Implementation and results of a risk-sharing scheme for enzyme replacement therapy in lysosomal storage diseases
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| Format: | Artículo científico |
| Language: | en |
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Sociedad Española de Farmacia Hospitalaria
2020
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| _version_ | 1876469995914919936 |
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| author | María Dolores Edo-Solsona |
| author_facet | María Dolores Edo-Solsona |
| contents | Implementation and results of a risk-sharing scheme for enzyme replacement therapy in lysosomal storage diseases María Dolores Edo-Solsona Isidro Vitoria-Miñana José-Luis Poveda-Andrés Medicina Risk Access Orphan drugs Effectiveness Health outcomes Objective: To describe a risk-sharing program’s implementation and results on enzyme replacement therapy for lysosomal diseases.Method: The program was designed and implemented in a referral hospital for congenital metabolic diseases. The conclusion of agreements required the following phases: 1) To define and agree on response variables and criteria to treatment; 2) to assign discount percentage to each stage of effectiveness; 3) to prepare and sign the agreement by all parties; 4) to implement the agreement; 5) to individualize purchases management; 6) to evaluate clinical results, and 7) to issue an annual report.Results: Eight patients were included in the program (four with Hurler’s disease, two with Pompe and two with Gaucher), five of them were women and three were men. After analyzing the defined variables and response criteria, all patients presented full effectiveness after two or three years of follow-up except one of them that could not be evaluated. Given the effectiveness achieved, the hospital made full payment of all administered therapies.Conclusions: The implanted risk-sharing program is Spain’s first published event of paying for clinical results using orphan drugs. Economic impact has been limited, and program implementation has gone through a complex process of formulation and management. However, the greatest achievement has been to reduce the knowledge gap between efficacy and effectiveness, stating that the therapies administered have shown the optimal benefits for which the funder is willing to pay. 2020 artículo científico 1130-6343 https://www.redalyc.org/articulo.oa?id=365963365003 https://www.redalyc.org/journal/3659/365963365003/ https://www.redalyc.org/journal/3659/365963365003/html/ https://www.redalyc.org/journal/3659/365963365003/365963365003.epub https://www.redalyc.org/journal/3659/365963365003/movil 10.7399/fh.11262 en http://www.redalyc.org/revista.oa?id=3659 Farmacia Hospitalaria application/pdf Sociedad Española de Farmacia Hospitalaria Farmacia Hospitalaria (España) Num.1 Vol.44 |
| format | Artículo científico |
| id | redalyc_365963365003 |
| institution | Redalyc |
| language | en |
| publishDate | 2020 |
| publisher | Sociedad Española de Farmacia Hospitalaria |
| spellingShingle | Implementation and results of a risk-sharing scheme for enzyme replacement therapy in lysosomal storage diseases María Dolores Edo-Solsona Medicina Risk Access Orphan drugs Effectiveness Health outcomes Implementation and results of a risk-sharing scheme for enzyme replacement therapy in lysosomal storage diseases María Dolores Edo-Solsona Isidro Vitoria-Miñana José-Luis Poveda-Andrés Medicina Risk Access Orphan drugs Effectiveness Health outcomes Objective: To describe a risk-sharing program’s implementation and results on enzyme replacement therapy for lysosomal diseases.Method: The program was designed and implemented in a referral hospital for congenital metabolic diseases. The conclusion of agreements required the following phases: 1) To define and agree on response variables and criteria to treatment; 2) to assign discount percentage to each stage of effectiveness; 3) to prepare and sign the agreement by all parties; 4) to implement the agreement; 5) to individualize purchases management; 6) to evaluate clinical results, and 7) to issue an annual report.Results: Eight patients were included in the program (four with Hurler’s disease, two with Pompe and two with Gaucher), five of them were women and three were men. After analyzing the defined variables and response criteria, all patients presented full effectiveness after two or three years of follow-up except one of them that could not be evaluated. Given the effectiveness achieved, the hospital made full payment of all administered therapies.Conclusions: The implanted risk-sharing program is Spain’s first published event of paying for clinical results using orphan drugs. Economic impact has been limited, and program implementation has gone through a complex process of formulation and management. However, the greatest achievement has been to reduce the knowledge gap between efficacy and effectiveness, stating that the therapies administered have shown the optimal benefits for which the funder is willing to pay. 2020 artículo científico 1130-6343 https://www.redalyc.org/articulo.oa?id=365963365003 https://www.redalyc.org/journal/3659/365963365003/ https://www.redalyc.org/journal/3659/365963365003/html/ https://www.redalyc.org/journal/3659/365963365003/365963365003.epub https://www.redalyc.org/journal/3659/365963365003/movil 10.7399/fh.11262 en http://www.redalyc.org/revista.oa?id=3659 Farmacia Hospitalaria application/pdf Sociedad Española de Farmacia Hospitalaria Farmacia Hospitalaria (España) Num.1 Vol.44 |
| title | Implementation and results of a risk-sharing scheme for enzyme replacement therapy in lysosomal storage diseases |
| topic | Medicina Risk Access Orphan drugs Effectiveness Health outcomes |
| url | https://www.redalyc.org/articulo.oa?id=365963365003 https://www.redalyc.org/journal/3659/365963365003/ https://www.redalyc.org/journal/3659/365963365003/html/ https://www.redalyc.org/journal/3659/365963365003/365963365003.epub https://www.redalyc.org/journal/3659/365963365003/movil |