Objectives of the meeting
In the preceding year, 2022, there were 89 positive EMA recommendations for authorisation, including 41 concerning new active substances. However, despite the fact that cardiovascular diseases remain the number one cause of death and morbidity in EU, representing the largest unmet medical need, there were no new cardiovascular drugs recommended for marketing approval via the centralised procedure.
The proposed revision of the EU medicines regulatory framework is aimed at strengthening investment in medicinal products where research is most needed, but investment is riskier. This newly proposed regulation aims to address the needs of patients' groups that are overlooked and that may therefore fall in the category of patients with an unmet medical need.
The objectives of the meetings are therefore to:
- Better understand interaction between regulation and innovation in the context of forthcoming pharmaceutical legislation to address the current crisis in cardiovascular drugs development
- Take a closer look at unmet medical needs in cardiovascular diseases, to better understand the prominent disconnect between high cardiovascular mortality and morbidity and declining innovation in cardiovascular therapeutics
- Seek for fit-for-purpose regulatory definitions and market access solutions, including appropriate incentivization of innovation and conditional approval of novel technologies
- Better define the patient’s role in the assessment of risk-benefit in drug development.
Related Reading
Reform of the EU Pharmaceutical Legislation
Session Recording
Final Programme
Academic Chairpersons
- Prof. Piotr Szymanski (Chairman of the ESC Regulatory Affairs Committee)
- Prof. Faiez Zannad
- Prof. Thomas F. Lüscher (ESC President-Elect and ESC CRT Co-chair).
Industry Chairpersons
- Prof. Anders Himmelmann (AstraZeneca)
- Dr. Bettina Kraus (Boehringer Ingelheim Pharmaceuticals Inc /BI),
- Dr. Narinder Bhalla (BMS)
- Prof. Alexandra Goncalves (BMS / Industry CRT Co-chair)
DAY 1: 21 November 2023 - 13:30-18:45
| Time | Session | Speaker |
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12:30-13:30 |
Arrival and buffet lunch |
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13:30-13:35 |
Welcome from the CRT Chairpersons |
Thomas F. Lüscher Alexandra Goncalves (BMS) |
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13:35-13:45 |
General introduction to the workshop |
Piotr Szymanski Faiez Zannad |
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13:45-15:20 - Session 1 – Interaction between regulation and innovations Moderated by Prof. Faiez Zannad and Dr. Narinder Bhalla (BMS) |
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13:45-14:05 |
The New EU Pharmaceutical Strategy: Implications for all stakeholders |
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14:05-14:25
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Is current EU legislation favorable to cardiovascular innovation? |
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14:25-14:45 |
Ongoing clinical trials and cardiovascular drugs in the pipeline - is there a shortage of innovation? |
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14:45-15:30 |
Discussion |
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15:30-19:00 - Session 2 – Definitions and their practical implications |
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15:30-15:50 |
How are unmet medical needs defined in the EMA guidelines? |
Patrick Vrijlandt |
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15:50-16:10 |
Clinical cardiologists' perspective on unmet needs in cardiovascular diseases. |
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16:10-16:30 |
Why there are many unmet medical needs in cardiovascular diseases and so few newly approved drugs? |
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16:30-16:45 |
Break |
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16:45-17:30 |
Breakout Sessions 1 - 45 min Group 1: (How) Should we redefine unmet medical needs?
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Lead: Patrick Vrijlandt Rapporteur: Piotr Szymanski |
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Group 2: How to incentivise innovation and market access?
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Group 3: How can the cycle of updating ESC Guidelines keep pace with innovation? |
Lead: Eva Prescott |
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17:30-17:40 |
Report from breakout session 1 – 10mins |
Rapporteur: Piotr Szymanski |
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17:40-17:50 |
Report from breakout session 2 – 10mins |
Rapporteur: Bettina Kraus (BI) |
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17:50-18:00 |
Report from breakout session 3 – 10mins |
Rapporteur: Narinder Bhalla (BMS) |
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18:00-18:40 |
General discussion |
Panelists: Bettina Kraus (BI), Alar Irs, Patrick Vrijlandt, Faiez Zannad, Thomas F. Lüscher |
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18:40-18:55 – Wrap-up and summary Day 1 – Outlook to Day 2 - End of Day 1 |
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20:00 |
Dinner |
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DAY 2: 22 November 2023 - 08:30 - 12:30
| Time | Session | Speaker |
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08:30 - 12:30 - Session 3 - Novel surrogate endpoints Moderated by Prof Thomas F. Lüscher and Dr. Bettina Kraus (Boehringer Ingelheim Pharmaceuticals Inc) |
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08:30-08:50 |
Regulatory approach to surrogate end-points in cardiovascular diseases – which are acceptable and why? |
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08:50-09:15 |
How and when surrogate endpoints may help in cardiovascular drug approval trials? What can be learnt from the use of surrogate endpoints in oncology approval trials ? |
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09:15 -09:35 |
Reimbursement based on surrogate end-points in cardiovascular diseases |
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09:35-09:55 |
Patient’s role in the assessment of risk-benefit in drug development – innovative cardiovascular drugs how much uncertainty is acceptable? |
ESC Patient Forum representative |
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09:55-10:55 |
Breakout Sessions 2 - 60 min Group 1: How and when surrogate endpoints may serve as primary endpoints in cardiovascular drug development? |
Lead: Tabassome Simon |
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Group 2: Should all surrogate end-points be validated and how? |
Lead: Jan Tijssen |
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Group 3: How to introduce conditional approval to CVD? |
Lead: Peter Mol |
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10:55-11:05 |
Break |
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11:05-11:15 |
Report from breakout session 1 – 10mins |
Rapporteur: Clemens Mittmann |
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11:15-11:25 |
Report from breakout session 2 – 10mins |
Rapporteur: Paulus Kirchhnof |
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11:25-11:35 |
Report from breakout session 3 – 10mins |
Rapporteur: Faiez Zannad |
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11:35-12:15 |
General discussion |
Panelists: Eva Prescott, Penilla Gunther, Clemens Mittmann, Peter Mol, Alar Irs, Borislava Mihaylova |
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12:15-12:30 – Wrap-up, conclusions, next steps for a publication - End of Day 2 |
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12:30 |
Buffet lunch and departures |
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Biographies
- Prof. Thomas F. LÜSCHER, UK
- Prof. Alexandra GONCALVES, BMS
- Prof. Piotr SZYMANSKI, Poland
- Prof. Faiez ZANNAD, France
- Prof. Anders HIMMELMANN, AstraZeneca
- Dr. Bettina KRAUS, BI
- Dr. Narinder BHALLA, BMS
- Prof. Eva PRESCOTT, Denmark
- Dr. Alar IRS, Estonia
- Prof. Peter MOL, Netherlands
- Associate Prof. Borislava MIHAYLOVA, UK
- Prof. Tabassome SIMON, France
- Dr. Clemens MITTMANN, Germany
- Prof. Jan TIJSSEN, Netherlands
- Prof. Eric BOERSMA, Netherlands
- Mr. Fabio D’ATRI, Belgium
- Prof. Paulus KIRCHHOF, Germany
- Mr. Mattias VAN HEETVERLDE, Belgium
- Ms. Penilla GUNTHER, Sweden
- Mr. Patrick VRIJLANDT, Netherlands
Our mission: To reduce the burden of cardiovascular disease.