Clinical research focused in European adult women with minority diseases: Do we try hard enough?

References

• Chilet-Rosell, E. (2014). Gender bias in clinical research, pharmaceutical marketing, and the prescription of drugs. Global Health Action, 7, 25484. https://doi.org/10.3402/gha.v7.25484
   PubMed Web of Science ®Google Scholar
• Cohen, D. (2017). Cancer drugs: High price, uncertain value. BMJ (Clinical Research ed.), 359, j4543. https://doi.org/10.1136/bmj.j4543
   PubMed Web of Science ®Google Scholar
• Davis, C., Lexchin, J., Jefferson, T., Gøtzsche, P., & McKee, M. (2016). "Adaptive pathways" to drug authorisation: Adapting to industry? BMJ (Clinical Research ed.), 354, i4437. https://doi.org/10.1136/bmj.i4437
   PubMedGoogle Scholar
• Davis, C., Naci, H., Gurpinar, E., Poplavska, E., Pinto, A., & Aggarwal, A. (2017). Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: Retrospective cohort study of drug approvals 2009-13. BMJ (Clinical Research ed.), 359, j4530. https://doi.org/10.1136/bmj.j4530
   PubMed Web of Science ®Google Scholar
• Duma, N., Vera Aguilera, J., Paludo, J., Haddox, C. L., Gonzalez Velez, M., Wang, Y., Leventakos, K., Hubbard, J. M., Mansfield, A. S., Go, R. S., & Adjei, A. A. (2018). Representation of minorities and women in oncology clinical trials: Review of the past 14 years. Journal of Oncology Practice, 14(1), e1–e10. https://doi.org/10.1200/JOP.2017.025288
   PubMed Web of Science ®Google Scholar
• EMA. (2018). Annual report on the use of the special contribution for orphan medicinal products. https://www.ema.europa.eu/en/documents/report/annual-report-use-specialcontribution-orphan-medicinal-products-2018_en.pdf
   Google Scholar
• Ermisch, M., Bucsics, A., Vella Bonanno, P., Arickx, F., Bybau, A., Bochenek, T., van de Casteele, M., Diogene, E., Fürst, J., Garuolienė, K., van der Graaff, M., Gulbinovič, J., Haycox, A., Jones, J., Joppi, R., Laius, O., Langner, I., Martin, A. P., Markovic-Pekovic, V., … Godman, B. (2016). Payers’ views of the changes arising through the possible adoption of adaptive pathways. Frontiers in Pharmacology, 7, 305. https://doi.org/10.3389/fphar.2016.00305
   PubMed Web of Science ®Google Scholar
• ESMO. (2017). Ovarian Cancer: An ESMO Guide for Patients. https://www.esmo.org/content/download/10097/201883/file/EN-Ovarian-Cancer-Guide-for-Patients.pdf
   Google Scholar
• European Commission. (2018). Eurostat [Data base online]. https://ec.europa.eu/eurostat/data/database
   Google Scholar
• European Medicines Agency. (2018). Clinical trial register [Data base online]. https://www.clinicaltrialsregister.eu/ctr-search/search
   Google Scholar
• Ferlay, J., Colombet, M., Soerjomataram, I., Dyba, T., Randi, G., Bettio, M., Gavin, A., Visser, O., & Bray, F. (2018). Cancer incidence and mortality patterns in Europe: Estimates for 40 countries and 25 major cancers in 2018. European Journal of Cancer, 103, 356–387. https://doi.org/10.1016/j.ejca.2018.07.005
   PubMed Web of Science ®Google Scholar
• Franco, P. (2013). Orphan drugs: The regulatory environment. Drug Discovery Today, 18(3–4), 163–172. https://doi.org/10.1016/j.drudis.2012.08.009
   PubMed Web of Science ®Google Scholar
• Gaeta, M., Campanella, F., Capasso, L., Schifino, G. M., Gentile, L., Banfi, G., Pelissero, G., & Ricci, C. (2017). An overview of different health indicators used in the European Health Systems. Journal of Preventive Medicine and Hygiene, 58(2), E114–E120.
   PubMedGoogle Scholar
• Gammie, T., Lu, C. Y., & Babar, Z. U. (2015). Access to orphan drugs: A comprehensive review of legislations, regulations and policies in 35 countries. PLoS One, 10(10), e0140002. https://doi.org/10.1371/journal.pone.0140002
   PubMed Web of Science ®Google Scholar
• González, P., Macho-Stadler, I., & Pérez-Castrillo, D. (2016). Private versus social incentives for pharmaceutical innovation. Journal of Health Economics, 50, 286–297. https://doi.org/10.1016/j.jhealeco.2015.12.003
   PubMed Web of Science ®Google Scholar
• Heemstra, H. E., de Vrueh, R. L., van Weely, S., Büller, H. A., & Leufkens, H. G. (2008). Orphan drug development across Europe: Bottlenecks and opportunities. Drug Discovery Today, 13(15–16), 670–676. https://doi.org/10.1016/j.drudis.2008.05.001
   PubMed Web of Science ®Google Scholar
• Husereau, D., Henshall, C., & Jivraj, J. (2014). Adaptive approaches to licensing, health technology assessment, and introduction of drugs and devices. International Journal of Technology Assessment in Health Care, 30(3), 241–249. https://doi.org/10.1017/S0266462314000191
   PubMed Web of Science ®Google Scholar
• Joppi, R., Bertele’, V., & Garattini, S. (2013). Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU. European Journal of Clinical Pharmacology, 69(4), 1009–1024. https://doi.org/10.1007/s00228-012-1423-2
   PubMed Web of Science ®Google Scholar
• Kaufmann, P., Pariser, A.R., Austin, C. (2018). From scientific discovery to treatments for rare diseases – the view from the National Center for Advancing Translational Sciences – Office of Rare Diseases Research. Orphanet Journal of Rare Diseases, 13(1), 196. https://doi.org/10.1186/s13023-018-0936-x
   Google Scholar
• Martinalbo, J., Bowen, D., Camarero, J., Chapelin, M., Démolis, P., Foggi, P., Jonsson, B., Llinares, J., Moreau, A., O’Connor, D., Oliveira, J., Vamvakas, S., & Pignatti, F. (2016). Early market access of cancer drugs in the EU. Annals of Oncology: Official Journal of the European Society for Medical Oncology, 27(1), 96–105. https://doi.org/10.1093/annonc/mdv506
   PubMed Web of Science ®Google Scholar
• Matesanz, R., Domínguez-Gil, B., Coll, E., Mahíllo, B., & Marazuela, R. (2017). How Spain reached 40 deceased organ donors per million population. American Journal of Transplantation: Official Journal of the American Society of Transplantation and the American Society of Transplant Surgeons, 17(6), 1447–1454. https://doi.org/10.1111/ajt.14104
   PubMed Web of Science ®Google Scholar
• Moliner, A. M., & Waligora, J. (2017). The European Union Policy in the Field of Rare Diseases. Advances in Experimental Medicine and Biology, 1031, 561–587. https://doi.org/10.1007/978-3-319-67144-4_30
   PubMed Web of Science ®Google Scholar
• Mullard, A. (2012). 2011 FDA drug approvals. Nature Reviews. Drug Discovery, 11(2), 91–94. https://doi.org/10.1038/nrd3657
   PubMed Web of Science ®Google Scholar
• Pauwels, K., Huys, I., Casteels, M., De Nys, K., & Simoens, S. (2014). Market access of cancer drugs in European countries: Improving resource allocation. Targeted Oncology, 9(2), 95–110. https://doi.org/10.1007/s11523-013-0301-x
   PubMed Web of Science ®Google Scholar
• Pinxten, W., Denier, Y., Dooms, M., Cassiman, J. J., & Dierickx, K. (2012). A fair share for the orphans: Ethical guidelines for a fair distribution of resources within the bounds of the 10-year-old European Orphan Drug Regulation. Journal of Medical Ethics, 38(3), 148–153. https://doi.org/10.1136/medethics-2011-100094
   PubMed Web of Science ®Google Scholar
• Prasad, V. (2017). Do cancer drugs improve survival or quality of life?BMJ (Clinical Research ed.), 359, j4528. https://doi.org/10.1136/bmj.j4528
   PubMedGoogle Scholar
• Rollet, P., Lemoine, A., & Dunoyer, M. (2013). Sustainable rare diseases business and drug access: No time for misconceptions. Orphanet Journal of Rare Diseases, 8, 109. https://doi.org/10.1186/1750-1172-8-109
   PubMed Web of Science ®Google Scholar
• Romaniuk, P., & Szromek, A. R. (2016). The evolution of the health system outcomes in Central and Eastern Europe and their association with social, economic and political factors: An analysis of 25 years of transition. BMC Health Services Research, 16, 95. https://doi.org/10.1186/s12913-016-1344-3
   PubMed Web of Science ®Google Scholar
• Weale, A. (2015). Reflecting on ‘Are health problems systemic? Politics of access and choice under Beveridge and Bismarck systems’. Health Economics, Policy and Law, 10(4), 431–435. https://doi.org/10.1017/S1744133115000171
   PubMed Web of Science ®Google Scholar