Rare diseases and orphan drugs

Melnikova, Irena

doi:10.1038/nrd3654

Cited by 162 publications

(128 citation statements)

References 2 publications

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“…Em alguns estudos, a população afirmou estar disposta a pagar mais por uma doença que é rara ou muito grave (Hughes-- Wilson et al, 2012). Por outro lado, existe um paradoxo relacionado às doenças raras, uma vez que essas doenças acometem pequena quantidade de pessoas, porém existem em grande número (Melnikova, 2012). Dessa forma, é preciso avaliar quanto a sociedade está disposta a pagar pelo tratamento dessas doenças e o custo de oportunidade, pois esses recursos vão representar a perda em algum outro setor, expressando os trade-offs enfrentados pelo gestor.…”

Section: Discussionunclassified

Análise de Decisão Multicritérios (MCDA): uma revisão rápida sobre os critérios utilizados na Avaliação de Tecnologias em Saúde

Souza¹,

Santos²,

Cintra³

2018

JBES

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RESUMOObjetivo: Identificar os vários critérios utilizados por agências e órgãos governamentais internacionais na Avaliação de Tecnologias em Saúde (ATS). Métodos: Foi realizada uma revisão rápida com busca estruturada na base de dados Medline (via PubMed) e Lilacs complementada por busca na literatura cinzenta, sem restrição de idioma. Incluíram-se estudos da área de saúde que descreviam critérios e que utilizaram o método MCDA (Análise de Decisão Multicritérios) na ATS. Excluíram-se estudos que avaliaram apenas uma tecnologia específica ou contexto restrito. Os critérios foram agregados e sumarizados conforme opinião de especialistas em ATS. Resultados: Foram identificadas 3.746 publicações, sendo 27 selecionadas. Os critérios extraídos foram categorizados e seus respectivos atributos foram listados. Dez categorias foram apresentadas: benefício e risco da intervenção (4 critérios); impacto da doença (4 critérios); impacto econômico (5 critérios); contexto terapêutico da intervenção (4 critérios); qualidade e incerteza da evidência (4 critérios); implementação da intervenção (5 critérios); benefícios para a sociedade (5 critérios); benefícios para o sistema de saúde público (5 critérios); benefícios para a indústria (1 critério); e outros (3 critérios). Os atributos mais prevalentes foram: custo-efetividade; segurança e tolerabilidade; impacto orçamentário para o sistema/plano de saúde; gravidade da doença; equidade; efetividade; custo. Conclusões: Os órgãos decisores utilizam grande variedade de critérios para o MCDA, refletindo visões e valores diferentes entre as culturas. O estudo fornece informação para discussão no contexto brasileiro de uma lista mais ampla e possível padronização de critérios a serem utilizados na tomada de decisão pública ou privada sobre a incorporação de novas tecnologias. ABSTRACTObjective: To identify the various criteria used by international government agencies and bodies in Health Technology Assessment (HTA). Methods: A rapid review was conducted with a structured search in the Medline database (via PubMed) and Lilacs supplemented by a search in the gray literature, without a restriction of language. Inclusion criteria were studies in health context, describing the criteria and using the MCDA (Multicriteria Decision Analysis) method in HTA. We excluded studies that assessed only one specific technology or restricted context. The criteria were aggregated and summarized according to the opinion of HTA experts. Results: A total of 3,746 publications were identified, of which 27 were selected. The extracted criteria were categorized, and their respective

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Section: Discussionunclassified

Análise de Decisão Multicritérios (MCDA): uma revisão rápida sobre os critérios utilizados na Avaliação de Tecnologias em Saúde

Souza¹,

Santos²,

Cintra³

2018

JBES

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“…Furthermore, it can be difficult to conduct useful clinical trials for these medicines because few participants may be available, and their conditions are often heterogeneous and poorly understood [6,7]. …”

Section: Introductionmentioning

confidence: 99%

“…These policies have successfully increased interest in rare disease treatment, resulting in the development of thousands of new medicines [1,2,7]. However, even these drugs may not be approved or reimbursed for patients unless the available benefit, risk and cost-effectiveness data is rigorous enough.…”

Section: Introductionmentioning

confidence: 99%

“…However, even these drugs may not be approved or reimbursed for patients unless the available benefit, risk and cost-effectiveness data is rigorous enough. Of over 2,500 medicines designated as orphan drugs since the US's Orphan Drug Act in 1983, only about 400 have been approved for use by patients [6,7]. …”

Section: Introductionmentioning

confidence: 99%

“…Therefore, the use of pre-existing data is often key for the development of rare disease drugs, partly in order to compensate for the limited number and lifespan of many patients and partly due to the importance of repurposing methods. Data availability from previous clinical trials is a key driver of the increasing interest and investment in rare disease treatment [5,6,7]. …”

Section: Introductionmentioning

confidence: 99%

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Clinical Trial Transparency and Orphan Drug Development: Recent Trends in Data Sharing by the Pharmaceutical Industry

Joly²,

Knoppers³

2013

Public Health Genomics

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Background: Data sharing from clinical trials can be key to the development and approval of medicines for rare diseases. Many events during the first half of 2013 have contributed to the movement for increased transparency. These include the development of the European Medicines Agency's new data publication policy, the creation of the AllTrials petition and GlaxoSmithKline's choice to sign it, the launch of GlaxoSmithKline's system for access to patient-level clinical trial data and Roche's commitment to create a similar system, the release of results from the Yale University Open Data Access project's first medicine analysis for Medtronic, and the creation of the Reg4All website. Aims/Objectives: This paper summarises major developments in clinical trial transparency between January and June 2013 and analyses the composition of datasets released by GlaxoSmithKline. Methods: GlaxoSmithKline's database of available trials was tabulated and graphs of relevant trial characteristics were produced. Results/Conclusions: Due to current transparency initiatives, it is likely that much more data will be made available over the next few years through systems similar to GlaxoSmithKline's. Although some aspects of GlaxoSmithKline's model could limit its usefulness, the data currently listed is diverse and could be promising for researchers interested in rare disease treatment.

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Characteristics and Public Availability of Results of Clinical Trials on Rare Diseases Registered at Clinicaltrials.gov

et al. 2016

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same period from 0.3% to 1.1% (P < .001). Euthanasia and ending of life without the patient's explicit request also increased, but the number of studied cases of the 2 categories was small, at 8 and 15 cases in 2001, and at 11 and 25 cases in 2013, respectively, and the increases, from 0.2% to 0.3% for euthanasia and from 0.5% to 0.8% for ending of life without the patient's explicit request, were not significant. The greatest increase was in the use of continuous deep sedation until death, which rose from 4.7% of all deaths in 2001 to 17.5% in 2013 (P < .001). Sedation was combined with forgoing life-prolonging treatment, alleviation of pain and symptoms, or physician-assisted death in 62%, 28%, and 3% of cases, respectively.

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Rare diseases and orphan drugs

Cited by 162 publications

References 2 publications

Análise de Decisão Multicritérios (MCDA): uma revisão rápida sobre os critérios utilizados na Avaliação de Tecnologias em Saúde

Análise de Decisão Multicritérios (MCDA): uma revisão rápida sobre os critérios utilizados na Avaliação de Tecnologias em Saúde

Clinical Trial Transparency and Orphan Drug Development: Recent Trends in Data Sharing by the Pharmaceutical Industry

Characteristics and Public Availability of Results of Clinical Trials on Rare Diseases Registered at Clinicaltrials.gov

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