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For Pharmaceutical Companies


Regarding minors, drug developers must deal with two layers of reality: the real world and the regulatory view. FDA and EMA approved drugs for “children” separately,  defining “children” as <18 years as if they were another species. The EMA demands a "pediatric investigation plan" (PIP) years before marketing authorization application, the FDA an initial pediatric study plan (iPSP) 60 days after the end-of-phase-2 meeting;

 

Newborns are really vulnerable and immature, but they grow and mature fast. The regulatory administrative age limits confer an inadequate physiological connotation to the 18th birthday. The word “child” has different meanings: we associate with children small, vulnerable persons; but administratively, anybody <18 years is a "child".

 

One resulting challenge is the negotiating of development plans for new drugs. Companies should try to avoid pointless separate “pediatric” efficacy studies that might even harm. Adolescents and school-age children can often be included in pivotal regulatory studies. In this, the FDA is today more pragmatic, but the Research to Accelerate Cures and Equity (RACE) for Children Act authorizes the FDA to demand “pediatric” studies for anticancer drugs in minors <12 years.

 

Another challenge is ongoing PIPs and resulting “pediatric” studies that cost millions per year and are mostly pointless. The EMA insists on their continuation. The time has come to challenge these studies in the public, with Institutional Review Boards (IRBs)/ ethics committees (ECs), and eventually in courts. The EMA will prefer not to openly challenge clinicians, IRBs, and ECs that question such studies.  To coordinate this, companies might consider professional support. I offer project-specific support, in-house general training, literature research, & more.

 

I have worked for thirty years in drug development. I was Global Head Pediatrics Novartis 2001 – 2005, Global Head Pediatrics Genentech/Roche 2005 – 2009, and am now independent. I am used to both operational and strategic, hands-on, and high-level challenges. Negotiating with EMA and FDA requires a solid background in developmental pharmacology, clinical trials methodology, pediatrics, regulatory affairs, and diplomacy. klaus.rose@klausrose.net or +41 61 312 0510.

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