OBJECTIVES

: To describe the actual patient access to orphan medicinal products (OMP) for the treatment of rare diseases in Catalonia over the past five years.

METHODS

: The main source used to perform the analysis was the Pharmaceutical Reimbursement File of the Catalan Health Service (CatSalut). The selection criteria used to define the study population was that of medicines dispensed during 2013 to 2017 that held orphan drug designation of the European Medicines Agency (EMA) by the time of marketing authorisation.

RESULTS

: Among the 142 OMP approved by the EMA, 83 had been prescribed and/or dispensed in CatSalut during 2013 to 2017. As expected, the increasing number of OMP financed overtime was found to correlate with the number of patients treated in Catalonia, being 3690 in 2013 and up to 8853 at 2017, the latest representing a 0.12 % of Catalonia population. The treatments represented 84 M € (4.3% of the total pharmaceutical expenditure in CatSalut) in 2013 and raised up to 157 M € (6.7% total pharmaceutical expenditure) in 2017. The main therapeutic areas that influenced to such increase were onco-haematology, pulmonary diseases and metabolic endocrine disorders.

CONCLUSIONS

: The expenditure in OMP in Catalonia has almost duplicated in five years, paralleling the raise of the number of OMP approved by the EMA, and consistent with the elevated cost associated with these pharmaceuticals. The growth in expenditure is very relevant within the overall pharmaceutical billing and is in line with the data published in comparable environments. In the light of these results, measures should be proposed to guarantee the sustainability of access to innovation for Catalan patients in the field of rare diseases, as well as methods to assess their effectivity in terms of health outcomes.