Comparison of lung cancer clinical trials treatment options and standard practice in Brazilian national health system over the last decade.

Abstract

e18678 Background: New cancer drugs for metastatic non-small cell (NSCLC) and small cell lung cancer (SCLC) have been approved worldwide in the past decade, but expensive pricing has led to unequal access. Despite being categorized as an upper-middle income country, Brazil's public health system is underfunded and has faced difficulties in incorporating new cancer treatments, which is concerning as two-thirds of the population depend on the public health system for their cancer-related medical needs. We hypothesized that the disparity between the standard of care (SOC) provided in developed nations and the prevailing practices in Brazil's public healthcare system is widening. Methods: We conducted a search of the ClinicalTrials.gov database from January 1st, 2012 to December 31th, 2021, for all phase III clinical trials on metastatic lung cancer that had at least one clinical site located in Brazil. We collected data on the experimental arm therapies, planned outcomes, and control arm therapies (used as a proxy for the standard of care worldwide). We then compared the control-arm drug therapy at the trial's start date with the standard practice for lung cancer in the Brazilian Unified Health System (SUS) at that time, using available governmental protocols on lung cancer (PCDT) and recent approvals from the technology health assessment committee (CONITEC). Additionally, we assessed the potential eligibility of patients treating in SUS for second-line therapy trials based on prior required treatments to trial enrollment. We analyzed two time periods: 2012-2016 and 2017-2021. Results: Out of 86 screened trials, 64 were included. 86% of them were on NSCLC, and 67% on first-line settings. Almost half had a biomarker inclusion criterion for enrollment. Immunotherapy was studied most frequently (52%), followed by targeted therapies (36%) and antibody-conjugate drugs (ADC) (9.4%). PD(L)1 was the target of 33% of drug therapies. Compared to 2012-2017, in the 2018-2021 period we observed an increase in available clinical trials (24 vs 40) with similar rates of NSCLC (83% vs. 88%, p = 0.7) and first-line therapies (68 vs 67%, p > 0.9). The proportion of trials with control group therapies better than the standard practice available at the time increased from 8.3% to 38% in the recent time-period (p = 0.011). Among second-line therapy trials, the proportion of trials with mandatory previous therapy unavailable in SUS rose numerically from 12% to 54% (p = 0.085). Conclusions: While the number of trials increased over time, there was a rising proportion of control group therapies better than available standard practice in Brazil. The widening difference between drug therapies in clinical trials and standard cancer treatments creates difficult ethical quandaries for oncologists enrolling patients, and also impacts patient eligibility in advanced settings.