Abstract
How to show in a convincing manner efficacy in the treatment of rare tumors and to advance treatment outcomes is a perpetual challenge to all stakeholders developing new medicinal products. Making pediatric anti-cancer medicines available to treat rare tumors is an important example of the public health agenda of the European Medicines Agency, as sadly, the much-quoted successes in treating some pediatric malignant diseases do not provide a fair representation of the outcomes of any such disease. In this context, stakeholders include pharmaceutical companies that are the developers and practically sole sources of new medicines, and academic researchers who may also be the treating physicians and who drive disease-specific iterative processes to improve cure. This chapter introduces the relevant scientific-regulatory guidelines, followed by a presentation of the principles and experience obtained in orphan medicine designations, to academic researchers as they contribute to medicinal product development. Then, this chapter presents available incentives and opportunities for scientific-regulatory interactions, aspects of trial conduct for the demonstration of efficacy, and finally, considerations on a lack of efficacy of a medicine in treating a rare tumor.
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Developing Products for Rare Diseases & Conditions. http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/default.htm
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Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on Orphan Medicinal Products. Official Journal of The European Communities. http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2000:018:0001:0005:en:PDF
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Herold, R., Llinares, J. (2012). Showing Efficacy in Treating Rare Pediatric Tumors: Introduction to European Regulatory and Scientific Support Available to Investigators. In: Schneider, D., Brecht, I., Olson, T., Ferrari, A. (eds) Rare Tumors In Children and Adolescents. Pediatric Oncology. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-04197-6_2
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