Banca de DEFESA: Fábio de Oliveira Aquino

Uma banca de DEFESA de DOUTORADO foi cadastrada pelo programa.
STUDENT : Fábio de Oliveira Aquino
DATE: 21/02/2025
TIME: 09:00
LOCAL: https://teams.microsoft.com/l/meetupjoin/19%3ameeting_ZTBkNzJiODAtYmZmYy00ODkxLTk1MmQt
TITLE:

Public Policies for Rare Diseases in Brazil, the USA and the EU: Implications for Research Development and Access to Orphan Drugs in Brazil

 

KEY WORDS:

“ Rare Diseases; Health policy; Health Systems; Health Care; Orphan Drug Production; Access to Essential Medicines and Health Technologies. ”

PAGES: 100
BIG AREA: Ciências da Saúde
AREA: Saúde Coletiva
SUMMARY:

“ Introduction: Rare diseases represent a significant public health challenge worldwide, affecting millions of people. With a wide range of symptoms and causes, it is estimated that there are between 6,000 and 8,000 different types of rare diseases, 80% of which are genetic in origin and 75% of which occur in childhood. In Brazil, around 13 million people live with this health condition. The aim is to strengthen national public policies and improve access to treatment for patients with rare and ultrarare diseases in Brazil. Objectives: To analyze the regulatory models and public policies related to rare diseases in Brazil, the United States and the European Union, in addition to investigating research protocols on the subject submitted to Plataforma Brasil between 2013 and 2023, presenting the profile of the studies and discussing the various forms of access to orphan drugs in Brazil. Methods: This descriptive study used a methodological approach that combined documentary research, analysis of official websites and a literature review, employing interpretative and statistical techniques in qualitative and quantitative approaches. The search covered official documents on public policies and ethical regulations, including legislation, guidelines, presentations and reports, with a focus on rare diseases. The sources consulted were the websites of the Ministry of Health, Anvisa, the European Medicines Agency (EMA) and the Food and Drug Administration (FDA). Results: The comparative analysis suggests that Brazil can adopt successful practices from the United States and the European Union, such as intersectoral partnerships, tax incentives and the involvement of patient associations. These findings indicate an increase in investment in research into rare diseases, which could boost the development of new therapies and the formulation of more effective public policies. The gradual harmonization of policies is especially advantageous for developing countries, improving the care of patients' needs. In Brazil, of the 1,500 research protocols analyzed, 9 were duplicates, resulting in 1,491 unique studies. Of these, 1,301 (87.26%) were approved, 38 (2.55%) were not approved and 152 (10.19%) were pending. The average sample size was 153 individuals, with a standard deviation of 817.03; the median was 22, with a minimum of 0 and a maximum of 20,000. Between 2013 and 2019, the number of studies increased, from 58 (4.46%) in 2013 to 160 (12.30%) in 2019. Although there was a decrease in 2020 and 2021, the number of studies grew again, reaching 176 in 2023. The statistical analysis revealed that 92.24% of the studies were single-center and 7.76% multicenter. In terms of study design, 87.78% were observational and 12.22% intervention/experimental. Conclusions: This study revealed the complexity and importance of public policies and regulatory models for rare diseases in Brazil, the United States and the European Union, and from a comparative analysis showed that Brazil can be significantly strengthened by adopting successful practices observed in the regions studied, such as intersectoral partnerships, tax incentives and the active involvement of social control. These elements are decisive for increasing investment in research into rare diseases, which in turn can drive the development of new therapies and the formulation of more effective public policies that meet the real needs of individuals, taking into account their socioeconomic, cultural and regional characteristics. Analysis of the research protocols submitted to Plataforma Brasil between 2013 and 2023 showed a significant increase in the number of studies, which may indicate an increase in interest and investment in research into rare diseases in Brazil. However, the variation in the number of studies over the years highlights the need for more stable and continuous policies to sustain this growth, which is reflected in our country through the fragility of having a “National Policy for Comprehensive Care for People with Rare Diseases (PNAIPDR), instituted in 2014 by the Ministry of Health, through Ordinance GM/MS No. 199, which does not give it the weight and stability of a federal law, although the data analyzed shows that Brazil has made progress in building public policies for rare diseases, but there are still challenges to be overcome, such as ensuring equitable access to orphan drugs, strengthening clinical research and promoting multidisciplinary care. In addition, the predominance of observational and single-center studies suggests the need for greater encouragement to carry out multicenter and intervention studies, which can provide more robust and generalizable data, which reinforces the importance and need for continuous support and adequate infrastructure to carry out high-quality research .”

COMMITTEE MEMBERS:
Interna - 404501 - DIRCE BELLEZI GUILHEM
Externo à Instituição - ERICA CARINE CAMPOS CALDAS ROSA - IESB
Presidente - ***.616.438-** - MARIA RITA CARVALHO GARBI NOVAES - UnB
Externa ao Programa - 1554093 - MARIE TOGASHI - nullExterna à Instituição - Thaís Bergmann de Castro - UnB