Inter-hospital heterogeneity in the authorizations of off-label cancer drugs: A project by Associazione Italiana di Oncologia Medica
PurposeThis project aimed to describe variability of local evaluations of off-label cancer drug requests by submitting a list of identical requests to different Italian centers.MethodsIn October 2024, oncology unit directors listed in the AIOM Libro Bianco were invited to participate, evaluating 11 clinical cases with solid tumors through their internal approval process. Supporting evidence was retrospective in two cases, based on phase II trials in seven, and phase III trials in two. Inter-rater agreement was measured using Fleiss' kappa.ResultsSeven centers completed evaluations. Two cases were approved by all centers, both with a rare cancer with scant alternatives. The mean number of centers approving the individual requests was 5.36/7 (76.6%) with a poor inter-rater agreement (kappa 0.009, 95% CI -0.120 to 0.138). Single centers approved a mean of 8.4 requests (76.6%). The mean number of centers that approved requests was 5.5 for requests based on retrospective studies, 5.3 for those based on phase II trials, and 5.5 for those based on phase III trials. There was no correlation between estimated cost and chance of approval.ConclusionsThere is a significant inter-hospital variability in off-label oncology drug evaluations, leading to potential disparities in patient's access to care.
Authors
- Massimo Di Maïo (ORCID: https://orcid.org/0000-0001-8906-3785)
- Matilde Scaldaferri (ORCID: https://orcid.org/0000-0002-5210-9783)
- Marina Campione (ORCID: https://orcid.org/0000-0003-0582-5303)
- Alessandra Gennari (ORCID: https://orcid.org/0000-0002-0928-2281)
- Giordano Beretta (ORCID: https://orcid.org/0000-0002-8427-7765)
- Alessandra Zanardi
- Gianmauro Numico (ORCID: https://orcid.org/0000-0003-3253-2960)
- Nicola Personeni (ORCID: https://orcid.org/0000-0002-7995-272X)
- Alessia Pisterna
- Claudia Fruttero
- Marcello Tiseo (ORCID: https://orcid.org/0000-0002-9553-8465)
- Livio Blasi (ORCID: https://orcid.org/0000-0001-7150-1302)
- Anna Amela Valsecchi (ORCID: https://orcid.org/0009-0007-4940-5761)
- Maria Rachele Chiappetta
- Gianluca Russo (ORCID: https://orcid.org/0000-0003-2698-9470)
- Francesco Perrone (ORCID: https://orcid.org/0000-0002-9738-0526)
- Giulia Dusi
Institutions
- Università degli Studi del Piemonte Orientale “Amedeo Avogadro” (IT)
- Azienda Ospedaliera Citta' della Salute e della Scienza di Torino (IT)
- Fondazione Ricerca Molinette (IT)
- Casa di Cura Villa Garda (IT)
- Associazione Italiana Di Oncologia Medica (IT)
- Azienda Ospedaliero Universitaria Maggiore della Carita (IT)
- Presidio Ospedaliero (IT)
- Ospedale di Parma (IT)
- Azienda Sanitaria Ospedaliera S.Croce e Carle Cuneo (IT)
- Istituto Nazionale Tumori IRCCS "Fondazione G. Pascale" (IT)
- Azienda Ospedaliera Ospedale Maggiore (IT)
- Azienda USL di Pescara (IT)
- La Maddalena (IT)
Publication Details
- Journal
- Tumori Journal
- Published
- 2025-10-18
- DOI
- https://doi.org/10.1177/03008916251386285
- Primary Topic
- Pharmaceutical studies and practices
- Type
- article
- Field-Weighted Citation Impact
- 0.00