their clinical significance in ways that can bring new drugs forward in a more expeditious manner,” Dr. Rothenberg said. Dr. Lowy responded that “what we actually need are validated surrogate end points. Clearly biomarkers end up being of critical importance.” He noted that in the HPV (human papilloma virus) vaccine trials, there was sufficient understanding of the pathogenesis of cervical cancer that researchers could use moderate and high-grade dysplasia as an end point. “Because this was a prevention trial, it would have been unethical to wait until cancer developed in these patients,” Dr. Lowy added.


As researchers continue to uncover the molecular complexity of the network of pathways that foster various cancers, there is increasing interest in combining multiple drugs, each of which targets a key player in the network. Such combination therapy will likely require precompetitive (i.e., premarket) collaboration if these therapeutic agents are not already on the market and are sponsored by different drug companies. Dr. Rothenberg noted that there are four major obstacles to developing combination therapy: determining targets, validating those targets, determining how those targets interact, and clinically validating the drugs aimed at those targets when used in combination. Each of these tasks can be more effectively addressed by collaboration, Dr. Rothenberg said.

Dr. Dalton added that “there are definite synergisms between what different technologies, different companies, can bring. Companies are also realizing that by partnering, they actually increase their market considerably. So some strategic collaborations and partnerships are developing.”

Dr. Pazdur suggested such collaborations could be fostered with appropriate incentives, such as increased market exclusivity. FDA successfully used incentives to encourage drug companies to test their adult drugs in pediatric populations, he said. The incentive FDA gave companies was increased exclusivity for six months for the entire drug moiety when it was used for a pediatric indication. “From a regulatory point of view, it is a suboptimal situation if we have to make somebody do something…. When you have a program that has incentives—the only thing we have, from an FDA point of view, is exclusivity—then that means something.” The NCPF recently held a workshop on precompetitive collaboration (IOM, 2010a).

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