Strategies used to access CFTR modulators in countries without reimbursement agreements

CFTR modulators represent the international standard of care for the treatment of cystic fibrosis (CF). Yet due to prices of over $250,000 per year they are functionally inaccessible for people with CF (pwCF) unless reimbursed by healthcare systems. Current prices are unaffordable for payors in almost all low- and middle-income countries (LMICs) worldwide, and resulting disparities in access are widening existing global health inequities. In comparable situations in other therapeutic areas, patients have successfully developed strategies to bypass national reimbursement systems and gain access to treatment. We therefore undertook an international survey of CF clinicians in 15 countries where CFTR modulators are not reimbursed, to characterise alternative means of accessing modulator therapy. Successful methods were identified in 11 countries, and could broadly be categorised into legal challenges to access originator modulators, use of generic formulations, and access via donations. Aside from domestically produced generics used in Argentina and an originator-led donation program in Ukraine, these methods were only able to provide treatment to limited proportions of the local CF population due to significant associated financial costs. Accordingly, they are generally not sustainable or widely applicable, and fail to address the underlying structural issues driving international disparities in access. Twelve years after the initial marketing of CFTR modulators, pwCF in LMICs are being forced to take extraordinary measures to access disease-modifying treatment. Corrective measures are urgently required to overcome barriers posed by restrictive patents and prohibitively high prices, and to promote global health equity for pwCF.