<?xml version="1.0" encoding="ISO-8859-1"?><article xmlns:mml="http://www.w3.org/1998/Math/MathML" xmlns:xlink="http://www.w3.org/1999/xlink" xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance">
<front>
<journal-meta>
<journal-id>1130-6343</journal-id>
<journal-title><![CDATA[Farmacia Hospitalaria]]></journal-title>
<abbrev-journal-title><![CDATA[Farm Hosp.]]></abbrev-journal-title>
<issn>1130-6343</issn>
<publisher>
<publisher-name><![CDATA[Grupo Aula Médica]]></publisher-name>
</publisher>
</journal-meta>
<article-meta>
<article-id>S1130-63432020000100003</article-id>
<article-id pub-id-type="doi">10.7399/fh.11262</article-id>
<title-group>
<article-title xml:lang="en"><![CDATA[Implementation and results of a risk-sharing scheme for enzyme replacement therapy in lysosomal storage diseases]]></article-title>
<article-title xml:lang="es"><![CDATA[Implantación y resultados de un programa de riesgo compartido para terapias de sustitución enzimática en enfermedades de depósito lisosomal]]></article-title>
</title-group>
<contrib-group>
<contrib contrib-type="author">
<name>
<surname><![CDATA[Edo-Solsona]]></surname>
<given-names><![CDATA[María Dolores]]></given-names>
</name>
<xref ref-type="aff" rid="Aff"/>
</contrib>
<contrib contrib-type="author">
<name>
<surname><![CDATA[Vitoria-Miñana]]></surname>
<given-names><![CDATA[Isidro]]></given-names>
</name>
<xref ref-type="aff" rid="Aff"/>
</contrib>
<contrib contrib-type="author">
<name>
<surname><![CDATA[Poveda-Andrés]]></surname>
<given-names><![CDATA[José-Luis]]></given-names>
</name>
<xref ref-type="aff" rid="Aff"/>
</contrib>
</contrib-group>
<aff id="Af1">
<institution><![CDATA[,Hospital Universitari i Politècnic La Fe Pharmacy Service ]]></institution>
<addr-line><![CDATA[ ]]></addr-line>
<country>Spain</country>
</aff>
<aff id="Af2">
<institution><![CDATA[,Hospital Universitari i Politècnic La Fe Nutrition and Metabolopathies Unit ]]></institution>
<addr-line><![CDATA[ ]]></addr-line>
<country>Spain</country>
</aff>
<pub-date pub-type="pub">
<day>00</day>
<month>02</month>
<year>2020</year>
</pub-date>
<pub-date pub-type="epub">
<day>00</day>
<month>02</month>
<year>2020</year>
</pub-date>
<volume>44</volume>
<numero>1</numero>
<fpage>10</fpage>
<lpage>15</lpage>
<copyright-statement/>
<copyright-year/>
<self-uri xlink:href="http://scielo.isciii.es/scielo.php?script=sci_arttext&amp;pid=S1130-63432020000100003&amp;lng=en&amp;nrm=iso"></self-uri><self-uri xlink:href="http://scielo.isciii.es/scielo.php?script=sci_abstract&amp;pid=S1130-63432020000100003&amp;lng=en&amp;nrm=iso"></self-uri><self-uri xlink:href="http://scielo.isciii.es/scielo.php?script=sci_pdf&amp;pid=S1130-63432020000100003&amp;lng=en&amp;nrm=iso"></self-uri><abstract abstract-type="short" xml:lang="en"><p><![CDATA[Abstract  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.]]></p></abstract>
<abstract abstract-type="short" xml:lang="es"><p><![CDATA[Resumen  Objetivo: Describir la implantación y los resultados de un programa de riesgo compartido para el tratamiento enzimático sustitutivo de enfermedades lisosomales.  Método: Se diseñó y aplicó el programa en un hospital de referencia para enfermedades congénitas del metabolismo. La consecución de los acuerdos requirió las siguientes fases: 1) Definir y consensuar las variables y criterios de respuesta al tratamiento; 2) asignar el porcentaje de descuento a cada escalón de efectividad; 3) elaborar y firmar el acuerdo por todas las partes; 4) implantar el acuerdo; 5) individualizar la gestión de compras; 6) evaluar los resultados clínicos, y 7) emitir un informe anual.  Resultados: Se incluyeron ocho pacientes en el programa (cuatro con enfermedad de Hurler, dos con enfermedad de Pompe y dos con enfermedad de Gaucher), siendo cinco de ellos mujeres y tres varones. Tras analizar las variables y criterios de respuesta definidos, todos los pacientes presentaron efectividad plena tras dos o tres años de seguimiento, excepto uno de ellos que no se pudo evaluar. Dada la efectividad alcanzada, el hospital realizó el pago íntegro de todos los tratamientos administrados.  Conclusiones: El programa de riesgo compartido implantado es la primera experiencia publicada de pago por resultados clínicos en medicamentos huérfanos en España. El impacto económico ha sido limitado y la implantación del programa no ha estado exenta de complejidad de formulación y de gestión. Sin embargo, el mayor logro ha sido reducir la brecha de conocimiento entre eficacia y efectividad, constatando que las terapias administradas han mostrado los beneficios óptimos por los que está dispuesto a pagar el financiador.]]></p></abstract>
<kwd-group>
<kwd lng="en"><![CDATA[Risk-sharing agreements]]></kwd>
<kwd lng="en"><![CDATA[Orphan drugs]]></kwd>
<kwd lng="en"><![CDATA[Enzyme replacement therapies]]></kwd>
<kwd lng="en"><![CDATA[Health outcomes]]></kwd>
<kwd lng="en"><![CDATA[Effectiveness]]></kwd>
<kwd lng="en"><![CDATA[Access]]></kwd>
<kwd lng="es"><![CDATA[Acuerdos de riesgo compartido]]></kwd>
<kwd lng="es"><![CDATA[Medicamentos huérfanos]]></kwd>
<kwd lng="es"><![CDATA[Terapias de sustitución enzimática]]></kwd>
<kwd lng="es"><![CDATA[Resultados en salud]]></kwd>
<kwd lng="es"><![CDATA[Efectividad]]></kwd>
<kwd lng="es"><![CDATA[Acceso]]></kwd>
</kwd-group>
</article-meta>
</front><back>
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