Maximizing Success In Early Stage Oncology Trials: Considerations And Strategies In The Era Of Molecularly Targeted Agents

By Luke Gill, MSc, MBA, Executive Director, Oncology, Strategic Development

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 I 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

Making a meaningful impact on the survival and quality of life of patients with cancer remains a significant challenge. This white paper discusses considerations and strategies for maximizing the likelihood of success in early phase oncology trials and developing significantly improved therapeutics for patients.