Abstract
Purpose
While some studies show improved outcomes in clinical trial participants as compared to non-participants, existence of such a trial effect has not been proved precisely.
Methods
This was a prospective cohort study to compare the prognoses for participants in the randomized controlled trial (SELECT BC) and non-participants. SELECT BC compared S-1 and taxane as first-line treatment for metastatic breast cancer. Non-participants were all patients who met the eligibility criteria of SELECT BC and who had been requested to participate in that trial by attending doctors and declined. The study aimed to compare the prognoses between participants and non-participants. The primary endpoint was median overall survival.
Results
The median OS in participants was significantly superior to that in non-participants with a statistically significant difference (36.8 months vs. 25.2 months. HR 1.48, p = 0.022). A similar result was obtained when only patients who received the same chemotherapy (S-1 or taxane) used in SELECT BC after declining participation were assumed as non-participants (36.8 months vs. 22.0 months. HR 2.03, p = 0.006).
Conclusions
This study may suggest the existence of a trial effect, in which, for a given treatment, participation in a clinical trial is associated with a better outcome.
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Acknowledgements
We thank all investigators and their collaborators who were dedicated to this study.
Funding
This study was sponsored by the Comprehensive Support Project for Oncology Research of the Public Health Research Foundation. The research funding was provided to the Comprehensive Support Project for Oncology Research by Taiho Pharmaceutical under the study contract. Taiho Pharmaceutical took no part in this study other than providing information relevant to proper use of the study drug.
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SO has received remuneration from Chugai, Eisai Pfizer, AstraZeneca, Kyowa Hakko Kirin, and Taiho; funding from Taiho, Eisai, Chugai, and Daiichi Sankyo. HM has received remuneration from Astra Zeneca, Pfizer, Daiichi Sankyo, Taiho, and Novartis; funding from Japanese government, Eisai, Daiichi Sankyo, and Nippon Kayaku, outside the submitted work. YM has received remuneration from Astra Zeneca, Chugai, Eisai, Taiho, Eli Lilly, Kyowa Hakko Kirin, and Novartis; funding from Eisai, Daiichi Sankyo, Kyowa Hakko Kirin, Chugai, Taiho, Eli Lilly, and MSD. HD has received funding from Eisai and Kyowa Hakko Kirin. YO has received consultant fee from Chugai, Daiichi Sankyo, and Sanofi. The other authors declare no competing interests.
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All procedures performed in study involving participants were in accordance with the ethical standards of the institutional or national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards.
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Ohno, S., Mukai, H., Narui, K. et al. Participants in a randomized controlled trial had longer overall survival than non-participants: a prospective cohort study. Breast Cancer Res Treat 176, 631–635 (2019). https://doi.org/10.1007/s10549-019-05276-y
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Keywords
- Overall survival
- Prospective cohort study
- Randomized control trial
- SELECT BC
- Trial effect