Balancing Risk and Efficiency in Drug Development for Rare and Challenging Tumors: A New Paradigm for Glioma

Abstract

The process of developing cancer therapies is well established and has enabled the incorporation of many new drugs and classes of agents into the standard of care for common cancers. Clinical drug development is fundamentally different for rare and difficult-to-treat solid tumors, such as glioma or pancreatic cancer. The failure to develop effective new agents for the latter diseases has discouraged the development of therapeutics for these cancers. Using glioma as an example, we describe a process toward obtaining more reliable early-stage signals of drug activity and a process toward translating those signals into clinical benefits with more efficient late-stage development. If linked together, these processes should increase the likelihood of benefit in late-stage settings at a lower cost and encourage more drug development for patients with rare and difficult-to-treat cancers.