Analysis of patient-reported outcomes in the approval of novel oncology drugs in the United States, 2017–2022Research in context
Summary: Background: With the growing notion of patient-focused drug development, the quality of life and other patient-reported outcomes (PROs) of cancer patients are gaining considerable attention. Several drug regulatory agencies, including the U.S. Food and Drug Administration (FDA), are callin...
| Main Authors: | , , , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2023-05-01
|
| Series: | EClinicalMedicine |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S258953702300130X |
| _version_ | 1827971461424349184 |
|---|---|
| author | Chenghao Ge Kaiyuan Guo Yi Li Guanqiao Li Hong Zhang Jiaxuan Yang Yang Liu Chen Yin Sen Liu Songmei Xie Xiaoyuan Chen |
| author_facet | Chenghao Ge Kaiyuan Guo Yi Li Guanqiao Li Hong Zhang Jiaxuan Yang Yang Liu Chen Yin Sen Liu Songmei Xie Xiaoyuan Chen |
| author_sort | Chenghao Ge |
| collection | DOAJ |
| description | Summary: Background: With the growing notion of patient-focused drug development, the quality of life and other patient-reported outcomes (PROs) of cancer patients are gaining considerable attention. Several drug regulatory agencies, including the U.S. Food and Drug Administration (FDA), are calling attention to PROs. This review aims to comprehensively characterise the application of PROs and regulatory considerations for PROs in the FDA-approved novel oncology drugs. Methods: The FDA review documents and labels for novel oncology drugs approved from July 2017 to July 2022 were retrieved. We collected and analysed drug approval information, types of endpoints for PROs, PRO measures, designs of trials including PROs, and regulatory comments on PRO-related contents. Findings: Results demonstrated that PROs were used more commonly for solid tumours than hematologic malignancies, which might be correlated with the disease characteristics. We further categorised and analysed existing PRO measures, providing insight for tool selection in future oncology trial design. Our findings also indicated that PROs currently do not play a significant role in oncology drug approvals. The major deficiencies related to PROs commented on by FDA reviewers were analysed, followed by recommendations for improvements. Interpretation: This review demonstrates that PROs currently do not play a significant role in oncology drug marketing review, and how they can be used to support the approval of new oncology drugs is still in the exploratory stage. This current situation is not only related to the deficiencies in the design and implementation of PRO-related contents in oncology trials, but more importantly, it is a reminder that we should pay more attention to patient experience in the development of oncology drugs. Funding: This study was not supported by any funding. |
| first_indexed | 2024-04-09T19:08:13Z |
| format | Article |
| id | doaj.art-90c7b09368774d6abbe3122499df0b7a |
| institution | Directory Open Access Journal |
| issn | 2589-5370 |
| language | English |
| last_indexed | 2024-04-09T19:08:13Z |
| publishDate | 2023-05-01 |
| publisher | Elsevier |
| record_format | Article |
| series | EClinicalMedicine |
| spelling | doaj.art-90c7b09368774d6abbe3122499df0b7a2023-04-07T06:50:50ZengElsevierEClinicalMedicine2589-53702023-05-0159101953Analysis of patient-reported outcomes in the approval of novel oncology drugs in the United States, 2017–2022Research in contextChenghao Ge0Kaiyuan Guo1Yi Li2Guanqiao Li3Hong Zhang4Jiaxuan Yang5Yang Liu6Chen Yin7Sen Liu8Songmei Xie9Xiaoyuan Chen10Tsinghua Clinical Research Institute, School of Medicine, Tsinghua University, Beijing, ChinaSchool of Medicine, Tsinghua University, Beijing, ChinaTsinghua Clinical Research Institute, School of Medicine, Tsinghua University, Beijing, ChinaVanke School of Public Health, Tsinghua University, Beijing, ChinaCenter for Drug Evaluation, National Medical Products Administration, Beijing, ChinaSchool of Medicine, Tsinghua University, Beijing, ChinaTsinghua Clinical Research Institute, School of Medicine, Tsinghua University, Beijing, ChinaTsinghua Clinical Research Institute, School of Medicine, Tsinghua University, Beijing, ChinaTsinghua Clinical Research Institute, School of Medicine, Tsinghua University, Beijing, ChinaCenter for Drug Evaluation, National Medical Products Administration, Beijing, China; Corresponding author.Tsinghua Clinical Research Institute, School of Medicine, Tsinghua University, Beijing, China; Office of Clinical Trial Institute, Beijing Tsinghua Changgung Hospital, Beijing, China; Corresponding author. Tsinghua Clinical Research Institute, Tsinghua University, Beijing, China.Summary: Background: With the growing notion of patient-focused drug development, the quality of life and other patient-reported outcomes (PROs) of cancer patients are gaining considerable attention. Several drug regulatory agencies, including the U.S. Food and Drug Administration (FDA), are calling attention to PROs. This review aims to comprehensively characterise the application of PROs and regulatory considerations for PROs in the FDA-approved novel oncology drugs. Methods: The FDA review documents and labels for novel oncology drugs approved from July 2017 to July 2022 were retrieved. We collected and analysed drug approval information, types of endpoints for PROs, PRO measures, designs of trials including PROs, and regulatory comments on PRO-related contents. Findings: Results demonstrated that PROs were used more commonly for solid tumours than hematologic malignancies, which might be correlated with the disease characteristics. We further categorised and analysed existing PRO measures, providing insight for tool selection in future oncology trial design. Our findings also indicated that PROs currently do not play a significant role in oncology drug approvals. The major deficiencies related to PROs commented on by FDA reviewers were analysed, followed by recommendations for improvements. Interpretation: This review demonstrates that PROs currently do not play a significant role in oncology drug marketing review, and how they can be used to support the approval of new oncology drugs is still in the exploratory stage. This current situation is not only related to the deficiencies in the design and implementation of PRO-related contents in oncology trials, but more importantly, it is a reminder that we should pay more attention to patient experience in the development of oncology drugs. Funding: This study was not supported by any funding.http://www.sciencedirect.com/science/article/pii/S258953702300130XPatient-reported outcomeNovel oncology drug |
| spellingShingle | Chenghao Ge Kaiyuan Guo Yi Li Guanqiao Li Hong Zhang Jiaxuan Yang Yang Liu Chen Yin Sen Liu Songmei Xie Xiaoyuan Chen Analysis of patient-reported outcomes in the approval of novel oncology drugs in the United States, 2017–2022Research in context EClinicalMedicine Patient-reported outcome Novel oncology drug |
| title | Analysis of patient-reported outcomes in the approval of novel oncology drugs in the United States, 2017–2022Research in context |
| title_full | Analysis of patient-reported outcomes in the approval of novel oncology drugs in the United States, 2017–2022Research in context |
| title_fullStr | Analysis of patient-reported outcomes in the approval of novel oncology drugs in the United States, 2017–2022Research in context |
| title_full_unstemmed | Analysis of patient-reported outcomes in the approval of novel oncology drugs in the United States, 2017–2022Research in context |
| title_short | Analysis of patient-reported outcomes in the approval of novel oncology drugs in the United States, 2017–2022Research in context |
| title_sort | analysis of patient reported outcomes in the approval of novel oncology drugs in the united states 2017 2022research in context |
| topic | Patient-reported outcome Novel oncology drug |
| url | http://www.sciencedirect.com/science/article/pii/S258953702300130X |
| work_keys_str_mv | AT chenghaoge analysisofpatientreportedoutcomesintheapprovalofnoveloncologydrugsintheunitedstates20172022researchincontext AT kaiyuanguo analysisofpatientreportedoutcomesintheapprovalofnoveloncologydrugsintheunitedstates20172022researchincontext AT yili analysisofpatientreportedoutcomesintheapprovalofnoveloncologydrugsintheunitedstates20172022researchincontext AT guanqiaoli analysisofpatientreportedoutcomesintheapprovalofnoveloncologydrugsintheunitedstates20172022researchincontext AT hongzhang analysisofpatientreportedoutcomesintheapprovalofnoveloncologydrugsintheunitedstates20172022researchincontext AT jiaxuanyang analysisofpatientreportedoutcomesintheapprovalofnoveloncologydrugsintheunitedstates20172022researchincontext AT yangliu analysisofpatientreportedoutcomesintheapprovalofnoveloncologydrugsintheunitedstates20172022researchincontext AT chenyin analysisofpatientreportedoutcomesintheapprovalofnoveloncologydrugsintheunitedstates20172022researchincontext AT senliu analysisofpatientreportedoutcomesintheapprovalofnoveloncologydrugsintheunitedstates20172022researchincontext AT songmeixie analysisofpatientreportedoutcomesintheapprovalofnoveloncologydrugsintheunitedstates20172022researchincontext AT xiaoyuanchen analysisofpatientreportedoutcomesintheapprovalofnoveloncologydrugsintheunitedstates20172022researchincontext |