2021 PKD Regulatory Summit
Register Now for This Virtual Workshop!
The Critical Path Institute’s Polycystic Kidney Disease Outcomes Consortium (PKDOC) and The PKD Foundation invite you to participate in the 2021 PKD Regulatory Summit on May 19-20, 2021. Together with key stakeholders from the pharmaceutical industry and academic setting, foundations, patient advocacy groups, individuals living with PKD and regulatory agencies from around the world, the consortium will address current unmet drug development needs for PKD, identify tools that are needed to deliver new therapies to patients, and catalyze the development of a regulatory framework to enable advancement of new treatments.
The Summit includes 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 will summarize content from the pre-recorded webinars, followed by panel discussions with speakers to facilitate dialogue among all participants.
Access to pre-recorded presentations coming soon!
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 | |
|---|---|
| 1. | Didactic information about ARPKD including hepatic fibrosis, clinical course, imaging, and trial endpoints |
| 2. | ARegPKD Registry: Findings and relationship to clinical trial endpoints |
| 3. | State of the field including registries, biomarkers, and translational opportunities |
| 4. | Patient/Family perspective |
SESSION 2: Advancing Drug Development Therapies for Autosomal Dominant Polycystic Kidney Disease (ADPKD)
| SESSION 2: Pre-recorded Presentation Topics | |
|---|---|
| 1 | Didactic information about ADPKD including pediatric ADPKD |
| 2 | ADPedKD Registry: ADPKD clinical impacts in pediatric population, cardiovascular issues, etc. |
| 3 | Challenge of identifying biomarkers and structural outcomes vs functional outcomes beginning in childhood |
| 4 | Patient/Family perspective |
| 2021 PKD Regulatory Summit Agenda Day 1: Wednesday, May 19 10:00 AM – 2:00 PM (ET) |
||
|---|---|---|
| 10:00 – 10:10 am | DAY 1 INTRODUCTION: Johm-Michael Sauer | |
| SESSION 1: Advancing Drug Development Therapies for Autosomal Recessive Polycystic Kidney Disease (ARPKD) Moderators: Ron Perrone and Lisa Guay-Woodford |
||
| 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 Patient/Family Member TBD |
| 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 |
||
| 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 Patient/Family Member TBD |
| 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: Wednesday, May 20
10 AM – 2 PM (ET)
SESSION 3: PKDOC 2.0: The Future of PKD Research
SESSION 3A: Data-driven Drug Development Tools
| Topics | |
|---|---|
| 1 |
Enabling a path to optimize clinical trial design in PKD:
a) The importance of TKV in evaluating the progression of PKD b) Developing disease-intervention models/simulation tools for PKD |
| 2 | Patient Reported Outcomes/Clinical Outcome Assessments (PRO/COA) activities |
| 3 | Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP): Strengthening research through data sharing |
| 4 | Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI): Experiences with data sharing for kidney measures and clinical outcomes |
| 5 | Patient perspective |
SESSION 3B: Toward a Regulatory Standard for ADPKD
| Topics | |
| 1 | Study proposal for a PKD clinical trial |
| 2 | Patient perspective |
| 2021 PKD Regulatory Summit Agenda Day 2: Wednesday, May 20 10:00 AM – 2:00 PM (ET) |
||
| 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 |
||
| 10:10 – 10:20 am | Session 3A Introduction | John-Michael Sauer |
| 10:20 – 10:35 am | Patient Speaker | TBD Patient/Family Member TBD |
| 10:35 – 11:20 am | Session 3A Panel Discussion | Featured speakers and panelists for discussion and Q&A about pre-recorded presentations |
| 11:20 am – 11:40 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 |
||
| 11:40 – 11:45 am | Session 3B Introduction | Frank Czerwiec |
| 11:45 – 12:00 pm | Patient Speaker | TBD |
| 12:00 – 12:15 pm | Overview of protocol | Frank Czerwiec |
| 12:15 – 12:45 pm | Regulatory Comment | Aliza Thompson and Romaldas Mačiulaitis |
| 12:45 – 1:50 pm | Session 3B Panel Discussion | Featured speakers and panelists for discussion and Q&A about pre-recorded presentations |
| 1:50 – 2:00 pm | CLOSING COMMENTS: John-Michael Sauer | |
Live presentations and panel discussion will be recorded and posted to the PKDOC web page following the workshop.
