1. Privacy Symposium / HIPAA Summit
Genomics and Privacy
Privacy Symposium / HIPAA Summit
August 20, 2008
Stan Crosley (Eli Lilly)
Dean Forbes (Schering-Plough)

2. Discussion Outline Brief Background on IPPC Discussion on Genomics
Questions & Answers

3. Background on IPPC

4. Members
The IPPC is an association of companies that face worldwide responsibility for the protection of personal health information and other types of personal data. Members of the IPPC include:

5. Mission
The IPPC works to promote responsible privacy and data protection practices by the research-based, global pharmaceutical industry.
Maintaining data confidentiality and subject privacy are essential to clinical research, pharmacovigilance, and other activities of the pharmaceutical industry.
The IPPC seeks to increase awareness of privacy and data protection issues and to engage government in a dialogue about the need for data to support cutting edge biomedical research and other public health activities.
The IPPC pursues opportunities to collaborate with government and other stakeholders to develop data protection practices that enhance data subject privacy.

6. Goals The IPPC goals are to:
Engage government and stakeholders in the biomedical research and healthcare communities in a constructive dialogue on significant issues of privacy and data protection.
Serve as a resource for sound analyses of privacy and data protection requirements and compliance tools tailored to the pharmaceutical industry.
Serve as a forum for industry dialogue and promote responsible privacy and data protection practices.
Promote consistent privacy and data protection standards that can be achieved on a worldwide basis.
Remain on the leading edge of privacy and data protection.

7. Scope of Activities
The IPPC advances understanding of existing and emerging data protection and security rules in Europe, the US, and other key countries. The Consortium engages regulators and policymakers in the following areas:
Biomedical research
Pharmacovigilance
Sales and marketing
Market research
Human resources programs
Other corporate programs

8. Background on Genomics

9. Blockbuster Business Model
Future success of a pharmaceutical company depends heavily on the number and quality of drugs in the pipeline
The industry has traditionally relied primarily on the “blockbuster model”, where a few key drugs make up the majority of the company’s revenue
Challenges presented by the blockbuster model
Industry has fully exploited “low hanging fruit”
Expiration of patent terms
Pricing/reimbursement pressures

10. Personalized Medicine Business Model
Utilizes pharmacogenomics, which benefits from the recent advances of genomics/proteomics technology
Potentially, reduced development costs; shorter development time from discovery to launch
Potentially, smaller clinical trials required to prove efficacy in target population (depends on regulatory requirements)
Greater probability of clinical compounds reaching market
Better safety profile
Treat specific populations based on biomarkers or molecular diagnostics/imaging results
Should not require blockbuster-sized sales to generate attractive returns on investment

11. Drug Discovery and Development
Target Selection, Assay Development & Highthroughput Screening
Lead Optimization
Candidate Selection Process
Exploratory Development
Safety / Feasibility
Tolerability / Efficacy
Registration
Market Introduction
DISCOVERY
DEVELOPMENT
LAUNCH
Lead Selection
CSP Initiation
Selection Proof of Concept
Proof of Concept Outcome
Full Development Point
Submission Decision Point
Phases II & III
Phase IV
Phase I
Pre-clinical Research
20 to 100 health subjects to test for safety.
6-12 mos.
100 to 1,000 patients to test for safety and efficacy in a disease state.
12-24 mos.
1,000 to 10,000 patients to test for safety, efficacy, dosing and comparative studies.
18-24 mos.
Medical safety officers record and assess risk from reported adverse events.
Further studies continue, including surveys, sampling and testing.
Targets identified for drug discovery for specific disease states.
Validation of drug discovery target
Possible molecules that have some properties of a drug are identified.
Initial lead is modified to produce drug candidate.
In vivo (animal) and in vitro pharmacology, metabolism, and toxicology studies are conducted to evaluate safety of drug candidate
IND application filed
NDA/BLA submitted
10-12 mos.

12. Protections Built into Biomedical Research
Informed Consent
IRB Examination of Research
Data Security
Biomedical Research

13. Pre-Clinical Testing DISCOVERY DEVELOPMENT LAUNCH
Target Selection, Assay Development & Highthroughput Screening
Lead Optimization
Candidate Selection Process
Exploratory Development
Safety / Feasibility
Tolerability / Efficacy
Registration
Market Introduction
DISCOVERY
DEVELOPMENT
LAUNCH
Lead Selection
CSP Initiation
Selection Proof of Concept
Proof of Concept Outcome
Full Development Point
Submission Decision Point
Phases II & III
Phase IV
Phase I
Pre-clinical Research
20 to 100 health subjects to test for safety.
6-12 mos.
100 to 1,000 patients to test for safety and efficacy in a disease state.
12-24 mos.
1,000 to 10,000 patients to test for safety, efficacy, dosing and comparative studies.
18-24 mos.
Medical safety officers record and assess risk from reported adverse events.
Further studies continue, including surveys, sampling and testing.
Targets identified for drug discovery for specific disease states.
Validation of drug discovery target
Possible molecules that have some properties of a drug are identified.
Initial lead is modified to produce drug candidate.
In vivo (animal) and in vitro pharmacology, metabolism, and toxicology studies are conducted to evaluate safety of drug candidate
IND application filed
NDA/BLA submitted
10-12 mos.

14. Phase I DISCOVERY DEVELOPMENT LAUNCH
Target Selection, Assay Development & Highthroughput Screening
Lead Optimization
Candidate Selection Process
Exploratory Development
Safety / Feasibility
Tolerability / Efficacy
Registration
Market Introduction
DISCOVERY
DEVELOPMENT
LAUNCH
Lead Selection
CSP Initiation
Selection Proof of Concept
Proof of Concept Outcome
Full Development Point
Submission Decision Point
Phases II & III
Phase IV
Phase I
Pre-clinical Research
20 to 100 health subjects to test for safety.
6-12 mos.
100 to 1,000 patients to test for safety and efficacy in a disease state.
12-24 mos.
1,000 to 10,000 patients to test for safety, efficacy, dosing and comparative studies.
18-24 mos.
Medical safety officers record and assess risk from reported adverse events.
Further studies continue, including surveys, sampling and testing.
Targets identified for drug discovery for specific disease states.
Validation of drug discovery target
Possible molecules that have some properties of a drug are identified.
Initial lead is modified to produce drug candidate.
In vivo (animal) and in vitro pharmacology, metabolism, and toxicology studies are conducted to evaluate safety of drug candidate
IND application filed
NDA/BLA submitted
10-12 mos.

15. Phase II/III DISCOVERY DEVELOPMENT LAUNCH
Target Selection, Assay Development & Highthroughput Screening
Lead Optimization
Candidate Selection Process
Exploratory Development
Safety / Feasibility
Tolerability / Efficacy
Registration
Market Introduction
DISCOVERY
DEVELOPMENT
LAUNCH
Lead Selection
CSP Initiation
Selection Proof of Concept
Proof of Concept Outcome
Full Development Point
Submission Decision Point
Phases II & III
Phase IV
Phase I
Pre-clinical Research
20 to 100 health subjects to test for safety.
6-12 mos.
100 to 1,000 patients to test for safety and efficacy in a disease state.
12-24 mos.
1,000 to 10,000 patients to test for safety, efficacy, dosing and comparative studies.
18-24 mos.
Medical safety officers record and assess risk from reported adverse events.
Further studies continue, including surveys, sampling and testing.
Targets identified for drug discovery for specific disease states.
Validation of drug discovery target
Possible molecules that have some properties of a drug are identified.
Initial lead is modified to produce drug candidate.
In vivo (animal) and in vitro pharmacology, metabolism, and toxicology studies are conducted to evaluate safety of drug candidate
IND application filed
NDA/BLA submitted
10-12 mos.

16. Post-Approval
Need to identify patient populations for whom drug is indicated (and ensure that drug not taken by populations for whom contraindicated)
Development of companion diagnostics
Pharmacovigilance
Phase IV studies

17. Challenges of ‘Information Based Medicine’

18. Key Privacy Issues What’s ‘identifiable’?
Current law
Developments in technology
Reference databases
Notice, choice, access & amendment, confidentiality, anonymization
Secondary research, biobanking
Specific vs. general consent
Risks of unauthorized disclosure
Potential for discrimination in employment and insurance (elsewhere?)
Psychological impact - stigmatization

19. Questions Stan Crosley - Crosley_Stanley_W@lilly.com
Dean Forbes -

20. Questions on IPPC? Please contact: Dede.Godstrey@dbr.com