Details

Autor(en) / Beteiligte

• Quittell, Lynne M.
• Simon, Richard H.
• Morgan, Wayne

Titel

A model for oversight of rare disease studies: The 25-year experience of the cystic fibrosis foundation data safety monitoring board

Ist Teil von

• Journal of cystic fibrosis, 2024-07, Vol.23 (4), p.734-738

Ort / Verlag

Netherlands: Elsevier B.V

Erscheinungsjahr

2024

Quelle

MEDLINE

Beschreibungen/Notizen

• •Safety oversight for rare disease studies.•Cystic fibrosis clinical trial oversight.•Role of DSMB's in rare disease studies. This manuscript addresses the development and operating procedures of the Cystic Fibrosis Foundation Data Safety Monitoring Board (CFF-DSMB) and its role in the development and approval of new therapies through complex clinical trials with an emphasis on ensuring patient safety and study integrity. The authors describe the processes that have been developed over the last 25 years including the development of educational curricula for DSMB members and patient representation on DSMBs. The experience and success of the CFF-DSMB can serve as a model for providing high quality oversight of clinical trials for other groups who are dedicated to developing treatments for rare and complex diseases.