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?
Session Recordings
Final Programme
Academic Chairpersons
- Prof. Lis Neubeck
- Prof. Piotr Szymanski
- Prof. Cecilia Linde (ESC President-Elect and ESC Chair of the CRT)
Industry Chairpersons
- Dr. Maria Ripa/Novo Nordisk
- Dr. Prodromos Anthopoulos/Cytokinetics
- Prof. Alexandra Goncalves /(BMS) (Industry Chair of the CRT)
Day 1: 19 February 2025 13:00–18:30 CEST
| Time and Session | Speakers |
| 12:00-13:00 Arrival and Buffet Lunch | |
| 13:00-13:05 Welcome from the CRT Chairpersons | Alexandra Goncalves (BMS) 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? | Elena Arbelo |
| 13:25-13:45 Current perspectives of using PED/PROs as primary end points for regulatory approval of drugs | 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 | 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/PROsModerated by: Lis Neubeck and Prodromos Anthopoulos (Cytokinetics) | |
| 15:00-15:20 Patients’ role and using PED/PROs in clinical trials- trialist perspective | Roxana Mehran (Via zoom) |
| 15:20-15:40 Patients’ role and using PED/PROs in drug development - industry perspective | Cecilie Merklin (Novo Nordisk) Anne Domdey (Novo Nordisk) |
| 15:40-16:00 Patient’s role in clinical trial design from perspective of PEDS/PROs – patient’s perspective | 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 | 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 | Rapporteur: Alar Irs (EMA) |
| 17:40-17:50 Report from breakout session 2 | Rapporteur: Paulus Kirchhof |
| 17:50-18:00 Report from breakout session 3 | Rapporteur: Alphons Vincent (Medtronic) |
| 18:00-18:25 PANEL DISCUSSION | |
| 18:25-18:30 Wrap-up and Summary Day 1 – Outlook to Day 2 | Lis NeubeckProdromos Anthopoulos (Cytokinetics) |
| END OF DAY 1 | |
| 19:30 APERITIF + DINNER | |
Day 2: 20 February 2025 08:30–12:30 CEST
| Time and Session | Speakers |
| 08:30-08:40 Summary of day 1 | Piotr Szymansky 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 | Maria Luisa Buzelli |
| 09:00-09:20 Patients’ role in the appraisal of clinical trial results | Wendy Davis (ESC Patient Forum) |
| 09:20-09:40 Regulatory viewpoint on future of generating, collecting and analyzing patients experience data in the EU | 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 | Rapporteur: Julie Sanders |
| 10:50-11:00 Report from breakout session 5 | Rapporteur: Tiny Jaarsma |
| 11:00-11:10 Report from breakout session 6 | 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 taking into account PED/PROs | Paulus Kirchhof |
| 11:30-11:50 Needs Examination, Evaluation and Dissemination: Identifying disease-specific health-related unmet needs of patients and society | Irina Cleemput |
| 11:50-12:10 Future of evidence generation - patient-centered, patient-initiated | Esther Kim |
| 12:10-12:35 PANEL DISCUSSION and NEXT STEPS | |
| 12:35-12:40 Wrap-up, conclusions |
Cecilia Linde Piotr Szymanski |
| END OF DAY 2 | |
| 12:40 Buffet Lunch and Departures | |
Biographies
- Prof. Cecilia LINDE, Sweden
- Prof. Alexandra GONCALVES, BMS
- Assoc. Prof. Elena ARBELO, Spain
- Dr. Prodromos ANTHOPOULOS, Cytokinetics
- Prof. Folkert ASSELBERGS, Netherlands
- Ms. Maria Luisa BUZELLI, Italy
- Ms. Wendy DAVIS, UK
- Dr. Anne DOMDEY, Novo Nordisk
- Ms. Kaisa IMMONEN, Netherlands
- Prof. John IOANNIDIS, USA
- Dr. Alar IRS, Estonia
- Prof. dr Tiny JAARSMA, Sweden
- Prof. Esther S.H. KIM, USA
- Prof. Paulus KIRCHHOF, Germany
- Dr. Bettina KRAUS, Boehringer-Ingelheim
- Prof. Thomas F. LÜSCHER, UK/Switzerland
- Prof. Roxana MEHRAN, USA
- Ms. Cecilie MERKLIN, Novo Nordisk
- Dr. Clemens MITTMANN, Germany
- Prof. Lis NEUBECK, UK
- Prof. Julie SANDERS, UK
- Dr. Maria Sejersten RIPA, Novo Nordisk
- Prof. Piotr SZYMANSKI, Poland
- Dr. Mattias VAN HEETVELDE, Belgium
- Ms. Mariëtte Verbakel, Netherlands
- Dr. Alphons VINCENT, Medtronic
- Mr. Patrick VRIJLANDT, Netherlands
Our mission: To reduce the burden of cardiovascular disease.