Roxanne Nelson

June 01, 2013

CHICAGO — The time has never been better for making advances in medical research, and significant progress has been made toward modernizing clinical cancer research. However, an expert panel warned that these advances are in danger of stagnating as a result of the recent across-the-board federal budget cuts.

Although a great deal of research is funded by the private sector, the panelists emphasized that publicly funded cancer clinical trials are necessary to maximize benefits to patients and society.

At the opening press briefing of the 2013 Annual Meeting of the American Society of Clinical Oncology (ASCO®), the panelists noted that although tremendous progress has been in cancer treatment, continued investments in cancer research are needed to translate recent scientific breakthroughs into new therapies that can address these remaining gaps.

"In 2011, ASCO's Blueprint for Transforming Clinical and Translational Cancer Research laid out a 10-year road map for clinical research to capitalize on our new understanding of biology," said Bruce J. Roth MD, ASCO Cancer Communications Committee chair. "ASCO has worked with stakeholders to help transform clinical cancer research and regulatory processes to encourage new approaches and more promising therapies into clinical practice with better efficiency."

But despite the important progress to date, cancer continues to remain a global health problem, and in the United States alone, approximately 500,000 people still die from the disease every year. It is expected to become the nation's leading cause of death in the not too far-off future. Globally, the number of new cases has been projected to jump from 12.7 million in 2008 to more than 20 million by 2030.

Changes in Cooperative Groups

One issue is the need to update the publicly supported clinical trial program. As a result of the 2010 report by the Institute of Medicine, which called for modernizing and strengthening the system, the National Cancer Institute (NCI) developed a plan to consolidate the then existing 9 adult cooperative cancer groups into 4 groups. "They made changes that would promote greater centralization and standardization that would lead to resulting efficiencies," said ASCO president elect Clifford Hudis, MD. "This program became known as the NCI National Clinical Trials Network."

The groups continued ongoing clinical trials that were underway during this massive reorganization, which included more than 14,000 investigators. The results of clinical trials coming from these groups have often led to changes in practice, Dr. Hudis noted, and he voiced concern about the impact of the federal budget cuts and how they may affect the continued success of the National Clinical Trials Network and their ability to continue enrolling patients in federally funded trials.

Breakthrough Therapies

Richard Pazdur, MD, director of Office of Hematology and Oncology Products in the US Food and Drug Administration's (FDA's) Center for Drug Evaluation and Research, and also a member of the panel, pointed out the need to update the regulatory process.

He pointed out that "drug development in oncology is alive and well," and although development of these drugs is proceeding in an orderly manner, there is a tremendous need for improvement.

"From a regulatory perspective, we recognize that the field is changing," said Dr. Pazdur. "One of our regulatory initiatives has been the designation of breakthrough therapies."

This is different from and faster than accelerated approval, he noted. "A designation of a breakthrough therapy is one where there is preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over existing therapies."

"This is a higher bar than fast-track or accelerated approval," he continued. "In my own interpretation of this, what we're looking for are transformative therapies — therapies that are going to offer patients an option where no other therapy existed or are a dramatic improvement that looks like they are really going to transform the core of a patient's life, or the quality of that life, or the quantity of that life."

Private vs Public

But in order for new drugs to enter the regulatory process, there is a need for clinical trials. Moncial Bertagnolli, MD, group chair, Alliance for Clinical Trials in Oncology, pointed out the need for public funding.

There is a great disparity in the amount of public spending and spending by the pharmaceutical industry for drug development, she said. And although the pharmaceutical industry is a vital partner in the development of therapeutics, the goals are different.

"For industry, the goal is obtain FDA approval to market a new drug or extend the label of an existing agent," she said, "and to create shareholder value. It's what companies do."

In contrast, publicly funded trials have a goal of maximizing the benefits to patients and society. "The approaches we use are also different," she noted. "We compare effective and promising regimens with each other, and that is not something that pharmaceutical companies are usually interested in doing."

She also emphasized that cancer treatment is not just drugs but includes multiple modalities, such as surgery, radiation therapy, and other strategies, and also involves specialties such as pathology. "There is a lot more to making cancer treatment better for a patient that doesn't have to do with drugs," she said.

In addition, there is also cancer prevention, screening, survivorship, and optimizing quality of life, all of which do not generate a lot of revenue.

"The new National Clinical Trials Network is poised as never before to realize the promise of translating scientific discovery into new treatments that will improve the lives of cancer patients," she said, adding that there are many vitally important research questions that cannot be answered without public funding.

"Private industry will not support studies to address many of the most important research questions," Dr. Bertagnolli added.

2013 Annual Meeting of the American Society of Clinical Oncology. Presented May 31, 2013.


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