Associação Brasileira de Hematologia, Hemoterapia e Terapia Celular Consensus on genetically modified cells. Special article: compassionate use and clinical trial on CAR-T cells

Abstract

There are only two ways for a patient to gain access to treatment with an experimental product, such as CAR-T cells: participate in a clinical trial or receive a product in a compassionate basis. In the first case, the main beneficiary is society itself, which may in turn obtain a new treatment paradigm for a specific disease. In the second case, the use of a medicinal product has the objective of care in benefit of patients in grave clinical condition, for which no approved medicinal products exist, or for which all the possibilities for benefit from standard therapies have been exhausted. The CAR-T cell therapy may be included in one or the other types of access. The compassionate use is not a specific type of clinical research and should therefore not have its use appreciated by a research ethics committee, but rather by the medical ethics committee at the institution where the treatment will take place and by the regulatory agency.

Keywords

• Compassionate use
• Clinical trial
• CAR T-cell therapy
• Cell therapy