by Tuula Rintala
Director, Quality of Care and Advocacy
EBMT, European Society for Blood and Marrow Transplantation
As the Joint Accreditation Committee ISCT-Europe & EBMT (JACIE) celebrates its 25th Anniversary, it is a good time to reflect on some of the changes in the field of haematopoietic stem cell transplantation and cellular therapies during these 25 years. Many of the challenges in cellular therapies such as regulatory frameworks, rapid product development, centre qualification and education, to name a few, present a challenge from the accreditation perspective. Further, as the field develops and the treatment options open up for other than haematology indications, new challenges and opportunities open up.
Therefore, it is vital that JACIE collaborates with other stakeholders in the field through initiatives such as the GoCART Coalition. Such collaboration helps JACIE to stay informed about the latest developments, and to share the learning and experiences JACIE has gained during our first 25 years.
The GoCART Coalition was founded in 2020 as an initiative from Jurgen Kuball (UMC Utrecht) and Christian Chabannon (Aix-Marseille University) on behalf of the European Society for Blood and Marrow Transplantation (EBMT) with the European Haematology Association (EHA) joining as a co-founder. Advancing the field of cell and gene-based therapies requires progress on multiple fronts, including creating a pre- and post-authorisation registry to monitor product safety and efficacy; harmonising data collection, centre qualification and educational requirements; overcoming barriers in regulations designed for more traditional pharmaceutical products that affect preparation, administration and patient access to these novel therapies. To address such a wide range of complex issues, there is a need for strong collaboration amongst all relevant stakeholders. The aim of the GoCART coalition is to support patients’ access to novel gene and cellular therapies manufactured from cells and tissues of hematopoietic origin, and to contribute to their health and well-being through innovation by multi-stakeholder collaboration on clinical data, standards of care, education and policy.
The GoCART Coalition’s mission is to be a leading force in the field of cellular therapies and trusted partner to promote patient access to novel cellular therapies and to contribute health and well-being through multi-stakeholder collaboration on clinical data, standards of care, education and policy. This is realised through GoCART’s Vision to create a platform driven by input of a diverse group of stakeholders who jointly develop projects that advance the field of cellular therapies in Europa.
The aims of the GoCART Coalition are to:
- Improve health outcomes for patients;
- Engage stakeholders and establish a sustainable European coalition in the field of cellular therapy;
- Collaborate and share data and knowledge to prevent duplication of effort and resources;
- Promote harmonization of data collection, education, standards of care and policies in Europe;
- Support the set-up and maintenance of a pre-and post-authorisation registry that informs decision-making and supports shared research purposes;
- Develop a cellular therapy Education and Information program for patients and health care professionals;
- Harmonise standards of care and centre qualification;
- Advance policies that further the shared mission and vision.
The GoCART Coalition is open to all stakeholders relevant to achieve its mission. There are two categories of participation: institutional participants and individual participants. An institutional participant is considered to be a legal entity such as an association or company. Individuals who support the Coalition’s mission and vision and want to participate in the Coalition’s activities can become individual participants. All participants contribute their time to the GoCART Coalition pro bono.
GoCART Work Packages
The GoCART Coalition organizes its activities through its work packages (WP). The work packages cover an area of interest of the Coalition and ensure the implementation of the strategy. Within work packages, smaller work groups may be established to conduct specific projects. The current work packages and the overall aims are listed below:
WP: Data harmonisation
In Europe, clinical data from patients treated with gene and cellular therapies are reported to many registries, each built for a limited purpose, with different governance rules and specific software tools managing the data. This results in siloed data, inefficiencies and duplication of efforts.
Overall aim: Create a central EU data registry for the harmonised collection of clinical data on patients treated with cellular therapies to support collaborative studies and regulatory decision making.
To date this Work Package has carried out a number of multi-stakeholder meetings and a revision of the EBMT Cellular Therapies data collection form between 2020-2022. Final draft was presented to stakeholders in March 2022. First key elements have been implemented in 2022 in the forms used for the new registry. Additional, yet not implemented fields will become part of the annual renewal process of updating forms. The process of upcoming changes will be discussed with all stakeholders and implemented in 2023/2024.
WP: Standards of care
Gene and cellular therapies are inherently complex products and treatment administration is restricted to qualified centres. With the rapid developments and pending product approvals, there is a need for developing treatment guidelines and harmonising centre qualification procedures across pharmaceutical companies, accreditation bodies and national requirements.
Overall aims:
1) to develop harmonised guidelines on patient and product management for health care professionals.
2) to reduce inspection burden and redundancies by developing and implementing consensus-driven requirements and qualification standards for clinical teams delivering gene and cellular therapies from cells and tissues of hematopoietic origin.
To date Work Package 2 worked on a number of projects in 2022.
The GoCART Coalition announced on 8th March 2022 a new initiative to allow apheresis and cell therapy treatment centres accredited by JACIE to leverage their accreditation as part of site qualification processes for Bristol Myers Squibb, Janssen Pharmaceuticals, Kite Pharma and Novartis. Subject to marketing authorisation holders' agreement and obligations, centres holding JACIE accreditation have the option of undergoing a reduced or remote audit focused on product-specific requirements for cell therapies.
GoCART Coalition Pharmacist Working Group has developed a practical guidance on implementation and safe operational use of marketed CAR-T cell products within hospital pharmacies primarily throughout Europe. The guideline outlines the key areas where pharmaceutical expertise should focus on as well as key considerations for the hospital pharmacies. A manuscript has been accepted for publication and is expected to be published shortly.
GoCART Coalition Apheresis Working Group has published recommendations for the collection of T-cells by apheresis for ATMP manufacturing.
Worel, N., Holbro, A., Vrielink, H. et al. A guide to the collection of T-cells by apheresis for ATMP manufacturing—recommendations of the GoCART coalition apheresis working group. Bone Marrow Transplant (2023). https://doi.org/10.1038/s41409-023-01957-x.
A second manuscript is in development focussing on processing issues including sampling and labelling and the cryopreservation of these products.
WP: Education and training
Gene and cellular therapies are complex products that require comprehensive and ongoing training of health care professionals as well as patients and caregivers. A plethora of training courses are already offered by MAHs as well as health organisations, which can lead to considerable overlap.
Overall aim: develop harmonised educational programmes for different groups of health care professionals and patients.
In 2022, GoCART continued with the two established series of monthly educational webinars. In the Clinical Case Discussion series for physicians two cases are presented and discussed with experts in the field of CAR T-cell therapies. The aim is to discuss real-life clinical cases as instructive examples, foster lateral thinking to meeting participant’s own cases, contribute to a broader understanding of CAR T-cell treatments, toxicity management and outcomes. Ten sessions and twenty clinical cases were discussed in 2022.
The Nurses Best Practice Sharing Sessions focus on different aspects of the patient journey and/or the care nurses provide. In the meetings a nurse expert will present the theoretical background information and present a real-life case. In 2022 the group hosted six Best Practice Sharing Sessions.
Plans for the future work include exploring harmonisation of training requirements with GoCART Industry partners.
WP: Policy and advocacy
Context: Gene and cellular therapies are subject to EU and national regulations affecting their preparation, administration and patient access. These new therapies challenge the regulations which were designed for more traditional pharmaceutical products and health authorities are assessing how they will adapt.
Overall aim: represent and promote the interests of the GoCART coalition and its stakeholders in EU policy making by engaging with EU institutions and other relevant stakeholders.
GoCART Coalition continued to contribute to the response by its founding organisations to the draft proposal by the European Commission on the Revision of the EU legislation on Blood, Tissues and Cells. Key areas identified were the introduction of the joint regulation approach promoting harmonisation across the Member States as well as the principles of voluntary unpaid donations and donor protection measures.
Further plans include developing a shared policy and advocacy strategy to advocate key coalition positions with the policy makers in order to contribute to European policy making on gene and cellular therapy topics including Substances of Human origin (SoHO) regulation and Hospital Exemption.
WP: Scientific excellence
Context: Scientific research on gene and cellular therapies increased substantially over the last years. With Real World Data becoming increasingly available, many scientific questions, from different perspectives, can be explored. Only by working together can we leverage enough data to conduct meaningful research. GoCART wants to maximise the use of data collected in the central Registry as well as data available to other stakeholders, and to facilitate further collaboration between stakeholders. While strongly protecting confidentiality, the guiding principle should be ‘collect once, use often’ to advance our knowledge in the field of gene and cellular therapies, support better decision making and drive efficiencies for all stakeholders.
Overall aim: stimulate scientific discussion across stakeholders, facilitate the set-up of joint research projects and avoid duplication of scientific efforts.
To date, GoCART Coalition has carried out two calls for research studies with six winning studies now in different phases of completion. An update on the current state of the studies was provided at the EBMT Annual Meeting in a Special Session on GoCART ongoing projects. The selected projects have been supported for biostatistical support to carry out their studies by a dedicated GoCART data team at the EBMT.
The Selection Committee, Prof Anna Sureda (President of the EBMT), Dr Annalisa Ruggeri (Chair EBMT Cellular Therapies and Immunobiology Working Party), Prof John Gribben (Past President of the EBMT), Prof Martin Dreyling (Board member of EHA) and Natacha Bolanos (Chair EBMT patient advocacy committee selected the winning three proposals from total of sixteen scientific projects in autumn 2022.
The next call for studies is planned for late 2023.
How to get involved:
If you are interested in joining the coalition and the GoCART activities and work with different stakeholders to maximise the potential of cellular therapies, please visit thegocartcoalition.com.
To join the coalition, please review the Governance Charter and complete the form with your details as well as how you would like to contribute to GoCART’s overall aims.
GoCART is also planning a limited series of ‘GoCART meets and connects’ – meetings with the national CAR-T/ATMP initiatives. The first of these meetings will take place in May with GoCART joining in the national meeting in Sweden.
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