<?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>0034-7418</journal-id>
<journal-title><![CDATA[Revista Colombiana de Ciencias Químico - Farmacéuticas]]></journal-title>
<abbrev-journal-title><![CDATA[Rev. colomb. cienc. quim. farm.]]></abbrev-journal-title>
<issn>0034-7418</issn>
<publisher>
<publisher-name><![CDATA[Departamento de Farmácia, Facultad de Ciencias, Universidade Nacional da Colombia]]></publisher-name>
</publisher>
</journal-meta>
<article-meta>
<article-id>S0034-74182022000200735</article-id>
<article-id pub-id-type="doi">10.15446/rcciquifa.v51n2.98783</article-id>
<title-group>
<article-title xml:lang="pt"><![CDATA[A lacuna regulatória de medicamentos órfãos impacta na disponibilidade de alternativas terapêuticas no Brasil?]]></article-title>
<article-title xml:lang="en"><![CDATA[Does the regulatory gap on orphan drugs impact on the availability of therapeutic alternatives in Brazil?]]></article-title>
<article-title xml:lang="es"><![CDATA[¿El vacío regulatorio sobre medicamentos huérfanos impacta en la disponibilidad de alternativas terapéuticas en Brasil?]]></article-title>
</title-group>
<contrib-group>
<contrib contrib-type="author">
<name>
<surname><![CDATA[Rodrigues de Matos Lopes]]></surname>
<given-names><![CDATA[Francyelle]]></given-names>
</name>
<xref ref-type="aff" rid="Aff"/>
</contrib>
<contrib contrib-type="author">
<name>
<surname><![CDATA[Sá]]></surname>
<given-names><![CDATA[Natan Monsores de]]></given-names>
</name>
<xref ref-type="aff" rid="Aff"/>
</contrib>
<contrib contrib-type="author">
<name>
<surname><![CDATA[Fonseca Lima]]></surname>
<given-names><![CDATA[Rodrigo]]></given-names>
</name>
<xref ref-type="aff" rid="Aff"/>
</contrib>
<contrib contrib-type="author">
<name>
<surname><![CDATA[Tavares]]></surname>
<given-names><![CDATA[Noemia Urruth Leão]]></given-names>
</name>
<xref ref-type="aff" rid="Aff"/>
</contrib>
<contrib contrib-type="author">
<name>
<surname><![CDATA[Santana]]></surname>
<given-names><![CDATA[Rafael Santos]]></given-names>
</name>
<xref ref-type="aff" rid="Aff"/>
</contrib>
</contrib-group>
<aff id="Af1">
<institution><![CDATA[,Universidade de Brasília Departamento de Farmácia ]]></institution>
<addr-line><![CDATA[Distrito Federal ]]></addr-line>
<country>Brazil</country>
</aff>
<aff id="Af2">
<institution><![CDATA[,Universidade de Brasília Departamento de Saúde Coletiva ]]></institution>
<addr-line><![CDATA[Distrito Federal ]]></addr-line>
<country>Brazil</country>
</aff>
<pub-date pub-type="pub">
<day>00</day>
<month>08</month>
<year>2022</year>
</pub-date>
<pub-date pub-type="epub">
<day>00</day>
<month>08</month>
<year>2022</year>
</pub-date>
<volume>51</volume>
<numero>2</numero>
<fpage>735</fpage>
<lpage>751</lpage>
<copyright-statement/>
<copyright-year/>
<self-uri xlink:href="http://www.scielo.org.co/scielo.php?script=sci_arttext&amp;pid=S0034-74182022000200735&amp;lng=en&amp;nrm=iso"></self-uri><self-uri xlink:href="http://www.scielo.org.co/scielo.php?script=sci_abstract&amp;pid=S0034-74182022000200735&amp;lng=en&amp;nrm=iso"></self-uri><self-uri xlink:href="http://www.scielo.org.co/scielo.php?script=sci_pdf&amp;pid=S0034-74182022000200735&amp;lng=en&amp;nrm=iso"></self-uri><abstract abstract-type="short" xml:lang="pt"><p><![CDATA[RESUMO  Introdução: A regulação de registros específicos para os chamados "medicamentos órfãos" tem sido uma estratégia das maiores agências de medicamentos do mundo para fomentar o acesso e monitoramento de tratamento para doenças e agravos de pouca prevalência ou interesse mercadológico.  Objetivos: Esse estudo visou identificar o perfil dos medicamentos que se enquadram nessa categoria internacionalmente explorar possíveis lacunas de registro gerados pela ausência de uma norma sanitária específica no Brasil.  Métodos: Foram analisadas as bases de dados de registro de medicamentos órfãos de países da União Europeia e dos Estados Unidos da América e os resultados foram comparados com a base registros da Agência brasileira.  Resultados: Foram identificados 369 medicamentos registrados como órfãos nos órgãos europeu e estadunidense totalizando 801 indicações clínicas. A maior parte dos medicamentos registrados no âmbito internacional era de agentes anti-neoplásicos e imunomoduladores (N=135; 36,59 %) e de medicamentos que agiam no aparelho digestivo e metabolismo (N=48; 13,01 %). Dos medicamentos órfãos registrados e comercializados no âmbito internacional, quase metade, 177 (47,97 %), não apresentavam registros ativos no Brasil e atendem a 327 indicações clínicas (40,82 %).  Conclusão: O Brasil deve analisar afundo os impactos da ausência de um fluxo de registro medicamentos órfãos, que pode afetar diretamente no acesso de tratamento para determinadas doenças raras e negligenciadas.]]></p></abstract>
<abstract abstract-type="short" xml:lang="en"><p><![CDATA[SUMMARY  Introduction: The regulation of specific registrations for the so-called "orphan drugs" has been a strategy of the largest drug agencies in the world to promote access and monitoring of treatment for diseases and conditions of low prevalence or market interest.  Aims: This study aimed to identify the profile of drugs that fall into this category internationally and explore possible gaps in registration generated by the absence of a specific health standard in Brazil.  Methods: Orphan drug registration databases from countries of the European Union and the United States of America were analyzed and the results were compared with the database of the Brazilian Agency.  Results: A total of 369 drugs registered as orphans in European and US agencies were identified, totaling 801 clinical indications. Most of the drugs registered internationally were antineoplastic agents and immunomodulators (N=135; 36.59 %) and drugs that acted on the digestive system and metabolism (N=48; 13.01 %). Of the orphan drugs registered and marketed internationally, almost half, 177 (47.97 %), did not have active registrations in Brazil and meet 327 clinical indications (40.82 %).  Conclusion: Brazil must analyze in depth the impacts of the absence of an orphan drug registration flow, which can directly affect access to treatment for certain rare and neglected diseases.]]></p></abstract>
<abstract abstract-type="short" xml:lang="es"><p><![CDATA[RESUMEN  Introducción: La regulación de registros específicos para los denominados "medicamentos huérfanos" ha sido una estrategia de las agencias de drogas más grandes del mundo promover el acceso y seguimiento del tratamiento de enfermedades y condiciones de poca prevalencia o interés de mercado.  Objetivos: Identificar el perfil de las drogas que entran en esta categoría a nivel internacional explorar posibles lagunas en los registros generadas por la ausencia de un estándar sistema de salud específico en Brasil.  Métodos: Las bases de datos de registro de medicamentos huérfanos de países de la Unión Europea y Estados Unidos da América y los resultados fueron comparados con la base de registros de la Agencia Brasileña.  Resultados: Se identificaron 369 medicamentos registrados como huérfanos en órganos europeos y americanos, totalizando 801 indicaciones clínicas. La mayor parte de los medicamentos registrados a nivel internacional fueron agentes antineoplásicos e inmunomoduladores (N=135; 36,59%) y fármacos que actuaron en el aparato digestivo y metabolismo (N=48; 13,01%). De medicamentos huérfanos registrados y vendidos internacionalmente, casi la mitad, 177 (47,97 %), no tenía registros activos en Brasil y atendió 327 indicaciones clínicas (40,82%).  Conclusión: Brasil debe analizar en profundidad los impactos de la ausencia de un flujo de registro de medicamentos huérfanos, que puede afectar directamente el acceso al tratamiento para ciertas enfermedades raras y desatendidas.]]></p></abstract>
<kwd-group>
<kwd lng="pt"><![CDATA[doenças raras]]></kwd>
<kwd lng="pt"><![CDATA[registro de medicamentos]]></kwd>
<kwd lng="pt"><![CDATA[doenças negligenciadas]]></kwd>
<kwd lng="en"><![CDATA[Rare diseases]]></kwd>
<kwd lng="en"><![CDATA[drug registration]]></kwd>
<kwd lng="en"><![CDATA[neglected diseases]]></kwd>
<kwd lng="es"><![CDATA[enfermedades raras]]></kwd>
<kwd lng="es"><![CDATA[registro de medicamentos]]></kwd>
<kwd lng="es"><![CDATA[enfermedades olvidadas]]></kwd>
</kwd-group>
</article-meta>
</front><back>
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