Clinical Outcomes In HTA Appraisal Reports Of Oncology Medicines In Portugal: A Cross-Sectional Analysis
Author(s)
Pedro Teodoro, PharmD1, Diogo Mendes, PhD2, Carlos Alves, PhD3.
1Faculty of Pharmacy, University of Coimbra, Coimbra, Portugal, 2Clevidence, Oeiras, Portugal, 3Laboratory of Social Pharmacy and Public Health, Faculty of Pharmacy, University of Coimbra, Coimbra, Portugal.
1Faculty of Pharmacy, University of Coimbra, Coimbra, Portugal, 2Clevidence, Oeiras, Portugal, 3Laboratory of Social Pharmacy and Public Health, Faculty of Pharmacy, University of Coimbra, Coimbra, Portugal.
OBJECTIVES: In Portugal, the pharmacotherapeutic evaluation from the Health Technology Assessment (HTA) process applies the GRADE methodology, ensuring that the outcomes with greater clinical impact are central to the assessment of therapeutic value. This study aims to characterize the efficacy and safety outcomes considered in the HTA processes of oncology medicines to support reimbursement decisions.
METHODS: Analysis considered public reimbursement reports of oncology medicines published in 2023 and 2024 on the website of the Portuguese authority (INFARMED, IP). The scopes of the assessments (i.e., PICO matrices) were analyzed to retrieve the efficacy and safety outcomes, as well as their clinical relevance.
RESULTS: Fifty-five reports were analyzed. Quality of Life (QoL) was identified as a critical outcome in 89.1% of cases (n=49 reports) but it was prioritized for reimbursement decisions in only 3.6% (n=2). Overall survival (OS) was a critical outcome in 96.4% (n=53) and it was prioritized in 54.5% (n=30) of the reports. Progression-free survival (PFS) was a critical outcome in only 3.6% of the reports (n=2), yet it was prioritized in 43.6% (n=24) of reimbursement decisions. Regarding safety, mortality was cited as a critical outcome in 90.9% of the reports (n=50), severe or grade 3-4 adverse events in 92.7% (n=51) and discontinuation due to adverse events in 90.9% (n=50). None of the reimbursement reports prioritized safety over efficacy outcomes for reimbursement decisions.
CONCLUSIONS: OS predominates as the most clinically relevant outcome in the reimbursement of oncology medicines. When absent, PFS is preferred, despite not always being critical. QoL, often critical, is rarely used due to data sparsity. Safety outcomes, though frequently critical, are underutilized in decisions, even when data on critical efficacy outcomes are missing. Greater integration of safety outcomes into HTA evaluations warrants debate, given their influence on patient quality of life and clinical outcomes.
METHODS: Analysis considered public reimbursement reports of oncology medicines published in 2023 and 2024 on the website of the Portuguese authority (INFARMED, IP). The scopes of the assessments (i.e., PICO matrices) were analyzed to retrieve the efficacy and safety outcomes, as well as their clinical relevance.
RESULTS: Fifty-five reports were analyzed. Quality of Life (QoL) was identified as a critical outcome in 89.1% of cases (n=49 reports) but it was prioritized for reimbursement decisions in only 3.6% (n=2). Overall survival (OS) was a critical outcome in 96.4% (n=53) and it was prioritized in 54.5% (n=30) of the reports. Progression-free survival (PFS) was a critical outcome in only 3.6% of the reports (n=2), yet it was prioritized in 43.6% (n=24) of reimbursement decisions. Regarding safety, mortality was cited as a critical outcome in 90.9% of the reports (n=50), severe or grade 3-4 adverse events in 92.7% (n=51) and discontinuation due to adverse events in 90.9% (n=50). None of the reimbursement reports prioritized safety over efficacy outcomes for reimbursement decisions.
CONCLUSIONS: OS predominates as the most clinically relevant outcome in the reimbursement of oncology medicines. When absent, PFS is preferred, despite not always being critical. QoL, often critical, is rarely used due to data sparsity. Safety outcomes, though frequently critical, are underutilized in decisions, even when data on critical efficacy outcomes are missing. Greater integration of safety outcomes into HTA evaluations warrants debate, given their influence on patient quality of life and clinical outcomes.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
CO49
Topic
Clinical Outcomes, Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Clinical Outcomes Assessment, Comparative Effectiveness or Efficacy
Disease
Oncology