Health-related quality of life outcomes reporting associated with FDA approvals in haematology and oncology
Objective Health-related quality of life (HRQoL) outcomes are important in making clinical and policy decisions. This study aimed to examine the HRQoL reporting in cancer drug trials leading to Food and Drug Administration (FDA) approvals.Methods and analysis This retrospective cohort study analysed...
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| Format: | Article |
| Language: | English |
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BMJ Publishing Group
2024-07-01
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| Series: | BMJ Oncology |
| Online Access: | https://bmjoncology.bmj.com/content/3/1/e000369.full |
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| author | Bishal Gyawali Pallawi Torka Kristopher Attwood Medhavi Gupta Othman Salim Akhtar Bhavyaa Bahl Angel Mier-Hicks Kayla Catalfamo |
| author_facet | Bishal Gyawali Pallawi Torka Kristopher Attwood Medhavi Gupta Othman Salim Akhtar Bhavyaa Bahl Angel Mier-Hicks Kayla Catalfamo |
| author_sort | Bishal Gyawali |
| collection | DOAJ |
| description | Objective Health-related quality of life (HRQoL) outcomes are important in making clinical and policy decisions. This study aimed to examine the HRQoL reporting in cancer drug trials leading to Food and Drug Administration (FDA) approvals.Methods and analysis This retrospective cohort study analysed HRQoL data for trials leading to FDA approvals between July 2015 and May 2020. Proportion of included trials that reported HRQoL, latency between FDA approval and first report of HRQoL data, HRQoL outcomes, and their correlation with OS (overall survival) and PFS (progression-free survival) were analysed.Results Of the 233 trials associated with 207 FDA approvals, HRQoL was reported in 50% of trials, of which only 42% had the data reported by the time of FDA approval. There were no changes in frequency of HRQoL reporting between 2015 and 2020. HRQoL data were first reported in the primary publication in only 30% trials. Of the 115 trials with HRQoL data available, HRQoL improved in 43%, remained stable in 53% and worsened in 4% of trials. Among the trials that led to FDA approvals based on surrogate endpoints (79%), HRQoL was reported in 45% and improved only in 18% trials. There was no association between OS and PFS benefit and HRQoL outcomes.Conclusion Rates of HRQoL reporting were suboptimal in trials that led to FDA approvals with no improvements seen between 2015 and 2020. HRQoL reporting was often delayed and not presented in the primary publication. HRQoL reporting was further sparse in trials with approvals based on surrogate endpoints and HRQoL improved in only a minority of them. |
| format | Article |
| id | doaj-art-6c90cd6e028a4986af6c7a2ef5131964 |
| institution | Kabale University |
| issn | 2752-7948 |
| language | English |
| publishDate | 2024-07-01 |
| publisher | BMJ Publishing Group |
| record_format | Article |
| series | BMJ Oncology |
| spelling | doaj-art-6c90cd6e028a4986af6c7a2ef51319642025-01-30T06:55:09ZengBMJ Publishing GroupBMJ Oncology2752-79482024-07-013110.1136/bmjonc-2024-000369Health-related quality of life outcomes reporting associated with FDA approvals in haematology and oncologyBishal Gyawali0Pallawi Torka1Kristopher Attwood2Medhavi Gupta3Othman Salim Akhtar4Bhavyaa Bahl5Angel Mier-Hicks6Kayla Catalfamo7Department of Oncology, Queen`s University, Kingston, Ontario, CanadaMemorial Sloan Kettering Cancer Center, New York, New York, USADepartment of Biostatistics and Bioinformatics, Roswell Park Comprehensive Cancer Center, Buffalo, New York, USAProgram in Women`s Oncology, Brown University, Providence, Rhode Island, USAMedical College of Wisconsin, Milwaukee, Wisconsin, USAUniversity of Colorado System, Denver, Colorado, USATexas Oncology San Antonio Area, San Antonio, Texas, USARoswell Park Comprehensive Cancer Center, Buffalo, New York, USAObjective Health-related quality of life (HRQoL) outcomes are important in making clinical and policy decisions. This study aimed to examine the HRQoL reporting in cancer drug trials leading to Food and Drug Administration (FDA) approvals.Methods and analysis This retrospective cohort study analysed HRQoL data for trials leading to FDA approvals between July 2015 and May 2020. Proportion of included trials that reported HRQoL, latency between FDA approval and first report of HRQoL data, HRQoL outcomes, and their correlation with OS (overall survival) and PFS (progression-free survival) were analysed.Results Of the 233 trials associated with 207 FDA approvals, HRQoL was reported in 50% of trials, of which only 42% had the data reported by the time of FDA approval. There were no changes in frequency of HRQoL reporting between 2015 and 2020. HRQoL data were first reported in the primary publication in only 30% trials. Of the 115 trials with HRQoL data available, HRQoL improved in 43%, remained stable in 53% and worsened in 4% of trials. Among the trials that led to FDA approvals based on surrogate endpoints (79%), HRQoL was reported in 45% and improved only in 18% trials. There was no association between OS and PFS benefit and HRQoL outcomes.Conclusion Rates of HRQoL reporting were suboptimal in trials that led to FDA approvals with no improvements seen between 2015 and 2020. HRQoL reporting was often delayed and not presented in the primary publication. HRQoL reporting was further sparse in trials with approvals based on surrogate endpoints and HRQoL improved in only a minority of them.https://bmjoncology.bmj.com/content/3/1/e000369.full |
| spellingShingle | Bishal Gyawali Pallawi Torka Kristopher Attwood Medhavi Gupta Othman Salim Akhtar Bhavyaa Bahl Angel Mier-Hicks Kayla Catalfamo Health-related quality of life outcomes reporting associated with FDA approvals in haematology and oncology BMJ Oncology |
| title | Health-related quality of life outcomes reporting associated with FDA approvals in haematology and oncology |
| title_full | Health-related quality of life outcomes reporting associated with FDA approvals in haematology and oncology |
| title_fullStr | Health-related quality of life outcomes reporting associated with FDA approvals in haematology and oncology |
| title_full_unstemmed | Health-related quality of life outcomes reporting associated with FDA approvals in haematology and oncology |
| title_short | Health-related quality of life outcomes reporting associated with FDA approvals in haematology and oncology |
| title_sort | health related quality of life outcomes reporting associated with fda approvals in haematology and oncology |
| url | https://bmjoncology.bmj.com/content/3/1/e000369.full |
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