Specialized Workshop M2 - Monday June 19, 2023: 7am to 5:30pm - Discussion Topic List

"Biomarkers & Companion Diagnostics - Latest Developments, Challenges, and Solutions"

NEW Case Studies on Biomarker Strategies for LBA & Mass Spec Method Development; Low Abundant Proteins/Exosomes Discovery Biomarkers; Implementing Clinical Biomarkers as Primary Endpoints (CLIA vs GCLP); Challenges with Regulatory BAV Guidance; Biomarker Validation as CDx and CLIA Approved Assays; Development in Vaccine Biomarkers Immunoassay, Interferences, Dilutional Linearity, RSM; Industry/Regulators Interactive Discussions on BAV, IVDR and CDx and Recent Controversial Issues

Session 1: Development/Validation Strategies for Biomarkers & IVD/CDx, CLIA Approved Assay and Regulatory Requirements

  • Topic 1:

    When a CLIA Approved Assay is required in Clinical Biomarker Development: Current Regulatory Requirements from CMS, FDA, CDC, Applications of LDT vs IVD/CDx, Understanding the Planned Use of Biomarkers Assay, the Medical Decisions and need for CLIA in Clinical Trials 
    Dr. Sean Downing, Senior Director, Takeda

  • Topic 2:

    Using Diagnostic/IVD Assays for Biomarker Support to Clinical Studies: CLIA vs GCLP, Validation (BAV) Strategy, Including Requirements to Use of Clinical Samples for Validation Experiments, Validation Parameters for Soluble Biomarker, Biomarker Assays for Patient Selection, Patient Stratification and potential Companion Diagnostics
    Dr. Kai Wang, Scientific Director, GSK

  • Topic 3:

    Requirements for Validating Biomarker Assay as CDx: Point to Consider in CDx Assay Development and When to start turning a Biomarker Assay into a CDx, Biomarker used to Inform Decisions about Patient Treatment and necessary for Safe & Effective use of a Drug 
    Ms. Jennifer Jones, Director, BMS

Session 2: EU IVDR 2022 Implementation and impact for the Global Biomarker Community: How to Comply with these NEW Regulations

  • Topic 4:

    EU IVDR 2022 and impact for the Biomarker Community: Changes/Updates, Assessment and Implementation, Risk and Mitigation, Requirements for IVDs Performance Studies, Review of the Biomarker Assays used to support Clinical Trials and Assessment whether they meet EU Regulatory Criteria, Need for Lab Developed Tests (LDT) to meet ICH M10 BMV and Related IVD Regulations 
    Dr. Luying Pan, Senior Director, Takeda

  • Topic 5:

    The In Vitro Diagnostic Regulation into force in the EU since May 26, 2022 (EU IVDR 2022): Review Process and Potential Impact on Clinical Programs, Application to all Tests used for Patient Management, Inclusion/Exclusion Criteria in Clinical Trials, Evolving Clinical Biomarker Landscape in the EU
    Dr. Gerard Sanderink, Global Head Biomarkers and Clinical Bioanalyses, Sanofi-Aventis R&D

  • Topic 6:

    Severe Impact of 2022 EU IVDR on Biomarker Development as Endpoints in Clinical Trials: Direct Experience & Lesson Learned for use of US-based LDT in EU, Biomarker Assays Strategies for Complex Performance Study Application and Recommendations
    Dr. Johannes Stanta, Global Director Molecular and Cell Biology, Celerion

Session 3: Biomarkers Development/Validation for Vaccine Study Endpoints: Focus on LBA Method Development Challenges, BAV and Sample Collection

  • Topic 7:

    How to cope with Interferences in Biomarker Immunoassays Development/Validation used for Vaccine Study Endpoints: Evaluation of Interferences and how it applies to the Characterization of Assay Linearity & Dilutability, Selection of the Most Appropriate Diluent, Considerations about the Statistical Methods used to Assess Assay Linearity  
    Mr. Francis Dessy, Clinical Assay senior Advisor, GSK

  • Topic 8:

    An in-depth Assessment of Dilutional Linearity during Biomarkers Immunoassays Validation (BAV) used for Vaccine Study Endpoints : Impact on Assay Performance and Method Development Strategies
    Dr. Ingrid Scully, Executive Director, Pfizer

  • Topic 9:

    Use of Reference Standards in Clinical Biomarker Assays supporting Vaccine Development: Advantages and Challenges, Lack of International Reference Standards, Use Human Sera as Reference Standard from Vaccinated Individuals, Development & Validation of an Assay with Improved Precision Measuring HBGA-blocking Antibody Elicited in Humans  
    Dr. Shannon Harris, VP, Clinical Assays, HilleVax

  • Topic 10:

    Challenges in Implementation of Home Sample Collection by Capillary Blood Draw Device for Antibody Testing and for Monitoring duration of anti-SARSCoV2 Antibodies Post-vaccination: Sample Collection Challenge and Application of High-throughput Multiplexed Ligand-binding Assay  
    Dr. Victor Hou, Senior Director, Moderna

Session 4: Fit for Purpose Biomarker Assay Validation (FFP BAV) Challenges for LBA & Mass Spec: Exosomes, Low Abundant Protein, Peptides/Small Molecule Biomarkers

  • Topic 11:

    Unique Challenges in Small Molecule Biomarkers Development/Validation: Unique Considerations in Assay Design, Similarities to BMV ICH M10 Section 7.1 Endogenous Compounds, Method Development, BAV Regulatory Expectations, Overcoming Interference, Stability Issues, Sample/Matrix Collection, Surrogate Analyte vs Surrogate Matrix
    Dr. John Meissen, Associate Director, AstraZeneca

  • Topic 12:

    Strategies for Developing/Validating Biomarker Peptide Panels: Approaches to Stabilization due to quick Degradation, Achieving Required Sensitivity, Challenges in the Quantitation of Neuropeptides in various Human Matrices, Best Platforms for Bioanalysis, Accurate/precise methods for large panel of neuropeptides
    Dr. Matthew Szapacs, Scientific Director - Precision Medicine Neuroscie, Abbvie

  • Topic 13:

    Challenges in Complex Neurodegenerative Diseases Biomarkers Development: Strategies for Assays Development and Fit for Purpose Validation (FFP BAV) by Mass Spec: Selecting the Right Assay Conditions, Surrogate Matrix, Validation Strategy, Overcoming selectivity issues due to Multiple Proteoforms
    Dr. Khader Awwad, Laboratory Head Bioanalysis, AbbVie

  • Topic 14:

    Development of Quantitative Biomarker Assays for Low Abundant Proteins / Exosomes in Bio Fluids: Mass Spec & LBA Approaches, Challenges and Solution, Impact on the Biomarker Strategy, State-of-the-art methods to quantitate Protein-based Biomarkers, Approaches for Target Protein Enrichment, Conventional and Novel technologies for Exosome Preparation, Isolation, Characterization, and Best Assay Format
    Dr. Ming Li, Associate Director, Stoke Therapeutics

Session 5: 2023 White Paper in Bioanalysis

  • 2023 White Paper on Biomarkers & Companion Diagnostics - Latest Developments, Challenges, and Solutions

    Consensus & Conclusions on Biomarkers & Companion Diagnostics - Latest Developments, Challenges, and Solutions for 2023 White Paper

Session Finale: ASK THE REGULATORS! Interactive Panel Discussion with All the Regulators

  • Regulatory Feedbacks on Submitted Studies and Inspection/Audit Outcomes on Biomarkers & CDx/BAV

    Regulatory Panelists:

    • Dr. Mary Thanh Hai (US FDA CDER)
    • Dr. Olga Kholmanskikh (Belgium FAMHP)
    • Ms. Tamara Pinkney (US FDA CDRH)
    • Dr. Joshua Xu (US FDA NCTR)
    • Dr. Yow-Ming Wang (US FDA CDER)
    • Mr. Abbas Bandukwala (US FDA CDER)
    • Dr. Xiulian Du (US FDA CDER)
    • Dr. Leslie Wagner (US FDA CBER)
    • Dr. Sandra Diebold (UK MHRA)
    • Dr. Yoichi Tanaka (Japan MHLW)

Agenda at a Glance Agenda at a Glance