Исторически преглед на дефиницията за редки тумори в контекста на здравните политики
Keywords:
редки тумори, здравни политики, редки болести, епидемиология, рак
Abstract
Злокачествени заболявания като събирателна група от нозологични единици представляват значим социален и здравен проблем. Въпреки това, разглеждането на всички видове рак в една категория при изграждането на здравни политики е поставено под критика. Поради клиничните и епидемиологичните си характеристики, пациентите с редки тумори срещат в по-голяма степен бариери в достъпа си до иновативни медикаменти, съчетано с липса на експертиза в диагностично-лечебния процес. Изграждането на самостоятелни политики за тези пациенти изисква точна дефиниция на понятието.
Настоящият обзор цели да представи хронологичното развитие при дефинирането на понятието за редки тумори в контекста на изграждането на здравни политики в тази област. За целите на обзора е осъществено търсене по ключови думи „rare cancers”, „rare tumors” и „rare neoplasm” в базите данни PubMed/MEDLINE и ScienceDirect и Google Scholar. Откритите статии за организирани в три основни контекста на използване на термина „редки тумори“ – клинично базирани дефиниции; дефиниции, базирани на епидемиологични критерии и дефиниции, използвани в детската онкология.
References
WHO. Cancer - definition. 2022. Available: https://www.who.int/health-topics/cancer
WHO. Global health estimates: Leading causes of death. World Health Organization. 2020. Available: https://www.who.int/data/gho/data/themes/mortality-and-global-health-estimates/ghe-leading-causes-of-death
Conceição P et al. Human development report. 2019: Beyond income, beyond averages, beyond today: Inequalities in human development in the 21st century. 2019. Available: https://hdr.undp.org/content/human-development-report-2019
Hofmarcher T, Lindgren P, Wilking N, et al. The cost of cancer in Europe 2018. Eur J Cancer. 2020 Apr;129:41-49.
НЦОЗА. Заболеваемост на населението. 2021. Available: https://ncpha.government.bg/index/3032-zabolevaemost-na-naselenieto.html
НСИ. Deaths by causes and mortality by causes. 2021. Available: https://nsi.bg/en/content/3348/deaths-causes-and-mortality-causes
Cree IA, Indave BI. Commentary: Cancer research quality and tumour classification. Tumour Biol. 2020 Feb;42(2):1010428320907544.
Jackson TJ, Michel JL, Roberts R, et al. Development of a validation algorithm for 'present on admission' flagging. BMC Med Inform Decis Mak. 2009 Dec 1;9:48.
Arnold M, Karim-Kos HE, Coebergh JW, et al. Recent trends in incidence of five common cancers in 26 European countries since 1988: Analysis of the European Cancer Observatory. Eur J Cancer. 2015 Jun;51(9):1164-87.
Maughan KL, Lutterbie MA, Ham PS. Treatment of breast cancer. Am Fam Physician. 2010 Jun 1;81(11):1339-46.
Ettinger DS, Wood DE, Aggarwal C, et al. NCCN Guidelines Insights: Non-Small Cell Lung Cancer, Version 1.2020. J Natl Compr Canc Netw. 2019 Dec;17(12):1464-1472.
Peairs KS, Choi Y, Stewart RW, et al. Screening for breast cancer. Semin Oncol. 2017 Feb;44(1):60-72.
Hoffman RM, Sanchez R. Lung Cancer Screening. Med Clin North Am. 2017 Jul;101(4):769-785.
Shenoy SR, Dey B. Funding for cancer research by an Indian funding agency, DBT. J Biosci. 2021;46:2.
Begum M, Lewison G, Lawler M, et al. Mapping the European cancer research landscape: An evidence base for national and Pan-European research and funding. Eur J Cancer. 2018 Sep;100:75-84.
Griggs RC, Batshaw M, Dunkle M, et al; Rare Diseases Clinical Research Network. Clinical research for rare disease: opportunities, challenges, and solutions. Mol Genet Metab. 2009 Jan;96(1):20-6.
Andrews EB, Gilsenan A, Midkiff K, et al. Challenges in studying very rare cancer outcomes and infrequent exposures: example of teriparatide and osteosarcoma. Ann Epidemiol. 2016 Nov;26(11):751-753.
Искров Г, Стефанов Р. Редките тумори – епидемиологични и общественоздравни аспекти. Редки болести и лекарства сираци. 2018;9: 9–13.
Kasper B. The challenge of drug approval in rare cancers. Cancer. 2021 Mar 15;127(6):837-839.
Gatta G, van der Zwan JM, Casali PG, et al; RARECARE working group. Rare cancers are not so rare: the rare cancer burden in Europe. Eur J Cancer. 2011 Nov;47(17):2493-511.
European parlament and the Concil. Europe’s beating cancer plan. 2022. Available: https://health.ec.europa.eu/system/files/2022-02/eu_cancer-plan_en_0.pdf
Танев Т. Анализ на публичните политики - науката за създаване на конкретни политики. Военно издателство ЕООД; 2008.
Chabner BA, Friedman MA. Progress against rare and not-so-rare cancers. N Engl J Med. 1992 Feb 20;326(8):563-5.
Adams EE. Rare Cancers in Persons Exposed to Internal Radium. JAMA. 1982;247(20):2778–2779.
Sikora K, Smedley H. Rare cancers and specialist centers. Br Med J (Clin Res Ed). 1986 Mar 29;292(6524):898.
Wilson L. PP70. The costs of pancreatic cancer: Methodology of costing rare cancers. Eur J Cancer. 1997;33: S40.
Patel A, Ozsahin M, Mirimanoff RO, et al. The Rare Cancer Network: achievements from 1993 to 2012. Rare Tumors. 2012 Jun 26;4(3):e35.
Mirimanoff RO, Ozsahin M, Thariat J, et al. History of the rare cancer network and past research. Rare Tumors. 2014 Aug 6;6(3):5462.
97th United States Congress. Orphan drug act. FDA; 2014. Available: https://www.fda.gov/media/99546
European Parlament and the Council of Eropean union regulation 141/2000. 2000.
Very rare cancers--a problem neglected. Lancet Oncol. 2001 Apr;2(4):189.
European cancer information system. ECIS; 2022. Available: https://ecis.jrc.ec.europa.eu/info/initiatives.html
International agency for research on cancer (IARC). Available: https://www.iarc.who.int/
European Commision. ENCR | european network of cancer registries. Available: https://encr.eu/
International association of cancer registries IACR. Available: http://www.iacr.com.fr/
Berrino F, Sant M, Verdecchia A, Capocaccia R, Hakulinen T. Survival of cancer patients in europe. Cancer Causes and Control. 1995;6: 469–470. doi:10.1007/bf00052188
De Angelis R, Francisci S, Baili P, et al; EUROCARE Working Group. The EUROCARE-4 database on cancer survival in Europe: data standardisation, quality control and methods of statistical analysis. Eur J Cancer. 2009 Apr;45(6):909-30.
Gatta G, Ciccolallo L, Kunkler I, et al; EUROCARE Working Group. Survival from rare cancer in adults: a population-based study. Lancet Oncol. 2006 Feb;7(2):132-40.
Maule M, Merletti F, Mirabelli D, et al. Spatial variation of mortality for common and rare cancers in Piedmont, Italy, from 1980 to 2000: a Bayesian approach. Eur J Cancer Prev. 2006 Apr;15(2):108-16.
Marty M, Rheims N. Cancers rares: Des entités différentes, des problématiques communes. Oncologie. 2008;10: 360–362.
Eng C. Mendelian genetics of rare--and not so rare--cancers. Ann N Y Acad Sci. 2010 Dec;1214:70-82.
Griffiths J, Willard C, Burgess A, et al. Meeting the ongoing needs of survivors of rarer cancer. Eur J Oncol Nurs. 2007 Dec;11(5):434-41.
Executive Agency for Health and Consumers (EAHC) of the European Commission. RARECARE - surveillance of rare cancers in europe. 2007. Available: https://rarecare.eu
Posada M, Taruscio D. Rare diseases epidemiology: Update and overview. Springer Netherlands; 2010. Available: https://link.springer.com/book/10.1007/978-3-319-67144-4
WHO. International classification of diseases for oncology, 3rd edition (ICD-O-3). Available: https://www.who.int/standards/classifications/other-classifications/international-classification-of-diseases-for-oncology
European Society For Medical Oncology. “Families” and List of Rare Cancers. 2022. Available: https://www.rarecancerseurope.org/what-are-rare-cancers/families-and-list-of-rare-cancers
Regulation (EU) No 536/2014 of the European Parliament and of the Council of 16 April 2014 on Clinical Trials on Medicinal Products for Human Use, and Repealing Directive 2001/20/EC. Official Journal of the European Union; 2014. Available: https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=celex:32000R0141
Ferrari A, Bisogno G, De Salvo GL, et al; Italian Study on Rare Tumours in Paediatric Age (TREP); Associazione Italiana Ematologia Oncologia Pediatrica (AIEOP). The challenge of very rare tumours in childhood: the Italian TREP project. Eur J Cancer. 2007 Mar;43(4):654-9.
Bisogno G, Ferrari A, Bien E, et al. Rare cancers in children - The EXPeRT Initiative: a report from the European Cooperative Study Group on Pediatric Rare Tumors. Klin Padiatr. 2012 Oct;224(6):416-20.
Aggarwal A, Ginsburg O, Fojo T. Cancer economics, policy and politics: What informs the debate? Perspectives from the EU, Canada and US. J Cancer Policy. 2014;2: 1-11.
European Cooperative Study Group For Pediatric Rare Tumors (EXPeRT), 2023. Available: https://www.raretumors-children.eu/
Ferrari A, Brecht IB, Gatta G, et al. Defining and listing very rare cancers of paediatric age: consensus of the Joint Action on Rare Cancers in cooperation with the European Cooperative Study Group for Pediatric Rare Tumors. Eur J Cancer. 2019 Mar;110:120-126.
Fantini B, Vaccaro CM. Value based healthcare for rare diseases: efficiency, efficacy, equity. Ann Ist Super Sanita. 2019 Jul-Sep;55(3):251-257.
Graessner H, Zurek B, Hoischen A, et al. Solving the unsolved rare diseases in Europe. Eur J Hum Genet. 2021 Sep;29(9):1319-1320.
Митева-Катранджиева Ц. Реални ползи от приложението на епидемиолoгични регистри в областта на редките болести. Редки болести и лекарства сираци. 2017;8: 8–12.
Rosell L, Wihl J, Hagberg O, et al. Function, information, and contributions: An evaluation of national multidisciplinary team meetings for rare cancers. Rare Tumors. 2019 May 8;11:2036361319841696.
Gatta G, Capocaccia R, Trama A, et al; RARECARE Working Group. The burden of rare cancers in Europe. Adv Exp Med Biol. 2010;686:285-303.
Limon J, Miettinen M, Lasota J. Rare cancers. Int J Biochem Cell Biol. 2014 Aug;53:461.
Консулова А, Йорданов А, Ел Шемери С и съавт. Колко редки са редките онкологични болести? Редки болести и лекарства сираци 2021;12(2):19-22.
WHO. Global health estimates: Leading causes of death. World Health Organization. 2020. Available: https://www.who.int/data/gho/data/themes/mortality-and-global-health-estimates/ghe-leading-causes-of-death
Conceição P et al. Human development report. 2019: Beyond income, beyond averages, beyond today: Inequalities in human development in the 21st century. 2019. Available: https://hdr.undp.org/content/human-development-report-2019
Hofmarcher T, Lindgren P, Wilking N, et al. The cost of cancer in Europe 2018. Eur J Cancer. 2020 Apr;129:41-49.
НЦОЗА. Заболеваемост на населението. 2021. Available: https://ncpha.government.bg/index/3032-zabolevaemost-na-naselenieto.html
НСИ. Deaths by causes and mortality by causes. 2021. Available: https://nsi.bg/en/content/3348/deaths-causes-and-mortality-causes
Cree IA, Indave BI. Commentary: Cancer research quality and tumour classification. Tumour Biol. 2020 Feb;42(2):1010428320907544.
Jackson TJ, Michel JL, Roberts R, et al. Development of a validation algorithm for 'present on admission' flagging. BMC Med Inform Decis Mak. 2009 Dec 1;9:48.
Arnold M, Karim-Kos HE, Coebergh JW, et al. Recent trends in incidence of five common cancers in 26 European countries since 1988: Analysis of the European Cancer Observatory. Eur J Cancer. 2015 Jun;51(9):1164-87.
Maughan KL, Lutterbie MA, Ham PS. Treatment of breast cancer. Am Fam Physician. 2010 Jun 1;81(11):1339-46.
Ettinger DS, Wood DE, Aggarwal C, et al. NCCN Guidelines Insights: Non-Small Cell Lung Cancer, Version 1.2020. J Natl Compr Canc Netw. 2019 Dec;17(12):1464-1472.
Peairs KS, Choi Y, Stewart RW, et al. Screening for breast cancer. Semin Oncol. 2017 Feb;44(1):60-72.
Hoffman RM, Sanchez R. Lung Cancer Screening. Med Clin North Am. 2017 Jul;101(4):769-785.
Shenoy SR, Dey B. Funding for cancer research by an Indian funding agency, DBT. J Biosci. 2021;46:2.
Begum M, Lewison G, Lawler M, et al. Mapping the European cancer research landscape: An evidence base for national and Pan-European research and funding. Eur J Cancer. 2018 Sep;100:75-84.
Griggs RC, Batshaw M, Dunkle M, et al; Rare Diseases Clinical Research Network. Clinical research for rare disease: opportunities, challenges, and solutions. Mol Genet Metab. 2009 Jan;96(1):20-6.
Andrews EB, Gilsenan A, Midkiff K, et al. Challenges in studying very rare cancer outcomes and infrequent exposures: example of teriparatide and osteosarcoma. Ann Epidemiol. 2016 Nov;26(11):751-753.
Искров Г, Стефанов Р. Редките тумори – епидемиологични и общественоздравни аспекти. Редки болести и лекарства сираци. 2018;9: 9–13.
Kasper B. The challenge of drug approval in rare cancers. Cancer. 2021 Mar 15;127(6):837-839.
Gatta G, van der Zwan JM, Casali PG, et al; RARECARE working group. Rare cancers are not so rare: the rare cancer burden in Europe. Eur J Cancer. 2011 Nov;47(17):2493-511.
European parlament and the Concil. Europe’s beating cancer plan. 2022. Available: https://health.ec.europa.eu/system/files/2022-02/eu_cancer-plan_en_0.pdf
Танев Т. Анализ на публичните политики - науката за създаване на конкретни политики. Военно издателство ЕООД; 2008.
Chabner BA, Friedman MA. Progress against rare and not-so-rare cancers. N Engl J Med. 1992 Feb 20;326(8):563-5.
Adams EE. Rare Cancers in Persons Exposed to Internal Radium. JAMA. 1982;247(20):2778–2779.
Sikora K, Smedley H. Rare cancers and specialist centers. Br Med J (Clin Res Ed). 1986 Mar 29;292(6524):898.
Wilson L. PP70. The costs of pancreatic cancer: Methodology of costing rare cancers. Eur J Cancer. 1997;33: S40.
Patel A, Ozsahin M, Mirimanoff RO, et al. The Rare Cancer Network: achievements from 1993 to 2012. Rare Tumors. 2012 Jun 26;4(3):e35.
Mirimanoff RO, Ozsahin M, Thariat J, et al. History of the rare cancer network and past research. Rare Tumors. 2014 Aug 6;6(3):5462.
97th United States Congress. Orphan drug act. FDA; 2014. Available: https://www.fda.gov/media/99546
European Parlament and the Council of Eropean union regulation 141/2000. 2000.
Very rare cancers--a problem neglected. Lancet Oncol. 2001 Apr;2(4):189.
European cancer information system. ECIS; 2022. Available: https://ecis.jrc.ec.europa.eu/info/initiatives.html
International agency for research on cancer (IARC). Available: https://www.iarc.who.int/
European Commision. ENCR | european network of cancer registries. Available: https://encr.eu/
International association of cancer registries IACR. Available: http://www.iacr.com.fr/
Berrino F, Sant M, Verdecchia A, Capocaccia R, Hakulinen T. Survival of cancer patients in europe. Cancer Causes and Control. 1995;6: 469–470. doi:10.1007/bf00052188
De Angelis R, Francisci S, Baili P, et al; EUROCARE Working Group. The EUROCARE-4 database on cancer survival in Europe: data standardisation, quality control and methods of statistical analysis. Eur J Cancer. 2009 Apr;45(6):909-30.
Gatta G, Ciccolallo L, Kunkler I, et al; EUROCARE Working Group. Survival from rare cancer in adults: a population-based study. Lancet Oncol. 2006 Feb;7(2):132-40.
Maule M, Merletti F, Mirabelli D, et al. Spatial variation of mortality for common and rare cancers in Piedmont, Italy, from 1980 to 2000: a Bayesian approach. Eur J Cancer Prev. 2006 Apr;15(2):108-16.
Marty M, Rheims N. Cancers rares: Des entités différentes, des problématiques communes. Oncologie. 2008;10: 360–362.
Eng C. Mendelian genetics of rare--and not so rare--cancers. Ann N Y Acad Sci. 2010 Dec;1214:70-82.
Griffiths J, Willard C, Burgess A, et al. Meeting the ongoing needs of survivors of rarer cancer. Eur J Oncol Nurs. 2007 Dec;11(5):434-41.
Executive Agency for Health and Consumers (EAHC) of the European Commission. RARECARE - surveillance of rare cancers in europe. 2007. Available: https://rarecare.eu
Posada M, Taruscio D. Rare diseases epidemiology: Update and overview. Springer Netherlands; 2010. Available: https://link.springer.com/book/10.1007/978-3-319-67144-4
WHO. International classification of diseases for oncology, 3rd edition (ICD-O-3). Available: https://www.who.int/standards/classifications/other-classifications/international-classification-of-diseases-for-oncology
European Society For Medical Oncology. “Families” and List of Rare Cancers. 2022. Available: https://www.rarecancerseurope.org/what-are-rare-cancers/families-and-list-of-rare-cancers
Regulation (EU) No 536/2014 of the European Parliament and of the Council of 16 April 2014 on Clinical Trials on Medicinal Products for Human Use, and Repealing Directive 2001/20/EC. Official Journal of the European Union; 2014. Available: https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=celex:32000R0141
Ferrari A, Bisogno G, De Salvo GL, et al; Italian Study on Rare Tumours in Paediatric Age (TREP); Associazione Italiana Ematologia Oncologia Pediatrica (AIEOP). The challenge of very rare tumours in childhood: the Italian TREP project. Eur J Cancer. 2007 Mar;43(4):654-9.
Bisogno G, Ferrari A, Bien E, et al. Rare cancers in children - The EXPeRT Initiative: a report from the European Cooperative Study Group on Pediatric Rare Tumors. Klin Padiatr. 2012 Oct;224(6):416-20.
Aggarwal A, Ginsburg O, Fojo T. Cancer economics, policy and politics: What informs the debate? Perspectives from the EU, Canada and US. J Cancer Policy. 2014;2: 1-11.
European Cooperative Study Group For Pediatric Rare Tumors (EXPeRT), 2023. Available: https://www.raretumors-children.eu/
Ferrari A, Brecht IB, Gatta G, et al. Defining and listing very rare cancers of paediatric age: consensus of the Joint Action on Rare Cancers in cooperation with the European Cooperative Study Group for Pediatric Rare Tumors. Eur J Cancer. 2019 Mar;110:120-126.
Fantini B, Vaccaro CM. Value based healthcare for rare diseases: efficiency, efficacy, equity. Ann Ist Super Sanita. 2019 Jul-Sep;55(3):251-257.
Graessner H, Zurek B, Hoischen A, et al. Solving the unsolved rare diseases in Europe. Eur J Hum Genet. 2021 Sep;29(9):1319-1320.
Митева-Катранджиева Ц. Реални ползи от приложението на епидемиолoгични регистри в областта на редките болести. Редки болести и лекарства сираци. 2017;8: 8–12.
Rosell L, Wihl J, Hagberg O, et al. Function, information, and contributions: An evaluation of national multidisciplinary team meetings for rare cancers. Rare Tumors. 2019 May 8;11:2036361319841696.
Gatta G, Capocaccia R, Trama A, et al; RARECARE Working Group. The burden of rare cancers in Europe. Adv Exp Med Biol. 2010;686:285-303.
Limon J, Miettinen M, Lasota J. Rare cancers. Int J Biochem Cell Biol. 2014 Aug;53:461.
Консулова А, Йорданов А, Ел Шемери С и съавт. Колко редки са редките онкологични болести? Редки болести и лекарства сираци 2021;12(2):19-22.
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2023-11-05
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Костадинов К, Мусурлиева Н. Исторически преглед на дефиницията за редки тумори в контекста на здравните политики. Редки болести и лекарства сираци [Internet]. 2023Nov.5 [cited 2024Nov.21];14(2):6-12. Available from: https://journal.raredis.org/index.php/RBLS/article/view/182
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