Year: 2023
Type: article
Abstract: CFTR modulators have drastically improved outcomes for many people with cystic fibrosis (CF), including improved lung function and increased life expectancy by targeting the defective CFTR protein 1,2 .As a result, many more people with CF are now fulfilling key life milestones, including pursuing education, careers, and parenthood 3 .We applaud the research and development efforts of Vertex Pharmaceuticals, the sole manufacturer of CFTR modulators.Vertex has been heralded as a shining star of pharmaceutical companies for personalized medicine and rare disease and has a stated commitment to the CF community.However, we are appalled at how Vertex is achieving financial success on the backs of vulnerable populations.Only a small percentage of people with CF worldwide have access to these disease-altering CFTR modulators due to Vertex not allowing the development of generic forms or lowering pricing for lower-income or middle-income countries (LMICs), as done by other pharmaceutical companies for HIV, Hepatitis C, and Covid-19 medications 4 .Due to Vertex's refusal to assist LMICs and Vertex holding the patent for elexacaftor/tezacaftor/ivacaftor until 2037, LMICs will not have generic ETI available for 14 more years.An entire generation of people with CF in LMICs may therefore miss out on the profound benefit Conflict of Interest: Dr. Sawicki
Source: Pediatric Pulmonology
DOI: 10.1002/ppul.26335
Authors: Meghan E. McGarry, Elizabeth R. Gibb, Theresa A. Laguna, Brian P. O'Sullivan, Gregory S. Sawicki, Jeffery T. Zobell
Language: English
Subfield: Economics and Econometrics
Domain: Social Sciences
Open Access status: hybrid
Estimated APC paid: $4,740
Awards: G2322230794, G4820951400
OpenAlex ID: W4318753584
