Clinical trials involving adult patients conducted within the National Cancer Institute’s National Clinical Trials Network (NCTN) over the past 4 decades are estimated to have extended the lives of U.S. patients with cancer by at least 14.2 million patient-years, according to a new study published by Joseph M. Unger, PhD, and colleagues in the Journal of Clinical Oncology. Importantly, the researchers estimated that the costs of those gains were quite modest: roughly $326 in federal investment for each patient-year added.
The research team also quantified some of the enormous clinical and scientific impacts that the results of NCTN trials have had. Overall, the researchers identified 162 phase III trials conducted since 1980 with positive results. The primary findings from those studies were cited in the scientific literature more than 165,000 times through the end of 2020, and 87.7% of the trials have been referred to as evidence in subsequent cancer care recommendations made in widely used guidelines.
Joseph M. Unger, PhD
“This work provides a clear statement about the impact of publicly funded cancer research,” explained lead study author Dr. Unger, a Southwest Oncology Group (SWOG) health services researcher and biostatistician at the Fred Hutchinson Cancer Center. “When we discuss investments in infrastructure, it’s important to also consider scientific infrastructure. In the [United States], the [National Cancer Institute]–sponsored network groups have been a vital element of the scientific infrastructure of clinical research for decades and have returned incredible value to patients [with cancer] and to the U.S. taxpayer.”
Background
To estimate the patient-years gained from NCTN research, the investigators looked at trials conducted by the Alliance for Clinical Trials in Oncology; the Eastern Cooperative Oncology Group (ECOG)–American College of Radiology Imaging Network (ACRIN) Cancer Research Group; the National Surgical Adjuvant Breast and Bowel Project, the Radiation Therapy Oncology Group; the Gynecologic Oncology Group (NRG) Oncology; and SWOG Cancer Research Network. Together with the Children’s Oncology Group, these groups have been the core of a network that—in conjunction with the National Cancer Institute Community Oncology Research Program—runs clinical trials at more than 2,200 academic and community treatment sites across the United States and beyond. The group has conducted publicly funded research into effective new cancer treatments for more than half a century.
“First and foremost, we commend the generosity and courage of patients who participate in clinical trials,” said Monica M. Bertagnolli, MD, Director of the National Cancer Institute, Professor of Surgery at Harvard Medical School, and a surgical oncologist at the Dana-Farber Brigham Cancer Center. “This work is a clear demonstration that those who do so make a tremendous contribution to society. We also acknowledge the work of clinical providers and other caregivers who go above and beyond usual care to make clinical trials possible. This result validates the key role of [the NCTN] and other networks in improving the outlook for patients with cancer and progressing toward our goal to reduce the cancer death rate by half within 25 years.”
Study Methods and Results
For the current analysis of the work of the adult NCTN groups, researchers identified randomized phase III studies with primary results published in or after 1980 that reported an improvement in a time-dependent outcome favoring the experimental treatment. The 162 trials they analyzed had enrolled at total of 108,334 patients.
For each trial showing an overall survival benefit in the experimental arm, the researchers mapped the benefit onto the U.S. population with cancer using national cancer registry and life table data.
The 14.2 million patient-years these trials added through 2020 represented 4.2% of the 336.8 million years of life lost in the United States as a result of cancer from 1980 to 2020. The researchers further projected that by 2030, this same set of already-completed studies will have added 24.1 million patient-years to the population of survivors.
Total federal investment to conduct these trials was calculated using public data on estimated funding for the four NCTN groups—a total of $4.63 billion in federal investment over those 40 years. To put this $4.63 billion figure (an average yearly investment of about $116 million) into perspective, in the year 2015 alone, the United States spent $183 billion on cancer care, and the total years of life lost to U.S. adult cancer deaths in 2015 resulted in an estimated $94.4 billion loss in future earnings.
The impact of the results on clinical care was measured by determining whether a trial’s results were cited as evidence in favor of a recommended treatment in major clinical guidelines or in package inserts for new drug approvals by the U.S. Food and Drug Administration. Overall, the findings from 87.7% of the trials were used to support recommended clinical care. The overall scientific impact of the trial findings was assessed by determining how often the primary report on each study’s results was cited in Google Scholar. In total, the 162 trials were cited 165,336 times through 2020, and 90.1% of the trials were published in high-impact scientific journals.
Conclusions
The investigators further indicated that the $326 per patient-year of gained federal investment did not encompass all costs, noting that the costs of initial drug discoveries and early testing were not included in the analysis, nor were the full costs of conducting these trials—including the costs endured by clinical institutions and the time and effort of the researchers. On the other hand, the investigators also discussed multiple additional benefits patients may have derived from these and other NCTN clinical trials that were not accounted for in these statistics.
“These trials have also made substantial contributions in ways that are harder to measure,” Dr. Unger concluded. “For instance, negative trials are critical for showing what treatments should not be used, and federally sponsored research groups also provide critical opportunities to mentor new generations of clinical researchers. And for patients, these trials routinely provide access to treatments for underserved patient populations that may not be as accessible in industry-sponsored trials.”
Disclosure: The research in this study was funded by the National Institutes of Health/National Cancer Institute. For full disclosures of the study authors, visit ascopubs.org.
