OBJECTIVES: To analyse and describe the managed entry agreements (MEAs) for orphan drugs in Belgium. In particular, the study aimed to identify the uncertainties that remained at the time of their reimbursement, and describe the real-world evidence (RWE) that was collected for their reassessment. In addition, specific recommendations were aimed at Belgian decision makers on evaluation of MEAs

METHODS: A literature review was carried out on the use of RWE in MEAs for orphan drugs. In addition, the public parts of the Belgian MEAs for all currently reimbursed orphan drugs in Belgium were analysed. Several characteristics of these MEAs were extracted, such as the type of MEA, the uncertainties included and the RWE collected in response to these uncertainties. Uncertainties were categorized according to the nomenclature as provided by the Federal Knowledge Center for Healthcare (KCE) in Belgium.

RESULTS: The MEAs of 31 orphan drugs were collected and analysed. The preliminary results show that the majority of uncertainties inquired about the budget impact (32%) and efficacy (28%) of the orphan drug, while questions on the quality of life and cost-effectiveness were least included (1%). All MEAs were extended at the end of of the contract agreement. Meanwhile, none of MEAs included information on the RWE that was collected in order to answer the uncertaintes. All MEAs included provisions on price discounts/risk-sharing, although details on discounts were not made public.

CONCLUSIONS: Given the high burden of orphan drugs on the Belgian health budget, the tax payer should be informed on whether orphan drugs meet the conditions as defined in the MEA. However, there is a need for more transparency around the content of these MEAs. In particular, reports on the reassessments of the uncertainties – and the RWE submitted by the pharmaceutical company in response to these uncertainties – should be made public.