Real-world results may differ from clinical trial results for some drugs approved to treat gastrointestinal cancers, according to research published in Clinical Oncology.

Researchers found that EGFR inhibitors and sorafenib performed as expected in the real world, but some chemotherapy drugs did not. 

For this study, researchers used the Systemic Anti-Cancer Therapy (SACT) dataset, which includes information on all patients receiving chemotherapy in England. The researchers noted that SACT data has largely gone unpublished, “despite concerns that real-world effectiveness of medicines may be inferior to that seen in clinical trials.”

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The researchers used local SACT data to evaluate real-world outcomes in patients with gastrointestinal cancers who were treated from May 2016 to March 2021. The team analyzed data on 13,422 patients — 1130 who had colorectal cancer (CRC), 299 who had pancreatic adenocarcinoma, and 45 who had hepatocellular carcinoma (HCC). 


The researchers first compared real-world data and clinical trial data for FOLFIRINOX (folinic acid, fluorouracil, irinotecan, and oxaliplatin), gemcitabine alone, and gemcitabine plus nab-paclitaxel as first-line treatment for metastatic pancreatic cancer.

In general, the overall survival (OS) rates were lower in the real world than in clinical trials. Furthermore, the real-world data suggest that adding nab-paclitaxel to gemcitabine may not provide an OS benefit at 6 months.

The 6-month OS rate with gemcitabine alone was 37% in the real world and 46% according to trial data. The 12-month OS rate was 7.6% and 18%, respectively. 

The 6-month OS rate with gemcitabine plus nab-paclitaxel was 36% in the real world and 67% according to trial data. The 12-month OS rate was 24% and 35%, respectively.

The 6-month OS rate with FOLFIRINOX was 71% in the real world and 75.8% according to trial data. The 12-month OS rate was 39% and 48.4%, respectively. 

The researchers also assessed sorafenib in HCC and found similar results between trial and real-world data. The 12-month OS rate was 44% in the SHARP trial and 43% in the real-world cohort.

When the researchers evaluated panitumumab or cetuximab plus chemotherapy as first-line treatment for metastatic, RAS wild-type CRC, trial results were slightly better than real-world results. 

The median OS with panitumumab plus FOLFOX (folinic acid, fluorouracil and oxaliplatin) was 23.9 months in the PRIME trial and 20 months in the real world.

The median OS with cetuximab plus FOLFOX was 25.8 months in a CECOG trial and 20 months in the real world. The median OS with cetuximab plus FOLFIRI (folinic acid, fluorouracil, and irinotecan) was 28.7 months in the FIRE-3 trial and 19 months in the real world.

When the researchers evaluated trifluridine/tipiracil as third-line chemotherapy for metastatic CRC, they found the 12-month OS rate was 27% in a trial and 29% in the real world. 

“Our results support the publication of national outcome data,” the researchers wrote. “If these results are confirmed [in] a larger cohort, it would support the reappraisal of certain drugs and provide further evidence to clinicians and patients when deciding the best treatment.”

Disclosures: One study author declared a relationship with AstraZeneca, and one is employed by the National Health Service in England. Please see the original reference for a full list of disclosures.  


Wadd N, Peedell C, Polwart C. Real-world assessment of cancer drugs using local data uploaded to the Systemic Anti-Cancer Therapy dataset in England. Clin Oncol. Published online May 16, 2022. doi:10.1016/j.clon.2022.04.012