TY - JOUR
T1 - Inclusion of Patient-Reported Outcomes in Adolescent and Young Adult Phase III Therapeutic Trials
T2 - An Analysis of Cancer Clinical Trials Registered on ClinicalTrials.gov
AU - Berkman, Amy M.
AU - Murphy, Karly M.
AU - Siembida, Elizabeth J.
AU - Lau, Nancy
AU - Geng, Yimin
AU - Parsons, Susan K.
AU - Salsman, John M.
AU - Roth, Michael E.
N1 - Funding Information:
Conflict of Interest Disclosures: Dr Berkman reported being supported by grant NIH R38-HL143612 during the course of this study. Dr Murphy reported being supported by grant NCI T32 CA193193 and NCI T32 CA122061 from the National Cancer Institute during the course of this study. Dr Lau reported being funded as an Implementation Science Scholar through the National Heart, Lung, and Blood Institute at the National Institutes of Health under grant 5K12 HL137940-02 during the course of this study. Dr Parsons reported receiving consultant fees for Seattle Genetics, receiving grants from the Leukemia & Lymphoma Society, and is on the advisory board for the Dana Farber/Harvard Cancer Center outside the submitted work. Dr Salsman reported receiving funding from the National Cancer Institute under grants UG1 CA189828-07S1 and R01 CA218398 during the course of this study, and being on the advisory board for True North Treks and Team Maggie outside the submitted work. No other conflicts were reported.
Funding Information:
Funding/Support: This work was supported by grants U10-CA180886 (MR), P30 CA016672 (MR), R38-HL143612 (AB), UG1-CA189955 (MR, SP), CA193193 (KM), CA122061 (KM), 5K12 HL137940-02 (NL), UG1 CA189828-07S1 (JS), and R01 CA218398 (JS) from the National Cancer Institute at the National Institutes of Health .
Publisher Copyright:
© 2021 ISPOR–The Professional Society for Health Economics and Outcomes Research
PY - 2021/12
Y1 - 2021/12
N2 - Objectives: There is a paucity of research on the impact of cancer treatment on the health-related quality of life (HRQOL) of adolescent and young adult (AYA) patients with cancer. Patient-reported outcomes (PROs) are self-report measures used to assess HRQOL and symptom burden. The extent to which PROs have been included in trials that include common AYA cancer types has not been previously assessed. Methods: Therapeutic phase 3 trials among common AYA cancer types (Hodgkin lymphoma, non-Hodgkin lymphoma, acute lymphoblastic leukemia, sarcomas, and germ cell tumors) initiated between 2007 and 2020 were identified on ClinicalTrials.gov. The proportions and characteristics of trials including a PRO endpoint were assessed. For comparison with an older population, the proportion of breast and colorectal therapeutic phase 3 trials including PRO endpoints were assessed. Results: Eighty-seven studies met the inclusion criteria. Overall, 20.7% of therapeutic phase 3 AYA trials included a PRO endpoint, and only one trial published PRO data. Germ cell tumors (42.9%) and non-Hodgkin lymphoma (40%) trials had the highest proportions of PRO inclusion. The European Organization for Research and Treatment of Cancer generic, cancer-specific quality of life questionnaire was the most commonly used PRO measure; nevertheless, the measures used varied within and between cancer types. The proportion of trials including a PRO endpoint did not change significantly between 2007 to 2013 and 2014 to 2020 (18.6% vs 22.7%, P=.79). Conclusions: Few therapeutic phase 3 AYA cancer trials include PRO endpoints, fewer publish PRO data, and there is no homogeneity in the measures administered. Therapeutic trials represent an underused opportunity to capture PRO data in the AYA population with the goal of improving HRQOL outcomes.
AB - Objectives: There is a paucity of research on the impact of cancer treatment on the health-related quality of life (HRQOL) of adolescent and young adult (AYA) patients with cancer. Patient-reported outcomes (PROs) are self-report measures used to assess HRQOL and symptom burden. The extent to which PROs have been included in trials that include common AYA cancer types has not been previously assessed. Methods: Therapeutic phase 3 trials among common AYA cancer types (Hodgkin lymphoma, non-Hodgkin lymphoma, acute lymphoblastic leukemia, sarcomas, and germ cell tumors) initiated between 2007 and 2020 were identified on ClinicalTrials.gov. The proportions and characteristics of trials including a PRO endpoint were assessed. For comparison with an older population, the proportion of breast and colorectal therapeutic phase 3 trials including PRO endpoints were assessed. Results: Eighty-seven studies met the inclusion criteria. Overall, 20.7% of therapeutic phase 3 AYA trials included a PRO endpoint, and only one trial published PRO data. Germ cell tumors (42.9%) and non-Hodgkin lymphoma (40%) trials had the highest proportions of PRO inclusion. The European Organization for Research and Treatment of Cancer generic, cancer-specific quality of life questionnaire was the most commonly used PRO measure; nevertheless, the measures used varied within and between cancer types. The proportion of trials including a PRO endpoint did not change significantly between 2007 to 2013 and 2014 to 2020 (18.6% vs 22.7%, P=.79). Conclusions: Few therapeutic phase 3 AYA cancer trials include PRO endpoints, fewer publish PRO data, and there is no homogeneity in the measures administered. Therapeutic trials represent an underused opportunity to capture PRO data in the AYA population with the goal of improving HRQOL outcomes.
KW - adolescent and young adult cancer
KW - cancer clinical trials
KW - patient-reported outcomes
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U2 - 10.1016/j.jval.2021.06.012
DO - 10.1016/j.jval.2021.06.012
M3 - Article
C2 - 34838280
AN - SCOPUS:85112234124
SN - 1098-3015
VL - 24
SP - 1820
EP - 1827
JO - Value in Health
JF - Value in Health
IS - 12
ER -