Pilot studies may seem unnecessary when your investigational diagnostic is performing superbly for lab professionals in your internal labs. Why waste time and money on a pilot study when you are witnessing excellent results in the lab day after day? After all, the goal of every development program is to obtain regulatory approval, market the product and generate revenue as rapidly as possible. Let’s be honest – it takes time to identify appropriate sites for a pilot study to evaluate real-world performance by intended operators, more time to conduct the pilot study and even more time to assess results. Meanwhile, competitors may beat your product to market.

The Performance Gap Between Lab and Clinical Settings

Lab to Clinic ImageThere is no denying that pilot studies require an investment of time and money. However, experience shows that proceeding directly to a large validation study in a clinical setting assumes the substantial risk of lengthening timelines, increasing costs and delaying regulatory approval and market entry. Why? Because the investigational device is very unlikely to perform superbly in the hands of everyday clinicians. Instructions that seem straightforward to experts in the developmental lab may not be clear to non-lab-professionals. You will see higher error rates in clinical studies: pilot or full-scale validation studies. At the end of the day, the costs and timelines of a small pilot study are nothing compared to being forced to return to the developmental lab to correct flaws in product design or instructions for use.

Pilot Studies Ensure a Sound Understanding of the Intended Use and User

Understanding the intended use and the intended-use setting are essential for planning and conducting diagnostics development programs. Intended use determines:

  • The appropriate regulatory pathway
  • The study design required
  • The choice of method-comparison or clinical outcome for product evaluation
  • The sample size
  • Program costs
  • Key content of the Product Label

Because intended use and the intended-use setting play such a central role in diagnostics development, the pilot study should follow the intended-use statement. Such pilot studies should have the following elements:

  • Realistic settings for the intended use of the investigational diagnostic
  • Operators who are not just naive with respect to the novel diagnostic but truly intended real-world users
  • An easy-to-interpret quick reference guide for intended users
  • User questionnaires to explore the usability of the diagnostic and the clarity of instructions
  • Focus groups to identify points of ambiguity and confusion.

Understanding the Intended Use Mitigates Diagnostics Development Risks

The worst case for a diagnostics development program is being forced to halt a large clinical validation study because of high error rates and return to the developmental labs. Carefully planned pilot studies are the best way to prevent such a disaster and keep diagnostics programs on track. Rather than being wasteful, pilot studies are an efficient way to ensure a realistic understanding of the intended-use setting and mitigate major risks in diagnostics development.

Jessica Kappes, MBA, CCRA is a clinical project manager at Health Decisions. Jessica has more than 7 years of project management experience on both the CRO and sponsor side. Her global experience includes trials in North America, Latin America, Asia-Pacific and the European Union. She has experience in both the neonatal and pediatric populations and therapeutic experience in IVDs, bacteremia, endocrinology and oncology. She holds an MBA from Indiana Wesleyan University and is a certified Clinical Research Associate through Association of Clinical Research Professionals.