Details

Autor(en) / Beteiligte

• Laird, Glen
• Yijie, Zhou
• Yang, Song
• Bo, Yang
• Yang, Bo
• Song, Yang
• Zhou, Yijie

Titel

Introduction to Rare Disease Therapy Development

Ist Teil von

• Drug Development for Rare Diseases, 2023, p.1-6

Auflage

1

Ort / Verlag

United Kingdom: CRC Press

Erscheinungsjahr

2023

Quelle

Alma/SFX Local Collection

Beschreibungen/Notizen

• We introduce the clinical development context of novel therapies for rare diseases. Areas of research progress are highlighted such as chronic myeloid leukemia and cystic fibrosis. Huntington's disease is given as an example of the many areas in which more progress is needed. Regulatory definitions of rare diseases are given. Key aspects of the setting include the genetic origin of many diseases, potential lack of clinical understanding of the diseases, a pediatric focus, funding challenges, regulatory uncertainties, privacy considerations, and difficulties identifying appropriate patients for study. A non-technical focus on quantitative issues such as statistics is brought to the forefront.