Pediatric Drug Development -  Masterclass

Introduction:

US and EU regulatory authorities demand pediatric studies for new drugs, in the EU within “pediatric investigation plans“ (PIPs). Initially, US companies benefited from patent extensions for pediatric studies, but PIPs are demanded early in development, and many PIP-demanded studies are questionable. Minors are not another species. They need correct dosing, not repeated proof of efficacy. The EMA calls its demands “pediatric drug development“ (PDD), but PDD produces separate labels in minors and has not improved clinical treatment. The training on this very controversial topic offers industry professionals essential background knowledge from the perspective of drug development. Understanding the logic of regulatory demands helps to negotiate with FDA & EMA. Pushing back exaggerated demands can save millions and can avoid damage lawsuits.

Trainer Profile:

Dr. Klaus Rose, M.D., M.S, Pediatric Drug Development & More CEO, Klausrose Consulting, Switzerland

Dr. Klaus Rose, CEO klausrose Consulting, studied medicine, psychology, and Latin languages. In 1997, he joined pharmaceutical industry for an international job. He was intrigued by the idea of drug development for children in the late 1990s, also because his older daughter had a very rare syndrome. He was Global Head Pediatrics Novartis 2001-2005, same position in Genentech/Roche 2005-2009, both based in Basel, Switzerland. He is self-employed since 2011. He speaks at international conferences, publishes in peer-reviewed journals, has co-edited three textbooks on pediatric drug development, and has authored five medical texbooks. Married, two daughters. His private interests include Mediterranean cooking, gardening, Latin languages, Hungarian, and classical guitar.

Methodology

• Historical background
• Overview US & EU pediatric legislation
• Analysis of FDA & EU pediatric decisions
• Science and pseudo-science in justifying separate studies & labels in minors
• Literature & internet research
• Interactive discussion with participants

Key Learnings

• Minors need correct doses, not repeated proof of efficacy
• Pediatric legislation blurs different meanings of the term “child”
• Companies waste time and money with questionable “pediatric” studies
• Companies might face damage lawsuits for questionable “pediatric” studies
• Most pediatric disciplines emerged “off-label” before the term “off-label” even existed
• Conflicts of interest behind the children-are-therapeutic-orphans and children-are-not-small adults mantras
• Most regulatory pediatric studies advance careers, not child health
• Minors need only reasonable studies
• Negotiating with EMA and FDA can save millions
• The FDA is gradually and partially relenting
  

Who Should Attend?

Professionals from pharmaceutical industry from:

• Regulatory affairs
• Clinical development
• Project management
• Communication
• Preclinical development
• Formulation development
• CMC
• Clinical pharmacology
• Safety
• Medical affairs
• Marketing

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Schedule:

Day One - Background and Overview
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14.00 Introductions of lecturer and participants‍

14.30 Background: drug development and the origins of US & EU pediatric laws

• Modern drug development triggered by World War II: penicillin
• Early reports about toxicities in babies in the 1950/60s
• The worldwide thalidomide desaster 1957-1961 with birth defects
• The 1962 amendment to the US Federal Food, Drug, and Cosmetic Act.
• Emergence of developmental pharmacology and separate pediatric studies
• Origins of the demand for separate paediatric studies
• US paediatric legislation since 1997, EU legislation since 2007

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15.30 Clinical studies and drug develoment

• Adult clinical development
• Pediatric drug development (PDD)

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15:45 The three key elements of PDD

• Pediatric formulations (tablets vs. fluids vs. orodispersible modern formulations)
• Juvenile animal studies
• Clinical studies, extrapolation, & more

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16:00 Coffee Break