Real World Data in Medical Research
What is real world data vs. on-paper data? Scientists create experiments in laboratories and present the best data they can in published papers for everyone to share. That’s paper data. In real life, clinical doctors prescribe the medication and the end results for people are the real-world data. Most of the time, this section highlights the “in laboratory” data. But here, we will discuss two examples of real world data.
In this first paper, the authors went back into the electronic data and looked for the answer to one question: “do our community medical systems follow the guidelines for biomarker testing if you are diagnosed with advanced non-small cell lung cancer (aNSCLC)”. ALK test use-rates in National Comprehensive Cancer Network (NCCN) ALK testing‐eligible patients rose from 59.5% in 2012 to 84.1% in 2019. This increase in ALK testing was also observed in patients ineligible for testing, ranging from 15.6% in 2012 to 50.8% in 2019. The entire population screened was 41,728 patients. They found that the histology type (squamous or non-squamous) and smoking history (yes or no) affected the frequency of testing for biomarkers. Other factors such as demographics, and the Eastern Cooperative Oncology Group (ECOG) performance score (how patients are functioning in terms of ability to take care of themselves, daily activities, and physical ability) at initial diagnosis, also affected the frequency of biomarker testing for patients. The conclusion is that one out of five of the patients eligible for ALK testing remained untested and potentially missed therapeutic options.
https://theoncologist.onlinelibrary.wiley.com/doi/abs/10.1002/onco.13779
In the second example, a German group studied the results of their Early Access Program (EAP) with third generation ALK and ROS1 inhibitor, Lorlatinib. Fifty-two patients with documented treatment failure of all approved ALK/ROS1-specific therapies, or with resistance mutations not covered by approved inhibitors, or with leptomeningeal carcinomatosis were enrolled and analyzed. The median age was 57 (ranging from 32-81 years old). 71% of the patients were ALK+ and 29% were ROS1+. Ten of 26 patients had the G1202R or G2032R mutations. Eleven of 39 patients showed the TP53 mutation. Thirty-six patients had brain metastases and 9% had leptomeningeal carcinomatosis. The median number of specific TKIs was 3 prior to enrolling. The response rate to Lorlatinib in this population was 54%. The median progression-free survival (PFS) was 8.0 months. Overall survival since primary diagnosis has reached 79.6 months (6.6 years!!!). Neither prior treatments with second generation TKIs, nor brain metastases had any significant influence on PFS or overall survival. So, the conclusion from this group is that Lorlatinib in heavily pretreated patients, those with G1202R and G2032R resistance mutations, and those with brain metastases or leptomeningeal carcinomatosis did provide a clinically meaningful option for patients with resistance mutations not covered by other targeted therapies.
https://journals.sagepub.com/doi/full/10.1177/1758835920980558
Author: Alice Chou