Strategies used to access CFTR modulators in countries without reimbursement agreements

Area of Science:

• Medical research
• Health equity
• Pharmaceutical access

Background:

• CFTR modulators are the standard CF treatment but cost over $250,000 annually.
• Prohibitive costs make these life-saving drugs inaccessible in low- and middle-income countries (LMICs).
• This widens existing global health inequities for people with CF (pwCF).

Purpose of the Study:

• To survey CF clinicians in 15 non-reimbursing countries.
• To characterize alternative access strategies for CFTR modulators.
• To assess the sustainability and applicability of these methods.

Main Methods:

• International survey of CF clinicians.
• Data collection on reimbursement status and access methods.
• Categorization of successful access strategies.

Main Results:

• Alternative access methods identified in 11 countries.
• Strategies include legal challenges, generic formulations, and donations.
• These methods provide limited access, are costly, and not widely applicable.

Conclusions:

• Current access methods for CFTR modulators in LMICs are unsustainable.
• Restrictive patents and high prices create significant global health disparities.
• Urgent corrective measures are needed to ensure global health equity for pwCF.