A major new study now underway aims to better incorporate patient feedback into clinical trials that help determine which new cancer treatments will be approved for use.
The project, supported by a five-year, $3.4 million grant from the National Cancer Institute, involves statisticians, clinicians and patient advocates. The team is analyzing data from previous and ongoing clinical trials to design new statistical measurement criteria for assessing how well trial participants tolerate experimental therapies.
“There is a pressing need to include the patient’s voice in the evaluation of the toxicity and tolerability of new cancer treatments,” saidAndré Rogatko, PhD, director of the Biostatistics and Bioinformatics Research Center at Cedars-Sinai Cancer. “As a consequence of our work, we expect that the future reporting of results from cancer treatment trials can include better evaluations.”
Rogatko is co-leading the study along with Patricia Ganz, MD, professor of Medicine in the David Geffen School of Medicine at UCLA.
New experimental cancer treatments are raising hopes among clinicians and patients for longer survival times and cures. But clinical trials that test such treatments also need to analyze the impact on patients of potentially harsh side effects, known as adverse events, Rogatko said. These side effects may include pain, fatigue, nausea, heart palpitations, skin reactions, mood changes, memory impairment and sexual dysfunction, among others.
“A big unknown is how adverse events affect patients over longer periods of time, particularly in immunotherapy, in which we only recently are learning about long-term toxicity and how it affects quality of life,” Rogatko said. Immunotherapy, which recently has revolutionized how cancer patients are treated, uses substances made by the body or in a lab to help the body’s immune system more effectively fight cancer.
“As we continue to improve immunotherapy and now combine it with other therapies to make it more effective, we will have to carefully study side effects so that we design combinations that are both more effective and less toxic. That would be a real advance, and this work will help with reaching this goal,” said Dan Theodorescu, MD, PhD, director of Cedars-Sinai Cancer.
In recent years, federal agencies have stressed the importance of increased data collection and scrutiny of adverse events experienced by patients while undergoing cancer treatments. The new study aims to advance that effort.
One goal of the study is to use existing and new methods for describing toxicity to show and foretell adverse events. The second goal is to predict the toxicity in a given clinical trial and whether a patient will complete the treatment.
Investigators are using a toxicity index previously developed by Rogatko plus PRO-CTCAE, a set of patient-reported outcome measures designed by the National Cancer Institute to evaluate symptomatic toxicity in patients in cancer clinical trials. The study takes advantage of data from three ongoing immunotherapy trials and three completed National Surgical Adjuvant Breast and Bowel Project trials.
Although the study’s research methods involve highly technical statistical analysis, the emphasis is on improving quality of life for cancer patients, Rogatko said. “We have a chance to give patients more power in how they want to be treated,” he explained.
Funding: Research reported in this publication was supported by the National Cancer Institute of the National Institutes of Health under Award Number U01CA232859. The grant was awarded in September, and the study will conclude in August 2023.