Single-Arm Studies For Use As A Registrational Study In Oncology

Randomized, controlled clinical trials (RCTs) are the preferred method endorsed by the FDA for conducting registrational studies in developing new drugs and biologics. However, in certain situations within the field of oncology, a single-arm trial may be deemed suitable due to the severity of the disease, the lack of effective treatments, and the practical challenges associated with conducting an RCT.

In these specific scenarios, opting for a single-arm trial can accelerate the development of new oncology treatments, shorten the time required for product approval and patient access, and provide evidence of the clinical benefits of innovative products on a smaller patient population.

Watch as Versistat's Vice President of Strategic Consulting Robin Bliss, PhD, Strategic Consulting Fellow, Biostatistician, Debora Manning, MPH, and Toni Marie Nearing, BS, Principal Regulatory Strategist, discuss single-arm oncology registrational studies, success stories, and lessons learned.

The following considerations are discussed during the webinar:

• How to know if there is an unmet medical need that may allow for a single-arm registrational study
• How to develop a single-arm study design for registration, including:
  • Defining the population
  • Selecting study endpoints and defining a clinically meaningful benefit
• How to determine a regulatory strategy, including:
  • Accelerated approval vs. full approval
  • Planning for agency interaction – when and how to approach the US FDA and what key questions to ask