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A Avaliação de Tecnologias em Saúde (ATS) considera os domínios de benefícios clínicos, perfil epidemiológico, inovação, custo-efetividade, ética e de equidade no processo de decisão dos gestores em saúde. No contexto dos medicamentos para doenças raras, é desafiador o trabalho da ATS, dada a baixa disponibilidade de evidências robustas e o alto custo unitário das tecnologias. O objetivo da revisão foi analisar as estratégias disponíveis de avaliação das demandas de incorporação de medicamentos para o tratamento de doenças raras em sistemas de saúde. Foi realizada uma revisão rápida com busca estruturada na base de dados MEDLINE (via PubMed), Cochrane Library e Health Systems Evidence. Incluíram-se estudos sobre estratégias de avaliação de medicamentos utilizados para tratamento de doenças raras. Adicionalmente, foram realizadas buscas nas Agências de ATS do Brasil, Austrália, Nova Zelândia, Canadá, Reino Unido, França, Estados Unidos e Alemanha. A síntese dos resultados foi qualitativa com o agrupamento dos achados nos seguintes eixos temáticos: Segurança e efetividade, Custo-efetividade, Impacto orçamentário e Perspectiva da sociedade. Foram identificadas 267 publicações, sendo selecionadas 16 das bases de dados indexadas e 7 da literatura cinzenta. Com a análise dos documentos, pode-se concluir que a adoção de critérios específicos harmonizada com o atual modelo de ATS é um possível caminho a ser seguido no contexto dos medicamentos para doenças raras. Concomitante a isso, abordagens no sentido de incentivo a pesquisa e produção de dados de mundo real e a criação de comitês específicos para tratativa do tema nas agências de ATS apresentam-se como alternativa para lidar com as fragilidades no contexto de doenças raras.
The Health Technology Assessment (HTA) considers evidence regarding clinical benefits, epidemiological profile, innovation, cost-effectiveness, ethics and equity in its assessment process to support managers' decisions. In the context of drugs in rare diseases, the work of the ATS is challenging given the low availability of evidence and the high cost of technologies. The objective of the review was to analyze the available strategies for evaluating the demands for incorporating drugs for the treatment of rare diseases in health systems. A rapid review was performed with a structured search in the MEDLINE database (via PubMed), the Cochrane Library and Health Systems Evidence. Studies on strategies for evaluating drugs used to treat rare diseases were included and, additionally, searches were carried out in ATS Agencies in Brazil, Australia, New Zealand, Canada, United Kingdom, France, United States and Germany. The synthesis of the results was qualitative, grouping the major ones into thematic axes: Safety and effectiveness, Cost-effectiveness, Budgetary impact and Society's perspective. 267 publications were identified, 16 selected from indexed databases and 7 from gray literature. With the analysis of the documents, it can be concluded that the adoption of specific criteria harmonized with the current ATS model is a possible path to be followed in the context of drugs for rare diseases. At the same time, approaches to encourage research and the creation of specific committees to deal with the issue in HTA agencies would complement actions towards the consolidation of this work.
Subject(s)
Orphan Drug Production , Technology Assessment, Biomedical , Rare DiseasesABSTRACT
Introducción: Las enfermedades raras conforman las afecciones de baja prevalencia que asociadas a los medicamentos huérfanos representan un problema sanitario y social mundial. Objetivo: Revisar los aspectos más sobresalientes relacionados con las enfermedades raras, con una visión gastroenterológica, y su repercusión en la infancia. Métodos: Se realizaron búsquedas no estructuradas de publicaciones en español e inglés en PubMed, Google Scholar, Scimago, SciELO, desde enero 2010 hasta agosto 2021. Se usaron los términos: enfermedades raras, conceptualización, prevalencia, epidemiología, medicamentos huérfanos y ética. Análisis y síntesis de la información: Se revisaron las enfermedades raras en la infancia, criterios conceptuales, epidemiología global, enfermedades más reconocidas con énfasis en gastroenterología. Se destacó la prevalencia, vínculo genético, importancia social, dilema diagnóstico y categorías; repercusión de los tratamientos con medicamentos huérfanos, sus costos y problemas éticos. Se resaltó la incidencia de enfermedades digestivas y el valor de la endoscopia y la biopsia en el diagnóstico. Conclusiones: Se documentaron las enfermedades raras en la infancia, y se analizaron como problema mundial, sanitario y social. El desarrollo de la técnica y de la ciencia, resultaron contribuciones decisivas que variaron criterios sobre diferentes afecciones catalogadas como raras(AU)
Introduction: Rare diseases are the conditions of low prevalence associated with orphan drugs and they represent a global health and social problem. Objective: To review the most outstanding aspects related to rare diseases, with a gastroenterological view, and their impact on childhood. Methods: Unstructured searches for publications in Spanish and English in PubMed, Google Scholar, Scimago, SciELO were conducted, from January 2010 to August 2021. The terms rare diseases, conceptualization, prevalence, epidemiology, orphan drugs and ethics were used. Analysis and synthesis of information: Rare diseases in childhood, conceptual criteria, global epidemiology, and the most well-known diseases with emphasis on gastroenterology were reviewed. Prevalence, genetic link, social importance, diagnostic dilemma and categories, also the impact of orphan drug treatments, their costs and ethical problems were highlighted. The incidence of digestive diseases and the value of endoscopy and biopsy in diagnosis were highlighted. Conclusions: Rare diseases in childhood were documented and analyzed as a global health and social problem. The development of technique and science were decisive contributions that varied criteria on different conditions classified as rare(AU)
Subject(s)
Humans , Child, Preschool , Concept Formation , Rare Diseases/drug therapy , Rare Diseases/epidemiology , Biopsy , Digestive System Diseases/epidemiology , Endoscopy/economicsABSTRACT
@#Rare diseases, also known as "orphan diseases", refer to diseases with very low incidence. Countries and regions define rare diseases according to epidemiological standards, economic standards of rare drugs and disease severity. The World Health Organization (WHO) has suggested the prevalence rate of less than 6.5 to 10 per 10 000 people to define rare diseases. In May 2018, "China's First List of Rare Diseases" was released, including 121 rare diseases. Most rare diseases are hereditary diseases with early onset, severe disease, and poor prognosis. About 75% of rare genetic diseases occur in the neonatal period or childhood, which are important part of human birth defects and brings a huge burden to society and families. The effective prevention and treatment of rare diseases is one of the important goals of building a "Healthy China". With the development of molecular biology technology and the continuous research and development of advanced medical products in the field of gene therapy, the level of clinical diagnosis and treatment of rare diseases has risen to a new level, which provides a possibility for the cure of some rare diseases. In China, most rare diseases rely on imported drugs, which cost a lot and bring heavy economic burden to patients. Improving the medical insurance system for rare diseases has become a difficult point in the current medical reform. This paper mainly discusses the definition of rare diseases, the research status, efforts and future development direction of rare diseases in China, in order to deepen the understanding and response of medical workers and the whole society to rare diseases.
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OBJECTIVE To systematically introduce the supply guarantee system of orphan drugs in South Korea， and to provide reference for improving the accessibility of orphan drugs in China. METHODS The basic characteristics and practical experience of supply guarantee system of orphan drugs in South Korea were summarized by studying the marketing incentive mechanism， reserve supply mechanism and emergency use mechanism. Then， based on the research on the current situation and existing problems of orphan drug supply in China， specific suggestions were put forward to improve the accessibility of orphan drugs in China. RESULTS & CONCLUSIONS South Korea has effectively improved the accessibility of orphan drugs through orphan drug identification channels and supporting incentive policies， relying on the reserve supply mechanism and the import route for emergency use. Therefore， it is suggested that China should guarantee the normal supply of orphan drugs from three aspects： improving the recognition and incentive policy of orphan drugs， building a full-time management department of orphan drugs， and optimizing the temporary import path.
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Rare diseases are characterized by extremely low incidence rate and small number of patients in total. The drugs used to treat rare diseases are called orphan drugs. Currently, 450 kinds of rare skin diseases have been identified, most of which lack in effective treatments. Supported by the policy-making from the country and pushed by all sectors of the society, drugs for rare skin diseases have been emerging continuously recently in China. This paper reviews the current accessibility of the orphan drugs for skin rare diseases that have been approved in China and in other countries and regions for a better understanding of rare dermatosis and orphan drugs for the diseases.
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Methylmalonic acidemia(MMA) is one of the common diseases in hereditary organic acid metabolism disorder. The disorder is mainly treated by using vitamin B12 which comes in various forms.The use of the methylcobalamin and adenosylcobalamin in China cannot meet the needs of children with MMA. Parenteral use of hydroxocobalamin of high concentration has significant advantages in the treatment of MMA. But, the accessibility to hydroxocobalamin is challenging now.Through the supervision and adjustment of hydroxocobalamin, we explored the strategy of using the drugs. Meanwhile, we are considering the oral use of the hydroxocobalamin as supplement to treatment. Solving the challenges of the hydrococobalamin is helpful to meet the treatment of MMA and to provide a new possibility for the use of medication for rare diseases. The improvement in policy and supervision measures will facilitate the development, involvement, and production of hydroxocobalamin, so that the patients will have a good chance of access to the treatment.
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The National Health Commission of the People′s Republic of China and six other departments jointly released the Second Catalog of Rare Diseases, which expanded the definition of rare diseases in China. The expansion of the catalog means greater attention to rare diseases at the national level, and is also expected to accelerate the launch of relevant rare disease drugs and benefit more rare disease patients. When the new rare disease catalog released, it is necessary to quickly sort out the availability of drugs and find out the base number for the advancement of subsequent relevant measures. This paper searched official medical information websites and medical databases such as the National Medical Products Administration (NMPA), the Center for Drug Evaluation (CDE), and foreign drug regulatory agencies, and sorted out and summarized the subject categories of diseases included in the Second Catalog of Rare Diseases, the corresponding global research and development and implementation of drugs, as well as the medical insurance access situation in China, elaborates on the differences in the accessibility of orphan drugs at home and abroad. As China′s prevention, treatment and protection of rare diseases has reached a new point, plans and prospects are made for the introduction of relevant policies in the future, as well as the promotion of research and development and protection and other key tasks.
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Políticas públicas que atendam a população de doenças raras no Brasil são cada vez mais discutidas, seja para acesso aos medicamentos, para atenção multidisciplinar, alternativas terapêuticas ou representatividade. Apesar de o Ministério da Saúde ter publicado em 2014 a Portaria nº 199, que instituiu a Política Nacional de Atenção Integral às Pessoas com Doenças Raras, aprovando as Diretrizes para Atenção Integral às Pessoas com Doenças Raras no âmbito do Sistema Único de Saúde (SUS), essa população segue desamparada frente às peculiaridades de seus tratamentos. Neste cenário, associações de pacientes passaram a buscar espaços de representação política, provocando cada vez mais iniciativas legislativas no Congresso Nacional com vistas a alternativas para os milhões de brasileiros que vivem ou convivem com doenças raras. Por meio de uma pesquisa documental qualitativa, o artigo considera todos os projetos apresentados até agosto de 2020 com foco neste tema, identificando os principais parlamentares que atuam nele, as características biográficas comuns entre eles, em qual etapa do ciclo de políticas públicas o tema está no Congresso Nacional e, com isso, pretende identificar um possível caminho para a consolidação de uma política. Observou-se que a atuação em doenças raras independe de alinhamento político partidário, sendo uma pauta compartilhada por grupos antagônicos, mas que enfrenta o desafio de apenas 18 parlamentares terem sidos os responsáveis por 50% de todas as matérias legislativas já apresentadas sobre isso. Além disso, observou-se também que os parlamentares preferem impulsionar o debate e dar visibilidade ao tema do que investir em novas tentativas de mudanças legais e regulatória.
Public policies that serve the Brazilian population with rare diseases are increasingly discussed, either regarding access to medicines, multidisciplinary care, therapeutic alternatives, or representativeness. Although the Brazilian Ministry of Health published in 2014 the Ordinance n. 199, which instituted the Brazilian National Policy of Integral Care for People with Rare Diseases and approving the Guidelines for Integral Care to People with Rare Diseases under the Brazilian Unified National Health System (SUS), this population remains unaided regarding the peculiarities of their treatments. In this scenario, patient associations began to seek spaces for political representation, increasingly provoking legislative initiatives in the Brazilian National Congress, seeking for alternatives for the millions of Brazilians living with rare diseases. By using a qualitative documentary research, the study considers all projects presented until August 2020 on this topic, identifying the main parliamentarians involved, their biographical characteristics, and at which stage of the public policy cycle this topic is in the Brazilian National Congress; thus, identifying a possible path for the consolidation of a policy. We observed that the actions regarding rare diseases are independent of any political party alignment, being an agenda shared by antagonistic groups. This agenda, however, faces the challenge of having only 18 parliamentarians responsible for 50% of all the legislative matters already presented. Furthermore, we also observed that parliamentarians prefer to boost the debate and give visibility to the subject than to invest in new attempts at legal and regulatory changes.
Las políticas públicas que asisten a la población que sufre enfermedades raras en Brasil son cada vez más discutidas, ya sea en cuanto al acceso a medicamentos, a la atención multidisciplinaria, a alternativas terapéuticas o a la representatividad. Aunque el Ministerio de Salud brasileño publicó la Ordenanza nº 199 en el 2014, que estableció la Política Nacional de Atención Integral a las Personas con Enfermedades Raras, y aprobando las Directrices para la Atención Integral a las Personas con Enfermedades Raras en el ámbito del Sistema Único de Salud (SUS), esta población permanece impotente ante las peculiaridades de sus tratamientos. En este escenario, las asociaciones de pacientes comenzaron a buscar espacios de representación política, dando lugar a cada vez más iniciativas legislativas en el Congreso Nacional brasileño con vistas a alternativas para los millones de brasileños que viven o conviven con enfermedades raras. Por medio de una investigación documental cualitativa, este artículo considera todos los proyectos presentados hasta agosto del 2020 centrados en este tema, identificando a los principales parlamentarios que actúan en él, las características biográficas comunes entre ellos, en qué etapa del ciclo de políticas públicas se encuentra el tema en el Congreso Nacional brasileño y, con ello, identificar un posible camino para la consolidación de una política. Se observó que la actuación relacionada con enfermedades raras no depende de la alineación político-partidista, ya que es una agenda compartida por grupos antagónicos, pero que se enfrenta al desafío de que solo 18 parlamentarios hayan sido los responsables del 50% de todos los asuntos legislativos que ya se presentaron al respecto. Además, también se observó que los parlamentarios prefieren impulsar el debate y dar visibilidad al tema que invertir en nuevos intentos de cambios legales y regulatorios.
Subject(s)
Rare Diseases/therapy , National Health Programs , Public Policy , Brazil , Government Programs , Health PolicyABSTRACT
We have analyzed the current status of recognization and qualification of orphan drugs in China and abroad, looking at the aspects of the authority institutions, identification and qualification process, and the number of orphan drugs identified and available in the market. By comparing and analyzing horizontally the differences in orphan drugs identification between representative developed countries vs. some developing countries, we discuss the inadequacy of orphan drugs supervision in China. We introduce the advanced experience from the developed countries and some developing countries to provide suggestions for the identification and management of orphan drugs, hoping to speed up the process of development and market availability of orphan drugs and to maximize patient's accessibility to treatment in China.
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@#Comprehensive clinical drug evaluation is an important technical tool for decision-making on drug supply. Traditional evaluation on health technology provides methodological references for comprehensive clinical evalu-ation of drugs. However, data from clinical trials for orphan drugs are often insufficient. It is difficult to evaluate the clinical and economic value of orphan drugs by using the criteria of ordinary drugs. Particularity of orphan drugs makes it difficult to use the traditional evaluation methods for health technology. Multiple criteria decision analysis explores the comprehensive value of drugs from different angles. It promotes the transparency and scientific thinking on the basis of evidence-based medicine, improves the quality of decision-making, and thus provides a possible solution to the comprehensive clinical evaluation of orphan drugs. However, its application in the comprehensive evaluation of orphan drugs in China is still in its infancy and trial stage, and no methodological guidelines is available. In order to optimize the construction of a diversified system for comprehensive clinical evaluation of orphan drugs in China, the Multi-disciplinary Team for Rare Diseases of Peking Union Medical College Hospital and China Alliance for Rare Disease organized experts in related fields to formulate this consensus, aiming to provide standardized methodological guideline to the comprehensive clinical evaluation of orphan drugs.
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Resumen En este estudio se analizaron las solicitudes de importación de Medicamentos vitales no disponibles (MVND) presentadas al INVIMA y los registros de solicitudes de recobros presentados en la ADRES por MVND, en el periodo 2016-2017. De las 2321 solicitudes de importación de MVND, el 76% fueron autorizadas. Se identificaron 88 solicitantes, 73 subgrupos terapéuticos y 195 principios activos, así como 368 diagnósticos. El 66% de los pacientes registrados en las solicitudes de importación se encuentran vinculados al régimen contributivo, en menor proporción al régimen subsidiado y al régimen especial o excepcional. En solicitudes de recobros asociados a MVND por vía judicial, se observó que, el monto solicitado por recobro fue de USD 8.577.583, equivalentes a 38483 UPCs. Los resultados mostraron que la implementación del decreto 481 de 2004 ha sido útil para garantizar el acceso a medicamentos para enfermedades raras, aunque no es ajena a la inequidad estructural de acceso a servicios de salud y medicamentos del SGSSS y que generan impacto, no solo en la salud pública, si no en el presupuesto asignado, bien sea por el alto costo que implica su importación o por la falta de regulación de estos dentro del mercado nacional.
Abstract This study analyzed the import applications for unavailable vital medicines (MVND) submitted to INVIMA and the records of MVND reimbursement requests submitted to the ADRES in the 2016-2017 period. Approximately 76% of the 2,321 MVND import applications were authorized. Eighty-eight applicants, 73 therapeutic subgroups, 195 active ingredients, and 368 diagnoses were identified. Most of the patients registered in the import applications (66%) are linked to the contributory regime, to a lesser extent to the subsidized regime and the Special or exceptional regimes. The total value of the reimbursement requests related to MVND granted by lawsuits, was USD 8,577,583, equivalent to 38,483 UPCs. The results showed that the implementation of Decree N° 481/2004 has ensured access to medicines for rare diseases. However, it is not alien to the structural inequality of access to health services and medicines of the Colombian Health System, which impacts public health and the allocated budget, either because of the high cost of importing MVND or because of the lack of MVND regulation within the national market.
Subject(s)
Humans , Rare Diseases , Government Programs , ColombiaABSTRACT
Budget impact analysis of drugs for rare diseases in different countries are reviewed. There are more than 7 000 kinds rare disease characterized by low incidence, serious influence on patients and society, difficulty in diagnosis, less effective intervention and therapeutic drugs. With the orphan drug legislation of the United States, the European Union and other countries, the number of drugs for rare diseases is increasing. At present, the United States has approved more than 600 drugs for rare diseases, and the European Union has approved more than 100 drugs for rare diseases. The types of rare diseases, the number of patients receiving treatment, the number of approved drugs for rare diseases, the annual cost of drug treatment for rare diseases and the availability of drugs are the main factors affecting the budget. The study shows that with the increase in the number of drugs approved for rare diseases, the proportion of drugs expenditure for rare diseases in the total drug expenses showed a slow rising trend, and there are national and regional differences. The overall budget impact of orphan drugs is small and fairly stable relative to total pharmaceutical expenditure. In addition, a small number of rare diseases drugs used to treat tumors and metabolic diseases have a greater impact on the total drug cost.
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OBJECTIVE: To explore the suggestions for the development of orphan drugs in China through comparative analysis of the status of orphan drugs in China and international developed countries or regions. METHODS: Based on “the first list of rare diseases” developed by the National Health Commission of the People′s Republic of China and other four departments,the accessibility of orphan drugs in China and abroad was compared. RESULTS: Until the end of 2017,the orphan drugs in China accounted for 38.33% of the 480 orphan drugs approved in the US, and 29.57% of the 115 orphan drugs approved in the EU. From the “the first list of rare diseases”, there are 60 orphan drugs for 36 diseases in the US and 27 orphan drugs for 18 diseases in the EU, and the orphan drugs approved in China accounted for 43.33% and 29.63, respectively. The 51.85% of the orphan drugs approved in China for the rare diseases of “the first list of rare diseases” are completely dependent on imports. CONCLUSION: There are still gaps of orphan drugs for “the first list of rare diseases”between China and the international developed countries or regions. Thus, it is urgent to promote legislation to speed up the research and limitation of orphan drugs in order to improve the development of orphan drugs in China.
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@#There had been increased and strong public interests in rare diseases and orphan drugs as well as the issue of compulsory licencing for expensive medications in Malaysia in the mass-media and social media. We reviewed the issues of orphan drugs and the challenges faced in many countries in developing appropriate health financial modelling as well as getting accurate data on rare diseases. We also reviewed the old off-patent medications and the developments on how policy-makers can intervene to make expensive treatment affordable and sustainable for patients and the country
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A partir de la década de los 80, las enfermedades raras han ido adquiriendo un lugar prioritario en los programas de salud y en la opinión pública. Se definen como aquellas que tienen una prevalencia menor a 1:2.000 individuos. En general son enfermedades crónicas, invalidantes y en más de un 80% de origen genético. Se estima que existen entre 7.000 y 8.000 enfermedades raras y que afectan al 6-8% de la población. Dada la baja prevalencia específica de cada afección, hay muy poco conocimiento de parte de la comunidad médica en relación a su diagnóstico y manejo. En este artículo se discute la importancia del diagnóstico de precisión, para su adecuado manejo y asesoramiento genético, también se muestra la importancia del trabajo en redes en las enfermedades de baja prevalencia y se destaca el rol fundamental de las agrupaciones de padres y familiares en promover políticas de salud para los afectados.
From the early 80's rare diseases had achieved a priority role in national health programs and in public opinion. Rare diseases are define as the ones who have a prevalence lower than 1:2000 habs. Generally, they are chronic and life threatening diseases and more than 80% of them are from genetic origin. It is estimated that there are between 7,000 and 8,000 different rare diseases affecting 6-8% of world population. Due to the low prevalence of each disease, there are very poor knowledge in the medical community about their diagnosis and management. In this review we discuss about the importance of the precise molecular diagnosis for the best treatment and genetic counselling; we also showed the importance of working in network in these diseases of low prevalence and we discuss about the fundamental role of parents associations in promoting public health politics for affected people.
Subject(s)
Humans , Rare Diseases/diagnosis , Rare Diseases/genetics , Rare Diseases/epidemiology , Orphan Drug Production , Phenotype , Prevalence , Genetic Counseling , International CooperationABSTRACT
The implementation of a specific policy for rare diseases in the Brazilian Unified Health System presents challenges in terms of its rationale. Recognizing the importance of rarity in the context of public health means understanding genetics as one of the dimensions of disease and accepting that Brazil is undergoing a period of transition in health indicators. Although most rare diseases lack pharmacological treatment and genetic counseling constitutes the best strategy for their prevention, the cost of "orphan drugs" and their consequent lack of cost-effectiveness are still claimed as hurdles to the implementation of public policies in this field. Epidemiological aspects should not be used as isolated criteria for prioritization in public policies
Subject(s)
Humans , Pharmaceutical Services/history , Rare Diseases/economics , Rare Diseases/drug therapy , Health Policy , Genetic Diseases, Inborn/drug therapy , Orphan Drug Production/legislation & jurisprudence , Research Support as Topic , Bioethics , Brazil , Cost-Benefit Analysis , Biomedical Research , Resource Allocation/organization & administration , Genetics, Medical/history , Health Services Accessibility/organization & administration , Genetic Diseases, Inborn/epidemiologyABSTRACT
Los medicamentos huérfanos son aquellos requeridos para diagnosticar, prevenir o tratar enfermedades poco frecuentes, pero que pueden ser graves y debilitantes. Este trabajo tiene como objetivo proponer la implementación de una normativa de registro sanitario específica para los medicamentos huérfanos en la República Bolivariana de Venezuela, considerando la situación actual de salud y las necesidades de la población, a fin llegar a todos los niveles, facilitando la disponibilidad del mayor número de medicamentos que son requeridos por un grupo reducido de pacientes. Es necesario que las autoridades sanitarias en Venezuela fortalezcan el sistema de salud pública estableciendo políticas para satisfacer las necesidades de la población en general pero que también incluya a las minorías. Se plantean las consideraciones técnicas, legales, fiscales y regulatorias necesarias para la implementación de esta normativa, así como los criterios de inclusión para clasificar un medicamento dentro la categoría de "huérfanos". La metodología utilizada fue de tipo documental, haciendo énfasis en el análisis teórico y conceptual del material a fin de elaborar la propuesta de la normativa para medicamentos huérfanos en la República Bolivariana de Venezuela. Se realizó un análisis teórico de la información obtenida para determinar la situación actual nacional e internacional a fin de obtener los resultados que sirvieron de base para desarrollar la propuesta.
Orphan drugs are those required to diagnose, prevent or treat rare diseases but that may be severe and debilitating; besides, reduce the quality of life of patients and their families. This project aims to propose a special regulation for Orphan Drugs in the Bolivarian Republic of Venezuela taking into account the current health situation and needs of the population in order to reach all levels by facilitating the availability of a bigger amount of drugs required by a small group of patients. It is necessary for health authorities in Venezuela to strengthen the public health system by establishing policies to meet the needs of general population but also include minorities. Technical, legal and regulatory considerations arise for the implementation of this normative, as well as the inclusion criteria for drugs within the category of "orphans". The methodology used is documentary, as it emphasizes the theoretical and conceptual analysis of the material for the proposed regulation for orphan drugs in the Bolivarian Republic of Venezuela. A theoretical analysis of the information was made to determine the current national and international situation in order to obtain the results as a basis to develop the proposal.
Subject(s)
Humans , Orphan Drug Production , Rare Diseases , Drug Industry , Products Registration , Pharmaceutical TradeABSTRACT
The orphan drug management system can significantly improve the quality of care and that of life of patients suffering from rare diseases, and accelerate the progress of medicinal science. With experiences of practicing the orphan drug management system in developed countries and regions,including certification of orphan drugs, supports for developing orphan drugs, market protection and clinic application management. Based on such studies, the authors discussed the feasibility and methodology of establishing an orphan drug management system in China, along with the significance of such a system.