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Drug development in dermatology: challenges in treating young patients
Klaus Rose1, Tomoko Maeda-Chubachi2, Jane M Grant-Kels3
1Klausrose Consulting, Lörrach, Germany.
Insights
Pediatric drug development faces regulatory hurdles, particularly in the EU, where the European Medicines Agency (EMA) has rigid pediatric investigation plan (PIP) demands. This creates challenges for drug sponsors, widening the gap between US and EU development.
Area of Science:
- Dermatology
- Pediatric Drug Development
- Regulatory Science
Background:
- Children are often 'therapeutic orphans' in drug development, necessitating specific pediatric requirements from regulatory bodies like the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
- The EMA mandates a 'pediatric investigation plan' (PIP) early in the drug development process.
- Skin maturation differs between age groups, impacting drug absorption and efficacy, yet regulatory approaches vary significantly between the US and EU.
Purpose of the Study:
- To analyze the differing pediatric drug development requirements between the FDA and EMA.
- To highlight the challenges posed by the EMA's stringent PIP process.
- To discuss the implications of these regulatory differences on the pace of pediatric drug development.
Main Methods:
- Comparative analysis of FDA and EMA pediatric drug development guidelines.
- Case study examples of crisaborole for atopic dermatitis and psoriasis drug development.
- Review of the impact of PIP negotiations on development timelines and strategies.
Main Results:
- The FDA accepted pivotal trials for crisaborole (atopic dermatitis) across a wide age range (2-79 years), which the EMA also accepted as part of its PIP.
- For psoriasis, the EMA's PIP specifically requires separate efficacy and safety data in children aged 6-17 years.
- EMA's rigid stance and lengthy PIP negotiations contrast with the FDA's more flexible approach, leading to frustration and delays.
Conclusions:
- Regulatory divergence between the FDA and EMA creates significant challenges for pediatric drug development, particularly for skin disorders.
- The EMA's stringent requirements, while aiming for safety, can hinder timely access to new treatments for pediatric populations.
- Pharmaceutical companies need a deep understanding of regulatory nuances, patience, and potentially external expertise to navigate these complex processes effectively.
Introduction:
Drug development for skin disorders is influenced by the concept of children as 'therapeutic orphans' and is subject to pediatric requirements of the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The EMA requires a 'pediatric investigation plan' (PIP) early in development. Healthy newborns' skin is as thick as that of adults. For crisaborole for atopic dermatitis, the FDA accepted pivotal trials involving patients 2-79 years; the EMA accepted this as part of its PIP. In contrast, for psoriasis, PIPs require separate evidence of efficacy and safety in children aged 6-17 years.
Areas Covered:
Adolescents' skin does not change overnight on their 18th birthday, only the legal status. Most FDA pediatric requirements are grounded in commonsense and reflect current scientific understanding. The EMA, however, continues to take rigid stance in its pediatric demands. PIP negotiations are often lengthy, complex, and frustrating.
Expert Opinion:
Companies must possess a deep understanding of the clinical landscape, considerable patience, and sometimes seek external experts' support. A gap has become wider between US and EU, with the EU increasingly falling behind in drug development.
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