Paediatric Oncology at the Crossroads: A Call for Change
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EDITORIAL
Paediatric Oncology at the Crossroads: A Call for Change Klaus Rose1
© Springer Nature Switzerland AG 2020
Cancer in children is fortunately rare. It was first recognised as a challenge when infectious diseases were increasingly prevented and/or became treatable. The first successes in patient care occurred not with new drugs, but rather with well-known chemotherapeutics from the 1970s onwards [1, 2]. For the treating physicians, the approval status of those drugs was irrelevant. Physicians’ focus was successful treatment; these studies were performed in the unique situation that effective drugs already existed. These studies were so successful in adult cancer patients that study participation became standard-of-care in paediatric oncology [3]. In accordance with US Federal law, the Food and Drug Administration (FDA) is not authorized to tell physicians what to prescribe, but it controls the promotional activities of pharmaceutical companies. In 1988, the term “offlabel” appeared [4], but the entire discipline of paediatric oncology emerged “off-label” decades before the term “offlabel” even existed [5, 6]. There is a fundamental difference between drug approval in general and approval/non-approval in “children” defined as younger than 17/18 years. Approval is based on science and data. But approval/non-approval in “children” is based on a misleading administrative definition [7]. There is no relevant physiological difference between 15- and 16-year-old persons. The physiological difference of 1 year is negligible in adolescents, while the difference of 1 month in babies is not [8]. Chemotherapy exposes the entire body to cell poisons in the hope of killing enough malignant cells so that the body can defeat the remaining cancer cells. Chemotherapeutics are unspecific. This helped when their use in adult cancer patients was transferred to young cancer patients, who have more reserves. Young leukaemia patients survived better than adults [1, 2]. In relation to their bodyweight,
* Klaus Rose [email protected] 1
klausrose Consulting, Pediatric Drug Development & More, Aeussere Baselstrasse 308, 4125 Riehen, Switzerland
they tolerated higher doses. Then followed the assumption that switching targets would also work with modern anticancer drugs. However, modern anticancer drugs are not chemotherapeutics. Paediatric oncology began in the unique situation that its key drugs existed already and “only” needed to be explored in young patients. In the most frequent childhood malignancy, acute lymphoblastic leukaemia (ALL), about 90% survived in 2000 [6, 9]. Further improvement by fine-tuning chemotherapy became limited. New drugs were needed [10], but “paediatric” laws have achieved the contrary. First, they triggered FDA-rewarded studies, that were amplified by the European Union (EU) paediatric investigation plans (PIPs) for virtually all new drugs. Now the FDA Reauthorization Act (FDARA) authorises mandatory cancer studies with new anticancer drugs in 0- to 11-year-old patients, copying the EU mo
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