Table 6.

Theoretical foundations of the European Union pediatric legislation.

1. The first official document “Better Medicines For Children” discusses pediatric off-label use without differentiation where this makes medical sense and where it might harm [EU Commission, 2002] 2. An EMA paper (2004) discusses risks of off-label use of drugs in children, without mentioning that off-label use is often necessary and lifesaving [EMA, 2004] 3. The sulfanilamide and thalidomide catastrophes triggered modern pharmaceutical legislation. No catastrophe triggered pediatric legislation [Rose, 2008; Wax, 1995; Taussig, 1962]. The US legislation intended to improve children’s healthcare 4. The EMA website gives a link to Rocchi that addresses a ‘lack of availability of appropriate medicines for children’. This is misleading. Off-label use is characterized as dangerous for children. A medical crisis of pharmaceutic treatment of children in Europe is thus invoked [Rocchi and Tomasi, 2011]. Past and present PDCO members have published comparable statements [Rocchi et al. 2010]. The American Academy of Pediatrics has issued a clear positive statement about off-label use of medicines [Fratarelli et al., 2014] 5. Drug development is moving into rare and neglected diseases. Pediatric multiple sclerosis, oncology and rheumatology are three major areas confronted with new medications [Chitnis et al. 2013; Niehues, 2015; Rose and Walson, 2015]. Pediatricians struggle to deal with this challenge, and the regulatory authorities do not prioritize. 6. The EMA’s approach is activism to enforce as many clinical trials in children as possible. In childhood diseases too rare for multiple trials this results in ‘ghost studies’, described exemplarily for multiple studies requested for metastasized melanoma in adolescents. For patients in ghost studies the term ‘therapeutic hostages’ has been proposed [Rose, 2014c; Rose and Senn, 2014; Rose and Kummer, 2015] 7. Academic research centers compete now with industry-sponsored, questionable clinical studies triggered by EMA/PDCO [Rose and Walson, 2015]

EMA, European Medicines Agency; EU, European Union; PDCO, pediatric committee.