OBJECTIVES

: Analyze the variables of administration, prescription, dispensing and financing of authorized orphan drugs in Spain, as well as the economic repercussion of the June 12, 2020 modification of Spanish legislation concerning the exemption from their appearing in the Reference Price Order (RPO).

METHODS

: Observational descriptive study of the prescription, administration, dispensing and financing of orphan drugs authorized in Spain registered in CIMA with a cut-off date of June 1, 2020. Likewise, was made a comparison of the marketing price of some examples of orphan drugs that currently there is the generic on the market and a price forecast with those who will soon lose their patent or marketing exclusivity.

RESULTS

: In Spain, at June 1, 2020, there were 174 authorized orphan drugs corresponding to 98 active ingredients. Most of them are for hospital use, oral administration route and, given the low prevalence of diseases, subject to additional monitoring. The number of authorizations has increased in recent years, being the maximum in 2018. The new policies try to guarantee the supply and sub-supply of orphan drugs and, in addition, allow the industries focused on rare diseases to maintain profit margins to continue with the development of new treatments. Comparing the price of Tracleer®(bosentan) and its generic, it decreased the mandatory 40%. On the contrary, Volibris® (ambrisentan) will soon have generic marketed with a possible decrease of 60% and, if it appears in the RPO, the presentation of less doses could decrease up to 80%.

CONCLUSIONS

: New economic models, such as risk-sharing agreements, could be a good alternative to favor the development of safe and effective treatments accessible to patients and, in turn, achieve the sustainability of the National Health System.