2023 White Paper on Recent Issues in Bioanalysis: EU IVDR 2017/746 Implementation/Impact, IVD/CDx/CLIA Approved Assays, High Dimensional Cytometry, Multiplexing Technologies, LBA Tissue Analysis, Vaccine Study Endpoints, Cell-Based Assays for Biomarkers, Cell Therapy and Vaccines (PART 2 - Recommendations on Development & Validation of Biomarkers, IVD, CDx, Cell-Based, Flow Cytometry, Ligand-Binding and Enzyme Assays; Advanced Critical Reagents Strategies)

doi:10.1080/17576180.2024.2340961

. 2024;16(8):179-220.

doi: 10.1080/17576180.2024.2340961. Epub 2024 May 22.

2023 White Paper on Recent Issues in Bioanalysis: EU IVDR 2017/746 Implementation/Impact, IVD/CDx/CLIA Approved Assays, High Dimensional Cytometry, Multiplexing Technologies, LBA Tissue Analysis, Vaccine Study Endpoints, Cell-Based Assays for Biomarkers, Cell Therapy and Vaccines (PART 2 - Recommendations on Development & Validation of Biomarkers, IVD, CDx, Cell-Based, Flow Cytometry, Ligand-Binding and Enzyme Assays; Advanced Critical Reagents Strategies)

Olga Kholmanskikh¹, Yow-Ming Wang², Sarah Hersey³, Meenu Wadhwa⁴, Katherine Block⁵, Abbas Bandukwala², Matthew Szapacs⁶, Russell Weiner⁷, Khader Awwad⁸, Francis Dessy^{9

10}, Sean Downing⁷, Xiulian Du², Fabio Garofolo¹¹, Shannon Harris¹², Victor Hou¹³, Jennifer Jones³, Sumit Kar¹⁴, Arvind Kinhikar¹⁵, Ming Li¹⁶, Joel Mathews¹⁷, John Meissen¹⁸, Giane Oliveira Sumner¹⁹, Luying Pan⁷, Gerard Sanderink²⁰, Ingrid Scully²¹, Johannes Stanta²², Yoichi Tanaka²³, Stephanie Vauleon²⁴, Leslie Wagner², Kai Wang²⁵, Liang Zhu¹³, Steven Eck²⁶, Yi-Dong Lin⁷, Mitra Azadeh²⁷, Vilma Decman²⁵, Sandra Diebold⁴, Xiulian Du², Polina Goihberg²⁸, Enrique Gomez Alcaide²⁴, Christele Gonneau²⁹, Michael Nathan Hedrick³, Gregory Hopkins³⁰, Sumit Kar¹⁴, Jakob Loschko²¹, Megan McCausland³¹, Luis Mendez³², Sarita Sehra³³, Erin Stevens³⁴, Yongliang Steve Sun³, Shabnam Tangri³⁵, Paul C Trampont¹³, Isabelle Cludts⁴, Mark Dysinger³⁶, Uma Kavita³⁷, Hiroshi Sugimoto⁷, Shannon Chilewski³, Christine Grimaldi¹⁹, Yong Jiang²⁵, John Kamerud²⁸, Susana Liu³⁸, Carolina Owen³⁴, Nisha Palackal¹⁹, Corinne Petit-Frere³⁹, Samuel Pine⁴⁰, Mohsen Rajabi Abhari², Kara Scheibner², LaKenya Williams³, Tao Xu⁴¹, Guodong Zhang⁴²

Affiliations

¹ Belgium FAMHP, Brussels, Belgium.
² US FDA, Silver Spring, MD, USA.
³ Bristol Myers Squibb, Lawrenceville, NJ, USA.
⁴ UK MHRA, London, UK.
⁵ Genentech, South San Francisco, CA, USA.
⁶ Abbvie, Philadelphia, PA, USA.
⁷ Takeda, Cambridge, MA, USA.
⁸ Abbvie, Ludwigshafen, Germany.
⁹ GlaxoSmithKline, Rixensart, Belgium.
¹⁰ Takeda, Cambridge, MA, USA.
¹¹ BRI Frontage, Vancouver, BC, Canada.
¹² HilleVax, Cambridge, MA, USA.
¹³ Moderna, Cambridge, MA, USA.
¹⁴ WRIB, Toronto, ON, Canada.
¹⁵ Novartis, Cambridge, MA, USA.
¹⁶ Stoke, Cambridge, MA, USA.
¹⁷ Ionis, Carlsbad, CA, USA.
¹⁸ AstraZeneca, South San Francisco, CA, USA.
¹⁹ Regeneron, Tarrytown, NY, USA.
²⁰ Sanofi Pasteur, Swiftwater, PA, USA.
²¹ Pfizer, Pearl River, NY, USA.
²² Celerion, Stevenage, UK.
²³ Japan MHLW, Tokyo, Japan.
²⁴ F. Hoffmann-La Roche Ltd, Basel, Switzerland.
²⁵ GlaxoSmithKline, Collegeville, PA, USA.
²⁶ AstraZeneca, Gaithersburg, MD, USA.
²⁷ Ultragenyx, Cambridge, MA, USA.
²⁸ Pfizer, Andover, MA, USA.
²⁹ Labcorp, Meyrin, Switzerland.
³⁰ 2seventy Bio, Cambridge, MA, USA.
³¹ Q2 Solutions, Atlanta. GA, USA.
³² Merck, Kenilworth, NJ, USA.
³³ Incyte, Wilmington, DE, USA.
³⁴ Pfizer, Groton, CT, USA.
³⁵ Navigate, a Novartis Subsidiary, San Diego, CA, USA.
³⁶ Alexion, New Haven, CT, USA.
³⁷ Spark Therapeutics, Plainsboro, NJ, USA.
³⁸ Pfizer, Kirkland, QC, Canada.
³⁹ Fresenius Kabi, Bad Homburg, Germany.
⁴⁰ Sanofi, Ghent, Belgium.
⁴¹ Frontage, Exton, PA, USA.
⁴² Alnylam, Cambridge, MA, USA.

PMID: 38899739
PMCID: PMC11216500
DOI: 10.1080/17576180.2024.2340961

2023 White Paper on Recent Issues in Bioanalysis: EU IVDR 2017/746 Implementation/Impact, IVD/CDx/CLIA Approved Assays, High Dimensional Cytometry, Multiplexing Technologies, LBA Tissue Analysis, Vaccine Study Endpoints, Cell-Based Assays for Biomarkers, Cell Therapy and Vaccines (PART 2 - Recommendations on Development & Validation of Biomarkers, IVD, CDx, Cell-Based, Flow Cytometry, Ligand-Binding and Enzyme Assays; Advanced Critical Reagents Strategies)

Olga Kholmanskikh et al. Bioanalysis. 2024.

. 2024;16(8):179-220.

doi: 10.1080/17576180.2024.2340961. Epub 2024 May 22.

Authors

Affiliations

¹ Belgium FAMHP, Brussels, Belgium.
² US FDA, Silver Spring, MD, USA.
³ Bristol Myers Squibb, Lawrenceville, NJ, USA.
⁴ UK MHRA, London, UK.
⁵ Genentech, South San Francisco, CA, USA.
⁶ Abbvie, Philadelphia, PA, USA.
⁷ Takeda, Cambridge, MA, USA.
⁸ Abbvie, Ludwigshafen, Germany.
⁹ GlaxoSmithKline, Rixensart, Belgium.
¹⁰ Takeda, Cambridge, MA, USA.
¹¹ BRI Frontage, Vancouver, BC, Canada.
¹² HilleVax, Cambridge, MA, USA.
¹³ Moderna, Cambridge, MA, USA.
¹⁴ WRIB, Toronto, ON, Canada.
¹⁵ Novartis, Cambridge, MA, USA.
¹⁶ Stoke, Cambridge, MA, USA.
¹⁷ Ionis, Carlsbad, CA, USA.
¹⁸ AstraZeneca, South San Francisco, CA, USA.
¹⁹ Regeneron, Tarrytown, NY, USA.
²⁰ Sanofi Pasteur, Swiftwater, PA, USA.
²¹ Pfizer, Pearl River, NY, USA.
²² Celerion, Stevenage, UK.
²³ Japan MHLW, Tokyo, Japan.
²⁴ F. Hoffmann-La Roche Ltd, Basel, Switzerland.
²⁵ GlaxoSmithKline, Collegeville, PA, USA.
²⁶ AstraZeneca, Gaithersburg, MD, USA.
²⁷ Ultragenyx, Cambridge, MA, USA.
²⁸ Pfizer, Andover, MA, USA.
²⁹ Labcorp, Meyrin, Switzerland.
³⁰ 2seventy Bio, Cambridge, MA, USA.
³¹ Q2 Solutions, Atlanta. GA, USA.
³² Merck, Kenilworth, NJ, USA.
³³ Incyte, Wilmington, DE, USA.
³⁴ Pfizer, Groton, CT, USA.
³⁵ Navigate, a Novartis Subsidiary, San Diego, CA, USA.
³⁶ Alexion, New Haven, CT, USA.
³⁷ Spark Therapeutics, Plainsboro, NJ, USA.
³⁸ Pfizer, Kirkland, QC, Canada.
³⁹ Fresenius Kabi, Bad Homburg, Germany.
⁴⁰ Sanofi, Ghent, Belgium.
⁴¹ Frontage, Exton, PA, USA.
⁴² Alnylam, Cambridge, MA, USA.

PMID: 38899739
PMCID: PMC11216500
DOI: 10.1080/17576180.2024.2340961

Abstract

The 17^th Workshop on Recent Issues in Bioanalysis (17^th WRIB) took place in Orlando, FL, USA on 19-23 June 2023. Over 1000 professionals representing pharma/biotech companies, CROs, and multiple regulatory agencies convened to actively discuss the most current topics of interest in bioanalysis. The 17^th WRIB included 3 Main Workshops and 7 Specialized Workshops that together spanned 1 week to allow an exhaustive and thorough coverage of all major issues in bioanalysis of biomarkers, immunogenicity, gene therapy, cell therapy and vaccines.Moreover, in-depth workshops on "EU IVDR 2017/746 Implementation and impact for the Global Biomarker Community: How to Comply with these NEW Regulations" and on "US FDA/OSIS Remote Regulatory Assessments (RRAs)" were the special features of the 17^th edition.As in previous years, WRIB continued to gather a wide diversity of international, industry opinion leaders and regulatory authority experts working on both small and large molecules as well as gene, cell therapies and vaccines to facilitate sharing and discussions focused on improving quality, increasing regulatory compliance, and achieving scientific excellence on bioanalytical issues.This 2023 White Paper encompasses recommendations emerging from the extensive discussions held during the workshop and is aimed to provide the bioanalytical community with key information and practical solutions on topics and issues addressed, in an effort to enable advances in scientific excellence, improved quality and better regulatory compliance. Due to its length, the 2023 edition of this comprehensive White Paper has been divided into three parts for editorial reasons.This publication (Part 2) covers the recommendations on Biomarkers, IVD/CDx, LBA and Cell-Based Assays. Part 1A (Mass Spectrometry Assays and Regulated Bioanalysis/BMV), P1B (Regulatory Inputs) and Part 3 (Gene Therapy, Cell therapy, Vaccines and Biotherapeutics Immunogenicity) are published in volume 16 of Bioanalysis, issues 9 and 7 (2024), respectively.

Keywords: WRIB; bioanalysis; biomarkers; cell therapy; gene therapy; immunogenicity; vaccine.

PubMed Disclaimer

Conflict of interest statement

The authors have no competing interests or relevant affiliations with any organization or entity with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, stock ownership or options and expert testimony.

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References

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[1] Savoie N, Booth BP, Bradley T, et al. . The 2nd Calibration and Validation Group Workshop on Recent Issues in Good Laboratory Practice Bioanalysis. Bioanalysis. 2009. doi:10.4155/bio.09.11 - DOI - PubMed

[2] Savoie N, Booth BP, Bradley T, et al. . The 2nd Calibration and Validation Group Workshop on Recent Issues in Good Laboratory Practice Bioanalysis. Bioanalysis. 2009. doi:10.4155/bio.09.11 - DOI - PubMed

[3] Savoie N, Garofolo F, Van Amsterdam P, et al. . 2009 White Paper on Recent Issues in Regulated Bioanalysis from the 3rd Calibration and Validation Group Workshop. Bioanalysis. 2010;2(1). doi:10.4155/bio.09.134 - DOI - PubMed

[4] Savoie N, Garofolo F, Van Amsterdam P, et al. . 2009 White Paper on Recent Issues in Regulated Bioanalysis from the 3rd Calibration and Validation Group Workshop. Bioanalysis. 2010;2(1). doi:10.4155/bio.09.134 - DOI - PubMed

[5] Savoie N, Garofolo F, Van Amsterdam P, et al. . 2010 White Paper on Recent Issues in Regulated Bioanalysis & Global Harmonization of Bioanalytical Guidance. Bioanalysis. 2010;2(12). doi:10.4155/bio.10.164 - DOI - PubMed

[6] Savoie N, Garofolo F, Van Amsterdam P, et al. . 2010 White Paper on Recent Issues in Regulated Bioanalysis & Global Harmonization of Bioanalytical Guidance. Bioanalysis. 2010;2(12). doi:10.4155/bio.10.164 - DOI - PubMed

[7] Garofolo F, Rocci ML, Dumont I, et al. . 2011 White Paper on Recent Issues in Regulated Bioanalysis and Regulatory Findings from Audits and Inspections. Bioanalysis. 2011. doi:10.4155/bio.11.192 - DOI - PubMed

[8] Garofolo F, Rocci ML, Dumont I, et al. . 2011 White Paper on Recent Issues in Regulated Bioanalysis and Regulatory Findings from Audits and Inspections. Bioanalysis. 2011. doi:10.4155/bio.11.192 - DOI - PubMed

[9] Desilva B, Garofolo F, Rocci M, et al. . 2012 White Paper on Recent Issues in Bioanalysis and Alignment of Multiple Guidelines. Bioanalysis. 2012;4(18). doi:10.4155/bio.12.205 - DOI - PubMed

[10] Desilva B, Garofolo F, Rocci M, et al. . 2012 White Paper on Recent Issues in Bioanalysis and Alignment of Multiple Guidelines. Bioanalysis. 2012;4(18). doi:10.4155/bio.12.205 - DOI - PubMed

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