Disparities in Access to Cystic Fibrosis Therapy Across Countries

ABSTRACT

Cystic fibrosis has been transformed by the development of CFTR modulator therapies, with substantial improvements in survival and quality of life. However, access to these therapies remains profoundly unequal worldwide. The greatest benefits have been realized in high‐income countries, while people with cystic fibrosis in low‐ and middle‐income countries and underserved populations within high‐income settings continue to face limited access and poorer outcomes. Underdiagnosis is a major contributor to these disparities, as limited newborn screening, restricted access to sweat testing, and incomplete genetic characterization directly limit treatment eligibility and registry inclusion. Beyond diagnosis, disparities are driven by differences in genetic variant distribution, pricing and reimbursement policies, regulatory processes, and health system capacity. This review examines how these interrelated factors shape global access to therapies, with particular emphasis on CFTR modulators. Emerging strategies—including differential pricing, licensing mechanisms, regulatory adaptation, international collaboration, and health system strengthening—are discussed. Achieving equitable access will require coordinated action across diagnostic, economic, and policy domains to ensure that advances in cystic fibrosis care benefit patients regardless of geographic or socioeconomic context.

1. Introduction

Cystic fibrosis (CF) is a multisystem autosomal recessive disease [1]. Over the past decade, its treatment and prognosis have changed dramatically with the development of CFTR modulator therapies [1, 2]. These treatments have led to earlier intervention, improved survival, and more standardized multidisciplinary care models [1, 3, 4]. However, these advances have been realized mainly in high‐income countries [5, 6]. People with CF (pwCF) in low‐ and middle‐income countries (LMICs) remain largely excluded from these therapeutic gains [5, 6]. This disparity reflects differences in healthcare infrastructure, economic resources, pharmaceutical access, and national prioritization of rare diseases [6, 7, 8]. Recent estimates suggest that approximately 200,000 pwCF live across 96 countries [5]. Of these, only 59% have been diagnosed, and 27% have access to elexacaftor/tezacaftor/ivacaftor (ETI). Most undiagnosed individuals are believed to live in LMICs [5]. These data show that the global transformation of CF care has been uneven.

Before the modulator era, CF management focused on symptom control, including treatment of particularly pulmonary infections and pancreatic insufficiency. The development of CF care centers and national registries improved outcomes, particularly in North America and Western Europe. Nonetheless, disparities persisted even within high‐income countries, often linked to socioeconomic status, race, insurance coverage, and geographic location [2, 6, 9]. The discovery of the CFTR gene in 1989 enabled targeted therapy development. Ivacaftor was approved in 2012 as the first CFTR modulator. Early modulator therapies primarily targeted individuals with at least one F508del mutation, the most common variant in Europe and North America [3, 4]. Regulatory label expansions in the United States and Europe have since broadened eligibility to include additional responsive variants. Despite these developments, global access remains unequal, and many variants more common in LMIC populations are not included in approved indications.

This review examines global disparities in access to CF therapies, with particular emphasis on CFTR modulators. Clinicians, researchers, and policy stakeholders are intended to be involved in CF care across diverse health systems. It focuses on four interconnected domains that shape therapeutic access: diagnostic capacity and genetic characterization, genetic diversity and its impact on eligibility, economic and regulatory barriers, and health system infrastructure, registries, and policy frameworks.

By integrating clinical, economic, and policy perspectives, this review provides a structured framework for understanding inequities in CF care. Diagnostic capacity is addressed throughout as a prerequisite for treatment eligibility, registry inclusion, and equitable therapy delivery.

2. Mapping the Disparities

Global access to CFTR modulators remains highly unequal [1, 5, 6]. In North America and Western Europe, most patients benefit from early diagnosis, national registry inclusion, multidisciplinary care, and reimbursement systems that support access to high‐cost therapies. In contrast, many pwCF in LMICs face delayed diagnosis, limited diagnostic capacity, fragmented care models, and restricted access to advanced treatment [1, 10, 11].

Equitable access to these therapies remains a global health priority, and no one should be left behind [5]. These disparities are not limited to differences between countries [1, 10]. Within middle‐income settings such as Turkey, Brazil, and South Africa, marked variation exists in drug availability, insurance coverage, and institutional capacity. In South Africa, access differs substantially between the private and public sectors. In Brazil and Turkey, advocacy efforts and legal decisions have enabled partial or recent reimbursement of triple‐combination therapy [12, 13, 14]. Similar diagnostic and reimbursement challenges have been described in parts of South Asia, where CF remains under‐recognized [10].

Inequities also persist within high‐income countries. In the United States, Canada, and parts of Europe, socially disadvantaged populations experience delayed diagnosis, lower lung function, poorer nutritional status, and reduced access to therapies compared with more affluent groups [2, 6]. Across Europe, countries with lower income levels have significantly higher CF‐related mortality than wealthier nations [9].

Differences in regional CFTR mutation distribution further contribute to unequal eligibility for modulator therapy [6]. Together, these findings show that disparities arise from interacting factors: diagnostic capacity, genetic eligibility, health system structure, and financing mechanisms. Addressing these gaps requires coordinated system‐level investment rather than isolated interventions.

3. Diagnostic Capacity and Genetic Diversity in LMICs

Delayed or missed diagnosis remains a major barrier to appropriate CF management in many LMICs [1, 10]. Diagnosis depends on three main components: newborn screening (NBS), access to sweat chloride testing, and comprehensive genetic analysis. Limitations in any of these areas delay diagnosis and restrict treatment eligibility.

Diagnostic inequities are not limited to LMICs. Ethnic minority populations in high‐income countries may also be underdiagnosed. A comparison of UK and Canadian registries showed large differences in reported CF prevalence among individuals of South Asian origin. The prevalence was 1 in 74,892 in Canada compared with 1 in 13,340 in the UK [15]. The authors suggested that current NBS panels failed to detect 40%–47% of CFTR variants in this population [15]. Model‐based estimates indicate that 26,000–146,000 pwCF may live in South Asia, although these figures are not based on direct population data [15]. In China, fewer than 200 pwCF have been reported, and recent studies identified only 67 individuals with novel variants [16]. These findings suggest substantial under‐recognition.

In many LMICs, NBS is not widely implemented [1, 10, 17]. Diagnosis often relies on clinical presentation. Studies from South Asia report median ages at diagnosis between 1.5 and 2.8 years [10, 18, 19]. Many children are diagnosed after a chronic infection has developed. Earlier acquisition of Pseudomonas aeruginosa has been reported compared with North American registry populations [3, 18]. In contrast, NBS allows diagnosis within the first 35–45 days of life and is associated with improved outcomes [4, 10]. Delayed diagnosis may also lead to missed opportunities for early initiation of disease‐modifying therapy [1, 7].

Sweat chloride testing remains the diagnostic gold standard [1]. Access is limited in many LMICs. Barriers include high equipment costs, lack of trained personnel, absence of quality control systems, unstable electricity supply, and shortage of consumables [10, 17]. Even when devices are available, maintenance challenges reduce reliability. As a result, CF is often misdiagnosed as recurrent pneumonia, malnutrition, or tuberculosis. This delays referral and appropriate care.

Genetic heterogeneity adds further complexity [11, 15, 20]. Commercial mutation panels and NBS algorithms are largely based on variants common in Europe and North America [1, 10, 11, 20]. In regions with more diverse mutation spectra, these panels may miss disease‐causing variants [11, 15, 20]. Next‐generation sequencing provides more complete analysis, but its cost and infrastructure requirements limit availability in many LMICs [7, 10]. Recent CF Foundation (CFF) guidelines recommend including all known disease‐causing CFTR variants in NBS panels to improve sensitivity [21]. Although this guidance is US‐based, it highlights the need to adapt screening strategies to regional genetic patterns.

These diagnostic limitations restrict registry enrollment and clinical trial participation. They also limit eligibility for CFTR modulators. Diagnostic inequity therefore, functions as a gatekeeper to subsequent advances in CF care.

4. Barriers to Access Basic CF Treatment

Access to standardized basic CF care remains inconsistent in many LMICs. Core components of CF management include nutritional support, pancreatic enzyme and vitamin supplementation, mucolytics, inhaled antibiotics, and airway clearance [1, 10]. These treatments are routine in high‐income countries but are not universally available or affordable in lower‐resource settings.

Cost is a major barrier. In high‐income countries, annual estimates for standard CF care may exceed $10,000 per patient [11, 22]. The burden may be greater may greater in the LMICs because of lower household income, limited insurance coverage, and reliance on out‐of‐pocket payments. In some settings, the cumulative cost of basic CF care may equal 3 to 10 times the average monthly income [10, 11]. As a result, families may ration medications, interrupt therapy, or stop treatment altogether.

Regulatory and supply factors further limit access. Pharmaceutical companies may be reluctant to register CF medications in LMICs because of small patient populations and administrative costs [1]. Drugs are often supplied through local distributors, which can increase prices. Supply chain instability and inconsistent availability disrupt continuity of care.

In response to limited access, alternative treatment approaches have been used. For example, off‐label nebulized intravenous gentamicin has been employed to treat early P. aeruginosa infection in resource‐limited settings [23]. Adherence to physiotherapy is another challenge [10]. Even it is free, airway clearance techniques may be performed inconsistently. Limited availability of trained physiotherapists often restricts patients to basic methods such as postural drainage. Structured exercise programs may help improve adherence and respiratory health [10].

These barriers show that disparities in CF outcomes are not driven only by access to advanced therapies. In many LMICs, limited access to basic, evidence‐based treatment remains a key determinant of morbidity and survival. Sustainable improvement requires reliable financing, stable supply chains, trained multidisciplinary teams, and long‐term health system investment.

5. Lack of CF Care Centers

Access to specialized CF care centers remains limited in many LMICs. Survival and long‐term outcomes improve when care is delivered by multidisciplinary teams [24]. Patients followed in dedicated CF centers have better clinical outcomes [24]. Effective CF management requires coordinated input from pulmonologists, nurses, dietitians, physiotherapists, psychologists, and trained support staff. In many LMICs, such teams are not available. Health systems often lack the infrastructure needed to establish centers of excellence. As a result, care may be fragmented and inconsistent.

Limited professional awareness further contributes to delayed diagnosis and inadequate referral [1, 10, 13]. Many healthcare providers have limited clinical experience with CF. Capacity‐building programs can help address this gap. Distance‐learning platforms, tele‐mentoring programs, short‐term observerships, and structured fellowship exchanges have improved knowledge and standardized care practices in several settings [1, 10, 13]. Twinning programs between higher‐ and lower‐resource centers have also strengthened local expertise. Telemedicine may provide additional support for patients in remote areas. It allows access to specialist advice without requiring travel and may be cost‐effective in resource‐limited settings [1].

Transition from pediatric to adult care is another challenge [1]. As survival improves, the number of adults living with CF is increasing in LMICs. Structured transition programs are therefore essential [25, 26]. A center in Turkey adapted the US CF Foundation CFF CFR.I.S.E. model to establish a multidisciplinary transition pathway [25, 26, 27]. This program improved patient knowledge and self‐confidence [25]. Without a structured transition, clinical deterioration may occur during early adulthood. Strengthening CF care centers requires sustained investment in training, infrastructure, and national health policy support. Long‐term improvement in outcomes depends not only on medication access but also on coordinated, lifelong multidisciplinary care.

6. Real‐World Consequences

Disparities in CF care lead to measurable differences in outcomes. These include delayed diagnosis, faster disease progression, and reduced survival in many LMIC settings. High treatment costs remain a central barrier. The annual cost of CFTR modulators often exceeds $250,000 per patient [7]. In many LMICs, therapies are unavailable not because of slow regulatory review, but because marketing applications are not submitted or pricing negotiations fail. In smaller markets, limited commercial incentives may delay entry. Access is therefore shaped by pricing strategies, market size, reimbursement capacity, and political prioritization.

These structural factors contribute to large survival gaps. In high‐income countries such as the United States, projected survival now extends into the sixth decade of life [3]. In contrast, studies from parts of Central America, North Africa, and South Asia report median survival below 25 years [18, 28, 29]. Across LMICs, overall survival remains at least 10–15 years lower than in wealthier countries [9, 29].

Clinical indicators also differ. In a multicenter study from India, the median age at diagnosis was 2.8 years [19]. Lung function monitoring was limited, and early acquisition of P. aeruginosa was common. The prevalence of chronic Pseudomonas infection was substantially higher than reported in the CFF Registry [3, 18, 19]. These findings reflect delayed diagnosis and limited access to standard care.

Inequities also exist within countries. In Mexico and the United Kingdom, lower socioeconomic status is associated with higher mortality [29, 30]. In middle‐income settings, household poverty and food insecurity reduce adherence and compromise nutritional status [31].

The impact extends beyond clinical measures. Caregivers often face financial strain and psychological stress [32]. Families report frustration with delayed diagnosis and exclusion from therapies available elsewhere. In a Turkish cohort, patients eligible for modulators but unable to access treatment had higher rates of pulmonary exacerbations and hospital admissions [32]. Individuals without access to disease‐modifying therapy also reported higher rates of depression compared with treated patients [32].

These findings show that unequal access to diagnosis and treatment leads to earlier disease progression, reduced survival, and increased psychosocial burden. These outcomes are not inevitable. They reflect structural differences in health systems, financing, and therapeutic access. Understanding how these structural factors shape the eligibility and availability of CFTR modulators is therefore essential.

7. Global Landscape of CFTR Modulator Access

The survival gaps described above reflect structural differences in access to CFTR modulators. Access depends on three main factors: genetic eligibility, economic affordability, and regulatory and policy frameworks. These factors interact and create major disparities between high‐income countries and LMICs.

Eligibility for modulator therapy depends on CFTR variant distribution. Mutation patterns vary across regions and ethnic groups [15, 20, 33, 34]. In Western Europe and North America, the F508del mutation is highly prevalent. Most patients carry at least one responsive variant [3, 4]. In contrast, populations in Turkey, India, Southeast Asia, and parts of Latin America show greater genetic diversity [18, 33, 35, 36, 37]. In Turkey, national data indicate that only about half of patients are eligible for the currently approved modulators [33]. In India and Southeast Asia, F508del frequency is reported at 25%–30%, resulting in lower predicted eligibility [18, 37].

A large international study by Burgel et al. analyzed 95,838 individuals across 69 countries [34]. They reported that 82% carried at least one F508del variant and 9.5% carried rare variants responsive to ETI. Overall, 91.5% were theoretically eligible when expanded labels were considered. However, F508del prevalence ranged from 7% to 98% across countries [34]. In countries with low F508del prevalence, label expansion significantly increased theoretical eligibility. These findings show that genetic diversity directly influences potential access.

Incomplete genotyping further limits eligibility assessment [20]. In Turkey, both CFTR alleles were identified in only 79% of patients [33]. Commercial panels are designed around variants common in Europe and North America. As a result, CF may be under‐detected in regions with diverse mutation spectra. Genetic heterogeneity, therefore, acts as both a biological and structural barrier.

Even when patients are eligible, therapy may not be available [4]. Access to ETI is reported in approximately 60 countries, but access varies in nature [5, 7]. In most high‐income countries, it is available through national reimbursement systems. In a small number of LMICs, access is limited to private insurance, individual litigation, compassionate use, or donation programs.

The annual list price of ETI in the United States is approximately $322,000 [5]. This exceeds average annual income in most LMICs many times over. Conventional cost‐effectiveness models in high‐income countries often conclude that modulators exceed standard thresholds. These models are even less transferable to LMIC settings with lower health budgets. Several policy mechanisms have been discussed to improve affordability. Economic modeling suggests that ETI could be manufactured for approximately $5000 per patient per year through voluntary licensing, which may allow controlled generic production [5]. In Argentina, generic production reduced prices by approximately 85%. Compulsory licensing is legally permitted under TRIPS agreements but remains politically complex [37]. In South Africa, limited private‐sector coverage has created a marked public‐private divide, and some patients have adopted cost‐saving “modulator sparing” strategies to reduce costs as using a twice‐weekly regimen combined with clarithromycin [13, 38]. However, current evidence is limited and insufficient to establish equivalence to standard dosing in terms of long‐term efficacy, safety, or durability of response [13, 38]. Conventional health technology assessment frameworks often conclude that modulators exceed standard cost‐effectiveness thresholds, even in high‐income countries, highlighting the limitations of applying traditional economic models in lower‐resource settings. Pooled procurement and regional negotiation mechanisms may increase bargaining power for smaller markets. However, these approaches require coordination and political commitment.

Legal action has provided access in some middle‐income countries [13]. Courts have mandated treatment in Turkey, Chile, Colombia, and Uruguay. These decisions may help individual patients, but do not represent sustainable system‐level solutions. They may also shift priority‐setting from health authorities to judicial systems.

Improved genetic testing may increase accurate eligibility assessment. Next‐generation sequencing can detect rare variants but is not widely available in many LMICs. Functional testing using organoid models has demonstrated responsiveness in some rare variants, such as E92K [1, 37, 39]. However, these approaches remain largely confined to research settings and are not yet widely available in most LMICs.

Barriers to modulator access often form a cycle. Limited diagnosis reduces registry data. Weak registry data reduces evidence for reimbursement. Lack of reimbursement discourages investment in diagnostic infrastructure. This cycle disproportionately affects LMICs. Breaking this cycle requires coordinated reforms rather than isolated interventions.

8. The Role of Research and Collaborative Registries

The structural barriers described in the previous section are reinforced by gaps in research and data systems. Clinical trials remain the main pathway for developing new CF therapies. However, most trials are conducted in high‐income countries [40]. Even within these settings, ethnic minorities and socially disadvantaged populations are underrepresented [40]. This imbalance limits evidence for rare variants and non‐classical disease presentations. Regulatory approvals and label expansions are based largely on these trial populations. As a result, variants more common in non‐European populations may be slower to gain recognition as responsive to therapy. In a survey of CF research coordinators, 64% reported little or no experience enrolling patients from minority groups [40]. Barriers related to language, socioeconomic status, and trust in research remain insufficiently addressed.

Expanding clinical trial participation to LMICs is therefore important. Broader inclusion would improve understanding of genetic diversity and ensure that future therapies are evaluated across different populations [41]. Without this expansion, therapeutic innovation risks reinforcing existing inequities.

High‐quality registry data are equally important. Registries document outcomes, monitor quality of care, and support policy decisions. However, substantial variation exists between national registries in genotype reporting, outcome definitions, and data completeness [3, 4, 42, 43]. Differences in diagnostic criteria and follow‐up practices limit cross‐country comparisons. Strengthening registries in LMICs requires technical and structural support. Electronic data systems, trained personnel, external audits, and integration with NBS and genetic laboratories can improve data quality. Transparent reporting of coverage rates and standardized definitions would increase comparability.

Registry data have already demonstrated their values. In Brazil, the proportion of adults with CF increased from 20% in 2009 to 30% in 2017, reflecting improvements in survival [10]. In Turkey, early registry data showed improvements in body mass index and height z‐scores between 2017 and 2019 [42]. A comparison between South Africa and Canada revealed significant differences in median age, lung function, and nutritional status, even before widespread modulator use [43]. Such findings illustrate how registries make disparities visible.

Established registries such as the CFF Patient Registry and the ECFS Patient Registry provide models for data‐driven quality improvement [3, 4]. Harmonization initiatives, including efforts to expand the ECFSPR to LMICs, represent important steps toward global standardization [37]. Adoption of uniform definitions for lung function, exacerbations, nutrition, and genotype classification would further improve comparability. Standardized annotation of CFTR variants is particularly important in the modulator era.

Ultimately, inclusive research and harmonized registries strengthen the evidence base needed for equitable regulatory approval, pricing negotiations, and policy decisions. Without representative data, disparities in access are likely to persist or widen.

9. The Role of International Organizations

The need for harmonized research and stronger registries naturally raises the question of global coordination. International organizations play an important role in shaping policy, standards, and capacity building. Historically, CF has received limited attention at the global level. The WHO has addressed NBS, essential diagnostics, rare diseases, and access to high‐cost medicines within broader non‐communicable disease frameworks [8]. However, CF has not been designated as a global priority. WHO reports emphasize that social determinants of health strongly influence inequities in outcomes [8]. Recently, the inclusion of CFTR modulators in WHO essential medicines discussions signaled growing recognition, although implementation remains dependent on national decisions [37].

Regional professional organizations have had a more direct impact on CF care. The ECFS and the CFF have led advances in registries, clinical guidelines, and research networks. However, most of these activities have been concentrated in Europe and North America. Broader international engagement is needed to reduce disparities in LMICs [7, 8]. Capacity‐building initiatives represent one practical strategy. International training programs, such as the International CF Trainers Network, provide mentorship and education for healthcare professionals in resource‐limited settings. Twinning models typically pair an established CF center in a high‐income country with a developing center in a lower‐resource setting. These partnerships focus on multidisciplinary training, implementation of standardized care protocols, quality improvement strategies, and registry development. Twinning initiatives should have measurable benefits, such as nutritional status, infection control policies, and improved CF care [5, 44].

Evidence from Eastern Europe, the Middle East, and Latin America suggests that sustained mentoring improves nutritional monitoring, infection control practices, and diagnostic capacity [44]. Importantly, twinning initiatives prioritize systems strengthening. They support the establishment of sweat testing services, genetic diagnostics, and local data registries. While these partnerships cannot replace national policy reform or long‐term financing, they provide a structured framework for building local expertise.

Humanitarian donation programs also contribute in selected settings. However, medication supply alone is insufficient. Sustainable improvement requires parallel investment in infrastructure and workforce development. For example, MECFA has supported sweat chloride testing equipment and training in several countries, resulting in increased confirmed diagnoses [45]. Such initiatives demonstrate that regional collaboration and technical support can strengthen foundational capacity. Without such foundational investments, isolated drug access is unlikely to result in sustainable improvements in CF outcomes.

10. Patient Advocacy and the Role of Civil Society

In addition to international organizations, civil society has influenced access to CF care in several countries. Patient advocacy groups have played a visible role in shaping national policy and reimbursement decisions. In Brazil, organized advocacy contributed to court‐mandated provision of CF medications [14]. In Turkey, collaboration between patient organizations, clinicians, and policymakers supported reimbursement of ETI [37]. These examples show that coordinated national advocacy can affect policy outcomes.

Media attention has also shaped public debate. In 2023, The New York Times reported on families in India and Turkey who were unable to access ETI, drawing international attention to disparities in treatment access [46]. In South Africa, India, and parts of Latin America, advocacy campaigns increased visibility around modulator pricing and prompted parliamentary discussions on rare disease access. Global advocacy networks have further amplified these concerns. Organizations such as CF Europe, Rare Diseases International, and “Vertex Save Us” initiative have raised CF access issues within broader equity discussions [7, 13, 37, 46, 47]. In August 2023, more than 100 CF clinicians signed a joint statement urging adoption of tiered pricing strategies for LMICs [37]. Although such actions do not directly determine pricing decisions, they can accelerate negotiations and increase transparency. Sustained engagement with governments remains essential. Advocacy efforts are most effective when aligned with structured policy dialogue, health technology assessment processes, and long‐term reimbursement planning. Civil society can therefore act as a catalyst, but durable access requires institutional commitment.

11. Ethical Considerations

The disparities described in the previous sections raise ethical questions about fairness and priority setting. Differences in life expectancy and access to treatment are not explained by biology alone. They are also shaped by health system capacity, regulatory choices, and economic resources. CF shows how medical innovation can widen inequalities when access is uneven. From a policy perspective, there is tension between three goals: supporting innovation, managing limited health budgets, and ensuring fair access to care. High‐cost orphan drugs challenge traditional priority‐setting frameworks, especially in countries with constrained resources.

Ethical decision‐making requires balance. Health systems must encourage pharmaceutical development while also protecting vulnerable patients and promoting fairness. Access cannot be treated as an unlimited entitlement. Instead, pricing, licensing, and reimbursement policies should be evaluated through principles of proportional burden‐sharing and global equity. Integrating CF into broader rare disease strategies and universal health coverage frameworks may support more consistent and ethically grounded policy decisions [47]. These considerations provide a foundation for the practical recommendations outlined in the next section.

12. Recommendations for Equitable Access

The disparities described in this review result from linked failures in diagnosis, health system capacity, pricing, and policy. As these problems are connected, solutions must also be coordinated. No single intervention can resolve global inequities in CF care. Progress depends on combining policies in a structured and realistic way [7, 8, 37].

First, diagnostic capacity must be strengthened. Expanded NBS and improved access to genetic testing are essential. Without an accurate diagnosis, patients cannot access appropriate treatment. Second, investment in CF care centers and workforce training is necessary. Multidisciplinary care models, infection control practices, and functional registries improve outcomes and support safe treatment delivery. Third, reimbursement strategies must reflect economic realities. Differential pricing, voluntary licensing, and coordinated regional negotiations may offer more sustainable pathways than litigation or temporary donation programs. Finally, CF should be integrated into national rare disease strategies and universal health coverage plans. This approach supports long‐term financing, regulatory alignment, and equitable implementation.

These recommendations are interdependent. They should be implemented together rather than in isolation. This framework offers a structured approach for clinicians, policymakers, and stakeholders seeking to reduce global disparities in CF care.

13. Conclusion

CF is now a treatable disease in many parts of the world. However, access to effective care remains uneven. Differences in outcomes between countries are not inevitable. They reflect policy decisions, economic structures, and variations in health system capacity.

Improving equity in CF care requires coordinated action across diagnosis, infrastructure, pricing, and policy. Clinicians have a role not only in providing care but also in supporting research, data collection, and informed advocacy. Sustained international collaboration and realistic policy solutions are necessary to reduce disparities. Advances in CF treatment should benefit patients regardless of where they are born or the resources of their health system.

Author Contributions

Bulent Karadag: conceptualization, investigation, writing – original draft, methodology, writing – review and editing, visualization, validation, software, project administration, resources, data curation, formal analysis.

Funding

The author received no specific funding for this work.

Conflicts of Interest

The author declares no conflicts of interest.

Data Availability Statement

Data sharing are not applicable to this article as no datasets were generated or analyzed during the current study.