Our customer is a specialized biopharmaceutical company, developing novel oncology therapies. Their breakthrough therapy had the potential to be first-in-class for a rare and aggressive hematological cancer and had shown great potential in earlier clinical trials. In areas of high unmet need, it is critical to bring vital new medicines to patients faster, yet conventional development pathways involving multiple stages can be slow, expensive and inefficient.
In many breakthrough treatment areas, where the patient population is small, or there is overwhelming evidence of efficacy at Phase 2, it has become common for drugs to be approved based on a pivotal single arm trial – however, this is not always optimal. With this in mind, our customer planned a registration pathway based on a single arm registrational study with a comparison to a synthetic control.