AMPLIFY: Estimating Clinical and Humanistic Benefits of Pembrolizumab in Portugal

BACKGROUND: Pembrolizumab has become a cornerstone in the treatment of multiple tumor types. In Portugal, it is currently reimbursed across 25 indications. However, a comprehensive holistic assessment of its cumulative clinical and humanistic impact is lacking. OBJECTIVE: The aim was to estimate the population-level clinical and humanistic outcomes associated with pembrolizumab use in Portugal from 2017 to 2024, and to project its potential future impact until 2030. METHODS: We developed a Microsoft Excel-based tool to aggregate outputs from validated, Portugal-adapted, cost-effectiveness models across all reimbursed indications for pembrolizumab. Using a real-world number of treated patients, clinical outcomes such as incremental life years (LYs), incremental quality-adjusted life years (QALYs), and avoided deaths were estimated. Forecasts through 2030 were developed using market and epidemiological projections. Alternative scenarios evaluated the additional benefits of accelerated reimbursement decisions and expanded patient access. RESULTS: Between 2017 and 2024, among 13,530 treated patients, pembrolizumab was associated with estimated lifetime outcomes of 14,042 incremental QALYs, 17,914 incremental LYs, and 2021 deaths avoided. Scenario analyses showed that with accelerated reimbursement, within 180 days of market approval, and full uptake, cumulative gains could have increased by up to 14,705 QALYs (total 28,746 QALYs) and an additional 1250 deaths could have been avoided (total 3271 avoided deaths). Forecasts project continued substantial benefits for patients treated between 2025 and 2030, with lifetime outcomes estimated at approximately 31,306 QALYs, 38,274 LYs, and 4841 deaths avoided for the currently reimbursed indications. CONCLUSION: Pembrolizumab has delivered significant clinical benefits in Portugal. Our results suggest that more timely reimbursement decisions and broader access could have yielded measurable additional health gains. These findings highlight the potential impact of access timelines on health outcomes, while acknowledging that broader assessments of value require consideration of cost-effectiveness, budget impact, and opportunity costs. Pembrolizumab's benefits to the Portuguese health system is expected to grow through 2030.