Cystic fibrosis•

Socioeconomic burden of cystic fibrosis in Canada

Abstract

Background Cost of illness studies are important tools to summarise the burden of disease for individuals, the healthcare system and society. The lack of standardised methods for reporting costs for cystic fibrosis (CF) makes it difficult to quantify the total socioeconomic burden. In this study, we aimed to comprehensively report the socioeconomic burden of CF in Canada.

Methods The total cost of CF in Canada was calculated by triangulating information from three sources (Canadian CF Registry, customised Burden of Disease survey and publicly available information). A prevalence-based, bottom-up, human capital approach was applied, and costs were categorised into four perspectives (ie, healthcare system, individual/caregiver, variable (ie, medicines) and society) and three domains (ie, direct, indirect and intangible). All costs were converted into 2021 Canadian dollars (CAD) and adjusted for inflation. The cost of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies was excluded.

Results The total socioeconomic burden of CF in Canada in 2021 across the four perspectives was $C414 million. Direct costs accounted for two-thirds of the total costs, with medications comprising half of all direct costs. Out-of-pocket costs to individuals and caregivers represented 18.7% of all direct costs. Indirect costs representing absenteeism accounted for one-third of the total cost.

Conclusion This comprehensive cost of illness study for CF represents a community-oriented approach describing the socioeconomic burden of living with CF and serves as a benchmark for future studies.

What is already known on this topic

  • Previous cystic fibrosis cost of illness studies have been limited to healthcare system costs (eg, hospitalisations and healthcare encounters), which likely underestimates the total costs of cystic fibrosis. Further, the lack of standardised tools and reporting of costs makes it difficult to compare studies or generalise findings.

What this study adds

  • A comprehensive estimate of the socioeconomic burden of cystic fibrosis in Canada before the widespread introduction of highly effective modulator therapies.

How this study might affect research, practice or policy

  • The current study summarises the socioeconomic cost of cystic fibrosis in Canada in a period prior to the availability of highly effective modulator therapies and serves a benchmark to evaluate the impact of future policies and interventions in the cystic fibrosis population.

Introduction

Policy-makers use the cost of illness studies to understand the financial and population impacts of their decisions. For rare conditions, cost of illness studies are difficult to conduct due to the fragmented nature of data sources, and challenges with collecting data from relatively small populations. Since health administrative records and insurance databases are generally easier to access, the cost of rare diseases is often solely reported from the perspective of the healthcare system. From the community perspective, the costs of illness incurred by individuals and societal are especially important to capture, and omission can lead to gross underestimates of the total socioeconomic burden.1

Cystic fibrosis (CF) is one of the most prevalent rare genetic conditions. CF is typically diagnosed at birth and requires daily supportive therapies, quarterly clinic visits and frequent hospitalisations which put substantial demands on healthcare system resources, individuals, caregivers and society.2 Historical, longitudinal CF registries capture almost all CF-related healthcare encounters and clinical outcomes for nearly all people living with CF and are essential tools to describe the burden of this condition.3–5 A recent systematic review summarised 39 previously published CF cost of illness studies reported between 1999 and 2022.6 24 of the 39 studies (61.5%) focused on the healthcare system or payer perspective. 12 of the 39 studies (30.8%) reported costs from all three perspectives (healthcare system, individual and society). A comprehensive cost analysis which includes direct costs to individuals with CF, indirect costs of time lost due to CF and other quality of life measures, were seldomly reported (n=4, 10.2%). Further, the specific items that were included in each study varied considerably making it challenging to compare costs between studies.

Given the heterogeneity of previous CF cost of illness studies, we aimed to comprehensively capture the socioeconomic burden of CF in Canada.

Methods

Patient and public involvement

People with CF were included as part of the steering committee and were consulted on six separate occasions in small group sessions and 1:1 interview. People living with CF were provided opportunities to inform the design, analysis and interpretation of the survey and study findings.

Data sources

The total cost of CF in Canada was calculated by combining information from three sources (the CCFR, the customised BoD survey and publicly available costing information). The BoD survey was conducted online between July and October 2021. The BoD survey aimed to capture the out-of-pocket costs (eg, drug benefit premiums, CF equipment costs, transportation costs), employment status and the time spent by individuals with CF and their caregivers on CF-related care. The BoD survey comprised two sections: one for people living with CF (or caregivers for those under 18) and one for caregivers. Participants were recruited via social media platforms (CF Canada’s Twitter and Facebook channels, CF Canada website and email list) and through word of mouth at CF clinics across Canada.

BoD survey responses were deterministically linked (name, date of birth, sex) with the CCFR. The CCFR includes demographic and clinical data for nearly all people with CF who receive care at one of Canada’s 42 CF clinics; it is estimated that less than 1% of the Canadians with CF decline consent for their data to be captured. There are incentives for people with CF to receive care at a CF centre and incentives for CF centres to enter data into the CCFR. For instance, the Canadian healthcare system provides publicly funded health services, including coverage for visits to specialised care such as that provided by CF centres. There are no direct financial barriers to receive care at a CF centre. In many jurisdictions, access to CF medications requires a prescription from the CF centre, and in some cases, the medication must be dispensed from the CF centre to obtain government reimbursement. All Canadian CF clinics participate and enter data into the Registry. CF Canada provides some support for activities related to the Registry including data entry. Furthermore, the CCFR undergoes routine data accuracy checks.

Study period

The socioeconomic burden was estimated for the year 2021. BoD survey respondents were asked to recall the costs incurred in year 2021 to minimise recall bias. The CF population captured in CCFR in the year 2019 was used to extrapolate the number of people with CF, new diagnoses, transplants and deaths, as well as the healthcare utilisation rates (eg, clinic visits and hospitalisations) for the year 2021. The year 2019 was chosen to reflect the CF population, health status and healthcare utilisation prior to the availability of elexacaftor-tezacaftor-ivacaftor in Canada and to mitigate the impact of the COVID-19 pandemic on healthcare utilisation.

Socioeconomic burden estimates

The socioeconomic burden of living with CF in Canada in 2021 was determined by applying a prevalence-based, bottom-up, human capital approach using data that were triangulated from multiple sources. Costs were categorised into four perspectives (healthcare system, individual/caregiver, variable (ie, medicines), and society) and three domains (direct, indirect and intangible).6 7 The cost of each item (table 1) was determined by multiplying the estimated dollar value for a single unit by the units observed. When the unit could not be obtained from the CCFR (eg, transportation mode to attend a CF clinic) the proportion of BoD survey respondents that reported item use was applied to the entire population. For example, if 2% of the survey respondents reported requiring air travel to attend a clinic visit, the estimated airfare costs were multiplied for 2% of all clinic visits. Minimum and maximum costs were calculated using the lower and upper bounds based on the 25th and 75th percentiles of all responses respectively. If minimum or maximum costs could not be found, the median cost was reported. Whenever possible costs were also stratified by disease severity (defined as mild: ≥70% predicted forced expiratory volume in 1 s (FEV1), moderate: per cent predicted 40%–69% predicted FEV1, severe: <40% predicted FEV1 and postlung transplant), age (paediatric and adult) and biological sex. All costs were converted to 2021 CAD using the Canada Inflation Calculator.8 A summary of all costs and sources is available in online supplemental table 1.

Table 1
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Summary of perspectives, domains and items used to estimate the total socioeconomic burden of CF

Direct costs

Direct costs refer to expenses directly attributed to a condition. Direct medical costs were subcategorised into four groups based on who paid for it: healthcare system, individual, caregiver and variable (ie, medications).

Healthcare system

Healthcare system utilisation such as the number of hospitalisations, clinic visits, tests and procedures were obtained from the CCFR. The costs of CF-specific hospital encounters (ie, hospitalisations, lung transplants) were obtained from the 2019–2020 Canadian Institute for Health Information Patient Cost Estimator.9 The staffing cost of a CF clinic was estimated based on the minimum staffing standards for CF healthcare teams according to CF Canada10 with healthcare provider salaries (eg, respirologist, nurse, dietician, pharmacist) obtained from Statistics Canada.11 Diagnostic and procedural costs incurred (eg, pulmonary function tests and chest X-rays) were informed by provincial physician billing codes.12–15 When costs could not be obtained from these sources, a literature search was conducted to identify studies that had previously published costs specifically for each item. If neither the CCFR nor BoD survey was sufficient to estimate a utilisation unit, the study steering committee (comprised CF researchers, healthcare providers, CF Canada staff and members of the CF community) arrived at a number through discussion and unanimous agreement (eg, the number of CT scans, bone mineral density test).

Variable

Payment for medications in Canada is covered by multiple payers which makes it very difficult to directly attribute the medication costs. In some jurisdictions, the government covers the cost of all medications, whereas in others the government only covers a proportion, and the remaining costs are covered by either private benefit plans and/or out of pocket by the individual. Therefore, medicines were included in the variable category. A comprehensive list of CF-specific medicines was developed by the study team, including CF physicians and pharmacists. The estimated unit price for each medicine was obtained from the Alberta Blue Cross Drug Benefit List.16 Participants who completed the BoD survey entered the name, formulation, dose and frequency of each prescribed medicine they used. The average (mean) per person annual cost for each medicine was calculated. The total annual cost of each medicine was calculated by multiplying the average cost of each medicine by the number of people prescribed each medicine. The proportion of Canadians taking each medication from the BoD survey was cross-referenced with data reported in the CCFR. The number of people taking each medicine was taken from the CCFR, or if not available in the CCFR, the proportion of respondents taking each medicine in the BoD survey was extrapolated to the entire CF population. The cost of all CFTR modulators was purposefully excluded.

Individual

Informed by community consultations, typical out-of-pocket expenses such as medical equipment, supplies, travel expenses to attend CF centres and out-of-hospital healthcare were included in the BoD survey. The average self-reported cost for each item from the BoD survey was cross-referenced with an online source (eg, Statistics Canada, vendor websites) to assess validity. Survey responses were reviewed for implausible values. The additional cost of a CF diet (ie, 110%–200% energy intake of a typical diet) was calculated by conservatively applying a 40% increase to the estimated cost of a typical diet for Canadians. The cost of a CF diet was cross-referenced with the literature and by calculating the extra cost per calorie, if the average person with CF consumes 500 extra calories per day.17 18

Indirect costs

Indirect costs refer to the costs due to loss of productivity, either due to absence from work (due to the disease), suboptimal productivity despite attending work and having become unemployed because of the disease. Indirect costs were subcategorised into two groups (individual and caregiver). The financial cost of missed days for school or work due to illness (sick days) was estimated using the human capital approach and a modified version of the Workplace Productivity and Activity Impairment Questionnaire;19 20 where the number of days missed in 2021 was recorded. The average time spent by an individual with CF or their caregiver on daily treatments, attending CF clinic visits and hospitalisations was summarised based on BoD survey responses and published literature.21 22 The national average hourly wage reported by Statistics Canada (2021; $C33.4) was used, whereas the unit cost of each hour for children was based on the cost of education (2021; $C12.4).23 24 The type and frequency (daily, weekly, monthly) of informal care responsibilities of caregivers included in the BoD survey. We were unable to estimate the time spent on informal caregiving responsibilities using the responses as collected

Intangible costs

Intangible costs were summarised quantitively based on survey responses. Quality of life was summarised based on responses to the Cystic Fibrosis Questionnaire Revised (CFQ-R) included in the BoD survey. Responses for individuals older than 14 years were converted to European Quality of Life Five Dimension (EQ-5D) based on the approach published by Acaster et al.25 Years of potential life lost due to premature death was calculated using the Global Burden of Disease approach.26–28 The mean age of death for each person with CF who died in 2019 was compared with the standard Canadian life expectancy using life tables provided by Statistics Canada (1980–2020).

Results

Study population

In 2019, there were a total of 4707 people reported on in the CCFR, and the socioeconomic burden of CF in 2021 was estimated for this population. A summary of the CF population included is summarised in online supplemental table 2. A total of 369 people with CF (or the caregivers of people younger than 18 years) completed the survey, representing 8% of the total CF population in Canada. A total of 241 caregivers completed the caregiver survey. Of the survey respondents, the majority (63%) of people with CF older than 18 years of age were employed; of those, 70% were employed full time and 28% part time, 2% did not answer this question. 20% of survey respondents reported having dependents. Most caregivers (85%) reported being the parents or guardians of someone with CF. 84% of CF survey respondents (n=309 out of 369) were linked to the CCFR. Overall, the survey respondents were representative of the Canadian CF population captured in the CCFR with the exception that more females completed the survey than males, a greater proportion of those 25–50 years, and slightly more people with moderate and severe disease (online supplemental table 3).

Cost of CF in Canada

The overall socioeconomic burden in 2021 for 4707 people living with CF and their caregivers in Canada was $C414 million (25th percentile $C230 million, 75% percentile $C653 million) (figure 1, table 2). Direct costs ($C270 million) accounted for two-thirds of the total cost and medications ($C134 million) accounted for half of all direct costs (one-third of the total cost). Indirect costs ($C140 million) accounted for one-third of the total cost (figure 2).

Figure 1
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Figure 1

Summary of the economic burden ($C) of CF in 2021 by cost domains. CF, cystic fibrosis.

Figure 2
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Figure 2

Total cost of cystic fibrosis by domain and perspective in Canada in 2021 (CAD million). Median cost is represented by dots, whereas the minimum and maximum costs are represented by the error bars. CAD, Canadian dollars; CF, cystic fibrosis; TB, tuberculosis

Table 2
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Summary of the economic burden of CF in 2021 by cost domains

Direct costs to the healthcare system (excluding medicines) contributed 21% of the total socioeconomic burden. Clinic visits ($C45 million) and hospitalisations ($C31.5 million) were the major contributors to direct healthcare costs. Medicines, as a separate domain, accounted for one-third of all costs; 90% of the total medicine cost ($C134 million) was attributed to four types of medications (inhaled antibiotics, mucolytics, pancreatic enzyme supplements and biologics (eg, omalizumab)) (table 2).

Individual out-of-pocket expenses accounted for $C48 million (on average $C10 198/person/year). Medical equipment (eg, compressor, nebulisers) was the greatest expense ($C27.8 million). Out-of-pocket expenses also included health services typically not covered by provincial payers ($C6 million, eg, psychological services, massage therapy, chiropractors). A majority (65%) of survey respondents reported having private drug plans (either through an employer or a personal plan), and 34% had more than one private drug plan. A quarter of survey respondents reported they had annual insurance coverage limits, and 18% had limitations on lifetime insurance coverage, such that after these limits are reached medication costs would need to be paid for out-of-pocket. The additional grocery costs to sustain a high-fat diet contributed an additional $C6.8 million annually ($C4/day/person). Travel, parking and food costs for attending quarterly clinic visits accounted for nearly $C1 million. Of the subset of people with CF who have been listed for a transplant or received a transplant, 23% indicated they had to relocate to be closer to a transplant centre. Caregivers’ out-of-pocket expenses totalled $C3.3 million with 70% of caregivers reporting an average expense of $C1000 per year.

Indirect costs accounted for one-third of the total costs of CF ($C140 million). An estimated 3 million hours annually (individual and caregiver) were spent managing daily CF treatments per year. School and/or work absenteeism contributed 10% of the indirect costs ($C7.3 million for people with CF, $C7 million for caregivers (figure 2, table 2)). Caregivers accounted for 31% ($C42.5 million) of the indirect costs.

Stratified costs

Canadians with CF with severe disease (8% of the CF population) had the highest total per person cost at $C117 233/year (figure 3). The cost was mainly attributable to higher healthcare and medicine costs (figure 4A). Indirect costs were similar across disease severity groups, averaging $C30 000 per person per year. Caregiver absenteeism contributed a higher proportion of the overall burden for children, whereas individual absenteeism contributed a higher burden cost for adults (figure 4B). Females with CF had a slightly higher cost per person/year than males, primarily driven by higher healthcare system (greater number of clinic visits and hospitalisations) and medication costs (figure 4C).

Figure 3
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Figure 3

Stratified cost of cystic fibrosis by disease severity. Total cost (A) and per person cost (B) is stratified using disease severity (mild: ≥70% predicted FEV1; moderate: 40%–69% predicted FEV1; severe: <40% predicted FEV1). CAD, Canadian dollars; FEV1, forced expiratory volume in 1 s.

Figure 4
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Figure 4

Percentage of total cost by perspective and by disease severity (A), age group (B) and biological sex (C). Disease severity, defined using lung function (mild: ≥70% predicted FEV1; moderate: 40%–69% predicted FEV1; severe: <40% predicted FEV1 and postlung transplant). FEV1, forced expiratory volume in 1 s.

Intangible costs

The median quality of life score, determined by the EQ-5D (where one indicates good health and 0 indicates poor health) derived from the CFQ-R score was 0.74 (IQR 0.58–0.86) and was similar by disease severity group except for a higher score in the post-transplantation group (0.83, IQR 0.53–0.85). In 2019, there were 49 CF-related deaths with a mean age of death of 41.9 years (range 14.5–71.5 years) corresponding to 2088 years of life lost due to premature death.

Financial challenges were also reported by people with CF and their caregivers; 22% of people with CF relied on federal or provincial income assistance, 15% were on disability programmes. Further, 14% of respondents reported skipping clinic visits because they could not afford to take time off work, and 19% of respondents reported skipping a medication or treatment because of costs. One-third of respondents used their savings or retirement funds to afford treatments. Caregivers also reported financial challenges; 25% were unable to seek employment, 39% declined job offers or promotions and 10% quit their jobs entirely because of caregiving responsibilities. The type and frequency of informal caregiving are summarised in online supplemental table 4. Although these were not included in the total cost, caregivers of children and young people with CF were more likely to report more informal caregiving responsibilities.

Excluded costs

Costs associated with assisted reproductive technologies were included in the BoD survey whereas utilisation was not recorded in the CCFR, therefore, the direct healthcare, and out-of-pocket costs associated with assisted reproductive technologies were not included in the total cost estimates. Based on 22% of survey respondents who reported requiring assisted reproductive technologies in their lifetime, the lifetime assisted reproductive technologies costs extrapolated to the total population totalled $C12 million. The total costs exclude the cost of all CFTR modulators.

Discussion

The total socioeconomic costs of CF in Canada from four perspectives (healthcare system, individual/caregiver, variable (ie, medicines) and society) across three domains (direct, indirect, intangible) were estimated by triangulating data sources. These estimates provide a comprehensive evaluation of the total socioeconomic burden of CF in Canada prior to the widespread introduction of highly effective modulator therapies. A comprehensive summary of the total socioeconomic burden, as well as the direct and indirect costs incurred by individuals and their families, will help to inform and support advocacy efforts and programme development aimed at provincial, territorial and federal governments, as well as insurers and group benefit plans. From a policy perspective, the socioeconomic cost estimates will help inform economic evaluations of existing and novel therapeutics.

There have been at least 40 CF cost-of-illness studies published to date, including four systematic reviews.6 29–31 Direct comparisons with previous literature are challenging since the perspective (healthcare system, individual, society) and specific domains and items included in each of the studies varied considerably.6 Direct out-of-pocket costs of CF to individuals and caregivers have been infrequently reported. Since 1990, there have been five Canadian studies published; three reported direct healthcare costs, one included indirect caregiver costs and one included both direct and indirect costs. The total direct healthcare costs estimated (including medications) in the current study ($C47 330/person/year in 2021 Canadian dollars) are consistent with a recently published study which reported the total direct healthcare costs in the province of British Columbia, including inpatient, outpatient and medication costs (adjusted to $C46 000/person/year in 2021 Canadian dollars) (7). The indirect costs of CF estimated in the current study ($C29 694/person/year in 2021 Canadian dollars) were similar to a 2006 study by Guerriere et al (adjusted to $C30 392/person/year in 2021 Canadian dollars).32 The indirect costs in the Guerriere et al, study represented 72% of the cost of CF, whereas in the current study indirect costs represented 33% of the total cost. The difference likely reflects that Guerriere et al, only included ambulatory care as direct costs. Interestingly, the average number of sick days reported by the BoD survey respondents was similar to the general Canadian population (mean of 10.3 days33). There have been a limited number of studies investigating the socioeconomic burden of rare diseases in Canada. The identified studies were heterogeneous in terms of perspectives and domains making comparisons with CF difficult. For example, a study of the economic costs of 14 inherited retinal conditions in Canada (~20 000–80 000 people) found per person costs to range from $C78 197 to $C319 281 per year, which included the direct and indirect costs as well as the cost of loss of well-being measured using disability-adjusted life-years.34

Medications accounted for half of the direct CF costs, which is consistent with previous literature.35 In Canada, medically necessary healthcare services (eg, clinic visits and hospitalisations) are covered by the single payer (ie, government). The coverage of prescription medications varies by province and individual’s private drug plan coverage. For many people with CF in Canada, medications are paid for out of pocket, or with high co-pays and deductibles. To cover the costs of medicines, many Canadians had supplemental health benefits, either from an employer or a private provider that cover a proportion or all the non-essential healthcare costs (including medications). Some provinces also have programmes that help to cover the costs of medications specifically for CF. The high costs of medications likely contributed to the fact that 19% of respondents reported skipping a medication because they could not afford it. This finding is consistent with other recent Canadian surveys of the general population.36 37 Affordability of medicines will further be exacerbated for individuals with lifetime caps on private benefits insurance, which will be exceeded after a few years of CFTR modulator coverage.

Using CFQ-R responses from the BoD survey and applying an algorithm to estimate EQ-5D,25 we report on quality of life using an established patient-reported measure. The average EQ-5D was 0.74 which was lower than what is expected in health (a score of 1) but higher than in the study by Acaster et al, which reported an EQ-5D of 0.67.25 The differences may be due to the reporting of EQ-5D as a median in our study and mean by Acaster et al.25 Further, the quality-of-life score was relatively consistent across disease severity, suggesting that the BoD survey respondents may be healthier overall.

Strengths and limitations

The comprehensive list of CF-related expenses was codeveloped with the CF community after systematic review of existing CF cost of illness studies and literature for other rare diseases. Linkage of the BoD survey with the CCFR reduced participant burden and improved coverage of items that could be included. The BoD survey respondents (8% of all people with CF) were generally representative of the Canadian population in terms of socioeconomic status (eg, annual household income).38 The BoD survey respondents represented a slightly older, with more female respondents, and more severe disease group compared with the CCFR, which may have led to an overestimate of some items. To mitigate this, BoD survey responses were cross referenced with either CCFR data or vendor websites and where possible disease severity stratum costs were applied.

The average cost of each medication was applied to all people taking that medication, which was appropriate for many, but not all medicines. Stratified costs, especially by disease severity, often included a small number of observations for each medication. The list of possible expenses incurred by the healthcare system or individuals with CF and their caregivers may be missing important items, or items that are needed by a smaller subset of the population not captured in the survey. Further, we were not able to quantify a cost associated with presenteeism (lower work productivity due to disease or caregiving responsibilities). Finally, the CCFR data were taken from the year 2019 to represent a time before the widespread availability of elexacaftor-tezacaftor-ivacaftor and before the COVID-19 pandemic, whereas the survey was conducted in 2021, during a time when elexacaftor-tezacaftor-ivacaftor was available to some people through the compassionate use programme. The choice of different sources was purposeful and pragmatic to reduce recall bias for the survey. Recall bias may have randomly affected the reported costs. Nonetheless, we cannot exclude the possible influences of either early use of elexacaftor-tezacaftor-ivacaftor or the pandemic on our estimates. These limitations may explain why the EQ-5D scores were higher in the severe group compared with previous literature.

The costs of CFTR modulators (which can be in excess of $C250 000 per person per year) were excluded from the medication costs as our primary aim was to characterise the burden of CF prior to the availability of highly effective modulators. A growing number of studies have demonstrated substantive reductions in hospitalisations rates and medication costs after the introduction of elexacaftor-tezacaftor-ivacaftor use.39–41 At the time of the survey, 15% of people with CF were prescribed a CFTR modulators, and less than 1% were prescribed elexacaftor-tezacaftor-ivacaftor. The majority of this group was prescribed lumacaftor-ivacaftor or tezacaftor-ivacaftor which only showed modest improvements in outcomes and minimal changes to healthcare utilisations.42 This comprehensive summary of the pre-elexacaftor-tezacaftor-ivacaftor socioeconomic costs of CF will allow for direct comparisons across all domains in the future.

Indirect costs included the time individuals with CF or their caregivers spent on treatment, attending clinical visits and during hospitalisations. We applied the human capital approach to estimate indirect costs. There are other approaches (eg, friction cost method) and informal caregiver ‘spillover’ effects that were not considered and may have produced different estimates.43–46 Common informal care activities are reported (online supplemental table 4) but not included in the indirect caregiver costs. In Chevreul et al, informal caregiving costs determined using the proxy good method attributed between 17% and 77% of the total cost of CF across eight European countries.47 Wherein Angelis et al also used the proxy good method and attributed 45% of the total costs to informal caregiving.48 Therefore, the omission of informal care may have substantially underestimated the total costs of CF in Canada.47 49 Direct comparisons between the studies are challenging because of the different approaches used to define each domain and estimate costs.

Intangible costs were estimated using the CFQ-R questionnaire for people with CF over the age of 14 years. Caregivers of people with CF experience unique challenges, which may impact quality of life as well. The BoD survey did not include an assessment of the quality of life of caregivers, which will be important to include in future studies.

Conclusion

This socioeconomic burden study represents a community-oriented approach to estimating the total costs of living with CF in Canada, prior to the widespread availably of highly effective CFTR modulators. The framework applied serves as a benchmark for post-CFTR modulator studies.

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