Critical Path Institute’s (C-Path) Predictive Safety Testing Consortium (PSTC) announced the second public workshop on evidentiary considerations for regulatory assessment and qualification of complex in vitro models (CIVMs). Building upon outputs from the first workshop held in September 2023, attendees included individuals from health authorities, academia, model developers, and the pharmaceutical industry. The workshop provided a platform for attendees to discuss and deliberate on the evidentiary considerations for regulatory assessment and qualification of complex in vitro models. Attendees worked towards achieving a consensus on the model standards and features to improve the performance of CIVMs as a tool for drug development and regulatory assessment.
The meeting included sessions from key opinion leaders on general considerations for qualification, as well as interactive breakout sessions focusing on specifics related to different organ systems and disease models. The breakout participants had the opportunity to engage in discussions and deliberations on the evidentiary considerations for regulatory assessment and qualification of complex in vitro models.
The public workshop was held on September 26 and 27, 2024 in Bethesda, Maryland at The Bethesdan Hotel (8120 Wisconsin Ave, Bethesda, MD 20814).
Click here to see speaker bios.
2024 Complex In Vitro Model (CIVM) Qualification Framework Public Workshop: Day 1
September 26, 2024: 9:00 AM – 5:30 PM EST
| Session | Time | Length (min) | Topics | Presenters and Panelists | Links |
| Opening Remarks | 9:00 AM | 15 | Welcome and Introduction (set stage) | Klaus Romero (C-Path) | Session Recording Slides |
| Keynote Address | 9:15 AM | 30 | FDA Perspective on Qualification (ISTAND) | York Tomita (US FDA – ODES/DBIRBD) | Session Recording Slides |
| 9:45 AM | 30 | European Perspective on Qualification | Sonja Beken (Belgian Federal Agency for Medicines and Health Products (FAMHP)) | Session Recording Slides |
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| Break | 10:15 AM | 15 | Refreshment Break | — | |
| Session 1: Global Perspective and Discussion of Use | 10:30 AM | 10 | C-Path Workshop #1 | Graham Marsh (C-Path) | Session Recording |
| 10 | Incorporation of New Approach Methodologies (NAMs) and/or Weight of Evidence (WoE) in guidance documents utilized by FDA/CDER: ICH-S5(R3) and ICH-S1B(R1) Addendum | Amy Avila (US FDA) | Session Recording Slides |
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| 10 | EURL ECVAM Perspective on OoC Standardization and Qualification | Monica Piergiovanni (European Commission) | |||
| 10 | Standardization of MPS Engineering: Challenges and Opportunities | Darwin Reyes-Hernandez (NIST) | |||
| 10 | CEN CENELEC Standardization Document | Andries van der Meer (EuROocs) | Session Recording Slides |
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| 10 | IQ MPS Qualification/Validation Perspective | Rhiannon Hardwick (BMS) | Session Recording Slides |
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| 10 | Japanese initiative for the proposal of an OECD Test Guideline: TG417 Toxicokinetics | Seiichi Ishida (NIHS) | Session Recording Slides |
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| 11:40 AM | 45 | Panel Discussion | Graham Marsh, Darwin Reyes-Hernandez, Klaus Romero, Session Speakers | Session Recording | |
| Break (Lunch) | 12:15 PM | 90 | Lunch | — | |
| Session 2: Applying Specific examples to broader application | 1:45 PM | 20 | Lessons Learned in Precision Medicine | Mike Pacanowski (US FDA) | Session Recording Slides |
| 20 | Human on a chip system to enable regulatory submission for efficacy | James Hickman (Hesperos) | |||
| 20 | Qualification of a Human 3D Liver-on-Chip Model: Establishing a Cross-pharma trial to evaluate ADME and Toxicity Predictions in Pre-clinical Development | Andre Rodrigues (UCB) | |||
| 2:45 PM | 45 | Panel Discussion – broad takeaways and how can we use those learnings | Session Speakers + Nakissa Sadrieh (US FDA) + Julie Frearson (Charles River) + Deidre Dalmas | Session Recording | |
| Break | 3:30 PM | 15 | Break | — | |
| Session 3: Context of Use | 3:45 PM | 10 | Translational Centers for Microphysiological Systems (TraCe MPS) – Qualifying MPS as Drug Development Tools (DDTs) | Passley Hargrove-Grimes (NIH/NCATS) | Session Recording Slides |
| 10 | TraCe MPS Drug Development Tools for Pregnancy and Women's Health | Ramkumar Menon (University of Texas) | Session Recording Slides |
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| 10 | University of Rochester and Duke University: Barrier MPS | Jim McGrath (University of Rochester) | Session Recording Slides |
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| 10 | University of Pittsburgh: Patient-derived Biometric Liver MPS | Mark Schurdak (University of Pittsburgh) | Session Recording Slides |
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| 10 | University of Washington and Mount Sinai School of Medicine: Kidney MPS | Jonathan Himmelfarb (Mount Sinai) | Session Recording Slides |
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| 4:45 PM | 45 | Panel Discussion – COU discussion and critiques, unmet regulatory need | Passley Hargrove-Grimes (NIH/NCATS), Dmitriy Krepkiy (NIH/NCATS), Ivan Rusyn (Texas A&M University), Ramkumar Menon, Jim McGrath, Joan Adamo, Mark Schurdak, Jonathan Himmelfarb, Mary McElroy (Charles River), Vanitha Sekar (US FDA), Rodney Rouse (US FDA) | Session Recording | |
| Closing Remarks | 5:30 PM | 15 | Day 1 Summary and Thanks | Nicholas King (C-Path) |
Complex In Vitro Model (CIVM) Qualification Framework Public Workshop: Day 2
September 27, 2024: 9:00 AM – 2:10 PM EST
| Session | Time | Length (min) | Topics | Presenters and Panelists | Links |
| Opening Remarks | 9:00 AM | 10 | Welcome | Graham Marsh (C-Path) | Session Recording |
| Keynote Address | 9:10 AM | 20 | Perspective on Translating New Drug Development Tools into Regulatory Use | David Strauss (US FDA) | Session Recording |
| 9:30 AM | 15 | General evidentiary considerations | Ksenia Blinova (US FDA) | Session Recording Slides |
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| Session 4: Breakout Sessions | 9:45 AM | 15 | Breakout session structure and goals | Graham Marsh | |
| 10:00 AM | 30 | Breakout session assignments / coffee break | — | ||
| 10:30 AM | 60 | Cardiac | Ivan Rusyn (Texas A&M University) Jordan Pomeroy (US FDA) Natalie Simpson (US FDA) | Session Recording | |
| GI / Gut | Tomasz Kostrzewski (CN-Bio) Kevin Ford (US FDA) | Session Recording | |||
| Kidney | Deidre Dalmas Aliza Thompson (US FDA) | ||||
| Neuro | Pelin Candarlioglu (Vivodyne) Amy Avila (US FDA) | ||||
| Disease Modeling | Tyna Dao (US FDA) James Hickman (Hesperos) | Session Recording | |||
| Break (Lunch) | 11:30 AM | 90 | Lunch | — | |
| Session 4: Breakout Sessions | 1:00 PM | 90 | Post-Lunch breakout session wrap up (5 breakouts) | ||
| 2:30 PM | 50 | Breakout Session Recaps | Breakout session facilitators (5) | Session Recording | |
| Closing and Meeting Summary | 3:20 PM | 15 | Closing and Next Steps | Nicholas King |
