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Kwiecień Marcin (Aestimo s.c., Kraków, Poland), Homa Monika (Aestimo s.c., Kraków, Poland), Budasz-Świderska Małgorzata (Roche Polska Sp. z o.o., Warsaw, Poland), Kaczor Marcin (Aestimo s.c., Kraków, Poland ; Uniwersytet Jegielloński Collegium Medicum, Kraków, Poland)
Idiopathic Pulmonary Fibrosis as a Challenge for the Polish Healthcare System
Journal of Health Policy and Outcomes Research, 2015, nr 2, s. 6-17, rys., bibliogr. 101 poz.
Słowa kluczowe
System ochrony zdrowia, Leki
Health care system, Drugs
Idiopathic pulmonary fibrosis (IPF) is a rare disease characterised by age-dependent incidence, unclear aetiology and progressive course. Given the complexity of the diagnostic pathway and limited therapeutic options, and according to the fact that the Polish society is ageing, healthcare and social security expenditure are considerable and likely to rise. Until recently, there was no effective treatment for patients with IPF. After diagnosis only procedures that relieve the symptoms of the disease and treat its complications or lung transplantation were offered. In recent years a huge interest in this clinical entity has been seen. This resulted in the introduction of new drugs, whose efficacy has been confirmed in reliable clinical trials and whose slow the progression of the disease and improve survival of patients. The first such drug was pirfenidone; however such treatment is not available in Poland. Furthermore, due to the inadequacy of Polish legislative reimbursement solutions, which do not take into account the specific nature of rare disease, there are not used any favourable solution during the assessment of public funding of orphan drugs. This is contrary to European consensuses on an egalitarian approach to the use of orphan drugs. It is necessary to develop standards of care for IPF patients, including systemic solutions and the inclusion of the drugs used in the treatment of IPF on the reimbursement list. In long term this will reduce the financial burden associated with the disease, and thus reduce the direct and indirect costs resulting from its prevalence (original abstract)
Pełny tekst
  1. Rowińska-Zakrzewska E, Bestry I. Idiopatyczne włóknienie płuc. Interna Szczeklika. Gajewski P. (ed.), Medycyna Praktyczna, Krakow 2015;729-734
  2. Orphanet Report Series. Rare disease collection. List of rare diseases and synonyms: Listed in alphabetical order. ​Available from:
  3. Demkow U. Immunopathogenesis of idiopathic pulmonary fibrosis. Pneumonol Alergol Pol. 2014; 82(1):55-60; doi: 10.5603/PiAP.2014.0009
  4. Wolters PJ, Collard HR, Jones KD. Pathogenesis of Idiopathic Pulmonary Fibrosis. Annu Rev Pathol. 2014; 9:157-179; doi: 10.1146/annurev-pathol-012513-104706
  5. Belkin A, Swigris JJ. Health-related quality of life in idiopathic pulmonary fibrosis: where are we now? Curr Opin Pulm Med. 2013; 19(5):474-9; doi: 10.1097/MCP.0b013e328363f479
  6. Swigris JJ, Kuschner WG, Jacobs SS, Wilson SR, Gould MK. Health-related quality of life in patients with idiopathic pulmonary fibrosis: a systematic review. Thorax. 2005; 60(7):588-94; doi:10.1136/thx.2004.035220
  7. Bellaye PS, Kolb M. Why do patients get idiopathic pulmonary fibrosis? Current concepts in the pathogenesis of pulmonary fibrosis. BMC Med. 2015; 13:176; doi: 10.1186/s12916-015-0412-6
  8. Tzouvelekis A, Bonella F, Spagnolo P. Update on therapeutic management of idiopathic pulmonary fibrosis. Ther Clin Risk Manag. 2015; 11:359-70; doi: 10.2147/TCRM.S69716. eCollection 2015
  9. Lee AS, Mira-Avendano I, Ryu JH, Daniels CE. The burden of idiopathic pulmonary fibrosis: an unmet public health need. Respir Med. 2014; 108(7):955-67; doi: 10.1016/j.rmed.2014.03.015
  10. Collard HR, Ward AJ, Lanes S, Cortney Hayflinger D, Rosenberg DM, Hunsche E. Burden of illness in idiopathic pulmonary fibrosis. J Med Econ. 2012; 15(5):829-35; doi: 10.3111/13696998.2012.680553
  11. European Respiratory Society. European Lung White Book. Part C. Chapter 22. Interstitial lung disease. ​Available from:
  12. Sergew A, Brown KK. Advances in the treatment of idiopathic pulmonary fibrosis. Expert Opin Emerg Drugs. 2015; 20(4):1-17; doi: 10.1517/14728214.2015.1102886
  13. Spagnolo P, Rossi G, Cavazza A. Pathogenesis of idiopathic pulmonary fibrosis and its clinical implications. Expert Rev. Clin. Immunol. 2014; 10(8):1005-1017; doi:10.1586/1744666X.2014.91705
  14. Demographic profile of the elderly and consequences of the Polish population ageing in light of the forecast for 2014-2050. ​Available from:,18,1.html
  15. Raghu G, Collard HR, Egan JJ, Martinez FJ, et al. An Official ATS/ERS/JRS/ALAT Statement: Idiopathic Pulmonary Fibrosis: Evidence-based Guidelines for Diagnosis and Management. Am. J. Respir. Crit. Care Med. 2011; 183(6):788-824; doi:10.1164/rccm.2009-040GL
  16. Elmufdi F, Henke CA, Perlman DM, Tomic R, Kim HJ. Novel mechanisms and treatment of idiopathic pulmonary fibrosis. Discov Med. 2015 Sep; 20(109):145-53
  17. Behr J, Kreuter M, Hoeper MM et al. Management of patients with idiopathic pulmonary fibrosis in clinical practice: the INSIGHTS-IPF registry. Eur Respir J. 2015; 46(1):186-96; doi: 10.1183/09031936.00217614
  18. Valeyre D. Towards a better diagnosis of idiopathic pulmonary fibrosis. Eur Respir Rev. 2011; 20(120):108-13; doi: 10.1183/09059180.00001611
  19. Spagnolo P, Tonelli R, Cocconcelli E, Stefani A, Richeldi L. Idiopathic pulmonary fibrosis: diagnostic pitfalls and therapeutic challenges. Multidiscip Respir Med. 2012; 7(1):42; doi: 10.1186/2049-6958-7-42
  20. Ryerson CJ, Donesky D, Pantilat SZ, Collard HR. Dyspnea in idiopathic pulmonary fibrosis: a systematic review. J Pain Symptom Manage. 2012; 43(4):771-82; doi: 10.1016/j.jpainsymman.2011.04.026
  21. Ziora D. Samoistne śródmiąższowe zapalenia płuc i zapalenia oskrzelików. Wielka Interna. Pulmonologia. Część II. Wyd. 1. Antczak A (ed.). Medical Tribune Polska, Warsaw 2010; 317-321
  22. Ley B, Harold R. Collard HR, King TE Jr. Clinical Course and Prediction of Survival in Idiopathic Pulmonary Fibrosis Am J Respir Crit Care Med. 2011; 183(4):431-40; doi: 10.1164/rccm.201006-0894CI
  23. Kim HJ, Perlman D, Tomic R. Natural history of idiopathic pulmonary fibrosis. Respir Med. 2015; 109(6):661-70; doi: 10.1016/j.rmed.2015.02.002
  24. Papla B. Śródmiąższowe idiopatyczne zapalenie płuc. Pol J Pathol 2010; 1(1):1323
  25. Hutchinson J, Fogarty A, Hubbard R, McKeever T. Global incidence and mortality of idiopathic pulmonary fibrosis: a systematic review. Eur Respir J. 2015; 46(3):795-806; doi: 10.1183/09031936.00185114
  26. Xaubet A, Ancochea J, Bollo E, Fernández-Fabrellas E, Franquet T, Molina-Molina M et al. A. Normativa sobre el diagnóstico y tratamiento de la fibrosis pulmonar idiopática. Arch. Bronconeumol. 2013; 49(8):343-353; doi:10.1016/j.arbres.2013.03.011
  27. Orphanet Report Series. Rare Diseases collection. Prevalence and incidence of rare diseases: Bibliographic data. Prevalence, incidence or number of published cases listed by diseases (in alphabetical order). Number 1. July 2015. ​Available from:
  28. Caminati A, Madotto F, Cesana G, Conti S, Harari S. Epidemiological studies in idiopathic pulmonary fibrosis: pitfalls in methodologies and data interpretation. Eur Respir Rev. 2015; 24(137):436-44; doi: 10.1183/16000617.0040-2015
  29. EMPIRE (European MultiPartner IPF Registry). ​Available from:
  30. American Thoracic Society. Idiopathic pulmonary fibrosis: diagnosis and treatment. International consensus statement. American Thoracic Society (ATS), and the European Respiratory Society (ERS). Am J Respir Crit Care Med. 2000; 161(2 Pt 1):646-64
  31. Behr J. Prednisone, azathioprine, and N-acetylcysteine for pulmonary fibrosis. N. Engl. J. Med. 2012; 367(9):869; author reply 870-871; doi:10.1056/NEJMc1207471#SA1
  32. Raghu G, Anstrom KJ, King TE Jr, Lasky JA, Martinez FJ. Prednisone, azathioprine, and N-acetylcysteine for pulmonary fibrosis. N Engl J Med 2012; 366:1968-1977; doi:10.1056/NEJMoa1113354
  33. Martinez FJ, de Andrade JA, Anstrom KJ, King TE, Raghu G. Randomized trial of acetylcysteine in idiopathic pulmonary fibrosis. N. Engl. J. Med. 2014; 370(22):2093-2101; doi:10.1056/NEJMoa1401739
  34. Homma S, Sakamoto S, Kawabata M et al. Cyclosporin treatment in steroid-resistant and acutely exacerbated interstitial pneumonia. Intern Med. 2005 Nov; 44(11):1144-50
  35. Raghu G, Brown KK, Bradford WZ et. al. Idiopathic Pulmonary Fibrosis Study Group. A placebo-controlled trial of interferon gamma-1b in patients with idiopathic pulmonary fibrosis. N. Engl. J. Med. 2004; 350(2):125-133; doi:10.1056/NEJMoa030511
  36. King TE, Albera C, Bradford WZ et. al. Effect of interferon gamma-1b on survival in patients with idiopathic pulmonary fibrosis (INSPIRE): a multicentre, randomised, placebo-controlled trial. Lancet 2009; 374(9685):222-228; doi:10.1016/S0140-6736(09)60551-1
  37. Raghu G, Brown KK, Costabel U et al.Treatment of idiopathic pulmonary fibrosis with etanercept: an exploratory, placebo-controlled trial. Am J Respir Crit Care Med 2008; 178:948-955; doi: 10.1164/rccm.200709-1446OC
  38. Raghu G, Rochwerg B, Zhang B et al. An Official ATS/ERS/JRS/ALAT Clinical Practice Guide-line: Treatment of Idiopathic Pulmonary Fibrosis. An Update of the 2011 Clinical Practice Guideline. American Thoracic Society Documents. Am J Respir Crit Care Med. 2015 Jul 15; 192(2):e3-19; doi: 10.1164/rccm.201506-1063ST
  39. Richeldi L. Clinical trials of investigational agents for IPF: a review of a Cochrane report. Respir Res. 2013; 14(Suppl 1):S4; doi: 10.1186/1465-9921-14-S1-S4
  40. Behr J, Günther A, Ammenwerth W et al. S2K-Leitlinie zur Diagnostik und Therapie der idiopathischen Lungenfibrose. Pneumologie 2013; 67(02):81-111; doi:10.1055/s-0032-1326009
  41. Cottin V, Crestani B, Valeyre D et al. Diagnosis and management of idiopathic pulmonary fibrosis: French practical guidelines. Eur. Respir. Rev. 2014; 23(132):193-214; doi:10.1183/09059180.00001814
  42. Myllärniemi M, Kaarteenaho R. Pharmacological treatment of idiopathic pulmonary fibrosis - preclinical and clinical studies of pirfenidone, nintedanib, and N-acetylcysteine. Eur Clin Respir J. 2015; 2. doi: 10.3402/ecrj.v2.26385
  43. Summary of Product Characteristics for Esbriet®. ​Available from:
  44. Summary of Product Characteristics Ofev®. ​Available from:
  45. King Jr TE, Bradford WZ, Castro-Bernardini S et al. A phase 3 trial of pirfenidone in patients with idiopathic pulmonary fibrosis. New Engl J Med. 2014; 370(22):2083-2092; doi: 10.1056/NEJMoa1402582
  46. Noble PW, Albera C, Bradford WZ et al. Pirfenidone in patients with idiopathic pulmonary fibrosis (CAPACITY): Two randomised trials. Lancet. 2011; 377(9779):1760-1769; doi: 10.1016/S0140-6736(11)60405-4
  47. Azuma A, Nukiwa T, Tsuboi E et al. Double-blind, placebo-controlled trial of pirfenidone in patients with idiopathic pulmonary fibrosis. Am J Respir Crit Care Med. 2005; 171(9):1040-1047
  48. Taniguchi H, Ebina M, Kondoh Y et al. Pirfenidone in idiopathic pulmonary fibrosis. Eur Respir J. 2010; 35(4):821-82; doi: 10.1183/09031936.00005209
  49. Kolb M, Collard HR. Staging of idiopathic pulmonary fibrosis: past, present and future. Eur. Respir. Rev. 2014; 23(132):220-224. doi:10.1183/09059180.00002114
  50. Homma S, Sugino K, Sakamoto S. The usefulness of a disease severity staging classification system for IPF in Japan: 20 years of experience from empirical evidence to randomized control trial enrollment. Respir. Investig. 2015; 53(1):7-12; doi:10.1016/j.resinv.2014.08.003
  51. Richeldi L, Costabel U, Selman M et al. Efficacy of a tyrosine kinase inhibitor in idiopathic pulmonary fibrosis. N Engl J Med. 2011; 365(12):1079-87; doi: 10.1056/NEJMoa1103690
  52. Richeldi L, du Bois RM, Raghu G et al. Efficacy and safety of nintedanib in idiopathic pulmonary fibrosis. N Engl J Med. 2014 May 29; 370(22):2071-82; doi: 10.1056/NEJMoa1402584
  53. Ogura T, Taniguchi H, Azuma A et al. Safety and pharmacokinetics of nintedanib and pirfenidone in idiopathic pulmonary fibrosis. Eur Respir J. 2015; 45(5):1382-92; doi: 10.1183/09031936.00198013
  54. Biała Księga. Samoistne włóknienie płuc - aspekty kliniczne, ekonomiczne i systemowe ze szczególnym uwzględnieniem rekomendacji dotyczących optymalnego zarządzania chorobą. Gałązka-Sobotka M, Gierczyński J (ed.). Łazarski University, Warsaw, 2015
  55. Lamas DJ, Kawut SM, Bagiella E et al. Delayed Access and Survival in Idiopathic Pulmonary Fibrosis; Am J Respir Crit Care Med. 2011; 184(7): 842-847; doi: 10.1164/rccm.201104-0668OC
  56. Loveman E, Copley VR, Colquitt J et al. The clinical effectiveness and cost-effectiveness of treatments for idiopathic pulmonary fibrosis: a systematic review and economic evaluation. Health Technol Assess. 2015; 19(20):i-xxiv, 1-336; doi: 10.3310/hta19200
  57. National Institute for Health and Care Excellence. ​Available from:
  58. Canadian Agency for Drugs and Technologies in Health. Pirfenidone. ​Available from:
  59. Canadian Agency for Drugs and Technologies in Health. Nintedanib. ​Available from:
  60. Haute Autorité de Santé. Pirfenidone. ​Available from:
  61. Haute Autorité de Santé. Nintedanib. ​Available from:
  62. Scottish Medicines Consortium. Pirfenidone. ​Available from:
  63. Scottish Medicines Consortium. Nintedanib. ​Available from:
  64. All Wales Medicines Strategy Group. ​Available from:
  65. Pharmaceutical Benefits Scheme. Nintedanib. ​Available from:
  66. Principality of Lichtenstein. Ministry for Social Affairs. ​Available from:
  67. Grand-Duché de Luxembourg. Ministre De La Santé. ​Available from:
  68. Ministry of Health and Care Services Norway. ​Available from:
  69. Federal Office of Public Health Switzerland. ​Available from:
  70. Denmark Ministry of Health. ​Available from:
  71. Sweden. Ministry of Health and Social Affairs. ​Available from:
  72. Ireland. Department of Health. ​Available from:
  73. Iceland. Ministry of Welfare. ​Available from:
  74. Netherlands. Ministry of Health, Welfare and Sport. ​Available from:
  75. Austria. Federal Ministry of Health. ​Available from:
  76. Finland. Ministry of Social Affairs and Health. ​Available from:
  77. Germany. Federal Ministry of Health. ​Available from:
  78. Belgium. Federal Public Service. Health, Food Chain Safety and Environment. ​Available from:
  79. Great Britain. Department of Health. ​Available from:
  80. France. Ministère des Affaires sociales, de la Santé et des Droits des femmes. ​Available from:
  81. Italy. Ministerio della Salute. ​Available from:
  82. Spain. Ministerio de Sanidad, Servicios Sociales e Igualdad. ​Available from:
  83. Cyprus. Ministry of Health. ​Available from:
  84. Republic of Slovenia. Ministry of Health. ​Available from:
  85. Malta. Ministry of Health. ​Available from:
  86. Portugal. Ministério da Saúde. ​Available from:
  87. Greece. Ministry of Health. ​Available from:
  88. Estonia. Sotsiaalministeerium. ​Available from:
  89. Czech Republic Ministry of Health. ​Available from:
  90. Ministerstvo zdravotníctva Slovenskej republiky. ​Available from:
  91. Ministry of Health of The Republic of Lithuania. Available from:
  92. Latvia Ministry of Health. ​Available from:
  93. Poland. Ministry of Health. ​Available from:
  94. Hungary. Ministry of Health. ​Available from:
  95. Ministry of Health of the Republic of Croatia. ​Available from:
  96. Romania. Ministerul Sanatatii. ​Available from:
  97. Bulgaria. Ministry of Health. ​Available from:
  98. Application for reimbursement of Ofev and setting its official price. Verification Analysis. AOTMiT. Warsaw 2015. ​Available from:
  99. The World Bank. GDP per capita. ​Available from:
  100. Position of the Parliamentary Group on Rare Diseases and the Parliamentary Group on Oncology on the criterion of the threshold for the cost of gaining an additional quality-adjusted life year and applying the results of classic economic analysis of the profitability threshold - 9 September 2014. ​Available from:
  101. Systemic assumptions in the development of the NATIONAL RARE DISEASES PLAN for 2013-2017. Available from:
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