In a rapidly changing market and regulatory landscape, pilot and feasibility studies have never been more important to drug and product development in the pharmaceutical industry. In particular, pilot and feasibility studies that collect real-world data with an eye to capturing a patient experience within a therapeutic area have the potential to make a significant impact at all stages of the development pipeline.
The move towards pilot studies, and why you should care.
A pilot study is the first step in assessing whether the research protocol can be done. A prerequisite to larger, more expensive studies, a pilot study can increase the likelihood of the landmark study success by examining the feasibility of interventions, sample recruitment strategies, and research techniques. Pilot studies are de-risking the pipeline and asset development by capturing data early, often and quickly. Preclinical research can be expensive and prone to failure. Running smaller pilot studies can allow researchers to quickly identify signals in data. Studies that collect patient-reported outcomes or generated data can incorporate an element of social listening into a study at the pre-indication stage, pointing researchers in the direction of interventions and asset development that are most meaningful for participants.
For example, a recent pilot study evaluated the feasibility, usability, and implementation context of a self-management digital program based on exercises and pain education in people with chronic musculoskeletal pain. By conducting an eight week pilot trial, the researchers were able to conclude that the users found the RehabilitaDOR program feasible and acceptable, and move forward with more complex (and expensive) studies with confidence. In this way, pilot studies can inspire innovation by providing the scaffolding for iterative and experimental studies in therapeutic areas across the clinical research ecosystem.
Pilot and feasibility highlight signals for commercialization, helping to justify budget and investment in particular areas. One example is in the area of opioid use disorders. The pandemic has exacerbated the opioid epidemic, particularly with the disruption of inpatient services; however, there has been little evidence on how to increase retention for opioid drug users in telehealth care treatment services. In a recent pilot program, researchers established the feasibility of delivering a COVID-19 telehealth care management program using text messages for patients receiving opioid use disorder treatment. In a broader sense, pilot studies pinpoint focuses in therapeutic areas that require additional attention, like a novel telehealth program addressing patient engagement and treatment needs.
In a decentralized era, pilot studies enhance research participant diversity, increasing study generalizability and representativeness. A single-arm pilot study evaluated the feasibility and acceptability of a mHealth technology implementation as a gender- and culturally sensitive weight loss intervention for overweight and obese Hispanic men. Through digital pilot studies, high-risk and minority groups are tested when they often are excluded from research participation. In this case, few weight loss interventions have been conducted on Hispanic men, despite experiencing disproportionate rates of obesity.
Finally, thanks to tools like Smart Omix, digital pilot studies are cost-effective and efficient, driving greater value for both sponsors and patients.
Looking to build a pilot or feasibility study? Here are a couple things to consider:
- Use the right tools that enable iteration and experimentation. Feasibility or pilot studies are key to improving the iterative nature of product or drug development, evaluation, and future implementation. Determine recruitment and retention rates prior to performing expensive large-scale studies.
- Incorporate as much rich real-world data from patients as possible. Pilot studies enable you to collect a plethora of rich data in both quantitative and qualitative studies from participants who provide you insights and data on the feasibility of a future study.
- Keep your budget controlled. A pilot study should be based on budgetary constraints to effectively assess the feasibility goals of a future proposed study. Avoid wasting time and monetary resources with a pilot study; instead use your pilot study as a way to assess time and budgetary issues that could occur during the larger study.
- Don’t expect study results, expect “signals” from the data.