Accessibility to cancer medicines in Europe:Towards equitable access and fair pricing

Abstract

This dissertation explores disparities in access to novel cancer medicines across Europe from the perspectives of patients, healthcare providers, and national authorities. It addresses challenges related to accessibility, pricing, and the socioeconomic impact of cancer, with the aim of informing evidence-based policies to support fair and sustainable cancer care.In the first part, access disparities were assessed through surveys with hospital pharmacists and oncology specialists across several European countries. Findings revealed significant variation in time to access and availability of novel cancer medicines, influenced by hospital type, country-specific regulations, and medicine type. Combination therapies were generally less accessible than monotherapies, and access was often faster in specialized hospitals and countries with strong early access programs. The second part of the dissertation examined medicine pricing. We analyzed managed entry agreements (MEAs), revealing differences in how countries negotiate and report discounts. Although MEAs aim to reduce costs, confidentiality limits transparency and comparability. We also presented confidential hospital-level pricing data from nine countries, showing wide disparities and emphasizing the need for standardized reporting to strengthen negotiations and collaboration.The third part investigated the socioeconomic impact of cancer from the patient perspective through the SEC study, involving over 3,000 patients in 25 countries. High levels of financial toxicity were reported, particularly among younger, lower-income, self-employed, and less-educated patients. A sub-study on adolescents and young adults (AYAs) found that 79% experienced financial difficulties, with limited support tailored to their needs. Many healthcare professionals remained unaware of these challenges.The dissertation concludes with six key recommendations, including harmonized EU regulations for early access, improved use of real-world evidence, discontinuation of external reference pricing, and enhanced price transparency. It also calls for the development of validated tools to identify vulnerable patients and inform targeted, supportive policies across Europe