Unmet Medical Needs

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.

Read the ESC Statement on the reform of the EU Pharmaceutical Legislation

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.

Final Programme

DAY 1: 21 November 2023 - 13:30-18:45

• 12:30-13:30 - Arrival and buffet lunch
• 13:30-13:35 - Welcome from the CRT Chairpersons - Thomas F. Lüscher, Alexandra Goncalves (BMS)
• 13:35-13:45 - General introduction to the workshop - Piotr Szymanski, Faiez Zannad

Session 1 – Interaction between regulation and innovations (Moderated by Prof. Faiez Zannad and Dr. Narinder Bhalla (BMS))

• 13:45-14:05 - The New EU Pharmaceutical Strategy: Implications for all stakeholders - Fabio D'Atri (by Zoom)
• 14:05-14:25 - Is current EU legislation favorable to cardiovascular innovation? - Thomas F. Lüscher
• 14:25-14:45 - Ongoing clinical trials and cardiovascular drugs in the pipeline - is there a shortage of innovation? - Anders Himmelmann (AstraZeneca)
• 14:45-15:30 - Discussion

Session 2 – Definitions and their practical implications (Moderated by Prof. Piotr Szymanski and Prof. Anders Himmelmann (AstraZeneca))

• 15:30-15:50 - How are unmet medical needs defined in the EMA guidelines? - Patrick Vrijlandt

• 15:50-16:10 - Clinical cardiologists' perspective on unmet needs in cardiovascular diseases - Faiez Zannad

• 16:10-16:30 - Why there are many unmet medical needs in cardiovascular diseases and so few newly approved drugs? - Bettina Kraus (BI)

• 16:30-16:45 - Break

• 16:45-17:30 - Breakout Sessions 1 - 45 min

  • Group 1: (How) Should we redefine unmet medical needs? - Lead: Patrick Vrijlandt, Rapporteur: Piotr Szymanski
  • Group 2: How to incentivise innovation and market access? - Lead: Thomas F. Lüscher, Rapporteur: Bettina Kraus (BI)
  • Group 3: How can the cycle of updating ESC Guidelines keep pace with innovation? - Lead: Eva Prescott, Rapporteur: Narinder Bhalla (BMS)

• 17:30-17:40 - Report from breakout session 1 – 10 mins - Rapporteur: Piotr Szymanski

• 17:40-17:50 - Report from breakout session 2 – 10 mins - Rapporteur: Bettina Kraus (BI)

• 17:50-18:00 - Report from breakout session 3 – 10 mins - Rapporteur: Narinder Bhalla (BMS)

• 18:00-18:40 - General discussion - Panelists: Bettina Kraus (BI), Alar Irs, Patrick Vrijlandt, Faiez Zannad, Thomas F. Lüscher

• 18:40-18:55 - Wrap-up and summary Day 1 – Outlook to Day 2 - End of Day 1

• 20:00 - Dinner

DAY 2: 22 November 2023 - 08:30 - 12:30

• 08:30 - 12:30 - Session 3 - Novel surrogate endpoints (Moderated by Prof. Thomas F. Lüscher and Dr. Bettina Kraus (Boehringer Ingelheim Pharmaceuticals Inc)
  
  
• 08:30-08:50 - Regulatory approach to surrogate end-points in cardiovascular diseases – which are acceptable and why? - Peter Mol
  
  
• 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? - Paulus Kirchhof, Eric Boersma
  
  
• 09:15-09:35 - Reimbursement based on surrogate end-points in cardiovascular diseases - Borislava Mihaylova
  
  
• 09:35-09:55 - Patient’s role in the assessment of risk-benefit in drug development – innovative cardiovascular drugs how much uncertainty is acceptable? - Mattias Van Heetvelde, ESC Patient Forum representative
  
  
• 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, Rapporteur: Clemens Mittmann
  • Group 2: Should all surrogate end-points be validated and how? - Lead: Jan Tijssen, Rapporteur: Paulus Kirchhof
  • Group 3: How to introduce conditional approval to CVD? - Lead: Peter Mol, Rapporteur: Faiez Zannad
    
    
• 10:55-11:05 - Break
  
  
• 11:05-11:15 - Report from breakout session 1 – 10 mins - Rapporteur: Clemens Mittmann
  
  
• 11:15-11:25 - Report from breakout session 2 – 10 mins - Rapporteur: Paulus Kirchhof
  
  
• 11:25-11:35 - Report from breakout session 3 – 10 mins - Rapporteur: Faiez Zannad
  
  
• 11:35-12:15 - General discussion - Panelists: Eva Prescott, Penilla Gunther, Clemens Mittmann, Peter Mol, Alar Irs, Borislava Mihaylova
  
  
• 12:15-12:30 - Wrap-up, conclusions, next steps for a publication - End of Day 2
  
  
• 12:30 - Buffet lunch and departures