Second quarterly report on access to orphan drugs in 2023

Second quarterly report on access to orphan drugs in 2023

The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) has published the second Quarterly Report on Access to Orphan Drugs in Spain in 2023, which analyzes the evolution of access to this type of products with 4 indicators, mainly.

In order to develop this analysis, only those treatments that have an orphan designation and trade name in force until August 31, 2023 have been taken into account.

According to the main results of the study, 148 orphan drugs currently have marketing authorization from the European Medicines Agency (EMA), of which 126 have already reached Spain and 77 are financed by the SNS.

During the second four-month period of the year, 2 new National Codes were assigned and 9 new orphan drugswere approved for financing , all of which were approved with conditions and 78% on a restricted basis.

Today, 85% of the orphan drugs authorized in the EU have arrived in Spain, but only 52% of them are financed . In addition, 49% of the products have been financed with restrictions, i.e., with limited indications or with unfunded indications.

Another very interesting indicator in the report is the waiting time to obtain financing. If we take as a sample the products approved in 2023, from the time they obtain the CN until they become part of the Nomenclator, the average time is 26 months.

Despite the fact that the number of orphan drugs funded in recent months has grown significantly in recent months, it is important to note that there are still 49 orphan drugs with a National Code that are not funded in our country.

Advanced therapies for rare diseases with orphan designation are increasingly present in the report. Despite this, the number of advanced therapies with price and reimbursement in Spain has not changed in recent months. At the close of this report, 15 advanced therapies for rare diseases have marketing authorization, 14 have arrived in Spain and 5 are financed by the SNS.

The sources used to prepare this report are the European Medicines Agency (EMA), the Community Register of Orphan Medicinal Products, the Spanish Agency for Medicines and Health Products (AEMPS), and the Ministry of Health.

You can consult the infographic of the report here.