COST-EFFECTIVENESS MODELS

The recent advent of CFTR modulator therapies represents a significant breakthrough in the management of Cystic Fibrosis (CF), transforming the approach from symptomatic treatment to one that targets the genetic root cause of the disease. These innovative treatments offer substantial improvements in lung function, reduce respiratory infections, and enhance nutritional status and quality of life. Most importantly, by initiating therapy early in life, irreversible organ damage can be prevented, profoundly altering the disease’s natural course and enabling many patients to reach a life expectancy close to that of the general population. However, given the high costs associated with these treatments, which can amount to several hundred thousand euros per patient annually, there is a critical need to assess their economic viability through rigorous health economic methods.

To address this need, the REMEDIA project uses cost-benefit analysis (CBA) and cost-effectiveness analysis (CEA), two complementary methods that provide robust frameworks for evaluating the efficiency of public health interventions. CBA is particularly valuable as it translates both the costs and the benefits of interventions into monetary terms, allowing a direct comparison. This approach assesses whether the benefits of CFTR modulator therapies, such as avoided complications and reduced hospitalizations, outweigh their costs, and by how much. It is especially well-suited to public health decision-making because it accounts for both direct and indirect benefits, including healthcare savings and gains in productivity. Moreover, it provides clear, interpretable results through absolute cost-benefit (net financial gain) and relative cost-benefit ratios (return per euro invested), enabling policymakers to determine if such interventions are economically justified. In the context of preventive strategies for respiratory diseases related to exposome risk factors, such as air pollution or early environmental exposures, the CBA approach is essential. It helps quantify the financial value of avoided disease burden, thereby supporting the implementation of long-term strategies aimed at reducing chronic respiratory conditions and their associated costs.

In parallel, CEA has been conducted to assess the impact of these treatments on survival, expressed as cost per life-year saved. Unlike cost-utility analysis (CUA), which depends on quality-adjusted life years (QALYs), a controversial and methodologically debated indicator, CEA avoids aggregating quality and length of life into a single, synthetic metric. QALYs and their derivative, the disability-adjusted life years (DALYs), have been widely criticized for relying on unproven assumptions, arbitrary thresholds, and potentially discriminatory disability weights. These indicators are not only difficult to interpret but also pose conceptual and ethical challenges, as demonstrated in previous analyses and confirmed by the WHO Advisory Committee on Health Research. By contrast, CEA offers a more transparent and robust evaluation, particularly when comparing early preventive interventions to later-stage, symptom-based treatments. It helps prioritize health strategies that maximize survival and long-term benefits within available budgets.

To account for uncertainty in cost and outcome estimates, the study uses Monte Carlo simulation simulations, running 100,000 probabilistic iterations. This allows for comprehensive sensitivity analysis by modeling variability in resource use, costs, and probabilities of health events, thereby delivering a realistic assessment of potential outcomes. Costs are expressed in euros and derived from published data on healthcare utilization and complication rates under both standard care and CFTR modulator treatments. Benefits are measured by the difference in complication-related costs between these two scenarios.

Altogether, the combined use of cost-benefit and cost-effectiveness analyses offers a powerful approach to guide health policy. It ensures that innovative treatments such as CFTR modulators, as well as preventive measures targeting exposome-related respiratory risks, are evaluated not only for their clinical effectiveness but also for their economic sustainability. By supporting informed, evidence-based decision-making, these methods promote the efficient allocation of healthcare resources and contribute to more equitable and impactful public health strategies.

Target users: Decision makers