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.
Sponsors of first-in-human trials of novel oncology compounds face significant challenges. Beyond the typical circumstances of limited budget, compressed timeline, scarcity of skilled clinical trial professionals, and limited knowledge of regulatory hurdles, for many innovative biotech and specialty pharma sponsors, the success of the company depends heavily on the outcome of the trial.
Often, these early-phase trials involve a class of drug or target that has never been tested in a clinical trial. 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.
Optimizing regulatory strategy
For emerging and small- to mid-sized biotech and pharma companies with a novel oncology product, the first major goal of clinical development is a first-in-human study – often a Phase 1 safety, tolerability, and pharmacokinetics study in a limited number of targeted patients with cancer. Unfortunately, these sponsors can be faced with a shortage of skilled clinical trial professionals and limited regulatory knowledge and experience.