Reimbursement of medicines in Belgium: role of evidence-based medicine.

The European Transparency Directive requires that pricing and reimbursement decisions must be taken in a transparent, objective and verifiable way with respect of strict timelines. The Belgian competent authority integrated on January 1st, 2002 Evidence-Based Medicine (EBM) principles in the reimbursement evaluation. The present work describes the procedures and investigates whether the introduction of the EBM principles indeed affects the decision and whether it compromised the respect of strict timelines. The reimbursement decision for all new submissions except for generic drugs is preceded by an evaluation of the relative therapeutic value. There were 1285 submissions handled within the period 2002-2004 of which 159 (12.4%) related to new molecular entities. For the 824 files with valuation of the therapeutic value, the reimbursement decision was positive in 80.8% of cases. The percentage of positive decisions was dependent on the type of submission with the lowest percentages for new molecular entities and submissions for new indications (64%-71%). Line extensions and generics received a positive decision in nearly all cases (> 95%). Proof of added value by at least 1 positive superiority trial against active comparator is a requirement for obtaining a price premium: this was granted in less than 50% of the 67 submissions claiming such superiority and the odds for a negative reimbursement decision increased significantly if the applicant failed to prove added value: O.R. = 9.1 (2.3 - 35.6), indicating that clinical evidence of added therapeutic value clearly facilitates reimbursement. The introduction of the new procedure did not jeopardize the timelines. Introducing EBM principles had a significant impact on reimbursement decisions in Belgium by facilitating reimbursement with a price premium of new drugs with added value addressing unmet medical needs.