Pilot Study Overview

When developing a new medical product, there are significant financial implications to launching a full scale clinical trial and if the major trial does not go as planned, the fiscal impact can be ruinous. To combat this issue, companies from start-ups to top 5 biotech firms employ a powerful preliminary research tool called a Pilot Study. The main purpose of a pilot study is to assess the feasibility of a sizable and expensive full-scale study. The benefit added by conducting such a study is for the developing institution to avoid the possibly disastrous financial consequences that result from the failure of a large-study. Though not an official pre-requisite to conducting to a phase III (or similar sized) trial, in the era of ever increasing R&D costs, these studies are becoming more frequent. Pilot studies can be utilized in every clinical arena and every major clinical trial have had at some point conducted some piloting (or at least a small scale study).

As stated above, one of the major uses of pilot studies is to assess the feasibility of massive clinical trials before they begin- this reality could not be more important than for Phase III clinical trials. The objective of this is to provide a brief overview of the various clinical trial stages, the main four reasons pilot studies are conducted, the common challenges, and how to determine the criteria for success.

Types of clinical Trials

While the maximal benefit of a pilot study is realized in the large-scale clinical trials such as phase III, there is still benefit to quantitative pilot studies done prior to full-scale trials. Phase I trials are non-randomized studies with the purpose of determining the pharmacokinetics of a particular drug (such as how it is metabolized, how it is absorbed, and importantly its toxicity). If these early trials prove productive, the study progresses to Phase II, which can be either randomized or non-randomized and can provide insight into the clinical efficacy of a drug (in a small group of patients). The next step in the process is Phase III trials, which compare two or more drugs to determine the overall clinical efficacy as well as the safety (in a large group of patients, 1k-5k+). Finally, Phase IV, which are studies that are generally conducted after the drug and/or intervention has hit the market and consists of a non-randomized observational study to document the side-effects as well as potential interactions with other prescription drugs.

Reasons for Conducting a Pilot Study

The overall reasons for conducting a pilot study are numerous, however, they can be grouped into several larger categories- development, resource management, human and data management, and scientific methods. Development: To assess the overall feasibility of the steps that have to occur as a part of the larger study- such as recruiting and subject retention. Resource Management: To determine the financial/budget and time allotment issues that can occur with the large study. The pilot serves in this case to discover potential issues on a small scale that could be prevented t in the main study. Human Management: To assess all human organizational problems that could arise with the main study. These can consist of researcher management issues that could arise from multiple testing sites or managing overall researcher organization. Scientific Methods: To assess how the main study will go about developing an effective evaluation process. An effective evaluation process can consist of such categories as estimation of treatment effect, developing dose and response curves, as well as the overall anticipated treatment effect.

Common Challenges

As a whole, pilot studies can be extremely beneficial and provide valuable insight to both those who are conducting the study itself as well as future researchers in that field- assuming that the study was published. However, successful pilot studies are not easily constructed and can be misleading if critical goals are not established. One such goal is ensuring that the emphasis of the pilot study is based solely on feasibility. This may appear obvious, but a large percentage of studies incorrectly end up placing emphasis on statistical significance- which does not accurately reflect the potential of the main study. It is important to note that a pilot study is not a hypothesis testing study. In addition, many pilot studies do not clearly define their feasibility objectives nor do they establish concrete criteria for what is successful feasibility.

How to determine Criteria for Success

The most important factor in a successful pilot study is determining the actual criteria for success- which should be centered on feasibility. There are generally four types of outcomes for a pilot study. 1: Halt study as the main study is no longer determined to be feasible. 2: Proceed with caution- only basic adjustments are needed. 3: Proceed with extreme caution- heavy adjustments are needed. In this case, the study is noted to have significant flaws but has not yet completely compromised feasibility, careful monitoring and planning need to occur, but the study can continue. 4: Continue on to the main study- the pilot proved that the model is viable in its present form.

How Does a Pilot Study Associated With and Impact Proof of Concept (PoC)

Proof of concept is the demonstration that a particular method or idea has the potential for real-world application. In terms of healthcare startups, PoC implies that not only is the product interesting, but that it will provide real clinical value and be able to perform its function as promised. A PoC study is extremely similar to a pilot study, with the exception that PoC tends to occur earlier in the clinical trial cycle- somewhere linking phase I and phase II. Overall, both types of studies exist to validate the product- the distinct difference being that a PoC study is not concerned so much with feasibility, but rather, if the concept/intervention has any utility at all.

Why is PoC Important?

In the present era of ever increasing competition for product spotlight as well as decreasing attention span for hospital purchasing officials- now more than ever, a startup must be armed with as much product data as possible. When a startup sales team gets facetime with a hospital purchasing official, it is essential that the team is efficient with their time. The rise of digital advertising has led to outstanding advances in product displays and presentation, however, no amount of sleek can substitute for concrete data of the product in question. In addition to digital media leading to increased presentation quality, it has also enabled hospital purchasing officials to be more informed than ever. It can be difficult for a startup’s product to break through all the “noise” created by flashy presentations, but leading with proof of concept and data that provides irrefutable evidence, it will ensure that purchasing officials will be able to pay attention to and quickly see value in the product.