LAUSR

Introduction The number of novel therapies that achieve registration in oncology based on limited clinical evidence like non-randomized studies (NRS) is increasing. Dutch health authorities determine the therapeutic value of medicines evaluated in randomized controlled trials based on formal criteria for minimal relative efficacy or clinical treatment guidelines. However, there are no criteria to determine the therapeutic benefit of treatments assessed in NRS, which impacts reimbursement and patient access. Additionally, clinicians in the Netherlands see potential clinical benefit in off-label use but are currently unable to prescribe these because of absent clinical evidence. This review aims to identify and summarize initiatives to improve patient access to oncology therapies with limited or no clinical evidence in the Netherlands. Methods Relevant public reimbursement documents for the Netherlands were identified through a pragmatic literature review. Results The main initiatives to improve access for therapies with limited clinical evidence are outside regular reimbursement pathways. The Drug Rediscovery Protocol (DRUP, initiated 2016) and drug access protocol (DAP, initiated 2021) are two initiatives to improve access in solid tumor oncology. DRUP collects real-world data and provides access for targeted therapies in patients with targetable mutations in off-label indications. At least 25 therapies have been included to date. DAP uses the same data framework to reimburse on-label solid tumor therapies through a managed entry agreement based on response. The first participating therapies are cemiplimab (NRS, failed ZiN reimbursement), entrectinib and larotrectinib (both NRS, tumor-agnostic). Finally, Dutch clinical experts proposed criteria for therapeutic value of therapies studied NRS in oncology. Conclusions Patient access to both off-label and on-label oncology therapies is addressed in DRUP and DAP. New criteria for the therapeutic value of therapies studied in NRS may decrease the relevance of DAP, although these criteria would not address the need for comparative evidence for cost-effectiveness analysis.