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

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

Background on IPPC

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:

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.

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.

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

Background on Genomics

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

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

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.

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

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.

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.

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.

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

Challenges of ‘Information Based Medicine’

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

Questions Stan Crosley - Crosley_Stanley_W@lilly.com Dean Forbes - dean.forbes@spcorp.com

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