PP147 Conditional Reimbursement Of Medicinal Products, A Procedure For Orphan Drugs, Conditionals and Exceptionals

Abstract

IntroductionIn 2018 the Dutch Ministry of Health (MoH) introduced a new policy regarding the conditional reimbursement (CR) of drugs in the basic health insurance package. This new policy offers patients with a serious, often rare disease for which no effective treatment is yet available, the possibility of obtaining controlled access to new promising drugs. In the meantime, additional data on (cost-) effectiveness is being collected. The aim was to assess whether this new policy allowed improved inclusion of drugs in the basic health insurance package.MethodsMarketing authorization holders (MAH) were able to apply for the CR. The drug had to be registered by the European Medicines Agency (EMA) as an orphan drug, conditional or exceptional and address an unmet medical need. The MAH had to submit a dossier which includes a study protocol together with the professionals’ associations, patients’ associations and a research institute. It was possible to engage an ongoing (international) study in the CR application. Based on the proposed study, the National Health Care Institute (ZIN) assessed whether it is possible to determine if the drug should be reimbursed at the end of the CR period. A reduced price was a condition for CR.ResultsFour drugs are currently reimbursed as part of the CR, being: parathyroid hormone, ataluren, larotrectinib and entrectinib. The proposed studies are ongoing and will generate data to support the final reimbursement decision. Progress will be monitored by the researchers and discussed with ZIN.ConclusionsFour drugs were successfully conditionally reimbursed, concluding the new CR procedure is feasible. Additional data is being collected to aid in the decision on the definitive reimbursement of these drugs. The upcoming period, the focus will be on the quality of the collected data and whether the inclusion of patients is proceeding as planned. The MoH will be informed by ZIN on the study progress annually. The final reimbursement decision is taken at the end of the CR period.