Oncology Drug Development

Applied Clinical Trials interviewed Cornerstone Pharmaceuticals' CEO Robert Shorr about challenges in oncology drug development. Last month, Cornerstone received an FDA orphan drug designation to CPI-613, the company's lead product candidate, for the treatment of acute myeloid leukemia.

Can you describe the current oncology drug development landscape?

To some extent the oncology drug development landscape will be driven by changes in the overall healthcare system: reimbursement policies for government and insurance, prevalence of particular indications, unmet needs, clinical trial strategies with the shortest time for development, incentive programs such as the orphan drug act. And of course advances in basic knowledge, diagnostic and therapeutic devices, and the emergence of new drug classes with new mechanisms of action to provide greater life and extension and improvement in quality of life over existing therapies.

Can you describe the largest hurdles to successful oncology clinical trials?

Phase II clinical trials that demonstrate efficacy in a particular indication when compared to current treatment protocols. Having done the clinical research to understand how any new drug with a new mechanism of action functions in patients and achieving proof of concept is essential in designing a clinical development plan that takes into account current measurements and modalities that defined benefit.

Are there solutions to address these hurdles?

Yes. However a greater burden to prove the science behind the drug and its benefits is likely to be placed on drug developers and pharmaceutical companies. I believe that collaborations between innovative small companies and the innovative groups of larger companies with strong development capabilities is going to be required to meet these objectives in a meaningful way.

Are there specific initiatives underway that would help oncology drugs move forward through clinical trials more quickly or with a smoother pathway?

Yes. I believe the various grants programs available through Federal Government sources and charitable institutions as well as through investment in higher risk, higher return approaches are emerging and will be fundamentally important in achieving breakthrough discoveries. I would expect some level of controversy regarding the benefit versus cost of drugs pending approval and ultimately their level of success in the market place. Perhaps third-party payers may consider the support of promising clinical trials to accelerate new treatments that offer overall improvement to a greater number of patients at lower costs to the healthcare system.

As last year's co-chair to CBI's Oncology Drug Development Congress, can you provide your major takeaways from the event?

By having speakers from academics, industry, regulatory agencies, reimbursement, and marketing groups I felt a higher level of confidence in planning strategies and tactics for clinical research that would lead to clinical development of products or indications with unmet needs. In particular to clinical trials, I left the conference with a sense that overall survival and quality of life as well as biomarkers linked to drug mechanism of action would become a standard for decisions of treatment and reimbursement.

Editor's Note: CBI and Applied Clinical Trials Pharma/Bio Congress on Oncology Drug Development will be held October 24-25 in Philadelphia, http://bit.ly/nRtyho.