Modeling strategy provided sponsor with rational basis for quantifying efficacy benefit of approved dose to avoid Phase IV trial, saving $6-20 Million and redirecting resources towards other programs.
A global biopharmaceutical company was seeking to fulfill a post-approval commitment to regulatory authorities for its currently approved oncology drug. Regulatory approval in the given indication was based on results of a pivotal Phase III study to evaluate safety and efficacy of the drug in combination with another agent as a first-line treatment. Dose selection for the pivotal trial was based on Phase II results of the drug as a single-agent, second-line treatment.
While the safety profile of the approved dose was not in question, the regulatory authorities noted that lower dose levels were not tested in the proposed indication and requested further investigation as a post-approval commitment. As the sponsor refined its strategy, it became critical to provide a quantitative basis for the approved, higher dose and to establish a comprehensive picture of the drug’s efficacy profile at intermediate doses in a first-line setting.