Nearly 75% of pivotal cancer trials in past 5 years exclude people living with HIV

Despite recent ASCO and FDA guidance recommending the inclusion of people living with HIV in pivotal cancer trials, nearly 75% of trials over the past 5 years excluded this population, according to study results.

Finding presented at ASCO Annual Meeting showed that trials for blood cancers and immunomodulatory agents, as well as trials funded by industry, were more likely to exclude people living with HIV.

“The topic is quite important because in the general U.S. population the rate of HIV is about 0.3% to 0.4%, which is about 1.2 million people at least, and 70% of trials, according to my research, are directly excluding these people,” Alberto Giovanni Leone, MD, an HIV/oncology fellow at Chelsea and Westminster Hospital NHS Foundation Trust, told Healio. “Prospective data are lacking, and because of that, we do not know if all the treatments that were FDA-approved in the last 5 years have the same efficacy and safety profile in these people.

“We don’t think that there is a biological rationale behind these exclusions,” he added. “The only way we have to use these drugs for this population is thanks to retrospective data that we can retrieve some safety and efficacy profile, but this means that there will be a gap of 5 or 6 years before we can safely administer these drugs for these people.”

Despite cancer being the most common cause of death for people living with HIV in high-income countries, trial protocols typically exclude this population without justification, according to study background.

Prior research by ASCO found that just 11% of clinical trials supporting FDA cancer therapy approvals from 2010 to 2014 allowed for inclusion of people living with HIV.

Such exclusions cause clinical uncertainty regarding the efficacy and safety of new cancer treatments for this population, which is particularly notable considering their increased risk for developing cancer compared with the general population.

Both ASCO, in 2017, and FDA, in 2020, issued specific recommendations to encourage inclusion of people living with HIV in cancer clinical trials.

To investigate the effectiveness of this guidance, Leone and colleagues conducted a study to review all new-FDA approved indications from 2020 through 2024 for inclusion of people living with HIV.

The researchers identified 268 clinical trials — with 253 new therapy indications — and included 252 clinical trials in analysis.

According to results, 72.6% of pivotal cancer clinical trials still excluded people living with HIV.

However, the inclusion rate significantly increased in protocols of studies published after 2020 vs. before 2020 (48% vs. 25.1%; OR = 2.75; 95% CI, 1.89-6.38).

Pivotal trials for blood cancers were significantly more likely to exclude people with HIV compared with trials for solid cancers (OR = 3.33; 95% CI, 1.6-6.94), as were trials of immunomodulatory agents compared with other therapies (OR = 4.73; 95% CI, 2.15-10.12).

Trials funded only by industry also were more likely to exclude this population compared with non-industry funded trials (OR = 2.6; 95% CI, 1.25-5.43).

Despite the improvement in inclusion of people living with HIV in pivotal cancer trials after ASCO and FDA guidance, Leone said much remains to be done.

“My opinion is it’s not enough,” he told Healio. “What we can do is [work] with pharmaceutical companies, because most of these pivotal cancer trials were industry-funded, to understand why they continue to exclude these people.

“We need more quantitative data just to be able to push more,” he added. “Pharmaceutical companies focus on academics to implement their protocols.”

By Matthew Shinkle

Source: Leone AG, et al. Abstract 1517. Presented at: ASCO Annual Meeting; May 30-June 3, 2025; Chicago.