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Published in: Applied Health Economics and Health Policy 2/2016

01-04-2016 | Scoping Review

A Scoping Review of Healthcare Costs for Patients with Cystic Fibrosis

Authors: Ilene L. Hollin, Karen A. Robinson

Published in: Applied Health Economics and Health Policy | Issue 2/2016

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Abstract

Background

Cystic fibrosis (CF), one of the most common life-shortening genetic diseases, has no cure, but people living with it have seen improvements in their health and survival. The rising life expectancy and increased availability of treatment options has likely increased the lifetime costs of people living with CF. In addition, a recent drug approval for a therapy that targets the cause of the disease is one of the most expensive drugs worldwide. In light of these circumstances, it is important to have an updated understanding of the costs of CF therapy and management. This study aims to determine the extent of available literature that quantifies CF costs.

Methods

We used a scoping review framework to identify the sources and types of evidence available to determine the costs of CF therapy and management compared to the general population or a comparable population of people with other complex chronic conditions. We searched 14 databases for peer-reviewed studies and grey literature published in English since 1998. The search was conducted in August 2013 and updated in October 2014.

Results

We identified 28 studies that estimated overall, general CF costs. Of these, three studies compare CF costs to healthcare costs of a general population and only one of those provides a direct comparison of CF costs to the general population in order to calculate the incremental cost associated with CF. We estimate there are 98 systematic reviews that quantify the costs of comparable conditions and potentially provide a comparison group for people with CF.

Conclusions

There is evidence available that attempts to quantify overall, general healthcare costs of people with CF, although much of it is outdated. However, there is much less evidence available that provides a comparison of these costs with either the general population or people with comparable conditions.
Appendix
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Literature
1.
go back to reference Cystic Fibrosis Foundation. Cystic Fibrosis Foundation Patient Registry 2012 Annual Data Report. Bethesda, MD; 2013. Cystic Fibrosis Foundation. Cystic Fibrosis Foundation Patient Registry 2012 Annual Data Report. Bethesda, MD; 2013.
2.
go back to reference Cohen-Cymberknoh M, Shoseyov D, Kerem E. Managing cystic fibrosis: strategies that increase life expectancy and improve quality of life. Am J Respir Crit Care Med. 2011;183:1463–71.CrossRefPubMed Cohen-Cymberknoh M, Shoseyov D, Kerem E. Managing cystic fibrosis: strategies that increase life expectancy and improve quality of life. Am J Respir Crit Care Med. 2011;183:1463–71.CrossRefPubMed
4.
go back to reference Arksey H, O’Malley L. Scoping studies: towards a methodological framework. Int J Soc Res Methodol. 2005;8(1):19–32.CrossRef Arksey H, O’Malley L. Scoping studies: towards a methodological framework. Int J Soc Res Methodol. 2005;8(1):19–32.CrossRef
5.
go back to reference Colquhoun H, Levac D, O’Brien KK, Straus S, Tricco AC, Perrier L, et al. Scoping reviews: time for clarity in definition, methods, and reporting. J Clin Epidemiol. 2014;67(12):1291–4.CrossRefPubMed Colquhoun H, Levac D, O’Brien KK, Straus S, Tricco AC, Perrier L, et al. Scoping reviews: time for clarity in definition, methods, and reporting. J Clin Epidemiol. 2014;67(12):1291–4.CrossRefPubMed
7.
go back to reference Davidoff AJ. Insurance for children with special health care needs: patterns of coverage and burden of families to provide adequate insurance. Pediatrics. 2004;114(2):394–402.CrossRefPubMed Davidoff AJ. Insurance for children with special health care needs: patterns of coverage and burden of families to provide adequate insurance. Pediatrics. 2004;114(2):394–402.CrossRefPubMed
8.
go back to reference Druss BG, Marcus SC, Olfson M, Tanielian T, Elinson L, Pincus HA. Comparing the national economic burden of five chronic conditions. Health Aff. 2001;20(6):233–41.CrossRef Druss BG, Marcus SC, Olfson M, Tanielian T, Elinson L, Pincus HA. Comparing the national economic burden of five chronic conditions. Health Aff. 2001;20(6):233–41.CrossRef
9.
go back to reference Health Resources and Services Administration, Maternal and Child Health Bureau Conducted by the National Center for Health Statistics, Centers for Disease Control and Prevention. (2009–2010) National survey of children with special health care needs. Questionnaire Version: December 14, 2011. Health Resources and Services Administration, Maternal and Child Health Bureau Conducted by the National Center for Health Statistics, Centers for Disease Control and Prevention. (2009–2010) National survey of children with special health care needs. Questionnaire Version: December 14, 2011.
10.
go back to reference Chen AY, Newacheck PW. Insurance coverage and financial burden for families of children with special health care needs. Ambul Pediatr. 2006;6(4):204–9.CrossRefPubMedPubMedCentral Chen AY, Newacheck PW. Insurance coverage and financial burden for families of children with special health care needs. Ambul Pediatr. 2006;6(4):204–9.CrossRefPubMedPubMedCentral
11.
go back to reference Ramsey BW, Pepe MS, Quan JM, Otto KL, Montgomery AB, Williams-Warren J, et al. Intermittent administration of inhaled tobramycin in patients with cystic fibrosis. Cystic Fibrosis Inhaled Tobramycin Study Group. NEJM. 1999;340(1):23–30.CrossRefPubMed Ramsey BW, Pepe MS, Quan JM, Otto KL, Montgomery AB, Williams-Warren J, et al. Intermittent administration of inhaled tobramycin in patients with cystic fibrosis. Cystic Fibrosis Inhaled Tobramycin Study Group. NEJM. 1999;340(1):23–30.CrossRefPubMed
12.
go back to reference Woodward TC, Brown R, Sacco P, Zhang J. Budget impact model of tobramycin inhalation solution for treatment of Pseudomonas aeruginosa in cystic fibrosis patients. J Med Econ. 2010;13(3):492–9.CrossRefPubMed Woodward TC, Brown R, Sacco P, Zhang J. Budget impact model of tobramycin inhalation solution for treatment of Pseudomonas aeruginosa in cystic fibrosis patients. J Med Econ. 2010;13(3):492–9.CrossRefPubMed
13.
go back to reference Colombo C, Dacco V, Loi S, Anelli M, Alicandro G. Analysis of cost drivers for health care services use in cystic fibrosis patients: the experience of the Milan cystic fibrosis center. J Cyst Fibros. 2014;13:S104.CrossRef Colombo C, Dacco V, Loi S, Anelli M, Alicandro G. Analysis of cost drivers for health care services use in cystic fibrosis patients: the experience of the Milan cystic fibrosis center. J Cyst Fibros. 2014;13:S104.CrossRef
14.
go back to reference Strong M, Harr B. Burden of disease and out-of-pocket costs associated with CF for patients and caregivers in the United States. Pediatr Pulmonol. 2010;45:447–8. Strong M, Harr B. Burden of disease and out-of-pocket costs associated with CF for patients and caregivers in the United States. Pediatr Pulmonol. 2010;45:447–8.
15.
go back to reference Krauth C, Jalilvand N, Welte T, Busse R. Cystic fibrosis: cost of illness and considerations for the economic evaluation of potential therapies. Pharmacoeconomics. 2003;21(14):1001–24.CrossRefPubMed Krauth C, Jalilvand N, Welte T, Busse R. Cystic fibrosis: cost of illness and considerations for the economic evaluation of potential therapies. Pharmacoeconomics. 2003;21(14):1001–24.CrossRefPubMed
16.
go back to reference Balinsky W, Zhu CW. Pediatric cystic fibrosis: evaluating costs and genetic testing. J Pediatr Health Care. 2004;18(1):30–4.CrossRefPubMed Balinsky W, Zhu CW. Pediatric cystic fibrosis: evaluating costs and genetic testing. J Pediatr Health Care. 2004;18(1):30–4.CrossRefPubMed
17.
go back to reference Lieu TA, Ray GT, Farmer G, Shay GF. The cost of medical care for patients with cystic fibrosis in a health maintenance organization. Pediatrics. 1999;103(6):e72.CrossRefPubMed Lieu TA, Ray GT, Farmer G, Shay GF. The cost of medical care for patients with cystic fibrosis in a health maintenance organization. Pediatrics. 1999;103(6):e72.CrossRefPubMed
18.
go back to reference Ouyang L, Grosse SD, Amendah DD, Schechter MS. Healthcare expenditures for privately insured people with cystic fibrosis. Pediatr Pulmonol. 2009;44(10):989–96.CrossRefPubMed Ouyang L, Grosse SD, Amendah DD, Schechter MS. Healthcare expenditures for privately insured people with cystic fibrosis. Pediatr Pulmonol. 2009;44(10):989–96.CrossRefPubMed
19.
go back to reference Baumann U, Stocklossa C, Greiner W, von der Schulenburg JM, von der Hardt H. Cost of care and clinical condition in paediatric cystic fibrosis patients. J Cyst Fibros. 2003;2(2):84–90.CrossRefPubMed Baumann U, Stocklossa C, Greiner W, von der Schulenburg JM, von der Hardt H. Cost of care and clinical condition in paediatric cystic fibrosis patients. J Cyst Fibros. 2003;2(2):84–90.CrossRefPubMed
20.
go back to reference Becker CC, Clements K, DeLong K, Harrow B, O’Sullivan A. Economic burden of cystic fibrosis in the US: costs of care by disease severity and age. Value Health. 2011;14(7):A490.CrossRef Becker CC, Clements K, DeLong K, Harrow B, O’Sullivan A. Economic burden of cystic fibrosis in the US: costs of care by disease severity and age. Value Health. 2011;14(7):A490.CrossRef
21.
go back to reference Briesacher BA, Quittner AL, Fouayzi H, Zhang J, Swensen A. Nationwide trends in the medical care costs of privately insured patients with cystic fibrosis (CF), 2001–2007. Pediatr Pulmonol. 2011;46(8):770–6.CrossRefPubMed Briesacher BA, Quittner AL, Fouayzi H, Zhang J, Swensen A. Nationwide trends in the medical care costs of privately insured patients with cystic fibrosis (CF), 2001–2007. Pediatr Pulmonol. 2011;46(8):770–6.CrossRefPubMed
22.
go back to reference Broder MS, Nasr SZ, Chang E, Villa KF. Utilization and healthcare charges in an insured US cystic fibrosis (CF) patient population. J Cyst Fibros. 2011;10:S97.CrossRef Broder MS, Nasr SZ, Chang E, Villa KF. Utilization and healthcare charges in an insured US cystic fibrosis (CF) patient population. J Cyst Fibros. 2011;10:S97.CrossRef
23.
go back to reference Colombo C, Daccò V, Alicandro G, Loi S, Mazzi S, Lucioni C, et al. Cost of cystic fibrosis: analysis of treatment costs in a specialized center in Northern Italy. Adv Ther. 2013;30(2):165–75.CrossRefPubMed Colombo C, Daccò V, Alicandro G, Loi S, Mazzi S, Lucioni C, et al. Cost of cystic fibrosis: analysis of treatment costs in a specialized center in Northern Italy. Adv Ther. 2013;30(2):165–75.CrossRefPubMed
24.
go back to reference Dewitt EM, Grussemeyer CA, Friedman JY, Dinan MA, Lin L, Schulman KA, Reed SD. Resource use, costs, and utility estimates for patients with cystic fibrosis with mild impairment in lung function: analysis of data collected alongside a 48-week multicenter clinical trial. Value Health. 2012;15(2):277–83.CrossRefPubMed Dewitt EM, Grussemeyer CA, Friedman JY, Dinan MA, Lin L, Schulman KA, Reed SD. Resource use, costs, and utility estimates for patients with cystic fibrosis with mild impairment in lung function: analysis of data collected alongside a 48-week multicenter clinical trial. Value Health. 2012;15(2):277–83.CrossRefPubMed
25.
go back to reference Eakin MN, Bilderback A, Ridge A, Quittner AL, Zhang J, Riekert KA. Out-of-pocket costs for medications in the I change adherence and raise expectations (iCARE) study. Pediatr Pulmonol. 2011;46:420. Eakin MN, Bilderback A, Ridge A, Quittner AL, Zhang J, Riekert KA. Out-of-pocket costs for medications in the I change adherence and raise expectations (iCARE) study. Pediatr Pulmonol. 2011;46:420.
26.
go back to reference Gu Y, Garcia-Perez 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. 2015;16(7):709–17.CrossRefPubMed Gu Y, Garcia-Perez 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. 2015;16(7):709–17.CrossRefPubMed
27.
go back to reference Harrow B, Becker CC, Buikema A. High cost cystic fibrosis patients as identified in a us claims database: a closer look at the tail. Value Health. 2011;14(7):A491.CrossRef Harrow B, Becker CC, Buikema A. High cost cystic fibrosis patients as identified in a us claims database: a closer look at the tail. Value Health. 2011;14(7):A491.CrossRef
28.
go back to reference Heimeshoff M, Hollmeyer H, Schreyogg J, Tiemann O, Staab D. Cost of illness of cystic fibrosis in Germany: results from a large cystic fibrosis centre. Pharmacoeconomics. 2012;30(9):763–77.CrossRefPubMed Heimeshoff M, Hollmeyer H, Schreyogg J, Tiemann O, Staab D. Cost of illness of cystic fibrosis in Germany: results from a large cystic fibrosis centre. Pharmacoeconomics. 2012;30(9):763–77.CrossRefPubMed
29.
go back to reference Huot L, Durieu I, Bourdy S, Ganne C, Bellon G, Colin C, et al. Evolution of costs of care for cystic fibrosis patients after clinical guidelines implementation in a French network. J Cyst Fibros. 2008;7(5):403–8.CrossRefPubMed Huot L, Durieu I, Bourdy S, Ganne C, Bellon G, Colin C, et al. Evolution of costs of care for cystic fibrosis patients after clinical guidelines implementation in a French network. J Cyst Fibros. 2008;7(5):403–8.CrossRefPubMed
30.
go back to reference Jarrett J, Lies R, Mugford M, Legh-Smith J. The cost of paediatric cystic fibrosis to the NHS in the Eastern Region. J Cyst Fibros. 2008;7:S117.CrossRef Jarrett J, Lies R, Mugford M, Legh-Smith J. The cost of paediatric cystic fibrosis to the NHS in the Eastern Region. J Cyst Fibros. 2008;7:S117.CrossRef
31.
go back to reference Klimes J, Dolezal T, Vavrova V, Turnovec M, Skalicka V, Mlcoch T. Economic burden of cystic fibrosis (CF): prevalence based cost of illness analysis related to the lung disease severity in Czech CF patients. J Cyst Fibros. 2014;13:S11.CrossRef Klimes J, Dolezal T, Vavrova V, Turnovec M, Skalicka V, Mlcoch T. Economic burden of cystic fibrosis (CF): prevalence based cost of illness analysis related to the lung disease severity in Czech CF patients. J Cyst Fibros. 2014;13:S11.CrossRef
32.
go back to reference Levy J, Rosenberg M, Farrell PM. The direct medical costs of pediatric cystic fibrosis. Pediatr Pulmonol. 2013;48:375. Levy J, Rosenberg M, Farrell PM. The direct medical costs of pediatric cystic fibrosis. Pediatr Pulmonol. 2013;48:375.
33.
go back to reference O’Sullivan A, Sullivan J, Higuchi K, Montgomery A. Health care utilization and costs for cystic fibrosis patients with pulmonary infections. Manag Care. 2011;20(2):37–44.PubMed O’Sullivan A, Sullivan J, Higuchi K, Montgomery A. Health care utilization and costs for cystic fibrosis patients with pulmonary infections. Manag Care. 2011;20(2):37–44.PubMed
34.
go back to reference Okumura MJ, Campbell AD, Nasr SZ, Davis MM. Inpatient health care use among adult survivors of chronic childhood illnesses in the United States. Arch Pediatr Adolesc Med. 2006;160(10):1054–60.CrossRefPubMed Okumura MJ, Campbell AD, Nasr SZ, Davis MM. Inpatient health care use among adult survivors of chronic childhood illnesses in the United States. Arch Pediatr Adolesc Med. 2006;160(10):1054–60.CrossRefPubMed
35.
go back to reference Rosenberg MA, Farrell PM. Assessing the cost of cystic fibrosis diagnosis and treatment. J Pediatr. 2005;147(3 Suppl):S101–5.CrossRefPubMed Rosenberg MA, Farrell PM. Assessing the cost of cystic fibrosis diagnosis and treatment. J Pediatr. 2005;147(3 Suppl):S101–5.CrossRefPubMed
36.
go back to reference Sansgiry S, Joish V, Boklage S, Goyal RK, Seal B, Sethi S. Pseudomonas aeruginosa related economic burden stratified by comorbidity in cystic fibrosis patients. Value Health. 2010;13(7):A433.CrossRef Sansgiry S, Joish V, Boklage S, Goyal RK, Seal B, Sethi S. Pseudomonas aeruginosa related economic burden stratified by comorbidity in cystic fibrosis patients. Value Health. 2010;13(7):A433.CrossRef
37.
go back to reference Sansgiry SS, Joish V, Boklage S, Goyal RK, Sethi S. Impact of pseudomonas aeruginosa infections on direct medical costs among cystic fibrosis patients. Pediatr Pulmonol. 2010;45:451. Sansgiry SS, Joish V, Boklage S, Goyal RK, Sethi S. Impact of pseudomonas aeruginosa infections on direct medical costs among cystic fibrosis patients. Pediatr Pulmonol. 2010;45:451.
38.
go back to reference Sansgiry SS, Joish VN, Boklage S, Goyal RK, Chopra P, Sethi S. Economic burden of Pseudomonas aeruginosa infection in patients with cystic fibrosis. J Med Econ. 2012;15(2):219–24.CrossRefPubMed Sansgiry SS, Joish VN, Boklage S, Goyal RK, Chopra P, Sethi S. Economic burden of Pseudomonas aeruginosa infection in patients with cystic fibrosis. J Med Econ. 2012;15(2):219–24.CrossRefPubMed
39.
go back to reference Schwerner H, Mellody T, Goldstein AB, Wansink D, Sullivan V, Yelenik SN, et al. Evaluating the impact of a disease management program for chronic complex conditions at two large northeast health plans using a control group methodology. Dis Manag. 2006;9(1):34–44.CrossRefPubMed Schwerner H, Mellody T, Goldstein AB, Wansink D, Sullivan V, Yelenik SN, et al. Evaluating the impact of a disease management program for chronic complex conditions at two large northeast health plans using a control group methodology. Dis Manag. 2006;9(1):34–44.CrossRefPubMed
40.
go back to reference van Gool K, Norman R, Delatycki MB, Hall J, Massie J. Understanding the costs of care for cystic fibrosis: an analysis by age and health state. Value Health. 2013;16(2):345–55.CrossRefPubMed van Gool K, Norman R, Delatycki MB, Hall J, Massie J. Understanding the costs of care for cystic fibrosis: an analysis by age and health state. Value Health. 2013;16(2):345–55.CrossRefPubMed
41.
go back to reference Zhang J, Marynchenko M, Chopra P, Ohashi E, Signorovitch J, Wu E. Annual healthcare costs for patients with cystic fibrosis from a medicaid perspective. Pediatr Pulmonol. 2012;47:443–4. Zhang J, Marynchenko M, Chopra P, Ohashi E, Signorovitch J, Wu E. Annual healthcare costs for patients with cystic fibrosis from a medicaid perspective. Pediatr Pulmonol. 2012;47:443–4.
42.
go back to reference Johnson JA, Connolly MA, Jacobs P, Montgomery M, Brown NE, Zuberbuhler P. Cost of care for individuals with cystic fibrosis: a regression approach to determining the impact of recombinant human DNase. Pharmacotherapy. 1999;19(10):1159–66.CrossRefPubMed Johnson JA, Connolly MA, Jacobs P, Montgomery M, Brown NE, Zuberbuhler P. Cost of care for individuals with cystic fibrosis: a regression approach to determining the impact of recombinant human DNase. Pharmacotherapy. 1999;19(10):1159–66.CrossRefPubMed
43.
go back to reference Cystic Fibrosis Foundation Patient Registry 2013 Annual Data Report to the Center Directors. In. Bethesda, MD: Cystic Fibrosis Foundation; 2014. Cystic Fibrosis Foundation Patient Registry 2013 Annual Data Report to the Center Directors. In. Bethesda, MD: Cystic Fibrosis Foundation; 2014.
Metadata
Title
A Scoping Review of Healthcare Costs for Patients with Cystic Fibrosis
Authors
Ilene L. Hollin
Karen A. Robinson
Publication date
01-04-2016
Publisher
Springer International Publishing
Published in
Applied Health Economics and Health Policy / Issue 2/2016
Print ISSN: 1175-5652
Electronic ISSN: 1179-1896
DOI
https://doi.org/10.1007/s40258-015-0211-4

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