Transcript terry
Clinical Trials Registration Sharon F. Terry, JAM President and CEO, Genetic Alliance, Inc. Founding President, Genetic Alliance BioBank Consumer Advocate Perspective PXE International, Inc. Non-profit Disease Research Foundation Washington, DC www.pxe.org Coalition of 600 Advocacy Organizations Increase the capacity of advocacy organizations so they can achieve their missions Washington, DC www.geneticalliance.org Building Translational Research Capabilities for Genetic Diseases Sharon F. Terry, Founding President What Matters? Improved Health Outcome Outcome Improved Health What Matters? Bench Bench Bedside Bedside Practice How Do we Get to Personalized Medicine Trials? Theranostics Mechanism of Action Targeted Agents Rational Trial Design Pt Selection Consumer Understanding of the Drug R&D Enterprise Development Stage Drug Discovery Gene or Protein Research Drug Targets Functional Genomics Genomics Positional Cloning Technology Preclinical Parallel Sequencing Cell Assays Proteomics Clinical Approval IV I II III Small Molecules Combinatorial Chemistry Drug Leads Screening Drug Tests Animal Models Cellular Assays Structural 2-D Gel, Model Organism, Mass Spec. Drug Design Gene Knock-outs Human Trials Drug Pharmacogenomics Genotyping, Phenotyping, SNPs Markers Differential Display, Expression Patterns, Reporter Gene Technologies Bioinformatics Molecular Informatics Chip Technologies, DNA Chips, Protein Chips, Microarrays High-Throughput and Ultra-High Throughput Screening TIME: Variable 10–20 Year Duration 10 yrs? The Vertical Pharma Industry Patient management Clinical development Preclinical development Drug screening Target discovery Pharma 1 Pharma 2 Pharma 3 Pharma 4 The New Pharma Industry Evolving Levels of Consumer Scrutiny Patient Management HealthOrgs Clinical Development Contract Research Orgs Preclinical Development Pharmacogenomics Drug Screening High-throughput screening Target Discovery Genomics Industry Clinical Trials Registration w/ Sufficient Information to Enable… 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 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