Topic 1:

Limitation of Biomarker Systemic Sample Collection through traditional blood draw techniques; Appropriate sample collection for Dermatologic Indication using Home Skin-prick Microsampling; Overcoming issues & sampling interference/bias in processing blood samples through skin-prick micro sampling versus fractionation to plasma; Great care needs to be taken in the choice of blood sample Collection Strategies, Processing Steps, and Analyte Understanding to ensure accurate measurement of such analytes to support drug development.

Topic 2:

Troubleshooting method performance issues for LBA & Chromatographic-based Assay used to measure long-term blood sugar control by assessing Glycated Hemoglobin Levels over 8-12 weeks; Current challenges  in using HbA1c Test even if it is a routine/standardized monitoring tool (IVD); Lack of data on HbA1c measurements performed on clinical trial samples; Impact of Long Transit Times coupled with High Ambient Temperatures on Biomarker Sample Stability; Unique challenges faced when conducting Large Global Clinical Studies; Unexpected high HbA1c test cancellations in specific geographies not due to the samples arriving at the testing facility out of stability. 

Topic 3:

Lessons learned on BAV in Neurological Rare Diseases and strategies to overcome challenges with limited disease state samples; Practical strategies for BAV using Cerebrospinal Fluid (CSF) disease state samples; CSF Sample Stability, emphasizing handling practices to ensure accurate and reliable stability assessments; Bioanalytical challenges & emerging strategies for in rare neurological diseases during method development, validation, and sample analysis when Disease State Samples are limited or unavailable. 

Topic 4:

Sophisticated Multi-Assay Strategy for a Pivotal Trial in Rare Disease: Primary Endpoint Readout and Patient Stratification with Global Regulatory Alignment; Deploying Methylmalonic Acid (MMA) Mass Spec Assay (MSA) for screening use not treated as IVD/IVDR; For stratification use an Off-shelf CAP/CLIA assay across regions to ensure speed, comparability, and operational simplicity; Customized assay meeting IVDR expectations as a contingency if EU EMA challenges the stratification rationale; Evaluation of Comparability across assays.

Topic 5:

Enhancement of IVD Assay Performance to quantify Disease Biomarkers for IgA Nephropathy (IgAN);Validation KM55-based assay in the Serum matrix to achieve acceptable performance; Validation of KM55-based assay in the Urine matrix highly relevant for IgAN renal diseases; Improvements original IVD assay for LBA Critical Reagents and potential alternative approaches; Challenges in detecting galactose-deficient proteins and KM55-based assay as viable alternative to lectin-based approaches.

Topic 6:

Regulatory Agencies' requirements for Companion Diagnostic Assay (CDx) for Gene Therapies to ensure Patient Eligibility; Challenges in incorporating CDx AAV Testing into Gene Therapy clinical trials due to process complexity requiring a thorough evaluation of risks/benefits; Considerations on preexisting Antibodies to AAV impact on therapeutic potential of GT and on risks to Patient Safety; Establishing the Clinical Cut-off for the AAV immunogenicity assay by including both human and non-human samples; Conducting AAV CDx Testing prior to treatment to detect the presence of pre-existing antibodies to Specific AAV Serotype.

Topic 7:

Current best practices for Method Development & FFP BAV and COU Limitations as Primary Driver of the Validation Design when consensus on the value of and how to set minimum standards is not reached; Practical applications on small molecule Neurodegenerative Disease Biomarkers in clinical studies; Important considerations for 4-steps biomarker quantification FFP process; Challenges with analyte-free biological matrix, determination below endogenous level, separation of  isobaric interferences; Lack of BAV Regulatory Guidelines.

Topic 8:

Current challenges to achieve Tissue Specificity & Assay Sensitivity (S&S) in Biomarker Assay Development. Can isolation of Extracellular Vesicles (EV) be a way to achieve assay S&S? Focus on 2-step approach to enrich for tissue specific EV. What are the challenges with this approach? The second step requires an Immunoprecipitation/Immunoaffinity (IP/IA) and thus requires the use of magnetic beads. What are the challenges with magnetic beads? Are there other ways to get tissue specificity with tissue specific Proteoforms/Isoforms assays?

Topic 9:

Overcoming Extensive Heterogeneity in Peptide Expression to select reliable Celiac Disease Biomarker Targets for Method Development, BAV and efficacy monitoring; Challenges with over 600 Gluten Immunogenic Peptides discovered in urine containing high levels of proline and glutamine residues; Application of novel HRMS Peptidomics Method coupled with an informatics-driven workflow for detection of unique gluten-derived peptides originating from distinct proteins in urine samples revealing candidate Biomarker Peptides differentially expressed between oral glutenase.

Topic 10:

Alternative methodology to assess Linearity in Vaccine Biomarker LBA, focusing on from Proportionality Deviation, which allows for Validation Acceptance Criteria considering both assay performance and clinical trial context; Vaccine immunogenicity assays play a critical role in vaccine licensure and must be Rigorously Validated to support clinical trials, ensuring reliable data for Regulatory Decisions; Limitation of traditional Linearity assessment by creating dilution series and comparing measured concentrations to those expected from the dilution factor.

Topic 11:

Latest strategies for the successful implementation of Fully Automated Singlicate LBA for clinical sample testing; How to integrate automated liquid handling, plate washing, incubation, ECL plate reader, to enabling streamlined, high-throughput assay execution with minimal manual intervention; Comparison of Automated versus Manual Platforms and Singlicate versus Duplicate Wells to evaluate consistency, precision, and overall analytical performance. Definition of the Optimal Fit For Purpose Biomarker Assay Validation (FFP BAV) Strategies for focusing on risks mitigation.

Topic 12:

Recent developments in Total Biomarker Assay Validation (BAV) Strategy; Collaborative Pharma/CROs' efforts to establish the LBA in human serum; Technical challenge encountered during BAV due to varied lots of Critical Reagents; Crucial updates required and implemented in order to mitigate the critical reagent challenge; Streamlined approach for Cross-validation Design and statistical analyses (modeled from PK) to complete the expectations for study use. 

Topic 13:

When Singlicate Testing is scientifically acceptable for HAI Assays supporting primary Vaccine Biomarker Endpoints; Evaluation for high assay precision, well-characterized platforms, large-scale pivotal studies, variability & precision control; FFP BAV/Risk-based Validation acceptance criteria and strategies to support singlicate testing; Impact on data interpretation and Regulatory Acceptability (WHO, US FDA, EU EMA); Evaluation of singlicate results statistical robustness and  intra- and inter-assay variability; Singlicate Testing efficiency gains, throughput improvements, and data management implications. 

2026 White Paper on Biomarkers Discovery, Development, Validation & Regulatory Approval

Consensus & Conclusions on Biomarkers Discovery, Development, Validation & Regulatory Approval for 2026 White Paper

Panel Discussion with All the Regulators:

Have an Open Dialogue with the Regulators including US FDA, EU EMA, UK MHRA, Belgium FAGG / EU EMA, Health Canada
Ask the Regulators any Questions You Have on Biomarkers & Companion Diagnostics and Hear Their Feedbacks on Submitted Studies and Inspections/Audits Outcomes