Marco Zampoli, Cathy Baird, Brenda Morrow, Janine Verstraete, Greg Calligaro, Mark Seale
{"title":"Cystic Fibrosis Care in South Africa: Facing the Challenge of Diversity and Inequality.","authors":"Marco Zampoli, Cathy Baird, Brenda Morrow, Janine Verstraete, Greg Calligaro, Mark Seale","doi":"10.1002/ppul.71338","DOIUrl":null,"url":null,"abstract":"<p><p>Low-and-middle-income countries (LMIC) like South Africa (SA) were left behind with advancements in cystic fibrosis (CF) care that followed re-imbursement agreements in high-income countries of CF transmembrane regulator protein modulators (CFTRm) for the treatment of CF. A combination of global monopoly, patent protection by international trade agreements and delays in regulatory approvals are factors keeping these transformative drugs out of reach for many LMIC due to their high cost. Unequal and delayed access to CFTRm is amplifying existing disparities in CF care and outcomes worldwide, including SA where currently only 200 (50%) of the 400 eligible people have access to CFTRm. However, the SA experience demonstrated how health professionals, legal experts and global CF community activists can collaborate to mobilize local and international resources to advocate for affordable and equal access to CFTRm In this paper we describe the journey followed in SA to overcome some of these inequalities and challenges and highlight the current and future impact on CF care in SA that may have much in common with other LMIC.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 10","pages":"e71338"},"PeriodicalIF":2.3000,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12522015/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Pediatric Pulmonology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1002/ppul.71338","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"PEDIATRICS","Score":null,"Total":0}
引用次数: 0
Abstract
Low-and-middle-income countries (LMIC) like South Africa (SA) were left behind with advancements in cystic fibrosis (CF) care that followed re-imbursement agreements in high-income countries of CF transmembrane regulator protein modulators (CFTRm) for the treatment of CF. A combination of global monopoly, patent protection by international trade agreements and delays in regulatory approvals are factors keeping these transformative drugs out of reach for many LMIC due to their high cost. Unequal and delayed access to CFTRm is amplifying existing disparities in CF care and outcomes worldwide, including SA where currently only 200 (50%) of the 400 eligible people have access to CFTRm. However, the SA experience demonstrated how health professionals, legal experts and global CF community activists can collaborate to mobilize local and international resources to advocate for affordable and equal access to CFTRm In this paper we describe the journey followed in SA to overcome some of these inequalities and challenges and highlight the current and future impact on CF care in SA that may have much in common with other LMIC.
期刊介绍:
Pediatric Pulmonology (PPUL) is the foremost global journal studying the respiratory system in disease and in health as it develops from intrauterine life though adolescence to adulthood. Combining explicit and informative analysis of clinical as well as basic scientific research, PPUL provides a look at the many facets of respiratory system disorders in infants and children, ranging from pathological anatomy, developmental issues, and pathophysiology to infectious disease, asthma, cystic fibrosis, and airborne toxins. Focused attention is given to the reporting of diagnostic and therapeutic methods for neonates, preschool children, and adolescents, the enduring effects of childhood respiratory diseases, and newly described infectious diseases.
PPUL concentrates on subject matters of crucial interest to specialists preparing for the Pediatric Subspecialty Examinations in the United States and other countries. With its attentive coverage and extensive clinical data, this journal is a principle source for pediatricians in practice and in training and a must have for all pediatric pulmonologists.