RAPS Convergence 2024

As the largest annual regulatory-focused event, RAPS Convergence unites stakeholders and decision makers across all facets of the regulatory field for three days of professional development, discussion, and relationship building.

Visit us at booth #810

Wednesday, Sept. 18 11:55 – 12:20 p.m.
Speaker: Emma Yang, Principal Regulatory Affairs Consultant
Title: The regulatory landscape of FDA-approved medicinal products for rare pediatric diseases

The majority of rare diseases affect children, and yet there remains an unmet need for approved therapies in most rare pediatric diseases. With many unique challenges and considerations faced with such drug development plans, this presentation will provide an overview of the regulatory environment and considerations within the US and EU to facilitate the development of rare orphan drugs for pediatrics. Successful approaches of drug development will be highlighted through evaluation of recent specific case studies.