4CPS-170 Health outcomes assessment of an interdisciplinary pharmaceutical onco-haematology committee

<h3>Background and Importance</h3> Antineoplastic treatments with a high health and/or economic impact (HHEI) request in ‘extraordinary’ use situations demand interdisciplinary assessment by a specialised committee on pharmaceutical onco-haematology. It is necessary to establish a standardised methodology that incorporates comprehensive patient assessment, clinical benefit and pharmacoeconomic analysis into clinical decision making. <h3>Aim and Objectives</h3> To evaluate the use of antineoplastic treatments with a HHEI request in ‘extraordinary’ use situations from an Inter-Multidisciplinary Committee of Pharmaceutical Onco-Haematology (CIMOF). To identify patients which clinical benefit similar outcomes to pivotal clinical trials. <h3>Material and Methods</h3> Retrospective observational 4 year (1 January 2020 to 30 December 2023) study conducted in a tertiary hospital. Antineoplastic treatments with a HHEI request in ‘extraordinary’ use situations have required an assessment by CIMOF were included. Follow-up was carried out until 1 October 2024. <h3>Variables collected</h3> demographic data (age, sex), clinical data (performance status (PS), diagnosis, type and line of treatment), ESMO clinical benefit scale, GRADE quality of evidence scale and resolution request. Treatment discontinuation and reason for discontinuation (Limitation of therapeutic effort (LTE), toxicity, progression) were also collected. Clinical benefit was determined as the number of antineoplastic treatments that had achieved clinical trial-like effectiveness results in real-life. Patients who discontinued treatment due to toxicity or insufficient follow-up were excluded. <h3>Results</h3> Out of 311 treatment requests received, 45 were rejected for various reasons before evaluation. Ultimately, 266 requests were evaluated, of which 261 were approved. Finally 254 treatments were started: mean age 64±12.9 years old, 52.0% men and 74.4% PS1. Of these, 16.1% and 9.4% were diagnosed with lung cancer and prostate cancer, respectively. Pembrolizumab (12.9%) and nivolumab (10.4%) were more frequently. Among all, 46.5% were requested in second-line and 78.4% with palliative intent. Relevant clinical benefit was estimated in 100% and 61.0% of curative (ESMOA-B) and palliative (ESMO4–5) requests, respectively. The quality of evidence was high in 79.8% of requests. Of the 74.8% of treatment discontinuations, the main reasons were progression (41.6%), LTE (25.3%) and toxicity (21.6%). A 52.0% of antineoplastic treatments achieved clinical benefit in real-life of patients. <h3>Conclusion and Relevance</h3> The assessment of HHEI antineoplastic treatments by an inter-multidisciplinary committee allows access to these therapies in extraordinary use situations. Moreover, 52% of these achieved clinical benefit in real-life of patients. <h3>References and/or Acknowledgements</h3> <h3>Conflict of Interest</h3> No conflict of interest