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New Drugs for Rare Diseases in Children

Klaus Rose1

  • 1klausrose Consulting, Pediatric Drug Development & More, Riehen, Switzerland.

Clinical Therapeutics
|February 6, 2017
PubMed

Insights

Pediatric Legislation in the US and EU aims to improve children's health by requiring pediatric studies for new drugs. However, innovative treatments for rare pediatric diseases require new market-based approaches beyond current legislation.

Area of Science:

  • Pediatric pharmacology
  • Drug development regulation
  • Rare disease therapeutics

Background:

  • United States (US) Pediatric Legislation (PL) enacted in 1997 and European Union Pediatric Legislation (EUPL) since 2007 mandate pediatric research for new drugs.
  • Both legislative frameworks aim to enhance children's health outcomes by ensuring pediatric drug safety and efficacy.
  • The EUPL mandates a pediatric investigation plan for new drug registration, highlighting market insufficiency for pediatric drug development.

Purpose of the Study:

  • To analyze the impact and limitations of current pediatric legislation (US PL and EUPL).
  • To explore novel strategies for facilitating drug development for rare pediatric diseases.
  • To discuss the evolution of medical decision-making from eminence-based to data-based approaches.

Main Methods:

  • Comparative analysis of US and EU pediatric legislation.
  • Discussion of modern drug labeling and its implications for clinical practice.
  • Review of historical successes in pediatric oncology and rare disease treatment.

Main Results:

  • Modern drug labels represent a shift towards data-driven medical decisions, but do not replace essential knowledge transfer.
  • Children have been successfully treated with off-label drugs, challenging the notion of "therapeutic orphans".
  • Significant therapeutic breakthroughs have been achieved in conditions like acute myelogenous leukemia and cystic fibrosis.

Conclusions:

  • Current pediatric legislation's focus on adult drug add-ons presents a limited perspective.
  • New global initiatives are needed to incentivize innovative treatment concepts for rare diseases through market-based mechanisms.
  • Rewarding novel therapeutic concepts developed via philanthropy and patient support, upon meeting regulatory milestones, is crucial for future breakthroughs.
Abstract

Keywords:
Creating Hope ActEuropean Medicines AgencaEuropean Pediatric Legislationpediatric drug developmentpediatric legislationpediatric ocology

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