Consumer Advocate Consumer Advocate PerspectivePerspective

ClinicalClinical TrialsTrials RegistrationRegistration

Sharon F. Terry, Sharon F. Terry, JAMJAMPresident and CEO, Genetic Alliance, Inc.

Founding President, Genetic Alliance BioBank

PXE International, Inc.Non-profit Disease Research Foundation

Washington, DC

www.pxe.org

Coalition of 600 Advocacy Coalition of 600 Advocacy OrganizationsOrganizations

Increase the capacity of advocacy organizations Increase the capacity of advocacy organizations so they can achieve their missionsso they can achieve their missions

Washington, DCWashington, DC

www.geneticalliance.orgwww.geneticalliance.org

Building Translational Building Translational Research Capabilities for Research Capabilities for

Genetic DiseasesGenetic Diseases

Sharon F. Terry, Founding Sharon F. Terry, Founding PresidentPresident

Improved Health Outcome

What Matters?Improved Health

Outcome

PracticeBench Bedside

Bench Bedside

What Matters?

How Do we Get toHow Do we Get toPersonalized MedicinePersonalized Medicine Trials? Trials?

TheranosticsTheranostics

Mechanism Mechanism of Actionof Action

Targeted Targeted AgentsAgents

Rational Trial Rational Trial DesignDesign

Pt SelectionPt Selection

Consumer Understanding of the Drug R&D Enterprise

ApprovalClinicalDevelopmentStage

Technology

Drug DiscoveryPreclinical

Bioinformatics

Positional Cloning

Parallel Sequencing

Differential Display, Expression Patterns, Reporter Gene Technologies

2-D Gel,Mass Spec.

Cellular AssaysModel Organism,Gene Knock-outs

Chip Technologies, DNA Chips, Protein Chips, Microarrays

SmallMolecules

Animal Models

FunctionalGenomics Proteomics

CellAssays

DrugTargets

Gene orProtein

DrugTests

Genomics Combinatorial Chemistry Screening

DrugLeads

I IIIII IV

Pharmacogenomics

HumanTrials

Genotyping,Phenotyping,SNPs Markers

Drug

High-Throughput and Ultra-High Throughput Screening

StructuralDrug Design

Molecular Informatics

Research

10–20 Year DurationVariable 10 yrs?TIME:

The Vertical Pharma Industry

Pharma 1 Pharma 2 Pharma 3 Pharma 4

Patient management

Clinical development

Preclinicaldevelopment

Drug screening

Target discovery

The New Pharma Industry

Genomics Industry

High-throughput screening

Pharmacogenomics

Contract Research Orgs

HealthOrgsPatient Management

ClinicalDevelopment

PreclinicalDevelopment

DrugScreening

Target Discovery

Evolving Levels of Consumer Scrutiny

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…

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

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.

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