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1.
Health Policy ; 144: 105080, 2024 Jun.
Article in English | MEDLINE | ID: mdl-38733643

ABSTRACT

Access to drugs for rare diseases constitutes a challenge to healthcare systems, especially those with public funding. This study aimed to map and summarize the criteria used by HTA agencies in different healthcare systems to evaluate reimbursement recommendations for orphan drugs. A comprehensive literature search was performed on the databases PubMed, LILACS, Scopus, and Embase and the gray literature (Google Scholar and websites of HTA agencies). Publications addressing the criteria used by HTA agencies in countries with public healthcare systems when evaluating reimbursement recommendations for orphan drugs were included. This scoping review included 23 studies published between 2014 and 2023, mostly consisting of reviews of HTA reports, guidance documents, and original articles. The criteria were mapped from 19 countries and ranked within three models of healthcare systems (National Health System, National Health Insurance, and Social Health Insurance). All models shared concerns about unmet needs and disease nature. In addition, NHS countries (e.g., United Kingdom, Sweden, and Italy) prioritized innovation and system-level impact, while SHI countries (e.g., Germany, France, the Netherlands) usually valued budget impact and employed expedited evaluation processes. This review provides a comprehensive understanding of the general tendencies of each healthcare system model in establishing differentiated criteria to address the challenges posed by the limited evidence and investment in the field of rare diseases.


Subject(s)
Delivery of Health Care , Orphan Drug Production , Rare Diseases , Technology Assessment, Biomedical , Orphan Drug Production/economics , Humans , Rare Diseases/drug therapy , National Health Programs
2.
Cien Saude Colet ; 26(11): 5547-5560, 2021 Nov.
Article in Portuguese, English | MEDLINE | ID: mdl-34852089

ABSTRACT

This study aims to describe the profile for the requested incorporation of rare disease drugs submitted to CONITEC and its recommendations, comparing the incorporation criteria employed by other HTA agencies globally. To this end, requests for the treatment of rare diseases submitted to CONITEC from July 2012 to June 2019 and its recommendations to the Brazilian Unified Health System (SUS) were included in this study. Subsequently, we compared the criteria used by CONITEC and other HTA agencies to incorporate these drugs. Sixty medicine incorporation requests to treat thirty rare diseases were submitted to CONITEC. Pharmaceutical companies made the most requests (66%). Budget impact analyses were presented in 85% of the requests and HT economic analyses in 68%. A total of 52% of the requests were incorporated into the SUS. CONITEC's justifications for the non-incorporation were the lack of quality clinical evidence, non-cost-effective technologies, and modest clinical benefits that do not justify the high prices. International HTA agencies (CAN, UK, FR, AUS) use different criteria for rare diseases assessments. The data indicate that most of the evaluated drugs were incorporated into the SUS, and adopting different criteria to assess the incorporation of rare diseases medicines will possibly strengthen decision-making.


Descrever o perfil de solicitações de incorporação de medicamentos para doenças raras (DR) enviadas à Comissão Nacional de Incorporação de Tecnologias no SUS (CONITEC) e suas recomendações, comparando critérios usados para incorporação com outras agências de avaliações de tecnologias em saúde (ATS) no mundo. Para tanto, foram avaliadas as solicitações submetidas à CONITEC e suas recomendações ao SUS, de julho de 2012 a junho de 2019, para tratamento de DR. A seguir, foi feita comparação dos critérios utilizados pela CONITEC e por outras agências de ATS para incorporação destes medicamentos. Houve 60 solicitações de incorporação para 30 DR à CONITEC. A maioria das solicitações (66%) foi feita por indústrias farmacêuticas. Análises de impacto orçamentário foram apresentadas em 85% das solicitações e análises econômicas de tecnologias em saúde em 68% delas. Trinta e duas (52%) do total de avaliações foram incorporadas ao SUS. As justificativas da CONITEC para a não incorporação foram ausência de evidência clínica, tecnologias não custo-efetivas e modestos benefícios clínicos que não justificam o preço. Agências de ATS internacionais (UK, FR, CAN, AUS) usam critérios diferenciados para avaliações de DR. Os dados apontam que a maioria dos medicamentos avaliados foi incorporada ao SUS e que a adoção de critérios diferenciados para avaliação da incorporação de medicamentos para DR possivelmente trará robustez à tomada de decisão.


Subject(s)
Pharmaceutical Preparations , Rare Diseases , Brazil , Government Programs , Humans , Rare Diseases/drug therapy , Technology Assessment, Biomedical
3.
Ciênc. Saúde Colet. (Impr.) ; 26(11): 5547-5560, nov. 2021. tab, graf
Article in English, Portuguese | LILACS | ID: biblio-1350459

ABSTRACT

Resumo Descrever o perfil de solicitações de incorporação de medicamentos para doenças raras (DR) enviadas à Comissão Nacional de Incorporação de Tecnologias no SUS (CONITEC) e suas recomendações, comparando critérios usados para incorporação com outras agências de avaliações de tecnologias em saúde (ATS) no mundo. Para tanto, foram avaliadas as solicitações submetidas à CONITEC e suas recomendações ao SUS, de julho de 2012 a junho de 2019, para tratamento de DR. A seguir, foi feita comparação dos critérios utilizados pela CONITEC e por outras agências de ATS para incorporação destes medicamentos. Houve 60 solicitações de incorporação para 30 DR à CONITEC. A maioria das solicitações (66%) foi feita por indústrias farmacêuticas. Análises de impacto orçamentário foram apresentadas em 85% das solicitações e análises econômicas de tecnologias em saúde em 68% delas. Trinta e duas (52%) do total de avaliações foram incorporadas ao SUS. As justificativas da CONITEC para a não incorporação foram ausência de evidência clínica, tecnologias não custo-efetivas e modestos benefícios clínicos que não justificam o preço. Agências de ATS internacionais (UK, FR, CAN, AUS) usam critérios diferenciados para avaliações de DR. Os dados apontam que a maioria dos medicamentos avaliados foi incorporada ao SUS e que a adoção de critérios diferenciados para avaliação da incorporação de medicamentos para DR possivelmente trará robustez à tomada de decisão.


Abstract This study aims to describe the profile for the requested incorporation of rare disease drugs submitted to CONITEC and its recommendations, comparing the incorporation criteria employed by other HTA agencies globally. To this end, requests for the treatment of rare diseases submitted to CONITEC from July 2012 to June 2019 and its recommendations to the Brazilian Unified Health System (SUS) were included in this study. Subsequently, we compared the criteria used by CONITEC and other HTA agencies to incorporate these drugs. Sixty medicine incorporation requests to treat thirty rare diseases were submitted to CONITEC. Pharmaceutical companies made the most requests (66%). Budget impact analyses were presented in 85% of the requests and HT economic analyses in 68%. A total of 52% of the requests were incorporated into the SUS. CONITEC's justifications for the non-incorporation were the lack of quality clinical evidence, non-cost-effective technologies, and modest clinical benefits that do not justify the high prices. International HTA agencies (CAN, UK, FR, AUS) use different criteria for rare diseases assessments. The data indicate that most of the evaluated drugs were incorporated into the SUS, and adopting different criteria to assess the incorporation of rare diseases medicines will possibly strengthen decision-making.


Subject(s)
Humans , Pharmaceutical Preparations , Rare Diseases/drug therapy , Technology Assessment, Biomedical , Brazil , Government Programs
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