CP-128 Analysis and evolution of spending on orphan drugs. (25th February 2017)

• Record Type: Journal Article
• Authors: Selvi-Sabater, P; Leon-Villar, J; Alonso-Dominguez, T; Espuny-Miró, A; Titos-Arcos, JC; Sanchez-Catalicio, MDM; Aranda-Garcia, A; Gorostiza-Frias, I; Manresa-Ramon, N; Soria-Soto, M
• Is Part Of: European journal of hospital pharmacy. Volume 24(2017)Supplement 1
• Page Start: A57
• Page End: A57
• Publication Date: 2017-02-25
• Subjects: Pharmacy -- Periodicals; Hospital pharmacies -- Periodicals; 615.1
• DOI: 10.1136/ejhpharm-2017-000640.127
• Languages: English
• ISSNs: 2047-9956

Abstract

Abstract : Background: Orphan drugs mean a high economic impact and cost per patient, and in the past years there has been an increase in the number of patients. Purpose: To describe and analyse the evolution of spending on orphan drugs based on diagnosis. Material and methods: A retrospective study including all patients receiving treatment with orphan drugs from January 2013 to December 2015 was conducted. Collected parameters were: number of patients per drug, diagnosis, economic spending (€) and percentage of total and annual expenditure. Data were obtained through SAVAC, a prescription and validation programme; Discoverer, its complementary programme for exploitation of the data and a designed Excel base were used for data collection. Results: A total of 25 drugs were identified. Evolution from 2013 2015 is shown in the table. Triennial analysis: the distribution of total spending over the 3 years for diagnosis (€15 121 487) was: Pompe disease 28%, oncological diseases 27% and multiple myeloma 22%. Other expenditures were allocated to various diagnoses. Regarding drugs, lenalidomide was 25% of the total expenditure, followed by alglucosidase alpha (24%), and azacitidine, bretuximab and nilotinib (<10% for each). Regarding average cost per patient/year, Pompe disease involved a cost per patient of €357 419, followed by Gauche disease with €172 808, and myelodysplastic syndrome and multiple myeloma with €34 835 and €32 781, respectively. Conclusion: In the past 3 years there has been an increase in the number of patients prescribed orphan drugs. This has led to an increase of about €2 million with regard to cost per patient. Pompe disease, oncological diseases and multiple myeloma represented approximately 80% of the total expenditure of orphan drugs. Only 2 drugs (lenalidomida and alglucosidase alpha) represented about 50% of the total expenditure. Pompe disease represents one of the largest expenses every year, a limited number of patients and a high cost per patient/year. No conflict of interest