European stakeholder perspectives on challenges to rare disease drug development – a qualitative study. (3rd June 2021)

• Record Type: Journal Article
• Authors: Joshi, Sarala; Chen, Jessica; Sultan, Mustafa; Singh, Shefali; Abedi, Syed; Zheng, Sifan
• Is Part Of: Expert opinion on orphan drugs. Volume 9:Number 6(2021)
• Page Start: 181
• Page End: 188
• Publication Date: 2021-06-03
• Subjects: Orphan disease -- orphan drug -- rare disease -- rare disease challenges -- stakeholder perspective; Orphan drugs -- Periodicals; Rare diseases -- Periodicals; Chemotherapy -- Periodicals; 615.1
• DOI: 10.1080/21678707.2021.1953469
• Languages: English
• ISSNs: 2167-8707

Abstract

ABSTRACT: Background: The multifaceted challenges of rare disease research remain, leaving a large proportion of rare disease patients without treatment options. This study aims to identify the challenges of rare disease drug development. Research design & methods: Ten semi-structured interviews were carried out with a range of stakeholders. Thematic analysis was conducted to identify common and important themes. Results: Four overarching themes of stakeholder engagement, regulatory structure, data, and patient support were identified, encompassing fifteen subthemes. Conclusion: The presence of an active patient group is a significant factor in reducing barriers to rare diseases treatment development. Effective collaboration between the various stakeholders of rare disease research is crucial.