Small- to mid-sized biotech and pharma companies face many challenges when planning and executing an early-phase oncology trial, from performing a regulatory gap analysis and developing a target product profile to identifying the right patients and selecting efficacy endpoints.
For the purposes of this white paper, we will focus on the following development scenario: Your target is a newly validated mutated receptor that is only present in a limited number of patients with cancer, with no diagnostic test yet approved. And the compound is an antibody-like molecule that inhibits the receptor’s activity but also stimulates a potent immune response. To further complicate matters, much of the preclinical data suggests the compound will synergize with unapproved newer molecules that are in later-stage trials. In this situation, you are co-developing – at a very early stage – biomarkers and diagnostic kits to define those patients who will best respond to your investigative therapy.
In this white paper, we explore the many aspects a company must consider in planning and executing an early-phase oncology trial of this hypothetical compound, from performing a regulatory gap analysis and developing a target product profile to identifying the right patients and selecting efficacy endpoints.