PMID- 36345809 OWN - NLM STAT- MEDLINE DCOM- 20230324 LR - 20230519 IS - 1099-1557 (Electronic) IS - 1053-8569 (Print) IS - 1053-8569 (Linking) VI - 32 IP - 4 DP - 2023 Apr TI - Effectiveness research in oncology with electronic health record data: A retrospective cohort study emulating the PALOMA-2 trial. PG - 426-434 LID - 10.1002/pds.5565 [doi] AB - PURPOSE: Oncology electronic health record (EHR) databases have increased in quality and availability over the past decade, yet it remains unclear whether these clinical practice data can be used to conduct reliable comparative effectiveness studies. We sought to emulate a clinical trial with EHR data in the advanced breast cancer population and compare our results against the trial. METHODS: This cohort study used EHR data from US oncology practices. All elements of the study were defined to mimic the PALOMA-2 trial as closely as possible. Patients with hormone-positive, HER-2 negative metastatic breast cancer with no prior treatment for metastatic disease were included. Patients initiating palbociclib and letrozole on the same day following the earliest record of metastasis were compared to those initiating letrozole only. The primary associational measure was the conditional hazard ratio for time-to-next treatment (TTNT). TTNT is well-measured in our data source and amenable for calibration against the randomized study results of the PALOMA-2 trial. We used multiple imputation for several patient characteristics with missing values. RESULTS: There were 3836 study-eligible women with advanced breast cancer. The hazard ratio for TTNT in the observational study (HR: 0.62; 95% CI: 0.56-0.68) was closely aligned with that of the randomized trial (HR: 0.64; 95% CI: 0.52-0.78). CONCLUSIONS: Under our assumptions on missing data and comparability of the two study populations, results from our non-randomized study closely matched that of the randomized trial. Further studies are needed to determine whether EHR data can yield reliable conclusions on treatment effects in oncology. CI - (c) 2022 John Wiley & Sons Ltd. FAU - Merola, David AU - Merola D AD - Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, USA. AD - Department of Epidemiology, Harvard TH Chan School of Public Health, Boston, Massachusetts, USA. FAU - Young, Jessica AU - Young J AD - Department of Epidemiology, Harvard TH Chan School of Public Health, Boston, Massachusetts, USA. AD - Department of Population Medicine, Harvard Medical School & Harvard Pilgrim Health Care Institute, Boston, Massachusetts, USA. AD - CAUSALab, Harvard TH Chan School of Public Health, Boston, Massachusetts, USA. FAU - Schrag, Deborah AU - Schrag D AD - Department of Medicine, Memorial Sloan Kettering Cancer Center, Weill Cornell Medical School, New York, New York, USA. FAU - Lin, Kueiyu Joshua AU - Lin KJ AD - Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, USA. AD - Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA. FAU - Alwardt, Sarah AU - Alwardt S AD - Avalere Health, Washington, District of Columbia, USA. FAU - Schneeweiss, Sebastian AU - Schneeweiss S AUID- ORCID: 0000-0003-2575-467X AD - Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, USA. AD - Department of Epidemiology, Harvard TH Chan School of Public Health, Boston, Massachusetts, USA. LA - eng GR - P30 CA008748/CA/NCI NIH HHS/United States GR - R01 LM012594/LM/NLM NIH HHS/United States PT - Journal Article PT - Observational Study PT - Research Support, Non-U.S. Gov't DEP - 20221114 PL - England TA - Pharmacoepidemiol Drug Saf JT - Pharmacoepidemiology and drug safety JID - 9208369 RN - 7LKK855W8I (Letrozole) RN - EC 2.7.10.1 (Receptor, ErbB-2) SB - IM MH - Humans MH - Female MH - Letrozole/therapeutic use MH - *Electronic Health Records MH - Retrospective Studies MH - Cohort Studies MH - Receptor, ErbB-2 MH - *Breast Neoplasms/drug therapy/pathology MH - Antineoplastic Combined Chemotherapy Protocols/therapeutic use PMC - PMC10038825 MID - NIHMS1853224 OTO - NOTNLM OT - comparative effectiveness OT - electronic health records OT - healthcare databases OT - metastatic breast cancer OT - oncology OT - real-world evidence COIS- Dr. Schneeweiss (ORCID# 0000-0003-2575-467X) is participating in investigator-initiated grants to the Brigham and Women's Hospital from Boehringer Ingelheim unrelated to the topic of this study. He is a consultant to Aetion Inc., a software manufacturer of which he owns equity. His interests were declared, reviewed, and approved by the Brigham and Women's Hospital in accordance with their institutional compliance policies. Dr. Alwardt reports that she is a shareholder of McKesson Corporation. Dr. Merola owns equity in and is an employee of Aetion, Inc. EDAT- 2022/11/09 06:00 MHDA- 2023/03/25 06:00 PMCR- 2023/04/01 CRDT- 2022/11/08 04:52 PHST- 2022/10/31 00:00 [revised] PHST- 2022/05/23 00:00 [received] PHST- 2022/11/02 00:00 [accepted] PHST- 2022/11/09 06:00 [pubmed] PHST- 2023/03/25 06:00 [medline] PHST- 2022/11/08 04:52 [entrez] PHST- 2023/04/01 00:00 [pmc-release] AID - 10.1002/pds.5565 [doi] PST - ppublish SO - Pharmacoepidemiol Drug Saf. 2023 Apr;32(4):426-434. doi: 10.1002/pds.5565. Epub 2022 Nov 14.