BY SARAH DIGIULIO
CHICAGO --The American Society of Clinical Oncology released a Progress Report on its 2011 Blueprint for Transforming Clinical and Translational Cancer Research, at this year’s Annual Meeting. The key message is that progress has been made towards meeting the Blueprint aims,
ASCO 2013-2014 President Clifford Hudis, MD, FACP, said in an interview. “In just two years since the first report came out there are tangible improvements. But, of course at the same time, we still have a lot more to do.”
The original Blueprint report (OT, 12/10/11) had laid out a vision for cancer research based on the then newly recognized more sophisticated understanding of cancer biology, potential of targeted therapies, and health information technology. The 2013 progress report says that advances have been made by ASCO and key stakeholders since 2011 in three areas:
(1) establishing new approaches to therapeutic development that are driven by the molecular characteristics of the cancer (rather than based solely on the cancer’s location in the body);
(2) promoting faster, smarter clinical trials driven by the molecular characteristics of the cancer; and
(3) harnessing health information technology to better inform research.
Redefining Clinically Meaningful Outcomes
The Society’s draft recommendations that identify specific clinically meaningful outcomes for breast, colon, lung, and pancreatic cancers were noted as a key step taken by ASCO, according to the report. The draft document (which can be accessed via a link on www.asco.org/advocacy-practice/clinically-meaningful-outcomes) were proposed by the ASCO Cancer Research Committee, posted publicly for a comment period, and then presented at the Annual Meeting. The completed recommendations are expected to be released later this summer.
The recommendations will help answer the question about defining value, Hudis said. “Is ‘better’ defined as a drug that helps somebody live four weeks longer? Four months longer? Cures them? Is better defined as a drug that costs less money? What if it’s a little more toxic but costs less money?
“Value is going to become a dominant question -- as it should -- when resources are constrained. It may be a hard question, but we may be the best group to tackle it while keeping the patients’ interests front and center.”
Another key step highlighted in the Progress Report is the National Cancer Institute’s consolidation of its Clinical Trials Cooperative Group Program. In a move to achieve more uniformity, greater collaboration, and faster completion of trials the nine previously existing adult cancer research groups were reorganized into four groups, with the Children’s Oncology Group continuing to serve as the NCI-funded group for pediatric studies (OT, 4/10/11).
“We’re very concerned with the impact of the federal funding cuts, and specifically we’re concerned about this transformation of the national clinical trials network and our ability to continue to enroll patients on these federally sponsored trials,” Hudis said. The idea is that the reorganization will lead to more standardization, efficiency, and help ensure sustained funding, he added.
“Every measure of our productivity, the real hard productivity -- the number of studies we can keep going, the number of patients we can study, and the number of publications that we can give to the American people -- is directly and dramatically affected by the 29 percent reduction in our federal funding support,”
Monica Bertagnolli, MD, Chair of the Alliance for Clinical Trials in Oncology (the group formed two years ago by the merger of the American College of Surgeons Oncology Group, Cancer and Leukemia Group B, and the North Central Cancer Treatment Group), explained at a news conference at the Annual Meeting.
The vast disparity in the amount of publically funded money versus pharmaceutical company-funded support that is directed toward cancer research has implications, she said. While both sources of funding are critical to the overall mission of cancer research, the goal of industry-funded trials is to obtain FDA approval to market a new drug or extend the label of an existing agent -- to create shareholder value.
Publicly funded trials, on the other hand, compare promising regimens with each other, optimize multimodality approaches, and evaluate questions about cancer prevention, screening, survivorship, and quality of life. “Unequivocally, our only goal [for publicly funded trials] is to maximize benefit to patients and society,” she said. “As a result, the approaches used are different.”
The NCI has also reorganized its community-based research programs to consolidate programs that support clinical research in practices where most U.S. cancer patients receive care. The efforts to increase clinical trial participation are important because the percentage of adult cancer patients who participate in clinical trials is still only three percent, Hudis noted. “This is a long-standing dilemma for us, obviously affecting our ability to advance the science and make new discoveries for our patients.”
Cancer Drug Development ‘Alive and Well’
And also noted in the report as a key advance was the creation of the U.S. Food and Drug Administration’s “breakthrough therapy designation.” The designation is intended for drugs (in preclinical or Phase I research) that show potential to offer substantial improvement over existing therapies for life-threatening diseases.
Speaking at the news briefing, Richard Pazdur, MD, Director of the FDA’s Office of Hematology and Oncology Products in the Center for Drug Evaluation and Research, said that the new designation brings needed attention to therapies that offer patients options where no other therapy existed or offer dramatic, transformative benefits to quantity or quality of life for patients.
The FDA will spend much energy to speed up the development of a drug that has been granted the designation, he added. “It’s a different pattern and communication structure and working relationship with the sponsor [versus the traditional standard regulatory meetings]. It’s a continuous dialogue.”
So far, the designation has been granted to seven therapies for cancers (since July 2012 when it became effective with the most recent PDUFA legislation -- OT, 8/10/12). Pazdur also noted that in 2012, the 14 new molecular entities approved for cancer represent 40 percent of all drugs that were approved in the U.S.-- “Drug development in oncology is alive and well,” he said.
In addition to the key steps already taken by the field’s key stakeholders listed above, the Progress Report listed these priorities for ASCO (and for oncology) for the next two years:
· Address federal research funding threats;
· Develop CancerLinQ and establish responsible pathways for research;
· Continue to establish consensus on new research endpoints;
· Implement ASCO’s upcoming recommendations for clinically meaningful outcomes and eligibility criteria (working with the field’s key stakeholders); and
· Modernize (working with the FDA) the oversight of molecular diagnostics that are vitally important for clinical decision-making in the era of precision oncology therapeutics.
Progress Report Quick-Takes
In addition to the advances discussed, the Progress Report also noted the following…
Key Steps Taken by ASCO:
· Completing and launching the CancerLinQ prototype in March, which includes de-identified data on more than 150,000 patients with breast cancer and demonstrates the feasibility of a rapid learning system;
· Collecting data and surveying experts to complete a draft recommendation on how to avoid unnecessary exclusion of patients from trials to help modernize and streamline eligibility criteria for molecularly driven clinical trials (draft to be completed this fall); and
· Sharing feedback with the FDA on the Agency’s draft guidance to eliminate the need for research sites to collect and companies to submit data on already-known, low-grade safety risks associated with expanded uses of already-approved drugs to help accelerate approvals of additional indications for marketed drugs.
Key Step Taken by the FDA:
· An FDA draft guidance is being created for breast cancer trials as a result of a series of public meetings over the last year and a half co-sponsored by ASCO, the FDA, and other specialty societies to discuss the use of surrogate endpoints for efficacy in cancer clinical trials, including various alternatives to overall or progression-free survival.
Full Report Online
The full Progress Report is available at http://www.cancerprogress.net/blueprint.html