Case Studies
Introduction
This case study compendium showcases real-world examples of implementable solutions to address three challenge areas to CAR T-cell therapy mentioned in the report:
- Awareness and understanding of CAR T-cell therapy
- Resources and capacity to deliver CAR T-cell therapy
- Sustainable and innovative financing approaches to manage the costs of treatment and care
Europe: Developing HTA models to capture the value of CAR T-cell therapies
One persistent challenge for CAR T-cell therapies is that traditional HTA models are often not fit-for-purpose to capture the characteristics of CAR T-cell therapy (e.g., as a one-time treatment).133, 152T2EVOLVE is a consortium working to accelerate the process of developing CAR T-cell therapy in the European Union (EU) to support patient access to this type […]
US: Reviewing clinical accreditation standards to ensure patient access
The Foundation for the Accreditation of Cellular Therapy (FACT) has announced the formation of a ‘Community CAR T Working Group’ (working group) to discuss and propose clinical accreditation standards for community-based providers of CAR T cell therapies. 151 The working group builds on discussions around the multiple, complex challenges surrounding eligible patient access to these innovative […]
Austria: Expanding equitable access to CAR T-cell therapy
Although CAR T-cell therapy has been available in Austria since 2019, access remained limited, with only a small proportion of eligible patients receiving treatment by 2021.141 Identified barriers included restrictive patient selection criteria, suboptimal referral pathways, and limitations in funding and infrastructure.140 To address these challenges, a national policy initiative was launched by stakeholders from […]
US: Streamlining onboarding processes for CAR T-cell treatment centers
The US Food and Drug Administration (FDA) mandates product-specific education and training to ensure the safe use of CAR T-cell therapies.104 This is delivered through the Risk Evaluation Mitigation Strategies (REMS) program, which involves manufacturers evaluating and monitoring treatment sites to ensure compliance.104 As the number of approved CAR T-cell therapies increases, treatment centers will […]
Europe: Driving coordinated European action on cellular therapy access
In 2020, the European Hematology Association (EHA) and the European Society for Blood and Marrow Transplantation (EBMT) founded the GoCART Coalition – a European group of patient representatives, HCPs, pharmaceutical companies, regulators and medical organizations working together to promote patient access to novel cellular therapies. The GoCART Coalition is organized around five key pillars: data […]
Italy: Enabling rapid funding decisions
In Italy, funding for CAR T-cell therapy and associated care must be approved by regional authorities.156 For patients referred to treatment centers outside of their home region, approvals must be obtained from authorities in the referring and receiving region, with costs ultimately billed to the patient’s home region.155 This process is time-consuming and can lead […]
UK: Leveraging the power of cross-center collaboration
Delivering innovative therapies often involves new ways of working for healthcare systems, including the National Healthcare Service (NHS) in the UK. CAR T-cell therapy is delivered by select treatment centers, which are formally evaluated and accredited. Standards are set by the Joint Accreditation Committee (JACIE) in Europe for consistent quality and safety.101 The UK’s Advanced […]
Austria: Expanding equitable access to CAR T-cell therapy
Although CAR T-cell therapy has been available in Austria since 2019, access remained limited, with only a small proportion of eligible patients receiving treatment by 2021. Identified barriers included restrictive patient selection criteria, suboptimal referral pathways, and limitations in funding and infrastructure.140 To address these challenges, a national policy initiative was launched by stakeholders from […]