Experts on clinical trials met last week to share ideas about how to improve a system that has served the nation well for decades but needs to be modified and updated to meet the needs of patients and its many stakeholders.

A major theme of this workshop was that the reform must be global and include regulatory agencies, third-party payers, and funding entities, such as NCI and pharmaceutical companies, noted NCI Director Dr. John Niederhuber, who delivered opening remarks.

More than 100 individuals representing government, regulatory agencies, academia, industry, and patient groups attended the Institute of Medicine's National Cancer Policy Forum event, held June 1-2 at the Keck Center of the National Academies. The focus was multicenter, final-stage clinical trials and the role of NCI's Clinical Trials Cooperative Group program.

The Cooperative Group program enrolls approximately 22,000 cancer patients each year in clinical studies at academic medical centers, cancer centers, community hospitals, and private research institutions. Congress started the program in 1955, and it has grown to include 1,700 institutions within 10 national groups.

The program has been instrumental in demonstrating the safety and effectiveness of many cancer therapies. But the current clinical trials system has not kept pace with the development of new combinations of therapies that target multiple pathways of cancer.

"The meeting generated a comprehensive and well-informed list of issues and needs that require attention," said Dr. Niederhuber. "It also produced some excellent ideas for positive change. These suggestions will certainly inform NCI's plan of action in the weeks ahead, as we continue our work to enhance this crucial - and rapidly evolving - clinical research enterprise."

Other topics discussed included the recruitment of patients and physicians; costs and payments; and regulatory issues such as data collection standards to establish safety and efficacy.

There seemed to be general agreement on some basic issues, including the need to prioritize the most important trials. Doing fewer trials could allow planners to shift precious resources from poorly accruing trials to other studies.

The annual funding for the Cooperative Groups is approximately $145 million, which is administered by the Cancer Therapy Evaluation Program of NCI's Division of Cancer Treatment and Diagnosis. In 1997, an independent review panel said that the Cooperative Group program needed to be properly funded, and since then the costs of running trials have risen without similar budget increases.

"We must confront not only the crucial scientific questions, but also address funding issues and long-range inefficiencies in the clinical trials system," said Dr. Niederhuber.

Dr. John Mendelsohn, president of the University of Texas M.D. Anderson Cancer Center, chaired the workshop planning committee. He predicted that Cooperative Groups are increasingly going to turn to industry for financial support. These groups are attractive to companies because they offer a greater diversity of patients than many companies can recruit. Another asset is that some groups have tissue banks, and some can look at the expression of genes and molecular targets in real time.

"As we're moving into more personalized medicines, these groups can carry out the kinds of trials we are all interested in," Dr. Mendelsohn said.

During a panel discussion about the collection of data on safety and efficacy, Dr. Richard Pazdur of the Food and Drug Administration (FDA) suggested that the topic could be explored in a future workshop or public forum, as has been done previously with the American Society of Clinical Oncology (ASCO).

The president of ASCO, Dr. Richard Schilsky, responded to the offer immediately by saying his organization would be "delighted" to work with the FDA and other stakeholders to discuss developing data sets required for regulatory approval.

Dr. Harold Moses, director emeritus of the Vanderbilt-Ingram Cancer Center and chair of the National Cancer Policy Forum, noted that previous reviews of NCI Cooperative Groups did not lead to action and said that this time would be different.

A workshop summary will discuss areas of broad agreement, and this could be the basis for an Institute of Medicine study committee. Such a group could form and make recommendations, potentially by next spring.

"Everyone here is interested in action," said Dr. Mendelsohn.

—Edward R. Winstead