The Critical Path Institute’s Polycystic Kidney Disease Outcomes Consortium (PKDOC), The PKD Foundation and Otsuka invite you view the 2021 PKD Regulatory Summit
The Summit included a series of three sessions with pre-recorded webinars available for registrants to view at their own pace two weeks prior to the meeting. On May 19 and 20, live presentations summarized content from the pre-recorded webinars, followed by panel discussions with speakers that facilitated dialogue among all participants.
Access Live Presentations Day 1
Access Live Presentations Day 2
Access pre-recorded presentations below.
AGENDA DAY 1: Wednesday, May 19
10 AM – 2 PM (ET)
SESSION 1: Advancing Drug Development Therapies for Autosomal Recessive Polycystic Kidney Disease (ARPKD)
| SESSION 1: Pre-recorded Presentation Topics and Speakers | ||
|---|---|---|
| 1. | Autosomal Recessive Polycystic Kidney Disease (ARPKD): Clinical course, trial endpoints, and imaging | Erum Hartung, MD, MTR Assistant Professor of Pediatrics, Division of Nephrology Children’s Hospital of Philadelphia (CHOP) |
| 2. | ARegPKD Registry: Recent Developments | Max Christoph Liebau, MD Pediatric Nephrology & Center for Chronically Ill Children, Center for Molecular Medicine University of Cologne |
| 3. | Translational Strategies for Autosomal Recessive Polycystic Kidney Disease (ARPKD) | Lisa M. Guay-Woodford, MD McGehee Joyce Professor of Pediatrics, Director, CTSI-CN/Director, Center for Translational Research Children’s National Research Institute |
| 4. | Patient/Family Perspective | Brandé and Ed Waldron, ARPKD Parents |
| 5. | Patient/Family Perspective | Jaina and Lisa Cormack, ARPKD Patient and Parent |
| SESSION 1: Panelists |
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Moderators: Ron Perrone, MD – Tufts Medical Center Lisa Guay-Woodford, MD – Children’s National |
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Panelists: Katherine Dell, MD – Case Western Reserve University, Cleveland Clinic David Eiznhamer, PhD, MBA – Kadmon Corporation Erum Hartung, MD, MTR – Children’s Hospital of Philadelphia (CHOP) Max Liebau, MD – University of Cologne Julie Marshall – Patient speaker Kirtida Mistry, MD – FDA Dimitar Roussinov, MD – EMA, Pediatric Committee (PDCO) Joseph Toerner, MD, MPH – FDA |
SESSION 2: Advancing Drug Development Therapies for Autosomal Dominant Polycystic Kidney Disease (ADPKD)
| SESSION 2: Pre-recorded Presentation TopicsTopics and Speakers | ||
|---|---|---|
| 1. | ADPKD: Diagnosis, Clinical Course, and health complications | Neera Dahl, MD, PhD Associate Professor, Section of Nephrology Yale University School of Medicine |
| 2. | Advancing in the research of pediatric ADPKD | Djalila Mekahli, MD, PhD Pediatric Nephrology and Organ Transplantation University Hospital Leuven |
| 3. | Assessing rapid progression in ADPKD | Roser Torra, MD, PhD Inherited Kidney Diseases, Nephrology Department Fundació Puigvert Barcelona, Spain |
| 4. | Patient Perspective | Lainie Esquivel, ADPKD Patient |
| 5. | Patient Perspective | Karen Edwards, ADPKD Patient |
| SESSION 2: Panelists |
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Moderators: Chris Rusconi, PhD – PKD Foundation Arlene Chapman, MD – University of Chicago |
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Panelists: Neera Dahl, MD, PhD – Yale University School of Medicine Ali Hariri, MD – Sanofi Pravin Jadhav, PhD – Otsuka Romaldas Mačiulaitis, MD, PharmD – EMA Djalila Mekahli, MD, PhD – University Hospital Leuven Kirtida Mistry, MD – FDA Craig Ostroff, PharmD, R.Ph. – Goldfinch Bio Norman Stockbridge, MD, PhD – FDA Joseph Toerner, MD, MPH – FDA Prof Roser Torra, MD, PhD – Fundació Puigvert Hayley Womack – Patient Speaker |
| 2021 PKD Regulatory Summit Agenda Day 1: Wednesday, May 19 10:00 AM – 2:00 PM (ET) |
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|---|---|---|
| 10:00 – 10:10 am | DAY 1 INTRODUCTION: John-Michael Sauer | |
| SESSION 1: Advancing Drug Development Therapies for Autosomal Recessive Polycystic Kidney Disease (ARPKD) Moderators: Ron Perrone and Lisa Guay-Woodford |
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| 10:10 – 10:20 am | Session 1 Introduction | Ron Perrone |
| 10:20 – 10:35 am | Patient Speaker | “ARPKD Patient’s Perspective” Real life aspects of ARPKD Julie Marshall |
| 10:35 – 11:50 am | Session 1 Panel Discussion | Featured speakers and panelists for discussion and Q&A about pre-recorded presentations |
| 11:50 am – 12:10 pm | BREAK (20 minutes) | |
| SESSION 2: Advancing Drug Development Therapies for Autosomal Dominant Polycystic Kidney Disease (ADPKD) Moderators: Chris Rusconi and Arlene Chapman |
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| 12:10 – 12:20 pm | Session 2 Introduction | Chris Rusconi |
| 12:20 – 12:35 pm | Patient Speaker | “ADPKD Patient’s Perspective” Real-life aspects of ADPKD Hayley Womack |
| 12:35 – 1:50 pm | Session 2 Panel Discussion | Featured speakers and panelists for discussion and Q&A about pre-recorded presentations |
| 1:50 – 2:00 pm | DAY 1 WRAP-UP: John-Michael Sauer | |
AGENDA DAY 2: Thursday, May 20
10 AM – 2:30 PM (ET)
SESSION 3: PKDOC 2.0: The Future of PKD Research
SESSION 3A: Data-driven Drug Development Tools
| SESSION 3A: Pre-recorded Presentation Topics and Speakers | ||
|---|---|---|
| 1. |
Enabling a path to optimize clinical trial design in PKD: a) Clinical trial design in PKD: Early TKV modeling for trial enrichment
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Vicente E. Torres, MD, PhD Klaus Romero, MD, MS, FCP |
| 2. | Clinical Outcome Assessments in ADPKD | Dorothee Oberdhan, MS Director, Patient-Centric Outcomes Global Value & Real-World Evidence Otsuka Pharmaceuticals |
| 3. | Potential Role of C-Path’s Clinical Outcome Assessment (COA) Program | Rebecca Speck, PhD, MPH Clinical Outcome Assessment (COA) Scientist, COA Program Critical Path Institute |
| 4. | Data Sharing – Value Proposition for the future of Rare Disease R&D | Jeff Barrett, PhD, FCP Senior Vice President, RDCA-DAP Lead Critical Path Institute |
| 5. | Lessons Learned Over Nearly Two Decades of CKD-EPI | Lesley A. Inker, MD, MS, FRCP Associate Professor Tufts Medical Center Tufts University School of Medicine |
| 6. | Patient Perspective | Heidi Cambareri, ADPKD Patient |
| SESSION 3A: Panelists |
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Moderators: John-Michael Sauer, PhD – Critical Path Institute Frank Czerwiec, MD, PhD – Goldfinch Bio |
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Panelists: Jeff Barrett, PhD – Critical Path Institute Ali Hariri, MD – Sanofi Lesley Inker, MD, MS, FRCP – Tufts University School of Medicine Pravin Jadhav, PhD – Otsuka Romaldas Mačiulaitis, MD, PharmD – EMA Cari Maxwell – Patient Speaker Dorothee Oberdhan, MS – Otsuka Klaus Romero, MD, MS, FCP – Critical Path Institute Rebecca Speck, PhD, MPH – Critical Path Institute Aliza Thompson, MD – FDA Vicente Torres, MD, PhD – Mayo Clinic |
SESSION 3B: Toward a Regulatory Standard for ADPKD
| SESSION 3B: Pre-recorded Presentation Topics and Speakers | ||
|---|---|---|
| 1. | Study proposal for a PKD clinical trial | Frank S. Czerwiec, MD, PhD Chief Medical Officer Goldfinch Bio Ronald D. Perrone, MD Division of Nephrology Scientific Director, Clinical and Translational Research Center Tufts Medical Center Tufts University School of Medicine |
| 2. | Patient Perspective | Greg Mainolfi, ADPKD Patient |
| SESSION 3B: Panelists |
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Moderators: Frank Czerwiec, MD, PhD – Goldfinch Bio Aliza Thompson, MD – FDA Romaldas Mačiulaitis, MD, PharmD – EMA |
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Panelists: Karl Cremer, PharmD – Regulus Therapeutics Peter Cody Fiduccia – Patient Speaker Kevin Fowler – The Voice of the Patient Inc. Ken Gruchalla, MD – Health Canada Tess Harris – PKD Charity Neil Shusterman, MD – Palladio Biosciences Liron Walsh, MD – Goldfinch Bio Alan Yu, M.B. B.Chir. – University of Kansas School of Medicine |
| 2021 PKD Regulatory Summit Agenda Day 2: Thursday, May 20 10:00 AM – 2:30 PM (ET) |
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|---|---|---|
| 10:00 – 10:10 am | DAY 2 INTRODUCTION: John-Michael Sauer | |
| SESSION 3A: PKDOC 2.0: The Future of PKD Research – Data-driven Drug Development Tools Moderators: John-Michael Sauer and Frank Czerwiec |
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| 10:10 – 10:15 am | Session 3A Introduction | John-Michael Sauer |
| 10:15 – 10:30 am | Patient Speaker | Cari Maxwell |
| 10:30 – 11:40 am | Session 3A Panel Discussion | Featured speakers and panelists for discussion and Q&A about pre-recorded presentations |
| 11:40 am – 12:00 pm | BREAK (20 minutes) | |
| SESSION 3B: PKDOC 2.0: The Future of PKD Research – Toward a Regulatory Standard for ADPKD Moderators: Frank Czerwiec, Aliza Thompson and Romaldas Mačiulaitis |
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| 12:00 – 12:05 pm | Session 3B Introduction | Frank Czerwiec |
| 12:05 – 12:20 pm | Patient Speaker | Peter Cody Fiduccia |
| 12:20 – 12:35 pm | Overview of protocol | Frank Czerwiec |
| 12:35 – 1:05 pm | Regulatory Comment | Aliza Thompson and Romaldas Mačiulaitis |
| 1:05 – 2:15 pm | Session 3B Panel Discussion | Featured speakers and panelists for discussion and Q&A about pre-recorded presentations |
| 2:15 – 2:30 pm | CLOSING COMMENTS: John-Michael Sauer | |
Funding for the Summit provided by The PKD Foundation and Otsuka.
