In recent years innovation in the pharmaceutical industry has been fuelled by small and mid-size biotech companies which have the benefit of cutting-edge science, nimble organizational processes and increased capital to fund research with a clear focus. More recently, regulatory processes like breakthrough designation have provided additional impetus to bringing new medicines forward rapidly. Despite these catalysts, some biotech’s face significant hurdles as they transition from discovery to development. This is especially true for small biotech’s that use a virtual model where most if not all the development work is outsourced and there is significant reliance on external partners. In this approach, one of the most common gaps is in the areas of pharmaceutical development that includes preformulation, formulation, drug delivery as well as CMC regulatory strategies. This could lead to conservative or erroneous strategies for early development studies and a negative impact on expediting transition to registrational studies. This presentation will outline several key areas of CMC focus for the discovery to development transition to establish POC and rapid transition to registrational studies.
Case studies highlighting smart risk taking and adoption of phase-appropriate development that enables rapid human assessment, speed to POC and transition to registrational studies will be covered.