Advancing Equitable Access to Cystic Fibrosis Treatment in Brazil: Strategies for Implementing National Clinical Guidelines
Author(s)
Lindemberg Assunção-Costa, MSc, PharmD, PhD1, Juliana Fernandes Ferreira Machado, Pharmacist2, Cleidenete G. Valli, Pharmacist2, Sofia Fernandes Vilas Boas Santos, Pharmacy Student3, Vitória Batista Calmon de Passos, Pharmacy Student3, Almirane Lima de Oliveria, Pharmacist2;
1Federal University of Bahia, Professor, Salvador, Brazil, 2National Institute of Pharmaceutical Assistance and Pharmacoeconomics, Salvador, Brazil, 3Federal University of Bahia, Medicine Department, Salvador, Brazil
1Federal University of Bahia, Professor, Salvador, Brazil, 2National Institute of Pharmaceutical Assistance and Pharmacoeconomics, Salvador, Brazil, 3Federal University of Bahia, Medicine Department, Salvador, Brazil
OBJECTIVES: Cystic Fibrosis (CF) is a rare autosomal recessive genetic disorder that affects exocrine glands. This study aims to describe initiatives by the National Institute of Pharmaceutical Assistance and Pharmacoeconomics (INAFF) to enhance access and promote the implementation of the CF PCDT nationally.
METHODS: A comprehensive qualification program was developed to support the implementation of the Cystic Fibrosis (CF) Clinical Protocol and Therapeutic Guidelines (PCDT) across 14 Brazilian states. The program aimed to standardize the understanding of the PCDT and enhance the capacity of state-level health departments in facilitating equitable access to CF treatments.The training program consisted of expert-led presentations and workshops focused on the pathophysiology of CF, the specific criteria for treatment access as outlined in the PCDT, and the clinical and administrative processes involved in implementing the protocol. In addition, the sessions emphasized the practical aspects of the PCDT, including the documentation and procedural requirements for prescribing and accessing CFTR protein modulator drugs.
RESULTS: The incorporation of CFTR protein modulator drugs into the PCDT has transformed CF treatment. A total of 191 healthcare professionals participated in the training, including 154 pharmacists, 33 physicians, and other professionals. Significant variations were observed between states regarding their understanding of criteria for providing treatments under the PCDT, potentially limiting access. Additionally, a need for clearer guidance for prescribers on properly completing documentation was identified to prevent delays in the approval process.
CONCLUSIONS: Equity in healthcare is a fundamental principle of Brazil’s Unified Health System (SUS), making equitable access to CF treatments a critical goal. INAFF’s initiatives aimed to standardize understanding of the CF PCDT across states, enhancing access to transformative CF treatments. By addressing disparities in knowledge and procedural challenges, these actions fostered more uniform implementation of the PCDT, helping to ensure that all patients, regardless of geographic location, benefit from timely and effective treatment.
METHODS: A comprehensive qualification program was developed to support the implementation of the Cystic Fibrosis (CF) Clinical Protocol and Therapeutic Guidelines (PCDT) across 14 Brazilian states. The program aimed to standardize the understanding of the PCDT and enhance the capacity of state-level health departments in facilitating equitable access to CF treatments.The training program consisted of expert-led presentations and workshops focused on the pathophysiology of CF, the specific criteria for treatment access as outlined in the PCDT, and the clinical and administrative processes involved in implementing the protocol. In addition, the sessions emphasized the practical aspects of the PCDT, including the documentation and procedural requirements for prescribing and accessing CFTR protein modulator drugs.
RESULTS: The incorporation of CFTR protein modulator drugs into the PCDT has transformed CF treatment. A total of 191 healthcare professionals participated in the training, including 154 pharmacists, 33 physicians, and other professionals. Significant variations were observed between states regarding their understanding of criteria for providing treatments under the PCDT, potentially limiting access. Additionally, a need for clearer guidance for prescribers on properly completing documentation was identified to prevent delays in the approval process.
CONCLUSIONS: Equity in healthcare is a fundamental principle of Brazil’s Unified Health System (SUS), making equitable access to CF treatments a critical goal. INAFF’s initiatives aimed to standardize understanding of the CF PCDT across states, enhancing access to transformative CF treatments. By addressing disparities in knowledge and procedural challenges, these actions fostered more uniform implementation of the PCDT, helping to ensure that all patients, regardless of geographic location, benefit from timely and effective treatment.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HTA91
Topic
Health Technology Assessment
Topic Subcategory
Systems & Structure
Disease
SDC: Rare & Orphan Diseases, SDC: Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory), STA: Personalized & Precision Medicine