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Review
. 2023 Apr 11;18(1):79.
doi: 10.1186/s13023-023-02693-7.

Decentralized clinical trials and rare diseases: a Drug Information Association Innovative Design Scientific Working Group (DIA-IDSWG) perspective

Mercedeh Ghadessi  1 , Junrui Di  2 , Chenkun Wang  3 , Kiichiro Toyoizumi  4 , Nan Shao  5 , Chaoqun Mei  6 , Charmaine Demanuele  7 , Rui Sammi Tang  8 , Gianna McMillan  9 , Robert A Beckman  10
Affiliations
Review

Decentralized clinical trials and rare diseases: a Drug Information Association Innovative Design Scientific Working Group (DIA-IDSWG) perspective

Mercedeh Ghadessi et al. Orphanet J Rare Dis. .

Abstract

Background: Traditional clinical trials require tests and procedures that are administered in centralized clinical research sites, which are beyond the standard of care that patients receive for their rare and chronic diseases. The limited number of rare disease patients scattered around the world makes it particularly challenging to recruit participants and conduct these traditional clinical trials.

Main body: Participating in clinical research can be burdensome, especially for children, the elderly, physically and cognitively impaired individuals who require transportation and caregiver assistance, or patients who live in remote locations or cannot afford transportation. In recent years, there is an increasing need to consider Decentralized Clinical Trials (DCT) as a participant-centric approach that uses new technologies and innovative procedures for interaction with participants in the comfort of their home.

Conclusion: This paper discusses the planning and conduct of DCTs, which can increase the quality of trials with a specific focus on rare diseases.

Keywords: Advanced Analytics; Clinical Trial; Decentralized Clinical Trials; Digital Health Technologies; Pilot Study; Rare Disease; Real World Data.

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Conflict of interest statement

The authors declare that they have no competing interests.

Figures

Fig. 1
Fig. 1
Flowchart - Key Steps for Conducting DCT
See this image and copyright information in PMC

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