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Learn more about In Review
Research Article
Silvana Nair Leite
Universidade Federal de Santa Catarina
Corresponding Author
License:
This work is licensed under a CC BY 4.0 License. Read Full License
Background
Rare diseases affect a small number of people compared to prevalent diseases' estimates. The vast majority of these diseases are of genetic origin, have no cure, are chronic and can lead to death. Although the right to access to medicines is included in the constitutionally guaranteed right to health in Brazil, problems in the supply of medicines for rare diseases are reported in the country.
Objective
This study aimed to describe and analyze the initiatives to promote access to medicines for treating rare diseases in Unified Health System, Brazil, after the publication of the National Policy on Comprehensive Care of People with Rare Diseases.
Methods
Based on the model published by The WHO Regional Office for Europe, which described access to medicines in pre-launch, per-launch and post-launch policies, the initiatives referring to each category were summarized, based on documentary research searched in online databases, in the period from January 2014 to December 2020.
Results
Different actions and policy interventions were identified, which went through the expansion of resources for research and development, health regulations, incorporation of new drugs, review and publication of clinical guidelines, expansion of the network of care facilities by the Ministry of Health. On the other hand, aspects related to care policies, pricing methods, technological development, and development of pharmaceutical services processes were not implemented.
Conclusions
Although it is impossible to determine the explicit motivation of such actions concerning the Policy, its publication certainly was a landmark in Brazilian society, allowing greater recognition of the needs of rare diseases patients and the treatment's specificities. However, the study suggests that the steps that make up the life cycle of medicines are not linked, lacking articulation and integration of the care network and, consequently, there is no evidence that the rare diseases policy publication has generated broad impact on the promotion of access to medicines to treat rare diseases in Brazil.
Keywords
Rare Diseases, Access to medicines, Health policy, Health Technologies, Brazil
No competing interests reported.
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This preprint is under consideration at BMC Health Services Research. A preprint is a preliminary version of a manuscript that has not completed peer review at a journal. Research Square does not conduct peer review prior to posting preprints. The posting of a preprint on this server should not be interpreted as an endorsement of its validity or suitability for dissemination as established information or for guiding clinical practice.
Learn more about In Review
Research Article
Silvana Nair Leite
Universidade Federal de Santa Catarina
Corresponding Author
Badges
Prescreen
This manuscript passed our Prescreen assessment
Complete author information
Appropriate declaration statements
Potential risks to human health
Prescreen
Peer Review Timeline
CURRENT STATUS: Under Review
Version 1
Posted 11 Nov, 2021
No community comments so far
Editor invited
On 08 Nov, 2021
Submission checks complete
On 08 Nov, 2021
First submitted
On 20 Oct, 2021
Metrics
Comments: 0
PDF Downloads: ...
HTML Views: ...
Subject Areas
Health Economics & Outcomes Research
License:
This work is licensed under a CC BY 4.0 License. Read Full License
Background
Rare diseases affect a small number of people compared to prevalent diseases' estimates. The vast majority of these diseases are of genetic origin, have no cure, are chronic and can lead to death. Although the right to access to medicines is included in the constitutionally guaranteed right to health in Brazil, problems in the supply of medicines for rare diseases are reported in the country.
Objective
This study aimed to describe and analyze the initiatives to promote access to medicines for treating rare diseases in Unified Health System, Brazil, after the publication of the National Policy on Comprehensive Care of People with Rare Diseases.
Methods
Based on the model published by The WHO Regional Office for Europe, which described access to medicines in pre-launch, per-launch and post-launch policies, the initiatives referring to each category were summarized, based on documentary research searched in online databases, in the period from January 2014 to December 2020.
Results
Different actions and policy interventions were identified, which went through the expansion of resources for research and development, health regulations, incorporation of new drugs, review and publication of clinical guidelines, expansion of the network of care facilities by the Ministry of Health. On the other hand, aspects related to care policies, pricing methods, technological development, and development of pharmaceutical services processes were not implemented.
Conclusions
Although it is impossible to determine the explicit motivation of such actions concerning the Policy, its publication certainly was a landmark in Brazilian society, allowing greater recognition of the needs of rare diseases patients and the treatment's specificities. However, the study suggests that the steps that make up the life cycle of medicines are not linked, lacking articulation and integration of the care network and, consequently, there is no evidence that the rare diseases policy publication has generated broad impact on the promotion of access to medicines to treat rare diseases in Brazil.
No competing interests reported.
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