Plain Language Summary

The study explores how real-world evidence (RWE) is used in the approval of cancer treatments in Europe. The research is important because it addresses the growing incorporation of RWE into regulatory and health technology assessment (HTA) decisions, which can help patients gain quicker access to new therapies. The study reviews the practices of the European Medicines Agency (EMA) and several HTA bodies, including the National Institute for Health and Care Excellence (NICE), Gemeinsamer Bundesausschuss (G-BA), and Haute Autorité de Santé (HAS).

The findings show that RWE is often utilized as a supplementary tool to support data from clinical trials, especially in oncology. However, the acceptance of RWE varies widely among the different organizations, leading to inconsistencies in how it is used in decision making. For example, while the EMA often accepts RWE as supportive evidence, other bodies like G-BA are more critical and may reject RWE due to concerns over its methodological quality.

Between 2020 and 2022, RWE appeared in about one-third of EMA's final reports on oncology medicines, and nearly half of the HTA appraisals by NICE included RWE. Despite this progress, the study highlights significant gaps. For instance, there are discrepancies in how RWE is perceived and utilized across different agencies. These differences can hinder the overall effectiveness of RWE in informing treatment decisions and ultimately affect patient care.

The article emphasizes the need for collaboration among regulatory bodies and HTA organizations to establish clearer and more standardized guidelines for using RWE. With the introduction of the European Union's Joint Clinical Assessment in 2025, it becomes even more crucial for these agencies to align their approaches to RWE. This alignment will help ensure that patients have equitable and timely access to innovative treatments.

In summary, while RWE holds promise for enhancing the approval process for cancer therapies in Europe, there are still significant challenges to address. Greater consistency in how RWE is evaluated and integrated into decision-making processes will be vital for improving patient outcomes and improving access to new medicines.

^{Note: This content was created with assistance from artificial intelligence (AI) and has been reviewed and edited by ISPOR staff. For more information or for inquiries on ISPOR’s AI policy, click here or contact us at info@ispor.org.}