Drug development in oncology: a regulatory perspective.

The ultimate goal of any drug development, including that in oncology, is to register the new medicinal product. A number of challenges remain in this field, primarily in demonstrating the efficacy of the new oncology products to the satisfaction of the regulatory authorities worldwide. Key aspects of clinical trial design, conduct, and analysis that require careful consideration by the sponsors before initiation of drug development programs in oncology include the choice of primary and secondary end points, the choice of comparators, the necessity for independent radiologic review of a disease-related end point such as progression-free survival and tumor response, and the relative value of the independently reviewed and investigator-assessed clinical end points. Many of these aspects, including the choice of comparators and choice of primary/secondary end points, may have a crucial impact on both the eventual registrability of the new medicine and on the feasibility of conducting the clinical trials in the first place. Although both the industry and the regulators share a common goal of developing promising new oncology products that make a tangible contribution to the care of patients with cancer, more harmonized expectations for "proof of efficacy" and more pragmatic, clinically driven decision-making approaches from regulatory authorities would go a long way in making oncology drug development process less fraught with uncertainties and will result in a more efficient path from clinical trials to the clinic for new medicines. An open dialogue and greater collaboration between the industry and the regulators may help in achieving clarity around these goals and in bringing these to fruition.