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International disparities in diagnosis and treatment access for cystic fibrosis

The elastacaftor/tezacaftor/ivacaftor (ETI) triple therapy has completely altered the course of treatment for cystic fibrosis (CF). Nonetheless, prior studies have shown that there are significant differences in the availability of diagnosis and therapy among countries. Existing health inequities could be exacerbated if these are left unchecked. Hence, the objective of this study was to compare high-income (HIC) nations with low- and middle-income (LMIC) nations in order to reevaluate deficiencies and assess advancements in equity of diagnosis and treatment.


Methods: Methods such as patient registries, thorough literature searches, and a worldwide poll of 14 CF specialists were used to estimate the global CF population in 158 countries. Epidemiological studies found through literature searches and data from CF registries were used to estimate the worldwide burden of undiagnosed CF. To determine the percentage of individuals receiving ETI, we surveyed 23 national drug pricing databases and used publicly accessible revenue data.


The estimated number of people with cystic fibrosis in 96 countries is 188,336 (163,421-209,204). Among them, 51,322 (or 27%) received ETI and 112,955 (or 60%) were diagnosed. Roughly 82% of the estimated 75,381 undiagnosed patients live in low- and middle-income countries. Thirty-five HICs cover ETI, although just one LMIC does. Even though ETI has been available for four years, 14,911 individuals with CF still reside in countries where it is not available. When the projected undiagnosed population is included, the number rises to 76,199.


The worldwide CF community must prioritize equitable access to CFTR modulators, according to the conclusions. While ETI can cost up to $322,000 annually, it is possible to make it for $5,000 and offer access under a voluntary license. Other methods of enhancing access that do not involve the manufacturer should also be investigated in light of the severity of the discrepancies.


Full article below:


Pediatric Pulmonology - 2024 - Guo - International disparities in diagnosis and treatment
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