ASCO is urging cancer researchers, clinical trial sponsors, and drug developers to employ clinical trial designs that aim to significantly extend the lives of people with cancer.
In a Special Article published today in the Journal of Clinical Oncology, ASCO outlines overall survival goals for cancer clinical trials in several diseases that researchers should aim for—and patients should expect. The recommendations, developed by the ASCO Cancer Research Committee working with other experts and cancer patient advocates, provide examples of "clinically meaningful outcomes" in four types of cancer: advanced pancreatic, lung, breast, and colon cancers.
"We're urging our colleagues to implement clinical trials that if successful, would provide a significant and clinically meaningful improvement in survival," said Lee M. Ellis, MD, FASCO, immediate past chair of the ASCO Cancer Research Committee and lead author of the article. "People with cancer are living longer due to new therapies that target specific molecular drivers of cancer. As our understanding of the molecular drivers of cancer expands, we should be able to design clinical trials that achieve better results."
The article, titled "Raising the Bar for Clinical Trials by Defining Clinically Meaningful Outcomes," notes that additional benefits associated with "raising the bar" include the ability to design smaller and smarter clinical trials that can be conducted faster than larger trials that aim for smaller benefits for patients.
"These aspirational trial goals must be based on sound, reliable preclinical studies that provide the foundation for significantly improving outcomes for our patients," Dr. Ellis said. "In our recommendations, we also address the importance of quality of life and toxicity issues. If a patient experiences more toxicity than with current therapies, the therapy should provide a greater benefit."
ASCO also recommends that clinical trial sponsors establish comprehensive biospecimen banks for each trial, with informed consent from patients, so researchers can connect biospecimens with treatment outcomes to better understand which patients are most likely to benefit from treatment. Access to information about patients' outcomes will enable investigators to ask scientific questions before and after trials are completed—a step that would better enable the discovery and validation of biomarkers, molecular markers in the body that are signs of normal or abnormal conditions, that can help guide treatment planning.
An editorial in the same issue of JCO by David M. Dilts of Oregon Health & Science University, is titled, "Time Has Come to Raise the Bar in Oncology Clinical Trials."
Development of the recommendations began during the 2012-2013 ASCO presidency of Sandra M. Swain, MD, FACP. "The Clinical Meaningful Outcomes project provides a beginning to a dialogue between investigators, industry, government, and patients intended to maximize the outcomes with new therapeutics for our patients with cancer," Dr. Swain said. "This work was complex and controversial, but I am very pleased that the ASCO Cancer Research Committee under Dr Lee Ellis' leadership took on this project with much enthusiasm and hard work."
The committee established working groups to study unmet needs in the four cancers, led by Emile Voest, MD, PhD (pancreas), Roy Herbst, MD, PhD (lung), Lowell Schnipper, MD (breast), and Alan Venook, MD (colon). The groups included experts and patient advocates for each cancer type.
The draft recommendations were released in April 2013 for public comment, discussed at ASCO's 2013 Annual Meeting, and reviewed by the ASCO Board of Directors.
In addition to being a priority topic area for the committee, the recommendations are connected to ASCO's research report "Accelerating Progress Against Cancer: ASCO's Blueprint for Transforming Clinical and Translational Cancer Research."