Decentralized clinical trials and rare diseases: a Drug Information Association Innovative Design Scientific Working Group (DIA-IDSWG) perspective
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...
| Main Authors: | , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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BMC
2023-04-01
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| Series: | Orphanet Journal of Rare Diseases |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s13023-023-02693-7 |
| _version_ | 1797845768900444160 |
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| author | Mercedeh Ghadessi Junrui Di Chenkun Wang Kiichiro Toyoizumi Nan Shao Chaoqun Mei Charmaine Demanuele Rui (Sammi) Tang Gianna McMillan Robert A. Beckman |
| author_facet | Mercedeh Ghadessi Junrui Di Chenkun Wang Kiichiro Toyoizumi Nan Shao Chaoqun Mei Charmaine Demanuele Rui (Sammi) Tang Gianna McMillan Robert A. Beckman |
| author_sort | Mercedeh Ghadessi |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-04-09T17:45:22Z |
| format | Article |
| id | doaj.art-c2f70ba80edf499990a6da6a4b0a44a2 |
| institution | Directory Open Access Journal |
| issn | 1750-1172 |
| language | English |
| last_indexed | 2024-04-09T17:45:22Z |
| publishDate | 2023-04-01 |
| publisher | BMC |
| record_format | Article |
| series | Orphanet Journal of Rare Diseases |
| spelling | doaj.art-c2f70ba80edf499990a6da6a4b0a44a22023-04-16T11:24:53ZengBMCOrphanet Journal of Rare Diseases1750-11722023-04-0118111110.1186/s13023-023-02693-7Decentralized clinical trials and rare diseases: a Drug Information Association Innovative Design Scientific Working Group (DIA-IDSWG) perspectiveMercedeh Ghadessi0Junrui Di1Chenkun Wang2Kiichiro Toyoizumi3Nan Shao4Chaoqun Mei5Charmaine Demanuele6Rui (Sammi) Tang7Gianna McMillan8Robert A. Beckman9Research and Early Development Statistics, Bayer U.S. LLCGlobal Product Development, Pfizer IncBiostatistics department, Vertex Pharmaceuticals, IncStatistics & Decision Sciences Department, Janssen Pharmaceutical K. KBiostatistics, Moderna, IncGlobal Biometrics and Data Sciences, Bristol Myers SquibbGlobal Product Development, Pfizer IncClinical Development, Global Biometric DepartmentBioethics Institute at Loyola Marymount UniversityLombardi Comprehensive Cancer Center and Innovation Center for Biomedical Informatics, Georgetown University Medical CenterAbstract 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.https://doi.org/10.1186/s13023-023-02693-7Decentralized Clinical TrialsClinical TrialPilot StudyRare DiseaseReal World DataDigital Health Technologies |
| spellingShingle | Mercedeh Ghadessi Junrui Di Chenkun Wang Kiichiro Toyoizumi Nan Shao Chaoqun Mei Charmaine Demanuele Rui (Sammi) Tang Gianna McMillan Robert A. Beckman Decentralized clinical trials and rare diseases: a Drug Information Association Innovative Design Scientific Working Group (DIA-IDSWG) perspective Orphanet Journal of Rare Diseases Decentralized Clinical Trials Clinical Trial Pilot Study Rare Disease Real World Data Digital Health Technologies |
| title | Decentralized clinical trials and rare diseases: a Drug Information Association Innovative Design Scientific Working Group (DIA-IDSWG) perspective |
| title_full | Decentralized clinical trials and rare diseases: a Drug Information Association Innovative Design Scientific Working Group (DIA-IDSWG) perspective |
| title_fullStr | Decentralized clinical trials and rare diseases: a Drug Information Association Innovative Design Scientific Working Group (DIA-IDSWG) perspective |
| title_full_unstemmed | Decentralized clinical trials and rare diseases: a Drug Information Association Innovative Design Scientific Working Group (DIA-IDSWG) perspective |
| title_short | Decentralized clinical trials and rare diseases: a Drug Information Association Innovative Design Scientific Working Group (DIA-IDSWG) perspective |
| title_sort | decentralized clinical trials and rare diseases a drug information association innovative design scientific working group dia idswg perspective |
| topic | Decentralized Clinical Trials Clinical Trial Pilot Study Rare Disease Real World Data Digital Health Technologies |
| url | https://doi.org/10.1186/s13023-023-02693-7 |
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