Study quantifies impact of NCI-sponsored trials on clinical cancer care

September 18, 2019

A new study shows that nearly half of phase 3 cancer clinical trials carried out by the National Cancer Institute (NCI)-sponsored SWOG Cancer Research Network, one of five groups in NCI's National Clinical Trials Network (NCTN), were associated with clinical care guidelines or new drug approvals. NCI is part of the National Institutes of Health.

The analysis was published in JAMA Network Open and conducted by researchers affiliated with SWOG from several institutions around the country. The study suggests that NCTN trials add value regardless of whether findings were positive or negative. In addition, the authors calculated the cost of running NCTN trials, and they also found that the cost of a U.S. Food and Drug Administration (FDA) approval from an NCTN trial was much less than the cost of an FDA approval from a trial run by pharmaceutical companies.

"We found that the NCTN program contributes clinically meaningful, cost-effective evidence to guide care of cancer patients," said Joseph Unger, Ph.D., a health services researcher and biostatistician for SWOG at the Fred Hutchinson Cancer Research Center, Seattle, and lead author of the study. "These trials are largely funded by the public, which is getting good value for their investment."

The researchers used data from 182 phase 3 trials enrolling 148,028 patients between 1980 and 2017. These included trials that were led by SWOG or that were led by other NCTN groups with SWOG participation. According to the analysis, 82 of the 182 trials, or 45%, were found to be "practice influential," meaning that they influenced cancer care, either by being reflected in the National Comprehensive Cancer Network (NCCN) clinical guidelines or by being associated with a new drug approval by the FDA. Of those 82 practice-influential trials, 70 influenced NCCN guidelines, six influenced new FDA drug approvals, and six influenced both.

"Federally funded cancer treatment trials fill an important gap in clinical research by seeking answers to treatment questions that might not otherwise be explored," said James Doroshow, M.D., director of NCI's Division of Cancer Treatment and Diagnosis, which oversees the NCTN. "This study sheds light on the critical role these trials have in guiding clinical cancer treatment, whether the findings from the trials are positive or negative."

In fact, the influence of negative trial results on cancer care seen in this study surprised the researchers. Of the 82 practice-influential trials identified, 35, or 43%, had negative findings, with nearly half of those 35 trials reaffirming standard of care compared with experimental therapies being tested in the trials. Such negative findings signal to the oncology community which new, and potentially expensive, drugs are not effective. Negative trials can also reveal harmful side effects caused by experimental therapies.

The researchers also sought to estimate the costs of the trials in the study and looked at differences in costs of getting FDA approvals between the publicly funded trials in the study and privately funded trials conducted by pharmaceutical companies, biotech firms, and other industry funders.

They estimated that total federal investment supporting the trials in the study was $1.36 billion. This suggests that for 182 trials, average costs were $7.5 million per completed phase 3 trial (all trials), $16.6 million per practice-influential trial, and $123.6 million per new drug approval. In a review of 10 studies of the cost of new drug approvals by industry, the researchers found that the mean inflation-adjusted cost for a single new drug approval was $1.73 billion.

The study authors wrote that this kind of cost comparison is imperfect because pharmaceutical company trials can be more expensive, in part because of regulatory costs. Still, this comparison highlights the value of the NCTN program for taxpayers and the patients and families that benefit, according to Dr. Unger.

"The take-home message from the study is that NCTN studies provide a lot of clinically meaningful evidence for patients that influences their care routinely and does so at a relatively cost-effective level," he said. "It's important that people appreciate just how valuable these trials are in terms of benefit to patients with cancer."
-end-
The SWOG study was supported by the NIH through the NCI under grants U10CA180888, U10CA180819, and UG1CA189974, and by The Hope Foundation for Cancer Research.

Along with Dr. Unger, the research team includes Van T. Nghiem, Ph.D., of the SWOG Statistics and Data Management Center and Fred Hutchinson; Dawn L. Hershman, M.D., of NewYork-Presbyterian/Columbia University Irving Medical Center; Riha Vaidya, Ph.D., of the SWOG Statistics and Data Management Center and Fred Hutchinson; Michael LeBlanc, Ph.D., of the SWOG Statistics and Data Management Center and Fred Hutchinson, the SWOG group statistician; and Charles D. Blanke, M.D., of Oregon Health & Science University, the SWOG group chair.

About the SWOG Cancer Research Network: SWOG was founded in 1956, and is a member of the National Cancer Institute's National Clinical Trials Network and the NCI Community Oncology Research Program, making it part of the oldest and largest publicly-funded cancer research network in the United States. SWOG has over 12,000 members in 47 states and six countries who design and conduct cancer prevention and treatment trials. SWOG trials have led to the approval of 14 cancer drugs, changed more than 100 standards of cancer care, and saved more than 3 million years of human life. Learn more at swog.org.

About the National Cancer Institute (NCI): NCI leads the National Cancer Program and NIH's efforts to dramatically reduce the prevalence of cancer and improve the lives of cancer patients and their families, through research into prevention and cancer biology, the development of new interventions, and the training and mentoring of new researchers. For more information about cancer, please visit the NCI website at cancer.gov or call NCI's contact center, the Cancer Information Service, at 1-800-4-CANCER (1-800-422-6237).

About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit nih.gov.

NIH/National Cancer Institute

Related Cancer Articles from Brightsurf:

New blood cancer treatment works by selectively interfering with cancer cell signalling
University of Alberta scientists have identified the mechanism of action behind a new type of precision cancer drug for blood cancers that is set for human trials, according to research published in Nature Communications.

UCI researchers uncover cancer cell vulnerabilities; may lead to better cancer therapies
A new University of California, Irvine-led study reveals a protein responsible for genetic changes resulting in a variety of cancers, may also be the key to more effective, targeted cancer therapy.

Breast cancer treatment costs highest among young women with metastic cancer
In a fight for their lives, young women, age 18-44, spend double the amount of older women to survive metastatic breast cancer, according to a large statewide study by the University of North Carolina at Chapel Hill.

Cancer mortality continues steady decline, driven by progress against lung cancer
The cancer death rate declined by 29% from 1991 to 2017, including a 2.2% drop from 2016 to 2017, the largest single-year drop in cancer mortality ever reported.

Stress in cervical cancer patients associated with higher risk of cancer-specific mortality
Psychological stress was associated with a higher risk of cancer-specific mortality in women diagnosed with cervical cancer.

Cancer-sniffing dogs 97% accurate in identifying lung cancer, according to study in JAOA
The next step will be to further fractionate the samples based on chemical and physical properties, presenting them back to the dogs until the specific biomarkers for each cancer are identified.

Moffitt Cancer Center researchers identify one way T cell function may fail in cancer
Moffitt Cancer Center researchers have discovered a mechanism by which one type of immune cell, CD8+ T cells, can become dysfunctional, impeding its ability to seek and kill cancer cells.

More cancer survivors, fewer cancer specialists point to challenge in meeting care needs
An aging population, a growing number of cancer survivors, and a projected shortage of cancer care providers will result in a challenge in delivering the care for cancer survivors in the United States if systemic changes are not made.

New cancer vaccine platform a potential tool for efficacious targeted cancer therapy
Researchers at the University of Helsinki have discovered a solution in the form of a cancer vaccine platform for improving the efficacy of oncolytic viruses used in cancer treatment.

American Cancer Society outlines blueprint for cancer control in the 21st century
The American Cancer Society is outlining its vision for cancer control in the decades ahead in a series of articles that forms the basis of a national cancer control plan.

Read More: Cancer News and Cancer Current Events
Brightsurf.com is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to Amazon.com.