Evaluación económica de tecnologías sanitarias en enfermedades raras y medicamentos huérfanos. Apreciaciones sobre la eficiencia y la equidad
Keywords:
Enfermedades raras, medicamentos huérfanos, tecnologías sanitarias, evaluación económicaAbstract
Introducción. La evaluación de tecnologías sanitarias en enfermedades raras y medicamentos huérfanos se ha convertido en objeto de atención mayor para científicos, directivos ysociedad en general. Dado su carácter polémico requiere de valoraciones y análisis desde diferentes perspectivas. Las evaluaciones económicas de tecnologías pueden contribuir a una mejora en la toma de decisiones y asignación de recursos. Objetivo: Describir y discutir acerca de un conjunto de temas considerados de relevancia para abordar la evaluación económica de tecnologías sanitarias en enfermedades raras y medicamentos huérfanos. Método: Se realizó una revisión bibliográfica y documental sobre el tema así como consultas a investigadores y directivos sobre debilidades relacionados con el tema. Resultados: Los aspectos más discutidos fueron la eficiencia, la equidad y la sostenibilidad de la financiación de tecnologías en enfermedades raras y medicamentos huérfanos. Se identificó un conjunto de debilidades a tener en cuenta para profundizar en el análisis económico de este temaDownloads
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References
1. Darbá Josep. Consideraciones económicas para la evaluación de los medicamentos huérfanos en las decisiones de financiación en España. Pharmacoeconomics Spanish Research Articles. 2013. DOI 10.1007/s40277-013-0014-7.
2. Kontoghiorghe NC, Andreou N, Constantinou K, Kontoghiorghes GJ. World Health dilemmas: orphan and rare diseases, orphan drugs and orphan patients. World J Methodol. 2014; 4(3): 163-88.
3. European Commission. European Commission Regulation (EC) No. 141/2000 of the European Parliament and the Council of 16 December 1999 on orphan medicinal products. Off J Eur Communities [Internet]. 2000 [cited 23 Nov 2015]; L 18: [about 5p.]. Available from: http://ec.europa.eu/health/files/eudralex/vol-1/reg_2000_141/reg_2000_141_en.pdf
4. Hughes DA, Tunnage B, Yeo ST. Drugs for exceptionally rare diseases: do they deserve special status for funding? Q J Med.2005;98:829–36.
5. Hyry HI, Manuel J, Cox TM, Roos JC. Compassionate use of orphan drugs. Orphanet J Rare Dis [Internet]. 2015 [cited 7 Dec 2015]; 10(100): [about 9p.]. Available from: http://www.ncbi.nlm.nih.gov/pubmed/26292942
6. Winstone J, Chadda S, Ralston S, Sajosi P. Review and comparison of clinical evidence submitted to support European Medicines Agency market authorization of orphan-designated oncological treatments. Orphanet J Rare Dis [Internet]. 2015 [cited 10 Dec 2015]; 10(139): [about 8p.]. Available from: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4625856/
7. Pastor Ma de las M. Modelo de gestión farmacéutica los medicamentos huérfanos. Consideraciones sobre el coste efectividad. Anexo IV. En: Rodríguez-Santana IP. El Modelo de Futuro de Gestión de la Salud. Propuestas para un debate [Internet]. Madrid: Bamberg; 2011 [citado 12 Jul 2011]. p. 38-43. Disponible en: http://static.correofarmaceutico.com/docs/2011/04/ modelo_futuro_gestion.pdf
8. National Institute for Health and Clinical Excellence. Appraising orphan drugs [Internet]. [cited Jun 12 2011]. Available from: http://www.nice.org.uk/niceMedia/pdf/smt/120705item4.pdf
9. Orofino J, Soto J, Casado MA, Oyagüez I. Global spending on orphan drugs in France, Germany, the UK, Italy and Spain during 2007. Appl Health Econ Health Policy. 2010;8(5):301–15.
10. Wang B, Kesselheim AS. Characteristics of efficacy evidence supporting approval of supplemental indications for prescription drugs in United States 2005-14: systematic review. BMJ [Internet]. 2015 [cited 1 Dec 2015]; 351(h4679): [about 8p.]. Available from: http://www.ncbi.nlm.nih.gov/pubmed/26400844
11. Gammie T, Lu CY, Babar ZU. Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries. PLoS One [Internet]. 2015 [cited 1 Dec 2015]; 10(10): [about 9p.]. Available from: http://www.ncbi.nlm.nih.gov/pubmed/26451948
12. Rhee TG. Policymaking for Orphan Drugs and Its Challenges. AMA J Ethics. 2015; 17(8):776-9.
13. Schuller Y, Hollak CE, Biegstraaten M. The quality of economic evaluations of ultra-orphan drugs in Europe - a systematic review. Orphanet J Rare Dis [Internet]. 2015 [cited 1 Dec 2015]; 10(92): [about 9p.]. Available from: http://www.ncbi.nlm.nih.gov/pubmed/26223689
14. Onakpoya IJ, Spencer EA, Thompson MJ, Heneghan CJ. Effectiveness, safety and costs of orphan drugs: an evidence-based review. BMJ Open [Internet]. 2015 Jun 24 [cited 2 Dec 2015]; 5(6):e007199. Available from: http://www.ncbi.nlm.nih.gov/pubmed/26109112
15. Orpha.net [Internet]. Paris: INSERM; c1997 [updated Nov 15 2011; cited Jun 8 2014]. Available from: http://www.orpha.net/consor/cgi-bin/index.php?lng=ES.
16. Gálvez A, Marrero M, Peralta J. Encuentro de Enfermedades Raras Cuba Ecuador. Universidad de Ciencias Médicas de la Habana. 2014.
2. Kontoghiorghe NC, Andreou N, Constantinou K, Kontoghiorghes GJ. World Health dilemmas: orphan and rare diseases, orphan drugs and orphan patients. World J Methodol. 2014; 4(3): 163-88.
3. European Commission. European Commission Regulation (EC) No. 141/2000 of the European Parliament and the Council of 16 December 1999 on orphan medicinal products. Off J Eur Communities [Internet]. 2000 [cited 23 Nov 2015]; L 18: [about 5p.]. Available from: http://ec.europa.eu/health/files/eudralex/vol-1/reg_2000_141/reg_2000_141_en.pdf
4. Hughes DA, Tunnage B, Yeo ST. Drugs for exceptionally rare diseases: do they deserve special status for funding? Q J Med.2005;98:829–36.
5. Hyry HI, Manuel J, Cox TM, Roos JC. Compassionate use of orphan drugs. Orphanet J Rare Dis [Internet]. 2015 [cited 7 Dec 2015]; 10(100): [about 9p.]. Available from: http://www.ncbi.nlm.nih.gov/pubmed/26292942
6. Winstone J, Chadda S, Ralston S, Sajosi P. Review and comparison of clinical evidence submitted to support European Medicines Agency market authorization of orphan-designated oncological treatments. Orphanet J Rare Dis [Internet]. 2015 [cited 10 Dec 2015]; 10(139): [about 8p.]. Available from: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4625856/
7. Pastor Ma de las M. Modelo de gestión farmacéutica los medicamentos huérfanos. Consideraciones sobre el coste efectividad. Anexo IV. En: Rodríguez-Santana IP. El Modelo de Futuro de Gestión de la Salud. Propuestas para un debate [Internet]. Madrid: Bamberg; 2011 [citado 12 Jul 2011]. p. 38-43. Disponible en: http://static.correofarmaceutico.com/docs/2011/04/ modelo_futuro_gestion.pdf
8. National Institute for Health and Clinical Excellence. Appraising orphan drugs [Internet]. [cited Jun 12 2011]. Available from: http://www.nice.org.uk/niceMedia/pdf/smt/120705item4.pdf
9. Orofino J, Soto J, Casado MA, Oyagüez I. Global spending on orphan drugs in France, Germany, the UK, Italy and Spain during 2007. Appl Health Econ Health Policy. 2010;8(5):301–15.
10. Wang B, Kesselheim AS. Characteristics of efficacy evidence supporting approval of supplemental indications for prescription drugs in United States 2005-14: systematic review. BMJ [Internet]. 2015 [cited 1 Dec 2015]; 351(h4679): [about 8p.]. Available from: http://www.ncbi.nlm.nih.gov/pubmed/26400844
11. Gammie T, Lu CY, Babar ZU. Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries. PLoS One [Internet]. 2015 [cited 1 Dec 2015]; 10(10): [about 9p.]. Available from: http://www.ncbi.nlm.nih.gov/pubmed/26451948
12. Rhee TG. Policymaking for Orphan Drugs and Its Challenges. AMA J Ethics. 2015; 17(8):776-9.
13. Schuller Y, Hollak CE, Biegstraaten M. The quality of economic evaluations of ultra-orphan drugs in Europe - a systematic review. Orphanet J Rare Dis [Internet]. 2015 [cited 1 Dec 2015]; 10(92): [about 9p.]. Available from: http://www.ncbi.nlm.nih.gov/pubmed/26223689
14. Onakpoya IJ, Spencer EA, Thompson MJ, Heneghan CJ. Effectiveness, safety and costs of orphan drugs: an evidence-based review. BMJ Open [Internet]. 2015 Jun 24 [cited 2 Dec 2015]; 5(6):e007199. Available from: http://www.ncbi.nlm.nih.gov/pubmed/26109112
15. Orpha.net [Internet]. Paris: INSERM; c1997 [updated Nov 15 2011; cited Jun 8 2014]. Available from: http://www.orpha.net/consor/cgi-bin/index.php?lng=ES.
16. Gálvez A, Marrero M, Peralta J. Encuentro de Enfermedades Raras Cuba Ecuador. Universidad de Ciencias Médicas de la Habana. 2014.
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Published
2016-01-06
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Gálvez González AM, Roldán E, Llera V, Fernández García A, Marrero Araujo M, Peralta Rojas JA. Evaluación económica de tecnologías sanitarias en enfermedades raras y medicamentos huérfanos. Apreciaciones sobre la eficiencia y la equidad. INFODIR [Internet]. 2016 Jan. 6 [cited 2025 Sep. 11];(22):3-8. Available from: https://revinfodir.sld.cu/index.php/infodir/article/view/72
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