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Shaping the future of cell therapies in Portugal
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Resumo(s)
Background: Chimeric Antigen Receptor T-cell (CAR-T) therapy is an innovative therapeutic approach in the treatment of B-lineage lymphoid neoplasms, especially in cases where first-line therapeutic options are ineffective or lose their efficacy over time. The journey of the patient eligible to receive CAR-T cell therapy is complex, presenting some significant challenges to its accessibility. Purpose: This study aims to reflect on and discuss the future of cell therapies in Portugal, with a particular focus on CAR-T cell therapy. It aims to promote the development of a strategic agenda with a potential impact on the definition of public policies that improve access to this technology and on scientific and clinical progress in CAR-T cell treatment. Method: A targeted literature review was conducted, followed by a Think Tank approach resorting to design thinking techniques. The study was led by a research team at NOVA Information and Management School (NOVA University of Lisbon) and guided by a Steering Committee of four national experts. The Think Tank consisted of three meetings involving 26 participants from the Portuguese healthcare sector, including health professionals, patients, caregivers, researchers, and policy and executive decision-makers. The first meeting focused on the evidence, diagnosis, and referral process for CAR-T therapy-eligible patients; the second examined financial models; and the third discussed the management and governance of the patient journey. Results: Following the results of the three Think Tank sessions, five key axes of action were identified to improve access to cell therapies in Portugal. The first axe of action is to invest in the capacity building of healthcare professionals and services. The second is to establish a centralized financing program. The third is to create a national referral network for cell therapies and standardize treatment eligibility criteria. The fourth is to provide comprehensive socio-economic, logistical, and training support for patients, caregivers, and families. Lastly, the fifth axe of action is to strengthen the ability to generate and share real-world evidence in the field of cell therapies. Implementing the proposed recommendations is expected to enhance the patient’s journey, which is crucial in the experience of a cancer patient whose first lines of treatment have already failed. Conclusion: The findings and recommendations from this study aim to inform policy decisions and foster adoption of cell therapies for patients living in Portugal. Although the study focuses on a national case study, its methodology, which involves engaging all parties' perspectives in reflecting on the adoption of personalized therapies, may offer valuable insights for adaptation in other countries.
Descrição
Moura, L., Castro, M. D., & Lopes, H. (2025). Shaping the future of cell therapies in Portugal: Defining a strategic agenda for improved access and policy development [abstract]. Pharmacy Education, 25(4), 5-6. https://doi.org/10.46542/pe.2025.254.vrs.129
Palavras-chave
SDG 3 - Good Health and Well-being