The following blog describes the basics of a pilot study and which tactics to use to overcome timeline and cost barriers.
Clinical trials are inherently challenging and require a substantial time commitment, but with proper planning and forethought, they can be carried out efficiently and on time. A well-designed pilot study is a fundamental component of a successful clinical trial, and without one, the odds of progressing to the pivotal phase is slim.
A pilot study is defined as “a small-scale test of the methods and procedures to be used on a larger scale” [1]. In other words, the goal of a pilot study is to determine whether a future study can and should be done, as well as providing valuable insight into how to design the main study. For example, a well-designed pilot study will do more than tell you if your idea is feasible; it will also inform on the most effective strategies for recruitment, randomization, treatment, and follow-up protocols [2].
Pilot studies deliver a wealth of data beyond whether the drug is safe. Data on the pharmacokinetics and pharmacodynamics of the drug can be collected, and pilot studies can also help determine the optimal drug dose, administration route and frequency, end points, and estimates for sample size calculations.
Identifying quantitative benchmarks ahead of time can help you determine whether your pilot study delivered accurate results. For example, if your protocol requires patients to self-administer an experimental drug, what is an acceptable compliance rate? Importantly, pilot studies do not test hypotheses or deliver preliminary conclusions about the efficacy of an intervention. That’s where pivotal studies come in.
In order to progress from a Phase II pilot study to a Phase III pivotal study, your pilot data must demonstrate that the drug is safe and exhibits potential efficacy in your small target population. The pivotal study then picks up where the pilot left off, with the additional goals of comparing the drug to a placebo and further ascertaining that the drug has the desired effect in the target population [3].
Identifying the quickest path from pilot to pivotal studies can be challenging, and it is common for sponsors to work with contract research organizations (CROs) that have experience in designing and executing clinical trials. CROs can also help you spot potentially weak or confounding data before you submit a new drug application (NDA) to the FDA, increasing the chance that your application will be successful.
Planning Your Pilot Study
Ready to learn more about launching a pilot study? Watch the on-demand webinar, “Zero Waste: The Quickest Path from Pilot to Pivotal.” Worldwide Clinical Trials is a global leader in the CRO industry, and our experts are equipped to execute your study accurately and efficiently. Contact Worldwide today to see how we can help support your pilot study.
