Costs of treatment of adult patients with cystic fibrosis in Poland and internationally

doi:10.1016/j.puhe.2017.03.003

Public Health

Volume 148, July 2017, Pages 49-55

https://doi.org/10.1016/j.puhe.2017.03.003 Get rights and content

Highlights

•
Cystic fibrosis (CF) represents a health problem with important societal impact in middle- and high-income countries.
•
Numerous cost of illness studies have been conducted over the past decades; however, few have addressed CF.
•
The analysis highlights the poor consensus of methodological approaches used by different investigators.

Abstract

Objectives

Despite its low prevalence, cystic fibrosis (CF) may have a considerable impact on healthcare system expenditures in terms of direct healthcare costs and lost productivity. This study was aimed at calculation of costs associated with CF treatment in Poland, as well as at comparison of average costs of treatment of CF patients in selected countries, taking into account the purchasing power parity.

Study design

Retrospective study.

Methods

The researchers undertook a retrospective study of adult patients with CF taking into account the broadest social perspective possible. Medical and non-medical direct costs as well as indirect costs were calculated. CF costs estimated by researchers from other countries over the last 15 years were also compared.

Results

Total annual treatment cost per one CF patient in Poland was on average EUR 19,581.08. Costs of treatment of CF patients over the last 15 years varied between the countries and ranged from EUR 23,330.82 in Bulgaria to EUR 68,696.42 in the United States.

Conclusions

CF is an international problem. The data in this study could be the baseline for integrated and harmonised approaches for periodical assessment of the future impact of new public policies and interventions for rare diseases at the national and international levels.

Introduction

In European Union (EU) countries, any disease affecting fewer than five people out of 10,000 is considered rare. That number may seem small, but it translates into approximately 246,000 people throughout the EU's 28 member countries.¹

One of the key rare diseases is cystic fibrosis (CF), with a prevalence in Europe is estimated at one case per 8000–10,000 people.² American researchers report its incidence in the United States between 1:1900 and 1:3700.³ In 2008, Farrell⁴ estimated the incidence of CF in the EU countries at between 1:1353 in Ireland and 1:25,000 in Finland. Polish register of patients with CF kept by the Polish Cystic Fibrosis Society lists over 1300 patients, of which around 300 are adults.⁵ Not all CF patients are listed in the register as registration is optional. The mean age of a CF patient was 20.6 years in 2014.⁶

Despite its low prevalence, CF may have a considerable impact on healthcare system expenditures in terms of direct healthcare costs and lost productivity as orphan drugs are frequently very expensive and CF symptoms tend to appear at a young age.7, 8, 9, 10

The aim of a cost of illness study is to identify and measure all the costs of a particular disease, including the direct, indirect and intangible dimensions. The output, expressed in monetary terms, is an estimate of the total burden of a particular disease to society.¹¹ Knowledge of the costs of an illness can help policymakers to decide which diseases need to be addressed first by healthcare and prevention policy. Additionally, these studies can indicate for which diseases cures would be valuable in reducing the burden of disease. For specific stakeholders, such as the federal government, cost of illness studies can show the financial impact a disease has on public budget.¹¹ Numerous cost of illness studies have been conducted over the past decades across a range of diseases, however, few have addressed rare diseases, in particular CF. In this context, this study was aimed at calculation of costs associated with CF treatment in Poland as well as at comparison of average costs of treatment of CF patients in selected countries, taking into account the purchasing power parity.

Section snippets

Literature review

We reviewed relevant literature on costs of CF from the last 15 years. The following databases were browsed: MEDLINE, Web of Science and EMBASE. The search words had to be part of the title or abstract. The search terms used were ‘cystic fibrosis’ AND ‘cost of illness’; ‘cystic fibrosis’ AND ‘economic evaluation’; ‘cystic fibrosis’ AND ‘cost study’ and ‘cystic fibrosis’ AND ‘cost analysis’. Language was restricted to English. No other limitations were specified. A search on the references cited

Costs of treatment of CF in selected countries

A total of 591 article titles were initially screened. Of these, 123 abstracts were retrieved for further investigation. Studies reporting any cost of illness data were included, while studies not providing specific data were excluded. Cost-effectiveness studies were only included if they reported information on the cost of illness or components of specific types of health care, individual or societal costs. A total of 35 full-text articles were accessed for eligibility (including cited

Discussion

Among rare diseases, CF represents a health problem with important societal impact in middle- and high-income countries.7, 10 Incidence and prevalence of CF and its effect on health and society in relation to morbidity, mortality, economic costs and quality of life justifies the attention given to the disease by both the society and its healthcare and health economics experts.¹⁸

As the presented results are the only results obtained in Poland, they could not have been compared with national

Ethical approval

The study was approved by the Bioethics Committee of the University of Medical Sciences, Poznan, Poland.

Funding

None declared.

Competing interests

None declared.

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Cited by (8)

Measuring the burden of cystic fibrosis: A scoping review
2023, Journal of Cystic Fibrosis
Cystic fibrosis (CF) contributes a significant economic burden on individuals, healthcare systems, and society. Understanding the economic impact of CF is crucial for planning resource allocation.
We conducted a scoping review of literature published between 1990 and 2022 that reported the cost of illness, and/or economic burden of CF. Costs were adjusted for inflation and reported as United States dollars.
A total of 39 studies were included. Direct healthcare costs (e.g., medications, inpatient and outpatient care) were the most frequently reported. Most studies estimated the cost of CF using a prevalence-based (n = 18, 46.2 %), bottom-up approach (n = 23, 59 %). Direct non-healthcare costs and indirect costs were seldom included. The most frequently reported direct cost components were medications (n = 34, 87.2 %), inpatient care (n = 33, 84.6 %), and outpatient care (n = 31, 79.5 %). Twenty-eight percent (n = 11) of studies reported the burden of CF from all three perspectives (healthcare system (payer), individual, and society). Indirect costs of CF were reported in approximately 20 % of studies (n = 8). The reported total cost of CF varied widely, ranging from $451 to $160,000 per person per year (2022 US$). The total cost depended on the number of domains and perspectives included in each study.
Most studies only reported costs to the healthcare system (i.e., hospitalizations and healthcare encounters) which likely underestimates the total costs of CF. The wide range of costs reported highlights the importance of standardizing perspectives, domains and costs when estimating the economic burden of CF.
Temporal trends in healthcare resource use and associated costs of patients with cystic fibrosis
2022, Journal of Cystic Fibrosis
Citation Excerpt :
Our study identified higher total costs in adults than in children, with adults having higher drug and hospitalisation costs, which was in agreement with the Chevreul study [27] and a recent US study conducted on the CF Medicaid population [28]. Outside France, several studies have estimated the cost of CF care [17–19,21, 25,28–30]. According to an observational cohort study using German claims data, pharmaceutical treatment and hospital care accounted for approximately 73.3% and 15.0% of the direct costs associated with CF, respectively, in 2016 [29].
Better insights into the natural course of cystic fibrosis (CF) have led to treatment approaches that have improved pulmonary health and increased the life expectancy of affected individuals. This study evaluated how the combination of modified demographics and changes in CF management impacted resource consumption and the cost of care.
Medical records of CF patients from 2006 to 2016 in the French CF Registry were linked to their corresponding claims data (SNDS). Medications, medical visits, procedures, hospitalisations, and indirect costs were annualized by calendar year from 2006 to 2017.
Of the 7,671 patients included in the French CF Registry, 6,187 patients (80.7%) were linked to the SNDS (51.9% male, mean age = 24.7 years). The average cost per patient was €14,174 in 2006, €21,920 in 2011 and €44,585 in 2017. Costs associated with hospital stays increased from €3,843 per patient in 2006 to €6,741 in 2017. In 2017, the mean cost per CF patient was allocated as follows: 72% for medications (of which 51% for modulator therapies), 15% for hospital stays, 7% for medical visits, 3% for indirect costs, 2% for medical devices, 1% for outpatient medical procedures.
There was a strong increase in the mean annual cost per CF patient between 2006 and 2017, mostly due to the cost of therapy after the introduction of cystic fibrosis transmembrane conductance regulator (CFTR) modulators. The combination of an increase in the number of CF patients – particularly adult patients – and an increase in the annual cost per patient led to a substantial increase in the total cost of CF disease care for the health systems.
Hospital costs of Canadian cystic fibrosis patients
2022, Canadian Journal of Respiratory, Critical Care, and Sleep Medicine
Cost-of-illness studies in rare diseases: a scoping review
2021, Orphanet Journal of Rare Diseases
Reflections on the importance of cost of illness analysis in rare diseases: A proposal
2021, International Journal of Environmental Research and Public Health
The Economic Burden of Cystic Fibrosis in Germany from a Payer Perspective
2019, PharmacoEconomics

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Original ResearchCosts of treatment of adult patients with cystic fibrosis in Poland and internationally

Highlights

Abstract

Objectives

Study design

Methods

Results

Conclusions

Introduction

Section snippets

Literature review

Costs of treatment of CF in selected countries

Discussion

Ethical approval

Funding

Competing interests

J Cyst Fibros

Value Health

Value Health

Lancet

J Cyst Fibros

J Cyst Fibros

J Cyst Fibros

Cystic fibrosis

Mukowiscydoza – Poradnik dla rodziców i chorych

Cost of illness of cystic fibrosis in Germany: results from a large cystic fibrosis centre

Pharmacoeconomics

Economic burden of non-cystic fibrosis bronchiectasis in the first year after diagnosis from a US health plan perspective

Appl Health Econ Health Policy

Original Research
Costs of treatment of adult patients with cystic fibrosis in Poland and internationally