Innovative Clinical Trial Study Design Adoption and Performance Impacts

The adoption of innovative clinical trial designs has the potential to provide benefits for patients, sponsoring and regulatory organizations. Despite many efforts by various organizations to help modernize the industry through higher rates of innovative design usage, adoption of these designs remains low. This research aims to investigate the and explore the landscape of innovative trial designs and potential factors that may influence the adoption and understanding of innovative design usage. As grounding and exploration across the industry, semi-qualitative interviews were conducted and influenced the development of the theories and analysis in this research. Registry data was utilized to explore variables measuring information disclosure and regulatory compliance to understand potential relatedness to innovative design usage. The performance impacts of innovative design use was then explored as measurements of cost and duration of study timelines. Overall, the results suggest that sponsors are unwilling to share investigational documents (protocol and statistical analysis plan) for innovative designs and are more likely to be overdue in reporting results. Trials that use an innovative design are also more likely to have more complete registrations at the time of entry into the registry based on required and voluntary fields at the time of registration. Innovative design usage does vary depending on the phase of the clinical trial which is supportive of appropriateness of fit of the innovation. The results also suggest that there is a tendency to do what is known or what was done before or to imitate competitor products. There are mixed views regarding the perceived cost of innovation, however there is a universal sense of hope that innovative designs have the potential for cost-savings. Finally, there are fears around failure and scrutiny from regulatory agencies which influence the process of design selection and development of a protocol. The performance model analysis showed that innovative designs cost more and take longer typically than traditional trial designs. This analysis provided results while counterintuitive perhaps suggest that cost and timeline savings are more on the overall clinical development plan than on the individual clinical trial. The limitations of the public registry have been acknowledged in this research and while this dissertation was being finalized a program to update the public registry was initiated. Hopefully the registry updates will include operational elements to support the need for further transparency. Future analysis could then leverage these factors and evaluate the robustness of these findings. Finally, further research should be conducted to evaluate impacts of innovative design use on the overall clinical program.