Reprioritizing Cancer Clinical Studies

“We have the perfect storm,” as Edward Benz Jr., MD, president of the Dana-Farber Cancer Institute (DFCI) in Boston, MA, put it.

Public funding for clinical trials is being eroded by a number of factors, including the rapidly changing U.S. health care environment, Benz explained at a public workshop held February 11–12 in Washington, DC, by the Institute of Medicine (IOM) and the American Society of Clinical Oncology.

“Nationally, people will begin to feel some of what we're already experiencing in Massachusetts, which is a real downward pressure on clinical reimbursements,” Benz said. That cuts down on the ability for centers such as DFCI to help cover the cost of clinical trials. In addition, as NIH funds tighten, trials may require higher contributions from the centers, he stressed.

Funding challenges were front and center at the workshop, which gathered clinical trials researchers, federal agency representatives, and other stakeholders to discuss ways to improve the nation's federally funded cancer clinical trials cooperative group system.

Since the IOM released the 2010 consensus report “A National Cancer Clinical Trials System for the 21^st Century,” the National Cancer Institute (NCI) has taken significant steps to make clinical trials more efficient, including encouraging consolidations among the NCI-sponsored Cooperative Oncology Groups and their integration into a National Clinical Trials Network (NCTN).

Sharp drops in public funding make it imperative to continue efforts to improve the ways in which trials are designed, workshop attendees stressed.

“We clearly live in an era of unparalleled ability to apply emerging science to clinical trials, but we can't do that if we continue to conduct 7,000-patient trials of toothpaste A versus toothpaste B,” said George W. Sledge Jr., MD, chief of oncology at Stanford University School of Medicine in Palo Alto, CA. “The current system is driven primarily by the cost of phase III trials, and that's something that we need to think about how we're going to change.”

Sledge and Robert Diasio, MD, director of the Mayo Clinic Cancer Center in Rochester, MN, cochair an NCTN Clinical Trials and Translational Research Advisory Committee working group that is looking at ways to prioritize trials based on accrual capability, clinical importance, societal benefit, scientific contribution, relative cost and time, and other criteria.

The working group also is considering such issues as a potential role for “smaller, more nimble randomized phase II studies” and the creation of a standard format for submitting trial concepts to the NCI, said Diasio. He emphasized that the group's efforts to date are best described as a learning exercise.

Workshop attendees also focused on issues involved in partnering with pharmaceutical companies.

“We absolutely want to partner with industry, but industry will always take a very drug-centric view,” commented Peter Adamson, MD, chair of the Children's Oncology Group and chief of the Division of Clinical Pharmacology and Therapeutics at the Children's Hospital of Philadelphia, PA. “For rare and ultra-rare diseases, which describes almost all of childhood cancers, we need to be able to decide what's the highest priority at any given time.”

“The pharmaceutical firms have different motives, and they may not be in tune with the whole picture of how cancer development should be going,” noted Richard Pazdur, MD, director of the U.S. Food and Drug Administration's Office of Hematology and Oncology Products. “For example, all of the pharma companies are working on PD-1 drugs at the present time. Is that in the best interest of the clinical trial system?”