
and ongoing clinical trials available?
• Adequately designed to achieve intended goal?
• Audited? GCP, again!
• IRB approval for the studies?
• Enough detail and care taken to review
IND/IMPD documentation and related updates?
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Phase I
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Assess the drug’s safety profile
Drug limiting toxicities
Maximum tolerated dose
Minimum effective dose/ “The Optimal Dose”
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Phase II
• Assess the drug’s efficacy
• (1) Proof of concept demonstrating biological
activity and (2) Clinical proof
• Historical comparisons may not suffice
• Usually reveals practical issues, e.g., handling
drug in routine hospital- or out-patient situations
• Orphan Drug potential indications may become
lead indication
• Efficacy been achieved? Risks?
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Phase III
• Selection of the primary and secondary
endpoints and comparator treatment
• Was the gold standard RCCT used?
• Is the delta clinically significant? Relevant?
Convincing?
• Regulatory requirements fulfilled? Previous
agreements with agencies met?
• New compound still favored? Other compounds
on the horizon?
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Marketing
• Trademark and trade dress usage
• Objections made by any agencies for reasons of
consumer protection?
• INN issues?
• How does the US, Europe, EU differ with social
program reimbursements?
• Pharmacoeconomic studies been performed?
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DISCUSSION/QUESTIONS?
• Richard T. Ruzich, J.D., LL.M.
• [email protected]
• Contact: http://www.taftlaw.com/attorneys/803richard-t-ruzich
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