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Regarding children, drug developers have to face two layers of reality: the real world and the regulatory view. US FDA and EU EMA approve drugs for „children“ separately,  defining „children“ as <17 years (US)/ <18 years (EU), as if children were another species. The FDA demands an initial pediatric study plan (iPSP) 60 days after the end-of-phase-2 meeting; the EU a „paediatric investigation plan“ (PIP) approved by the EMA pediatric committee (PDCO) long before marketing authoristation application.

Newborns are indeed vulnerable and immature, but they grow and mature fast. The FDA/EMA age limits are administrative. Their definitions confer an inadequate physiological connotation to administrative age limits. The word „child“ has different meanings: we associate small, vulnerable persons; but administratively anybody <17/18 years is a „child“.

One regulatory „pediatric“ challenge is the negotiating of development plans for new drugs. Companies should try to register drugs without having to perform separate „pediatric“ studies that are senseless or even harm patients. Adolescents should be included into pivotal regulatory studies, even if this requires additional paperwork. The FDA is often more pragmatic, but the US Research to Accelerate Cures and Equity (RACE) for Children Act will lead to FDA „pediatric“ demands in anticancer drugs from 2020 on. Another challenge are ongoing PIPs and „pediatric“ clinical studies. These studies cost millions per year and are medically questionable, but the EMA insists on their continuation. But the EMA will not dare to openly challenge clinicians involved in such studies and ethics committees that can suspend them. To address challanges, companies might consider professional support.

Dr. Rose works since thirty years in drug development. He was Global Head Pediatrics Novartis 2001 – 2005, Global Head Pediatrics Roche/Genentech 2005 – 2009, and is now independent. His work exposes him to both operational and strategic, hands-on and high level challenges. He established internal pediatric matrix structures and negotiates pediatric development with the FDA and the EMA across all clinical areas. He co-founded and chaired the EFGCP children’s medicines working party, was the first chairman of the DIA pediatric SIAC and of the IFPMA pediatric taskforce. He speaks at international conferences, has co-edited textbooks, and publishes. He identified key flaws of the US + EU pediatric laws in peer-reviewed journals. His papers are open source + can be downloaded, see publications.

Negotiating with FDA + EMA requires a solid background in developmental pharmacology, pediatrics, regulatory affairs; a well-founded position towards regulatory demands; and diplomacy. Dr. Rose is used to work independently, to maintain communication with and receive input + guidance from clients. He advises pharmaceutical companies and academic institutions. For support, e-mail  klaus.rose@klausrose.net or call +41 61 312 0510.