Improved access to orphan drugs in 2021
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Spain: AELMHU reports improved access to orphan drugs in 2021


According to the Spanish association for orphan and ultra-orphan drugs (La Asociación Española de Medicamentos Huérfanos y Ultrahuérfanos, AELMHU), 14 new orphan drugs were made available in the national health service (Sistema Nacional de Salud, SNS) in 2021, compared to 5 in 2020 (9 in 2019), bringing the total number of orphan drugs available in the SNS to 56.  

The average time to obtain funding for orphan drugs was 24 months in 2021, down from 33 months in 2020 (but up from 14 months in 2019). Of the new products funded in 2021, just 7% obtained a price and funding in less than one year, whilst another 7% required more than three years.  

The AELMHU also indicates that, of the 14 new orphan drugs funded in the SNS in 2021, 43% (6 out of the 14) were financed subject to restrictions (ie the number of reimbursed indications is less than the number of indications approved by the European Medicines Agency, EMA). Furthermore: 

  • 43% are subject to 3 or more financing conditions 
  • 93% (13 out of 14) are “subject to follow-up” 
  • 50% are subject to a spending ceiling or maximum cost per patient 
  • 21% are subject to payment by results/risk-share arrangements.  

22% of orphan drugs funded in 2021 were hematologic agents or antithrombotics, 22% were indicated for metabolic disorders, 14% for oncology and 14% for diseases of the musculoskeletal system.  

The AELMHU also indicates that as at 31 December 2021, there were 55 orphan drugs awaiting a decision on price and reimbursement, two more than in 2020. Of these 55 drugs, 51% have been waiting for a decision for more than three years.  

For the AELMHU, the data “reflects an improvement in access, in the approval times and in the financing of orphan drugs, as a result of efforts made by the administration and the flexibility shown by pharmaceutical manufacturers”. However, it emphasises that the improvement should be seen in the context of the “negative indicators” observed in 2020 which can be largely attributed to the impact of the COVID-19 pandemic. The association goes on to say that, whilst the results for 2021 are encouraging, less than half of orphan drugs with marketing authorisation in Europe are funded by the SNS in Spain.  


  1., “Informe Anual de Acceso a los Medicamentos huérfanos 2021”, 21st February 2022
  2., “Solo el 50% de los medicamentos huérfanos que llegan a España está financiado, según Aelmhu”, 21st February 2022

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