Access to anticancer medicines in the Netherlands and other high-income European countries. A clinical perspective

Access to innovative anticancer medicines is increasingly challenged by uncertainty regarding clinical benefit, rising medicine expenditure, and differences in national reimbursement processes. This thesis examines access across three successive phases: prior to EU marketing authorization (EU-MA), between EU-MA and national reimbursement, and after reimbursement, with particular emphasis on the Netherlands. Before EU-MA, substantial differences were observed between seven high-income European countries in the availability, organization, and reimbursement of early-access mechanisms. Following EU-MA, time to reimbursement varied considerably between countries. Shorter time to reimbursement was associated with higher gross domestic product, absence of a pre-assessment procedure, and submission by a large pharmaceutical company. In the Netherlands, medical oncologists frequently sought access to EU-authorized but non-reimbursed medicines through alternative routes. Variation in whether and how physicians pursued and discussed these options may contribute to unequal access between patients. Uncertainty regarding treatment benefit persists after reimbursement. In 2022, 29.7% of treatments with the 30 highest-budget-impact anticancer medicines in the Netherlands were discontinued within 90 days, accounting for €77 million, or 9.9% of medicine expenditure for these treatments. Early treatment discontinuation, however, may be clinically justified or unavoidable. Overall, uncertainty remains throughout the access pathway. Addressing this requires better alignment between regulatory approval, reimbursement, and clinical practice, continued evidence generation after market entry, and improved patient selection. These approaches may support timely and equitable access, rational use of medicines, and financially sustainable cancer care. Show more