Budgetary impact of new label indications after initial drug registration in Brazil: A case study of pembrolizumab and trastuzumab deruxtecan

Abstract

The pharmaceutical landscape has undergone a transformative shift due to emerging technologies, leading to a projected value of USD 405.52 billion for the global pharmaceutical manufacturing market in 2020. Brazil, representing 2% of the global pharmaceutical market, boasts a comprehensive public health system called Sistema Único de Saúde (SUS), ensuring universal health coverage. However, the public and private health sectors in Brazil operate independently, with 23% of Brazilians possessing private health insurance plans. Regulated by the Brazilian Drug Market Regulation Chamber (CMED), which controls drug prices based on international retail prices and other factors, the pharmaceutical market in Brazil faces significant challenges in maintaining the sustainability of the Brazilian health-care system, particularly due to the high costs of cancer medications, such as pembrolizumab and trastuzumab deruxtecan. This study aims to assess the budgetary impact of expanding indications for pembrolizumab and trastuzumab deruxtecan in Brazil after their initial registration by the Brazilian Health Regulatory Agency. Utilizing epidemiological data from the National Cancer Institute (INCA) and clinical trial data, we estimated the eligible patient populations and associated costs for these medications. Trastuzumab deruxtecan’s eligible patient population increased from 383 to 23,000 across various indications, resulting in a total cost increase from USD 90.6 million to USD 5.0 billion. Similarly, pembrolizumab’s eligible patient population expanded from 1796 to 99,544 for multiple indications, causing the total cost increase from USD 121.1 million to USD 6.7 billion. The study highlights the substantial expansions in indications and patient eligibility for trastuzumab deruxtecan and pembrolizumab in Brazil, with significant budgetary implications potentially jeopardizing the health-care system’s sustainability. To ensure the long-term sustainability of health-care systems and to provide equitable access to optimal treatments, it is crucial for policymakers to monitor and adjust drug prices following their initial registration. Policymakers must be aware of these challenges and consider them in health-care policy development and decision-making processes.