Advertisement

The European Journal of Health Economics

, Volume 18, Issue 1, pp 73–82 | Cite as

Cost-of-illness analysis and regression modeling in cystic fibrosis: a retrospective prevalence-based study

  • Tomáš Mlčoch
  • Jiří Klimeš
  • Libor Fila
  • Věra Vávrová
  • Veronika Skalická
  • Marek Turnovec
  • Veronika Krulišová
  • Jitka Jirčíková
  • Dana Zemková
  • Klára Vilimovská Dědečková
  • Alena Bílková
  • Vladimíra Frühaufová
  • Lukáš Homola
  • Zuzana Friedmannová
  • Radovan Drnek
  • Pavel Dřevínek
  • Tomáš Doležal
  • Milan MacekJr.
Original Paper

Abstract

Background

Economic data pertaining to cystic fibrosis (CF), is limited in Europe generally, and completely lacking in Central and Eastern Europe. We performed an analysis of all direct costs associated with CF relative to key disease features and laboratory examinations.

Methods

A retrospective prevalence-based cost-of-illness (COI) study was performed in a representative cohort of 242 CF patients in the Czech Republic, which represents about 65 % of all Czech CF patients. Medical records and invoices to health insurance companies for reference year 2010 were analyzed.

Results

The mean total health care costs were €14,486 per patient, with the majority of the costs going towards medicinal products and devices (€10,321). Medical procedures (€2676) and inpatient care (€1829) represented a much smaller percentage of costs. A generalized linear model showed that the strongest cost drivers, for all cost categories, were associated with patient age and lung disease severity (assessed using the FEV1 spirometric parameter), when compounded by chronic Pseudomonas aeruginosa airway infections. Specifically, maximum total costs are around the age 16 years; a FEV1 increase of 1 % point represented a cost decrease of: 0.9 % (medicinal products), 1.7 % (total costs), 2.8 % (procedures) and 7.0 % (inpatient care).

Conclusions

COI analysis and regression modeling using the most recent data available can provide a better understanding of the overall economic CF burden. A comparison of our results with other methodologically similar studies demonstrates that although overall costs may differ, FEV1 can nonetheless be utilized as a generally transferrable indicator of the relative economic impact of CF.

Keywords

Cystic fibrosis Cost-of-illness Disease severity Health care costs FEV1 Generalized linear model 

JEL Classification

I10 Health: General I18 Health: Government policy·Regulation·Public Health 

Notes

Acknowledgments

Supported by the Czech Ministry of Health 00064203 to MM and PD; NT/13770-4; NF-CZ11-PDP-3-003-2014, COST - LD14073 and CZ.2.16/3.1.00/24022OPPK to MM. We acknowledge administrative help from Ms. Ivana Funkova and support from the Czech CF Association (www.cfklub.cz).

Disclosure

This study was partially supported by Vertex Pharmaceuticals (USA) to the Institute of Health Economics and Technological Assessment in Prague (TM, JK, JJ, and TD). The remaining authors do not have any conflict of interest that could influence the content or processing of this manuscript.

Supplementary material

10198_2015_759_MOESM1_ESM.docx (56 kb)
Supplementary material 1 (DOCX 55 kb)

References

  1. 1.
    Goss, C.H., Ratjen, F.: Update in cystic fibrosis 2012. Am. J. Respir. Crit. Care Med. 187, 915–919 (2013). http://www.atsjournals.org/doi/abs/10.1164/rccm.201301-0184UP?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%3dpubmed
  2. 2.
    Lomas, P.: Enhancing adherence to inhaled therapies in cysic fibrosis. Ther. Adv. Respir. Dis. 8, 39–47 (2014). http://tar.sagepub.com/content/8/2/39.long
  3. 3.
    Klimeš, J., Doležal, T., Kubáčková, K., Turnovec, M., Macek Jr., M.: Health-economic aspects of cystic fibrosis screening and therapy. Eur. Respir. Soc. monogr. 64, 304–319 (2014)Google Scholar
  4. 4.
    Gu, Y., Pérez-García, S., Massie, J., van Gool, K.: Cost of care for cystic fibrosis: an investigation of cost determinants using national registry data. Eur. J. Health Econ. 16, 709–717 (2015). http://link.springer.com/article/10.1007%2Fs10198-014-0621-5
  5. 5.
    De Boeck, K., Zolin, A., Cuppens, H., Olesen, H.V., Viviani, L.: The relative frequency of CFTR mutation classes in European patients with cystic fibrosis. J. Cyst. Fibros. 13, 403–409 (2014). http://www.cysticfibrosisjournal.com/article/S1569-1993(13)00228-2/abstract
  6. 6.
    Mehta, G., Macek, M. Jr., Mehta, A.: Cystic fibrosis across Europe: EuroCareCF analysis of demographic data from 35 countries. J. Cyst. Fibros. 9(Suppl. 2), S5–S21 (2010). http://www.cysticfibrosisjournal.com/article/S1569-1993(10)00101-3/abstract?cc=y
  7. 7.
    McCormick, J., Mehta, G., Olesen, H.V., Viviani, L., Macek, M. Jr., Mehta, A., on behalf of the European Registry Working Group.: Comparative demographics of the European cystic fibrosis population: a cross-sectional database analysis. Lancet 375, 1007–1013 (2010). http://linkinghub.elsevier.com/retrieve/pii/S0140-6736(09)62161-9
  8. 8.
    Stephenson, A., Hux, J., Tullis, E., FRCPC, Austin, P.C., Corey, M., Ray, J.: Socioeconomic status and risk of hospitalization among individuals with cystic fibrosis in Ontario, Canada. Pediatr. Pulmonol. 46, 376–384 (2011). http://onlinelibrary.wiley.com/doi/10.1002/ppul.21368/abstract;jsessionid=4FA0A2E4BC67FB89E860D14A309C4065.f02t04
  9. 9.
    Larg, A., Moss, J.R.: Cost-of-illness studies: a guide to critical evaluation. Pharmacoeconomics 29, 653–671 (2011). http://link.springer.com/article/10.2165%2F11588380-000000000-00000
  10. 10.
    Vasiliadis, H.M., Collet, J.P., Penrod, J.R., Ferraro, P., Poirier, C.: A cost-effectiveness and cost-utility study of lung transplantation. J. Heart Lung Transplant. 24, 1275–1283 (2005). http://www.jhltonline.org/article/S1053-2498(04)00557-1/abstract
  11. 11.
    Farrel, P.M., Rosenstein, J.B., White, T.B., Accurso, F.J., Castellani, C., Cutting, R.G., Durie, P.R., LeGrys, A.V., Massie, J., et al.: Guidelines for diagnosis of cystic fibrosis in newborns through older adults: cystic fibrosis foundation consensus report. J. Pediatr. 153, S4–S14 (2008). http://www.jpeds.com/article/S0022-3476(08)00398-3/abstract
  12. 12.
    Balascakova, M., Holubova, A., Skalicka, V., Zemkova, D., Kracmar, P., Gonsorcikova, L., Camajova, J., Piskackova, T., Lebl, J., Drevinek, P., et al.: Pilot newborn screening project for cystic fibrosis in the Czech Republic: defining role of the delay in its symptomatic diagnosis and influence of ultrasound-based prenatal diagnosis on the incidence of the disease. J. Cyst. Fibros. 8, 224–227 (2009). http://www.cysticfibrosisjournal.com/article/S1569-1993(09)00004-6/abstract
  13. 13.
    Zapletal, A., Šamánek, M., Paul, T.: Lung function in children and adolescents. Methods, reference values. Prog. Respir. Res. Vol. 22. Karger, Basel (1987). http://www.karger.com/Book/Toc/221224
  14. 14.
    Miller, M.R., Hankinson, J., Brusasco, V., Burgos, F., Casaburi, R., Coates, A., Crapo, R., Enright, P., van der Grinten, C.P.M., Gustafsson, P., et al.: Standardisation of spirometry. Eur. Respir. J. 26, 319–338 (2005). http://www.ersj.org.uk/content/26/2/319.full.pdf
  15. 15.
    Harmonized index of consumer price, Eurostat. http://ec.europa.eu/eurostat/web/hicp/data/database
  16. 16.
  17. 17.
    Wang, J., Chen, H.: Use of percentiles and Z-scores in anthropometry. In: Handbook of anthropometry, pp. 29–48 (2012). http://link.springer.com/chapter/10.1007/978-1-4419-1788-1_2#page-1
  18. 18.
    Marchetti, F., Giglio, L., Candusso, M., Faraguna, D., Assael, B.M.: Early antibiotic treatment of Pseudomonas aeruginosa chronic infection in cystic fibrosis: a critical review of the literature. Eur. J. Clin. Pharmacol. 60, 67–74 (2004). http://link.springer.com/article/10.1007%2Fs00228-004-0735-2
  19. 19.
    Snell, C., Fernandes, S., Bujoreanu, S., Garcia, G.: Depression, illness severity, and healthcare utilization in cystic fibrosis. Pediatr. Pulmol. 49, 1177–1181 (2014). http://onlinelibrary.wiley.com/doi/10.1002/ppul.22990/abstract;jsessionid=7409EB9B976250A532D75548451DE03F.f02t01
  20. 20.
    Křenková, P., Piskáčková, T., Holubová, A., Balascakova, M., Krulisova, V., Camajova, J., Turnovec, M., Libik, M., Norambuena, P., Stambergova, A., et al.: Distribution of CFTR mutations in the Czech population: positive impact of integrated clinical and laboratory expertise, detection of novel/de novo alleles and relevance for related/derived populations. J. Cyst. Fibros. 12, 532–537 (2013). http://linkinghub.elsevier.com/retrieve/pii/S1569-1993(12)00235-4
  21. 21.
    van Gool, K., Norman, R., Delatycki, M.B., Hall, J., Massie, J.: Understanding the costs of care for cystic fibrosis: an analysis by age and health state. Value Health 16, 345–355 (2013). http://www.sciencedirect.com/science/article/pii/S1098301512042684
  22. 22.
    Kerem, E., Conway, S., Elborn, S., Heijerman, H., for the Consensus Committee.: Standards of care for patients with cystic fibrosis: a European consensus. J. Cyst. Fibros. 4, 7–26 (2005). http://www.cysticfibrosisjournal.com/article/S1569-1993(04)00213-9/abstract
  23. 23.
    Yankaskas, R.J., Marshall, R.C., Sufian, B., Simon, H.R., Rodman, D.: Cystic fibrosis adult care: consensus conference report. Chest 125(1Suppl), 1S–39S (2004). http://journal.publications.chestnet.org/article.aspx?articleid=1082187&resultClick=1
  24. 24.
    Heimeshoff, M., Hollmeyer, H., Schreyögg, J., Tiemann, O., Staab, D.: Cost of illness of cystic fibrosis in Germany. Pharmacoeconomics 30, 763–777 (2012). http://link.springer.com/article/10.2165/11588870-000000000-00000#page-1
  25. 25.
    Colombo, C., Daccó, V., Alicandro, G., Loi, S., Mazzi, S., Lucioni, C., Ravasio, R.: Cost of cystic fibrosis: analysis of treatment costs in a specialized center in northern Italy. Adv. Therapy 30, 165–175 (2013). http://link.springer.com/article/10.1007/s12325-013-0008-5#page-1
  26. 26.
    Reid, D.W., Blizzard, C.L., Shugg, D.M., Flowers, C., Cash, C., Greville, M.H.: Changes in cystic fibrosis mortality in Australia, 1979–2005. Med. J. Aust. 195, 392–395 (2011). http://www.mja.com.au/public/issues/195_07_031011/rei11229_fm.html
  27. 27.
    Zemkova, D., Skalicka, V., Barosova, J., Macek, M. Jr., Vavrova, V.: Forty years isolation from W. Europe and its influence on the course of CF: an unwanted experiment. J. Cyst. Fibros. 7, S114 (2008). http://www.cysticfibrosisjournal.com/article/S1569-1993(08)60433-6/abstract
  28. 28.
    The UK Cystic Fibrosis Trust Microbiology Laboratory Standards Working Group. Laboratory standards for processing microbiological samples from people with cystic fibrosis. Cystic Fibrosis Trust, September 2010. https://www.cysticfibrosis.org.uk/media/82034/CD_Laboratory_Standards_Sep_10.pdf
  29. 29.
    ECFS Patient Registry Annual Data Report 2010. European Cystic Fibrosis Society patient registry (ECFSPR). https://www.ecfs.eu/files/webfm/webfiles/File/ecfs_registry/ECFSPR_Report10_v12014_final_020617.pdf
  30. 30.
    Chevreu, K., Brigham, B.K., Michel, M., Rault, G., BURQOL-RD Research Network.: Costs and health-related quality of life of patients with cystic fibrosis and their carers in France. J. Cyst. Fibros. (2015). http://linkinghub.elsevier.com/retrieve/pii/S1569-1993(14)00267-7
  31. 31.
    Purchasing power parities (Auxiliary indicators to National Accounts), Eurostat—http://epp.eurostat.ec.europa.eu/portal/page/portal/national_accounts/data
  32. 32.
    Konstan, M.W., Plant, B.J., Elborn, J.S., Rodriguez, S., Munck, A., Ahrens, R., Johnson, C.: Efficacy response in CF patients treated with ivacaftor: post-hoc analysis. Pediatr. Pulmonol. 50, 447–455 (2015). http://onlinelibrary.wiley.com/doi/10.1002/ppul.23173/abstract

Copyright information

© Springer-Verlag Berlin Heidelberg 2016

Authors and Affiliations

  • Tomáš Mlčoch
    • 1
  • Jiří Klimeš
    • 2
  • Libor Fila
    • 5
  • Věra Vávrová
    • 3
  • Veronika Skalická
    • 3
  • Marek Turnovec
    • 4
  • Veronika Krulišová
    • 4
  • Jitka Jirčíková
    • 1
  • Dana Zemková
    • 3
  • Klára Vilimovská Dědečková
    • 6
  • Alena Bílková
    • 3
  • Vladimíra Frühaufová
    • 7
  • Lukáš Homola
    • 8
  • Zuzana Friedmannová
    • 7
  • Radovan Drnek
    • 7
  • Pavel Dřevínek
    • 6
  • Tomáš Doležal
    • 1
  • Milan MacekJr.
    • 4
  1. 1.Institute of Health Economics and Technological AssessmentPragueCzech Republic
  2. 2.Department of Social and Clinical Pharmacy, Faculty of PharmacyCharles University in PraguePragueCzech Republic
  3. 3.Department of Pediatrics, 2nd Faculty of MedicineCharles University in Prague and University Hospital MotolPragueCzech Republic
  4. 4.Department of Biology and Medical Genetics, 2nd Faculty of MedicineCharles University in Prague and University Hospital MotolPrague 5Czech Republic
  5. 5.Department of Pneumology, 2nd Faculty of MedicineCharles University in Prague and University Hospital MotolPragueCzech Republic
  6. 6.Department of Medical Microbiology, 2nd Faculty of MedicineCharles University in Prague and University Hospital MotolPragueCzech Republic
  7. 7.Division of Health InsuranceUniversity Hospital MotolPragueCzech Republic
  8. 8.Department of Children’s Infectious Diseases, Faculty of Medicine of Masaryk UniversityUniversity HospitalBrnoCzech Republic

Personalised recommendations