In a conversation with my colleague, I was telling him that it would be nice to have more real world practice data.
It would be interesting to see if results from real world practice is similar (or not) to those reported in clinical trials, which are conducted and tightly monitored for treatment effectiveness and patient safety.
Just to be clear, the main reason for clinical research, is to reflect the treatment effectiveness and safety in target population (eg. lung cancer patients) in an unbiased manner.
In phase 3 clinical trials, the new study drug is always compared to ‘standard of care’ treatment.
Patients who participate in clinical trial are randomised into either investigational product (IP) or ‘standard of care’ treatment but patients themselves and the doctors won’t know which treatment they are getting to prevent bias during assessment.
Real World Data
Real world practice data is data collected from large centre(s) and published after the medication had been approved for commercial use by government regulatory authorities such as FDA, European Medicines Agency (EMA) and in Malaysia, the National Pharmaceutical Regulatory Agency (NPRA).
Few real world practice data are available.
For example, in two large phase 3 clinical research trials, LUX-Lung 3 and in LUX-Lung 6, the median/ average overall survival for stage 4 lung cancer patients who were on afatinib (2nd generation EGFR-TKI) was 23.1 – 28.2 months.
There was a real world practice data for afatinib from National Cancer Centre Singapore, 2017 which showed median/ average survival duration of 29.6 months (95% CI: 24.0 to 46.4 months) for 125 patients who were on the medication.
It showed that both results from clinical research trials and real world practice were at least comparable (if not better) and gave us clinicians confident when using it to treat our patients.
- Average/ median progression free survival (PFS) for total 165 patients was around 19 months.
- EGFR mutation Del 19 vs EGFR mutation L858R (average/ median PFS): 19.1 vs 15.1 months (works better for mutation Del 19).
- Overall survival rate at 12 & 24 months: 91.0% & 70.7%, respectively.
The full paper above is interesting and I suggest those who is interested to read more about it.