PhD defence B. (Brenda) Leeneman

For many years, treatment options for patients with unresectable stage III and stage IV cutaneous melanoma (hereafter: advanced melanoma) were limited. Chemotherapy was the standard of care, but it never demonstrated to improve survival. Advances in immunotherapy and targeted therapy drastically changed the treatment landscape for advanced melanoma. The first two novel drugs, ipilimumab (an immunotherapy) and vemurafenib (a targeted therapy), received a marketing authorization valid throughout the European Union in 2011 and 2012, respectively. Since then, multiple other drugs and combinations of drugs have been approved for advanced melanoma.

To decide on reimbursement, national reimbursement authorities need valid evidence regarding a drug’s (long-term) effects and costs. Although it is widely acknowledged that randomized controlled trials (RCTs) are the gold standard for assessing efficacy and safety, it is also well known that not all evidence can be obtained from RCTs. Therefore, in recent years, reimbursement authorities have become increasingly interested in real-world evidence (i.e., evidence obtained from real-world data).

The aim of this thesis is to evaluate how and to what extent real-world evidence can complement evidence from RCTs in order to support evidence-based decision making on drug reimbursement in melanoma.

The public defence will take place at the Senate Hall, 1st floor Erasmus Building, location campus Woudestein. The ceremony will begin exactly at 10.30 AM. In light of the solemn nature of the ceremony, we recommend that you do not take children under the age of 6 to the first part of the ceremony.