Clinical Research: Phase 1 - Phase 4

Maximizing Success in Early Stage Oncology Trials

The success rate of investigational compounds eventually approved for clinical use in cancer remains the lowest among all diseases. Of the more than 750 drugs currently under development for the treatment of cancer, it is predicted based on past performance that only a few will ultimately demonstrate sufficient efficacy and safety for regulatory approval and clinical use. The likelihood of approval for investigational oncology drugs tested in Phase 1 trials is only 6.7 percent, the lowest among all therapeutic areas. Furthermore, the drug development process in oncology is estimated to take 1.5 years longer than in other diseases, likely due to slow recruitment and the long study duration needed to assess survival endpoints.

However, progressive regulatory policies, a favorable political climate, increased funding for cancer start-ups, and improved scientific and clinical approaches to developing cancer therapeutics are beginning to alter the status quo. These trends promise to increase approval rates, bringing better and more numerous therapeutic options to cancer patients.

Today, due to a global focus on cancer, most therapeutics are molecularly targeted agents (MTAs) which are less toxic, more effective, and tailored toward specific mutations or pathways altered in patients. MTAs comprise a vast array of different molecules and approaches, but all are generally designed to engage specific cancer-dependent targets and specifically kill cancer cells. Advances in our understanding of the molecular pathogenesis of cancer have led to increased interest and use of in MTAs.

The first two MTAs – imatinib and rituximab – were launched in 1997, and now, molecularly targeted agents represent the vast majority of new oncology drug approvals. Over the past decade, the number of novel anticancer drugs in development has risen

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