Abstract 82P
Background
Patients in a low-middle income country such as India do not have access to adequate clinical trials. Hence despite many newer drugs showing significant impact in cancer disease control in clinical trials they are not accessible to our patients. Here we present the impact of these drugs for the few of these patients who received them through compassionate access.
Methods
We conducted a retrospective audit of patients who received compassionate access drugs at the Medical Oncology Department, Tata Memorial Hospital, Mumbai, between August 2020 and July 2023. We analysed the data primarily for progression free survival, overall survival, and safety profile.
Results
A total of 36 patients received compassionate access drugs in the specified period. Of these 36 patients, 78.4% (n=29) had metastatic NSCLC, 8.1% (n=3) had metastatic medullary thyroid cancer with RET fusion, 5.4% (n=2) had metastatic bladder cancer with FGFR alteration, 2.7% (n=1) had metastatic nasal cavity adenocarcinoma with EGFR exon 20 insertion, and 2.7% (n=1) had metastatic vaginal skene gland adenocarcinoma with RET fusion. Among 29 patients with metastatic NSCLC, 34.5% (n=10) had MET alteration, 27.5%% (n=8) had EGFR exon 20 insertion, 24.1% (n=7) had RET gene fusion, 10.3% (n=3) had ROS 1 gene fusion, and 3.4% (n=1) had NTRT gene fusion. Targeted drugs received included selpercatinib 25% (n=9), capmatinib and tepotinib each 13.9% (n=5), amivantamab, mobocertinib and entrectinib each 11.1% (n=4), pralsetinib and erdafitinib each 5.5% (n=2) and poziotinib 2.6% (n=1). Median of 3.1 lines of therapy were received by the patients before starting the compassionate access drug. Median progression-free survival was 12.18 months (95% CI 4.27-20.10). Median overall survival was 42.28 months (95% CI 20.74-63.82). Median duration of response was 21.88 months. Overall, 86% patients had any grade of drug-related adverse event, but only 25% (n=9) had grade 3, and no grade 4 or grade 5 adverse events were seen.
Conclusions
Those who received compassionate access drugs have shown significant disease control and survival improvement even though they received this drug after multiple lines of therapy with fair tolerance. So, it is worth increasing the access to these drugs to populations from low-middle income country such as India.
Clinical trial identification
Editorial acknowledgement
Legal entity responsible for the study
The authors.
Funding
Has not received any funding.
Disclosure
All authors have declared no conflicts of interest.