Clinical evaluation of new drugs against orphan diseases in oncology – the current situation in Europe and in our country

Czech version

Authors: Jaroslav Štěrba ¹; Sylva Štěrbová ²; Daniela Kodytková ¹; Dalibor Valík ^3,4; Regina Demlová ^3,4
Authors‘ workplace: Klinika dětské onkologie LF MU a FN Brno, pracoviště Dětská nemocnice, přednosta prof. MUDr. Jaroslav Štěrba, Ph. D. ¹; Katedra Ústavního práva PrF UP Olomouc, vedoucí katedry doc. JUDr. Jiří Jirásek, CSc. ²; Masarykův onkologický ústav Brno, ředitel prof. MUDr, Jiří Vorlíček, CSc. ³; Farmakologický ústav LF MU Brno, přednostka MUDr. Regina Demlová, Ph. D. ⁴
Published in: Vnitř Lék 2014; 60(Suppl 2): 80-85
Category: 70th Birthday - prof. MUDr. Jiří Vorlíček, CSc.

Overview

Cancer represents one of the main causes of death among diseases across the age spectrum. Tumors in children, however, represent less than 1% of the total number of cancers in the population and in terms of the definition of orphan diseases in Europe are all children's cancers considered as orphan diseases. This is the reason why the research and development of new agents against cancer in childhood stands outside of the main interest. Every year around 30,000 new cases of cancer in children and adolescents are diagnosed in the European Unioun (EU) and approximately 80% of them achieve long-term remission using mainly conventional methods of treatment. However, almost 6,000 children and adolescents die every year of malignant tumors therefore cancer remains a major cause of morbidity and mortality. Consequently, there is a demand for new and safe drugs for children suffering from cancer which would lead to improved survival and to risk reduction of late adverse effects of cancer treatment. In the past 10 years in the EU, more in the EU-15 than in our country, 20 performed oncology trials in phase I involving adults account for only one trial in pediatric patients. The issue of new drugs clinical testing in rare cancers is very complex, complicated and for current unsatisfactory situation might be responsible various aspects. These aspects contain the legislative field, the problem of determining the correct dose of testing drug as a single agent or in combination therapy, the use of testing drug in advanced disease or already in de novo diagnosed patients, as well as equity (equal) access to new drugs being tested, the goal set for each molecule/drug in clinical trials, the conflict of interest balanced with sufficient professionalism and last but not least, the need for new methodologies and statistical approaches. The aim of this article is to describe the issue complexity of incorporation of new, modern drug for cancer patients with orphan diseases, including children.

Key words:
clinical evaluation – new drugs – orphan diseases in oncology

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