Clinical outcomes in HTA appraisal reports of oncology medicines in Portugal: A cross-sectional analysis

IntroductionReimbursement appraisals of medicines should emphasize outcomes clinically relevant to patients and clinicians. This study aims to characterize the outcomes selected in oncology reimbursement appraisal reports in Portugal.MethodsA cross-sectional analysis of public INFARMED pharmacotherapeutic appraisal reports for oncology medicines published in 2023-2024 was conducted. For each included report, we extracted efficacy and safety outcomes, their clinical relevance rating (critical/important), whether data were presented for analysis, and which outcomes were prioritized in the final decision rationale. Descriptive statistics were employed.ResultsFifty-four oncology reimbursement appraisals met the inclusion criteria. Overall survival was rated critical in 52/54 reports (96.3%) and prioritized in 29 (53.7%). Progression-free survival was rarely rated critical (n = 2, 3.7%) and was more often deemed important (n = 49, 90.7%), being prioritized in 15 (27.8%) reports. Quality of life was rated as critical in 52 (96.3%) reports but prioritized in only one (1.8%) decision. For safety, grade 3 to 4 AEs and mortality were graded as critical in 51 (94.4%) reports each, followed by discontinuation due to AEs (n = 50, 92.6%), although none of the appraisals prioritized safety outcomes in the final reimbursement decision.ConclusionsOverall survival predominates as the most influential outcome in Portuguese oncology drug reimbursement decisions. Progression-free survival is frequently used as a practical surrogate. Although quality of life and safety outcomes are commonly prioritized during scope-setting, they are not consistently reflected in decision rationales, underscoring the need to strengthen outcome reporting and evidence generation to better inform patient-centered reimbursement decisions.