{"entity":{"id":"paper-trias-hospital-exemption-ari-0001-bmt-2022","kind":"paper","name":"The hospital exemption pathway for advanced therapies and the case of the CAR-T ARI-0001","aka":[],"tldr":"How Hospital Clínic got its own CAR-T authorised in Spain through a route in European law that lets a hospital make an advanced therapy for its own patients, and what stands in the way of others doing the same.","summary":"Published as \"The hospital exemption pathway for the approval of advanced therapy medicinal products: an underused opportunity? The case of the CAR-T ARI-0001\". The authors, from Hospital Clínic de Barcelona's Advanced Therapies Unit, describe how in February 2021 the advanced therapy medicinal product ARI-0001 (CART19-BE-01), developed at the hospital, received authorisation from the AEMPS under the hospital exemption pathway for patients aged over 25 with relapsed or refractory acute lymphoblastic leukaemia. The hospital exemption, foreseen in the European regulation on advanced therapies, allows access to products prepared on a non-routine basis, to quality standards, for an individual patient, limited to the member state where the product was developed and used in a hospital under a medical practitioner's responsibility. Such products must meet national traceability, pharmacovigilance and quality requirements.","asOf":"2026-11-03","links":[{"label":"Bone Marrow Transplant 2022","url":"https://doi.org/10.1038/s41409-021-01463-y"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/35046545/"}],"tags":[],"related":[],"cancers":[],"sections":[],"technologies":["car-t","point-of-care-cell-manufacturing"],"targets":[],"drugs":["varnimcabtagene-autoleucel"],"companies":[],"institutions":["hospital-clinic-barcelona","aemps"],"pathways":[],"terms":[],"trials":["cart19-be-01"],"people":["manel-juan"],"bottlenecks":["b-manufacturing-cell-therapy"],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"journal":"Bone Marrow Transplantation","year":2022,"doi":"10.1038/s41409-021-01463-y","pmid":"35046545","authors":"Trias E, Juan M, Urbano-Ispizua A, Calvo G","paperType":"review","findings":["ARI-0001 was authorised by the AEMPS under the hospital exemption in February 2021 for patients aged over 25 with relapsed or refractory acute lymphoblastic leukaemia.","Use under the hospital exemption is limited to the member state where the product was developed, in a hospital, under the responsibility of a medical practitioner.","The authors argue the route offers close contact with clinical practice, the quality and rapid access patients need, and lower cost than a regular marketing authorisation, while barriers remain because member states implement it differently."],"whatItMeans":"Spain's use of the hospital exemption is a working model for public hospitals making cell therapies at lower cost; the paper is the account of how it was done by the people who did it.","caveats":["A perspective article by the hospital that developed the product; it reports no new patient outcomes and makes no cost figure public."]},"route":"/key-papers/paper-trias-hospital-exemption-ari-0001-bmt-2022/","neighbours":{"technology":[{"id":"car-t","kind":"technology","name":"CAR-T cell therapy","route":"/technologies/car-t/"},{"id":"point-of-care-cell-manufacturing","kind":"technology","name":"Point-of-care and decentralised cell manufacturing","route":"/technologies/point-of-care-cell-manufacturing/"}],"drug":[{"id":"varnimcabtagene-autoleucel","kind":"drug","name":"Varnimcabtagene autoleucel","route":"/drugs/varnimcabtagene-autoleucel/"}],"institution":[{"id":"hospital-clinic-barcelona","kind":"institution","name":"Hospital Clínic de Barcelona / IDIBAPS","route":"/institutions/hospital-clinic-barcelona/"},{"id":"aemps","kind":"institution","name":"Spanish Agency of Medicines and Medical Devices (AEMPS)","route":"/institutions/aemps/"}],"trial":[{"id":"cart19-be-01","kind":"trial","name":"CART19-BE-01 (ARI-0001 CAR-T, Hospital Clínic de Barcelona)","route":"/trials/cart19-be-01/"}],"person":[{"id":"manel-juan","kind":"person","name":"Manel Juan","route":"/people/manel-juan/"}],"bottleneck":[{"id":"b-manufacturing-cell-therapy","kind":"bottleneck","name":"Manufacturing cost and time for living and radioactive medicines","route":"/bottlenecks/b-manufacturing-cell-therapy/"}]}}