OBJECTIVES: Between 2002 and 2016, 94 orphan drugs (OD) designations were approved by the European Medicines Agency (EMA). In Spain, only 49 of them are reimbursed. Those OD have not been reimbursed due to 2 reasons: a) high treatment costs; and; b) limited clinical data ensuring reproducibility of clinical trials in real practice. Our objective was to identify tools to derive fast and broad access conditions for nusinersen

METHODS

: A benchmark of OD was done to set expectations and to identify important drivers of nusinersen’s reimbursement. The following OD were excluded:

- Products with oncological indications

- Products that have not applied National Code in Spain

- Products for which other therapeutic alternatives are available

We assessed the following data: status of reimbursement, level of price cuts/discounts relative to other countries, and types of agreements/contracts used to reimburse OD in Spain.

RESULTS: In total, 7 OD were identified. Only one OD achieved reimbursement in Spain, the rest were under several rounds of price negotiations since 2014. Initial discounts around 30% to 50% of first offer were identified. Spanish Authorities wanted to achieve a reasonable annual treatment costs and financial agreements that help control their budget. With that information, nusinersen’s pillars for reimbursement process in Spain were: 1) communicate the clinical value and robustness of data from pivotal clinical trials and real-world experience via a global expanded access program; 2) maintain open dialog with Spanish Ministry of Health (MoH) and their needs; 3) propose innovative financial contract agreements based on spending and budget predictability.

CONCLUSIONS: Overall, nusinersen’s reimbursement process focused on reaching an end-to-end solution between health authorities and the company. This resulted in a significantly reduced reimbursement time and in a recognition by Spanish MoH that nusinersen process became benchmark for future price and reimbursement processes in Spain