More than 5,700 trials worldwide are investigating PD-1/PD-L1 inhibitors, and new trials of these therapies were of combination regimens.
Oncology trials are a significant portion of all clinical trials. Oncology trial starts reached historically high levels in 2021, up 56% from 2016 and mostly focused on rare cancer indications, according to a new report by IQVIA Institute for Human Data Science.
“In a record-setting year, more novel cancer medicines became available for the first time than in any year in history, and many of them employ immunology or precision biomarkers to transform the way patients are treated,” IQVIA researchers wrote.
IQVIA’s analysis found that most cancer research focuses on metastatic or advanced cancers, but early cancer trials and vaccines have more than doubled in 10 years. About 80% of ongoing trials have molecular targets that require studies in pediatric patients.
In biotherapeutics, the oncology pipeline is focused on cell therapies, especially in hematological cancers, although hematological cancers are a declining share of oncology trials. Additionally, antibody drug conjugates (a class of targeted biopharmaceuticals) and bispecific antibodies (antibodies that bind to two different sites on cancer cells) are emerging as well.
More than 5,700 trials worldwide are investigating PD-1/PD-L1 inhibitors, according to IQVIA. The FDA approved the first PD-1/L1 checkpoint inhibitor, Merck’s Keytruda (pembrolizumab), for patients with melanoma in 2014, and since then five additional PD-1/L1 inhibitors — Pfizer’s Bavencio (avelumab), AstraZeneca’s Imfinzi (durvalumab), Regeneron/Sanofi’s Libtayo (cemiplimab), BMS’s Opdivo (nivolumab), Roche’s Tecentriq (atezolizumab) have been approved across a range of hematological cancers and solid tumors. In addition, the FDA has also approved the another checkpoint inhibitor focused on CTLA-4 , BMS’s Yervoy (ipilimumab).
Nearly 90% of clinical trials with PD-1/L1 inhibitors that started in 2021 are investigating their use in combination with other drugs while monotherapy trials have been declining. This is a big market, with market research company Technavio predicting that the market for PD-1/PD-L1 inhibitors could reach $29.97 billion by 2025.
IQVIA research also assessed trial success rates, defined as those that reach the next phase of development. Oncology had one of the lowest success rates of all therapies, and those trials that addressed rare and hematological cancers had higher success rates than non-rare cancer or solid tumors.
Since 2010, oncology trials have included an increasing number of endpoints. Overall, the complexity of these trials has been declining since 2015, mostly due to a drop in the number of sites used because of the COVID-19 pandemic. IQVIA found, however, that the novel designs, such as adaptive, basket, umbrella, and master protocols, have quadrupled in the last decade. And while these designs may be more complex, they provide program efficiencies and can shorten timelines.
The number of patients recruited for these trials has include globally, growing by 10% from 2020 to 2021 and 59% over the last 10 years. Globally, about 40,000 patients are enrolled in oncology clinical trials.
In 2021, 22 novel oncology drugs were launched in the United States, and in the last five years, there were 83 unique new cancer medicines launched. Oncology drugs increasingly receive accelerated approvals, orphan or breakthrough designations. Of those approved last year, 64% were approved through accelerated approval, meaning confirmatory trials are required. This is up from 36% in 2017.
And while many of those therapies launched year were significantly, many were based on limited trial evidence, with single arm trials and evidence submitted from earlier phase studies.
Significant last year was the FDA’s re-evaluation of previous accelerated approvals, which saw nine indications either withdrawn or revoked.