- 08:30-08:40 - Summary of day 1 by Piotr Szymanski and Prodromos Anthopoulos (Cytokinetics)
- 08:40-09:50 - SESSION 2.1: Patient Centered Trial and PED/PROs Risk and Benefit Assessment, moderated by Prodromos Anthopoulos (Cytokinetics) and Piotr Szymanski
- 08:40-09:00 - The role of patients and PED/PROs in the early feasibility studies of medical devices by Maria Luisa Buzelli
- 09:00-09:20 - Patients’ role in the appraisal of clinical trial results by Wendy Davis (ESC Patient Forum)
- 09:20-09:40 - Regulatory viewpoint on future of generating, collecting and analyzing patients experience data in the EU by Rosa Gonzalez-Quevedo (EMA) (via Zoom)
- 09:40-10:20 - BREAKOUT SESSIONS: Risk and Benefit Assessment
- Group 4: Should patients take the leading role in the benefit-risk assessment of new technologies before their approval? Lead: Piotr Szymanski, Rapporteur: Julie Sanders
- Group 5: Who and how should educate patients (which patients) willing to be in steering committees in trial design and evaluation of clinical trials? Lead: Andre Krueger (Novartis), Rapporteur: Tiny Jaarsma
- Group 6: How should patients be involved in the regulatory process? Lead: Kaisa Immonen (EMA), Rapporteur: Bettina Kraus (Boehringer Ingelheim)
- 10:20-10:40 - Coffee Break
- 10:40-10:50 - Report from breakout session 4 by Rapporteur: Julie Sanders
- 10:50-11:00 - Report from breakout session 5 by Rapporteur: Tiny Jaarsma
- 11:00-11:10 - Report from breakout session 6 by Rapporteur: Bettina Kraus (Boehringer Ingelheim)
- 11:10-12:40 - SESSION 2.2: Future directions and shared vision for addressing the unmet needs, moderated by Lis Neubeck and Maria Ripa (Novo Nordisk)
- 11:10-11:30 - Should discussion around unmet medical needs in the future take into account PED/PROs by Paulus Kirchhof
- 11:30-11:50 - Needs Examination, Evaluation and Dissemination: Identifying disease-specific health-related unmet needs of patients and society by Irina Cleemput
- 11:50-12:10 - Future of evidence generation - patient-centered, patient-initiated by Esther Kim
- 12:10-12:35 - PANEL DISCUSSION and NEXT STEPS
- 12:35-12:40 - Wrap-up, conclusions by Cecilia Linde and Piotr Szymanski
- END OF DAY 2
- 12:40 - Buffet Lunch and Departures
Unmet Medical Needs Focusing on Patient-Benefit Risk and Patient-Reported Outcomes
CRT meeting with EMA
19 Feb 2025
-
20 Feb 2025
+
2025-02-19T13:00
2025-02-20T12:45
Europe/Paris
Unmet Medical Needs Focusing on Patient-Benefit Risk and Patient-Reported Outcomes
Add to calendar
2025-02-19T13:00
2025-02-20T12:45
Europe/Paris
Unmet Medical Needs Focusing on Patient-Benefit Risk and Patient-Reported Outcomes
Objectives of the CRT meeting
1. Advantages and disadvantages of utilizing Patient Experience Data (PED) and Patient Reported Outcomes (PROs) as endpoints and perhaps as primary endpoints? What can be improved and how?
2. How can we involve patients in the horizon scanning of new technologies and how PED contribute to that?
3. What should be the role of patients in inspiring pharmaceutical development and in the benefit-risk assessment system? Additionally, how should we balance PED/PROs against traditional efficacy and safety measures in regulatory decision making and clinical guidelines?
Programme
Day 1: 19 February 2025 13:00–18:30 CEST
- 12:00-13:00 - Arrival and Buffet Lunch
- 13:00-13:05 - Welcome from the CRT Chairpersons by Alexandra Goncalves (BMS) and Cecilia Linde
- 13:05-15:00 - SESSION 1.1 – Current State of the Art Regarding PED (Patient Experience Data) / PROs (Patient Reported Outcomes) in the Cardiovascular Field, moderated by Piotr Szymanski and Maria Ripa (Novo Nordisk)
- 13:05-13:25 - Definition of PED/PROs and what are the advantages? by Elena Arbelo
- 13:25-13:45 - Current perspectives of using PED/PROs as primary end points for regulatory approval of drugs by Patrick Vrijilandt (EMA)
- 13:45-14:05 - Patient perspectives of using PED/PROs as primary versus supportive endpoints in the approval of new drugs and devices by Mattias Van Heetvelde (ESC Patient Forum)
- 14:05-14:30 - PANEL DISCUSSION
- 14:30-15:00 - Coffee Break
- 15:00-18:30 - SESSION 1.2 – Patients’ Role in Designing Clinical Trials from the Perspective of PED/PROs, moderated by Lis Neubeck and Prodromos Anthopoulos (Cytokinetics)
- 15:00-15:20 - Patients’ role and using PED/PROs in clinical trials - trialist perspective by Roxana Mehran (Via Zoom)
- 15:20-15:40 - Patients’ role and using PED/PROs in drug development - industry perspective by Cecilie Merklin (Novo Nordisk) and Anne Domdey (Novo Nordisk)
- 15:40-16:00 - Patient’s role in clinical trial design from perspective of PEDs/PROs – patient’s perspective by Mariette Verbakel (ESC Patient Forum)
- 16:00-16:20 - PED/PROs as primary endpoints in clinical trials – What is a clinical meaningful change statistician’s view by John Ioannidis (Via Zoom)
- 16:20-17:10 - BREAKOUT SESSIONS: PED/PROs in the design of clinical trials
- Group 1: Are PED/PROs ready to use as primary end points in clinical trials? Lead: Thomas F. Lüscher, Rapporteur: Alar Irs (EMA)
- Group 2: How to overcome the obstacles and limitations in using PED/PROs as measures in CVD Clinical Trials. Lead: Clemens Mittmann (EMA), Rapporteur: Paulus Kirchhof
- Group 3: The role of new technologies in the collection and assessment of PED/PROs. Lead: Elena Arbelo, Rapporteur: Alphons Vincent (Medtronic)
- 17:10-17:30 - Coffee Break
- 17:30-17:40 - Report from breakout session 1 by Rapporteur: Alar Irs (EMA)
- 17:40-17:50 - Report from breakout session 2 by Rapporteur: Paulus Kirchhof
- 17:50-18:00 - Report from breakout session 3 by Rapporteur: Alphons Vincent (Medtronic)
- 18:00-18:25 - PANEL DISCUSSION
- 18:25-18:30 - Wrap-up and Summary Day 1 – Outlook to Day 2 by Lis Neubeck and Prodromos Anthopoulos (Cytokinetics)
- END OF DAY 1
- 19:30 - APERITIF + DINNER
Day 2: 20 February 2025 08:30–12:30 CEST
Chairpersons
Doctor Maria Ripa
Novo Nordisk
Professor Cecilia Marianne Linde
ESC President-Elect and ESC Chair of the CRT
Doctor Alexandra Goncalves
BMS - Industry Chair of the CRT
Doctor Prodromos Anthopoulos
Cytokinetics
Ressources
Biographies