Details

Autor(en) / Beteiligte

• Palmisano, Joanne
• Yijie, Zhou
• Yang, Song
• Bo, Yang

Titel

Developing Drugs for Rare Diseases: Regulatory Strategies and Considerations

Ist Teil von

• Drug Development for Rare Diseases, 2023, p.17-33

Auflage

1

Ort / Verlag

United Kingdom: CRC Press

Erscheinungsjahr

2023

Link zum Volltext

Quelle

Alma/SFX Local Collection

Beschreibungen/Notizen

• Regulatory agencies, such as US Food and Drug Administration and the European Union European Medicines Agency, play a critical role in drug development. This chapter elaborates various regulatory pathways that enabled a specialized environment for rare and orphan disease-related drug development. Regulatory agencies, such as US Food and Drug Administration and the European Union European Medicines Agency, play a critical role in drug development. This chapter elaborates various regulatory pathways that enabled a specialized environment for rare and orphan disease-related drug development. It focuses on established regulatory pathways of the US Food and Drug Administration (FDA) and the European Union (EU) European Medicines Agency (EMA), which represent the major health authorities governing rare and orphan disease drug development. Both the FDA and EMA have provisions for the compassionate use of unapproved/unauthorized medicines. Under specific agreements and conditions, compassionate use programs are reserved for those patients with seriously debilitating disease or life-threatening diseases that are not satisfactorily treated and who are unable or ineligible to participate in ongoing clinical trials. To advance the global development of medicines for children, both EMA and the US FDA have agreed on principles for interaction and exchange of information on pediatric matters.