Post on 18-Jan-2018
description
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