ESMO Real World Data and Digital Oncology最新文献

Background and purpose

Materials and methods

Results

Conclusions

Background

Materials and methods

Results

Conclusions

Background

Patients and methods

Results

Conclusions

Background

Materials and methods

Results

Conclusion

Background

Limited real-world data exist on managing locally advanced/metastatic urothelial carcinoma (la/mUC), crucial for predicting outcomes and integrating new treatments. This study explores la/mUC in Northern Spain.

Methods

Retrospective observational analysis from 16 hospitals across 12 provinces. The study population included adult patients diagnosed with la/mUC (1 January 2007-31 December 2019). Median overall survival and progression-free survival were determined using the Kaplan–Meier method.

Results

A total of 1230 patients were included, 83% were males, median age was 68, and 70% had a smoking history. The most common primary tumor was bladder (89.2%) followed by renal pelvis (6.4%), ureter (4.3%), and urethra (0.1%) and most of them (91%) were pure urothelial tumors. Cystectomy was the predominant treatment of localized disease (69%) and 30% received perioperative chemotherapy. Twenty-four percent of la/mUC patients never received systemic therapy and out of 934 patients treated with first-line (1L) therapy, 55% were fit for cisplatin. Progression to further lines of treatment was poor, only 53% (n = 492) progressed to receive a second line and 22% (n = 209) to a third line. Median overall survival (95% confidence interval) was 12.2 (11.3-12.9) months for the entire cohort and 14.5 and 10.8 months for patients who received first-line cisplatin or carboplatin-based chemotherapy, respectively. No unexpected toxicity was reported.

Conclusions

This Spanish real-world data analysis echoes previous findings and highlights unmet needs, including the proportion of patients not receiving systemic treatment and the limited progression to subsequent lines of therapy, hampering access to new effective treatments.

Background

Understanding of comparative efficacy of treatments can inform clinical decision-making. This study compared overall survival (OS) and progression-free survival (PFS) across patients with metastatic BRAFV600-mutant melanoma initiating encorafenib + binimetinib (ENCO + BINI), dabrafenib + trametinib (DAB + TRAM), and vemurafenib + cobimetinib (VEM + COBI).

Materials and methods

In this hybrid study, we contextualized OS and PFS between patients with metastatic BRAF V600E/K-mutant melanoma receiving ENCO + BINI in the phase III COLUMBUS trial (enrollment: December 2013 to April 2015) versus real-world data (RWD) from a nationwide electronic health record-derived deidentified database (treatment initiation: 2014-2021). After observing consistent outcomes across trial and RWD, we compared OS and PFS for a pooled ENCO + BINI cohort across these settings versus comparable DAB + TRAM and VEM + COBI cohorts from the real-world database.

Results

Of 716 patients [ENCO + BINI (n = 275; n = 192 from COLUMBUS, n = 83 from RWD), DAB + TRAM (n = 387), VEM + COBI (n = 54)], mean age was 56-60 years. OS and PFS were similar for ENCO + BINI-treated patients in COLUMBUS and RWD [adjusted hazard ratios: 1.03 (95% CI 0.62-1.72) for OS, 1.10 (0.69-1.75) for PFS]. Relative to the pooled ENCO + BINI group, adjusted OS and PFS were significantly worse for DAB + TRAM [OS: 1.32 (1.05-1.65), PFS: 1.49 (1.20-1.87)] and comparable for VEM + COBI [OS: 1.17 (0.76-1.79), PFS: 1.20 (0.79-1.82)]. Results were similar in comparisons based on the RWD groups alone, when trial data were excluded.

Conclusions

OS and PFS were longer with ENCO + BINI relative to DAB + TRAM and comparable to VEM + COBI, after adjusting for differences in patient profiles. These findings add to evidence informing the use of combination v-Raf murine sarcoma viral oncogene homolog B protein (BRAF)/mitogen-activated protein kinase kinase (MEK) inhibitors in metastatic BRAF V600-mutant melanoma.

Through the Clinical Innovation Network, Japan’s regulatory authorities have enhanced the development of registries that utilize real-world data (RWD). The Ministry of Health, Labour and Welfare has issued guidelines, whereas the Pharmaceuticals and Medical Devices Agency has conducted consultations to manage and verify the integrity of these registries, thus improving the framework for the effective use of RWD. The use of cancer registry data as an external control group has been promoted by regulatory bodies and academic institutions. Given the aforementioned background, several high-quality cancer registries, such as the ‘SCRUM-Japan Registry’, ‘MASTER KEY project’, and ‘GALAXY registry’, have been established. The SCRUM-Japan Registry has been instrumental in achieving the world’s first regulatory approval for human epidermal growth factor receptor 2 (HER2)-positive colorectal cancer, demonstrating the value of regulatory-grade registries in managing rare molecular subtypes. However, the broader adoption of registry data for regulatory use in Japan remains limited, primarily owing to the lack of clear standards for using RWD/real-world evidence (RWE) for drug approval. This uncertainty has made pharmaceutical companies hesitant to use such data for regulatory submissions. This review aimed to elucidate the perspectives and related guidelines of the regulatory authorities concerning cancer registries in Japan. In response, the ‘REALISE study’ was initiated to define the ‘relevancy’ and ‘reliability’ of data necessary for new drug approvals and to develop methodologies to ensure data reliability retrospectively. The findings of this study will inform the creation of draft guidelines aimed at broadening the application of RWD/RWE throughout Japan.

Background

The COVID-19 pandemic impacted outpatient clinic services globally. It is unknown how the pandemic affected the follow-up of surgically treated colorectal cancer (CRC) patients. This population-based study aimed to assess the trends in CRC follow-up consultations before and during the COVID-19 pandemic in the Netherlands.

Materials and methods

Nationwide health care activities data between January 2018 and July 2021 were merged with patient-level data from the Netherlands Cancer Registry of stage I-III CRC patients treated with surgical resection. The number of follow-up consultations per patient per year was calculated, and between-group differences were assessed with descriptive statistics. Trends in the number and setting of follow-up consultations were assessed using joinpoint regression analyses. Out-of-hospital follow-up was defined as written, telephone or video consultations.

Results

In total, 42 970 CRC patients were included. The median number of follow-up consultations per year per patient was 2.9 (interquartile range: 2.0-4.7). The median number of follow-up consultations increased with disease stage (P < 0.001) and was higher for patients <60 years of age (P < 0.001). The total number of follow-up consultations did not change during the study period (P = 0.333). The percentage of out-of-hospital follow-up increased from 23% to 80% between January and April 2020 (P < 0.001), and remained between 48% and 59% until the end of the study period.

Conclusions

This population-based study showed a great increased use of out-of-hospital consultations during CRC follow-up, which predominantly corresponded to the severity of the COVID-19 pandemic. Future studies should assess whether the use of out-of-hospital follow-up consultations has persisted after the pandemic.