1. Consumer Advocate Perspective Clinical Trials Registration Sharon F. Terry, JAM Sharon F. Terry, JAM President and CEO, Genetic Alliance, Inc. Founding President, Genetic Alliance BioBank

2. PXE International, Inc. Non-profit Disease Research Foundation Washington, DC www.pxe.org

3. Coalition of 600 Advocacy Organizations Increase the capacity of advocacy organizations so they can achieve their missions Washington, DC www.geneticalliance.org

4. Building Translational Research Capabilities for Genetic Diseases Sharon F. Terry, Founding President

5. Improved Health Outcome What Matters? Improved Health Outcome

6. PracticeBenchBedside BenchBedside What Matters?

7. How Do we Get to Personalized Medicine Trials? Theranostics Mechanism of Action Targeted Agents Rational Trial Design Pt Selection

8. Consumer Understanding of the Drug R & D Enterprise Approval Clinical Development Stage Technology Drug Discovery Preclinical Bioinformatics Positional Cloning Parallel Sequencing Differential Display, Expression Patterns, Reporter Gene Technologies 2-D Gel, Mass Spec. Cellular Assays Model Organism, Gene Knock-outs Chip Technologies, DNA Chips, Protein Chips, Microarrays Small Molecules Animal Models Functional Genomics Proteomics Cell Assays Drug Targets Gene or Protein Drug Tests Genomics Combinatorial Chemistry Screening Drug Leads IIIIII IV Pharmacogenomics Human Trials Genotyping, Phenotyping, SNPs Markers Drug High-Throughput and Ultra-High Throughput Screening Structural Drug Design Molecular Informatics Research 10–20 Year Duration Variable 10 yrs? TIME:

9. The Vertical Pharma Industry Pharma 1 Pharma 2 Pharma 3 Pharma 4 Patient management Clinical development Preclinical development Drug screening Target discovery

10. The New Pharma Industry Genomics Industry High-throughput screening Pharmacogenomics Contract Research Orgs HealthOrgs Patient Management Clinical Development Preclinical Development Drug Screening Target Discovery Evolving Levels of Consumer Scrutiny

11. Clinical Trials Registration  Transparency of Opportunities & Options – Not Limited by Clinician Referral, or Commercial Interest  A New Level of Consumerism for Clinical Trials – Designs, Clinical End-points, Mechanism of Action, Delivery, Quality, & Data Management – Safety, efficacy, and surveillance – Risk vs. Benefit – Access to Information for Informed Choice  Redefine the patient’s relationship with the “System”  The ability to do this: – Make a shared research, clinical, or treatment decision – As a truly informed volunteer – For medications & risks I choose to take – I can have confidence in the process w/ Sufficient Information to Enable…

12. NEJM 351;12 Sept. 16, 2004 The Ethics of Ensuring Public Confidence in Clinical Trials Should be The Motivation for Disclosure. NOT Publication Obstacles or Federal Funding Restrictions

13. Clinical Trials Registration  “Publication” of Negative Data or Failed Endpoints is very important for improving clinical practice  MY RISK weighed against unique circumstances of MY BENEFIT should be the measure for trials  “My Health depends on a new level of engagement and understanding of my healthcare options.”  The ‘system’ will not ‘take care or protect me.’  The GOAL is EFFECTIVE TREATMENTS – The Goal of Research is Not Safe Products – The Method by which to Reach Evidence-based Medicine – Shed the Past of: Repeat Trial & Error, Redundancy, Similar “Me – To” Products, & Minimum Standards performance – A New social engagement in altruism to advance medicine and health outcomes. Increased trial participation.

14. Clinical Trials Registration  IDEALLY: There Should be a Centralized Viewing Portal for All Clinical Trials that Meets a Required Minimum Data-Set Standard.  That Would Permit Enhanced Reporting by Sponsors & Trialists  That Would Enable Trial Sorting & Comparison  Dynamic Searchable Data Architecture  That Would Enable Opt-In Patient Registration & Re-Contact  That it would be an Active Resource for Accrual  Structured for FDA Equivalence, Compliance, Monitoring, Reporting, and Surveillance  Managed Data Center for Trial Progress, Recruiting Status, and Event Reporting