As more gene and cell therapies enter the market, rare disease advocates in the European Union want to help pave the way for patients to access potentially life-changing treatments.
In two new episodes of the New Horizon Talks podcast, experts explore the EU’s leadership in rare disease innovation. CSL Behring created the audio series in 2021 to feature discussions about cell and gene therapy among leading researchers and advocates and policy stakeholders. The new episodes launch amid political debates on setting up the European Health Data Space – a major pillar of ongoing efforts to build a European Health Union. Debate at the European Health Forum Gastein also explored the role of this ambitious European health data ecosystem, in relation to real world evidence and rare disease.
Episode 5 features Yann Le Cam, CEO of EURORDIS and Elisabetta Zanon, Director, EU Public Affairs & Advocacy, Alliance for Regenerative Medicine. On the podcast, they discuss how Europe can address the unmet medical needs of rare disease patients. "Europe has an unequivocal need for a regulatory ecosystem that supports innovation and ensures patients" access, Le Cam says. Speeding up access will require collaboration and exploration of outcomes-based payment models – alongside quality evidence on long-term effectiveness, the experts said.
“In terms of speeding up access to innovative therapies, innovative payment models should be considered,” Zanon said. “Evidence on the long-term effectiveness of innovative therapies is lacking at the moment of launch so it is important to look at models that help addressing uncertainties, for example, using real-world data.”
Episode 6 digs in further on real world data and features Marcus Guardian, Chief Operating Officer of EUnetHTA and Professor Marijke van den Berg, Director of the European Paediatric Network for Haemophilia Management (PEDNET). They discuss paths to a qualitative data exchange that fosters research, innovation, and ultimately, positive patient outcomes. Collaboration will be key.
“In order to collect the high-quality data for the treatment of rare diseases, a more direct collaboration between regulators and stakeholders is required. Only by making this link can we allow for better and greater data generation,” van den Berg said.
Regarding the potential of the future European Health Data Space, Guardian notes that several regulatory challenges will have to be addressed. They include harmonizing standards for evidence creation and monitoring long-term outcomes of innovative therapies.