Brussels, 19 May 2021 - Advanced therapies have ground-breaking potential by opening up new treatment horizons in diseases with limited or absent available treatments. To better explain the transformational value of cell and gene therapies, EuropaBio is launching a series of events and other public communications this year. An inaugural event ‘Taking health up a gear: value of advanced therapies’ took place on 11 May 2021 and engaged patient representatives, policy makers, and therapy developers in a discussion on the benefits of Advanced Therapy Medicinal Products (ATMPs) to patients, their families, and healthcare systems.

The first part of the discussion focused on the break-through science behind the development of gene therapies for rare diseases. Dirk Vander Mijnsbrugge from Pfizer Rare Diseases explained why gene therapies are the medicine’s most suitable tool so far to tackle complex genetic rare diseases and proposed existing barriers. “With gene therapy, we have an exceptional opportunity to target rare diseases which in the end may become curative.”, said Vander Mijnsbrugge.

Simone Boselli, EURORDIS stressed that the EU Pharmaceutical Strategy opens an excellent window of opportunity to make gene therapies more accessible to patients. ’To realise the full potential of ATMPs, we need innovation not only in scientific development, but also in legislative and policy processes. The current regulation creates barriers to treatment in Europe.’, said Boselli.

In order to understand and overcome these challenges and prosper in tomorrow’s bioeconomy, for more than ten years, industry experts throughout the vibrant and innovative biobased community have come together to one central location every year to discuss the key issues and gain a snapshot of the most significant developments on scale up of commercial biorefineries around the world.

The second panel focused on the innovative cancer treatment cell-based therapies, the so-called CAR-Ts. Dick Sundh from Kite Pharma, a Gilead Company stressed the importance of employing a holistic approach on how cancer is treated and cured. Europe supports scientific collaboration between Member States very well, but improvement is needed, especially in terms of better cooperation on health technology assessment, infrastructure, and logistics for innovative European manufacturing of cell therapies. Sundh emphasised the importance of informing citizens : ‘People have to be informed of the awaiting opportunities arising from innovation. Patient organisations, the scientific community and experts have a crucial role to play in this’, he added. His remarks were supported by Sara Cerdas, MEP who gave a short overview of the EU4Health instrument and the objectives of the EU Pharmaceutical Strategy. ‘Health literacy provides citizens with the capacity to understand the choices they make and how they affect their health.’, concluded Cerdas.

As EuropaBio, we aim to raise the awareness of how biotechnology-driven innovation in cell and gene therapies is delivering transformative treatments to patients at critical junctures, and how this wave of medical innovation necessities policy and regulatory changes to fully tap into its potential to address unmet patient needs in the EU. The next event of the EuropaBio Advanced Therapies Series will focus on the topic of EU patient access to cross-border healthcare with gene and cell therapies. More information will follow.