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Objective: To describe the authorisations and funding resolutions for new onco-hematological drugs in Spain between 2017 and 2020, as well as the results of their main trials., Methods: Observational, cross-sectional, descriptive study conducted between October and December 2022. Onco-hematology drugs approved by the European Medicines Agency between 2017 and 2020 were included, according to EFPIA patients W.A.I.T Indicator 2021 Survey. Authorisation information was obtained from the main study of the European Public Assessment Report (EPAR). Data were collected on medicines, their authorisation and main study, benefit shown, cost, and status and time to reimbursement., Results: Forty-one new drugs authorised for 49 indications were identified. More than half (58.5%) were targeted therapies, and 61.2% were for the treatment of solid tumors (61.2%). Most had palliative intent (71.4%) and were indicated in relapsed or refractory disease (55.1%). Of the clinical trials, 57.1% were phase III and 63.3% were randomised. The primary endpoint was overall survival in 16.3%, increasing to 25.8% among randomised clinical trials. Regarding licensed drugs based on response rate, the median response rate was 56.4% (IQI 40.0-66.3). In those authorised on the basis of surrogate time-to-event endpoints, the median Hazard Ratio was 0.54 (IQI 0.38-0.57), and among those using overall survival was 0.71 (IQI 0.59-0.77). Globally, 22.4% had shown benefit in overall survival, with a median gain of 4 months (IQI 3.6-16.7). One third (33.3%) of the indications evaluable according to the European Society for Medical Oncology Magnitude of Clinical Benefit Scale showed substantial clinical benefit. Of the indications, 75.5% were funded, half (48.6%; 36.7% of the total) with restrictions. The median time to funding was 19.5 months (IQI 11.4-29.3)., Conclusions: Most main clinical trials of new onco-haematology drugs approved in Spain used surrogate primary endpoint and, at the time of authorisation, few had shown to prolong overall survival. More than a third were uncontrolled clinical trials., (Copyright © 2024 Sociedad Española de Farmacia Hospitalaria (S.E.F.H). Publicado por Elsevier España, S.L.U. All rights reserved.)

Objective: To describe the authorisations and funding resolutions for new onco-haematological drugs in Spain between 2017 and 2020, as well as the results of their main trials., Methods: Observational, cross-sectional, descriptive study conducted between October and December 2022. Onco-haematology drugs approved by the European Medicines Agency between 2017 and 2020 were included, according to EFPIA patients W.A.I.T Indicator 2021 Survey. Authorisation information was obtained from the main study of the European Public Assessment Report. Data were collected on medicines, their authorisation and main study, benefit shown, cost, and status and time to reimbursement., Results: Forty-one new drugs authorised for 49 indications were identified. More than half (58.5%) were targeted therapies, and 61.2% were for the treatment of solid tumours (61.2%). Most had palliative intent (71.4%) and were indicated in relapsed or refractory disease (55.1%). Of the clinical trials, 57.1% were phase III and 63.3% were randomised. The primary endpoint was overall survival in 16.3%, increasing to 25.8% among randomised clinical trials. Regarding licensed drugs based on response rate, the median response rate was 56.4% [IQI 40-66.3]. In those authorised on the basis of surrogate time-to-event endpoints, the median hazard ratio was 0.54 [IQI 0.38-0.57], and among those using overall survival was 0.71 [IQI 0.59-0.77]. Globally, 22.4% had shown benefit in overall survival, with a median gain of 4 months [IQI 3.6-16.7]. One-third (33.3%) of the indications evaluable according to the European Society for Medical Oncology Magnitude of Clinical Benefit Scale showed substantial clinical benefit. Of the indications, 75.5% were funded, half (48.6%; 36.7% of the total) with restrictions. The median time to funding was 19.5 months [IQI 11.4-29.3]., Conclusions: Most main clinical trials of new onco-haematology drugs approved in Spain used surrogate primary endpoint and, at the time of authorisation, few had shown to prolong overall survival. More than a third were uncontrolled clinical trials., Competing Interests: Declaration of competing interest Hilario Martínez-Barros has received support from AMGEN to travel to and attend the Midyear Clinical Meeting 2023 of the American Society of Health-System Pharmacists, held in December 2023. The other authors declare no conflicts of interest., (Copyright © 2024 Sociedad Española de Farmacia Hospitalaria (S.E.F.H). Publicado por Elsevier España, S.L.U. All rights reserved.)