Pre-Conference Workshop Day | Tuesday, 25 October

WORKSHOP A: 9.00 am - 12.00 pm

Tackling Safety Concerns Surrounding Gene Therapy Development & Administration

Several gene therapies currently in development are targeting rare and ultra-rare diseases. Given the significant challenges associated with developing trials with small patient numbers compounded with the complexity of genetic therapies, qualifying risk, and knowing how to meet regulatory requirements, understanding how to demonstrate product safety is paramount to product approval chances.

Attend this workshop to learn about:

• How to better qualify patient risk-benefit ratios and maximise regulatory approval
• How to finetune preclinical and clinical research to maximise patient safety
• Navigating a lack of available data in the rare disease space to define clinical endpoints
• What efforts are being made to standardise regulatory safety requirements?
• How to effectively set up safety and efficacy follow-up strategies for ATMPs

Workshop Leaders:

Ivan Gorbachov - Novartis

Ivan Gorbachov, Senior Director, Novartis

Oscar Segurado - ASC Therapeutics

Oscar Segurado, Chief Medical Officer, ASC Therapeutics

WORKSHOP B: 1.00 pm - 4.00 pm

The Committee for Orphan Medicinal Products of the EMA – the Role of Members Representing Patient Organisations

Analysis conducted by the EMA has proven that participation in their COMP meetings of patient organisation representatives improves not only the quality of the scientific advice and opinions given by the committee, but also increases transparency and trust in regulatory processes by the community. To explore this further, we are excited to have on-board from COMP, members representing patients’ organisations, as well the Committee’s Chair herself.

Attend this workshop to learn about:

• An outline of the role and how they give voices to patient needs in the decision making process of the committee
• How their advocacy helps to introduce real-life implications into regulatory decisions
• Their role in communicating the risk-benefit attitudes of patients
• Their interactions with the committee and how this shapes its decision-making process
• Examples of previous COMP cases with patient organisation representative involvement

Workshop Leaders:


Violeta Stoyanova-Beninska, Chair of Committee for Orphan Medicinal Products, EMA


Inês Alves, Patient Expert & Member of the Committee for Orphan Medicinal Products, EMA


Angelo Loris Brunetta - IAAC

Angelo Loris Brunetta, Coordinator, Italian Associations Advocacy Council