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Small But Mighty: ASCPT Convenes Discussion of Innovative Clinical Trial Designs in Small Patient Populations

This week, the American Society of Clinical Pharmacology and Therapeutics (ASCPT) held a two-day satellite session dedicated to discussing the challenges of conducting clinical trials in patient populations of limited size—a topic that is gaining attention for several reasons, in part because drug development in rare diseases is more common than it was a decade or two ago. But our understanding of the pathophysiology of diseases, which were historically considered to be large indications, is also changing rapidly due to a deeper understanding of the genetic and molecular backgrounds of diseases and phenotypes. Many diseases are really a constellation of different conditions with somewhat overlapping symptoms. For example, pediatric allergic asthma is a very different disease than adult corticosteroid refractory asthma. Heterogeneous symptoms can often require studies in patient subpopulations as well.

The ASCPT satellite session brought together a diverse group of scientists to discuss this challenge, including drug metabolism and pharmacokinetic scientists, clinical pharmacologists, pharmacometricians, statisticians, regulatory scientists, biomarker clinical scientists, and digital technology service providers. Our knowledge continues expanding. The approaches to solving this problem include using Bayesian statistics, historical data and synthetic study arms, surrogate endpoints and digital biomarkers, AI tools, and methodologies to handle missing data. Patient-centricity is at the forefront, from finding disease features meaningful to patients to utilizing clinically meaningful endpoints to interpreting results by contextualizing them with patient narratives. The session included both scientific presentations as well as breakout groups who worked hard using innovative thinking to come up with clinical study designs for drug development with a limited number of study subjects. The discussions were invigorating, as participants challenged the status quo of conventional clinical trials.

At Koneksa, we are committed to designing, developing and deploying digital biomarkers in clinical studies. As highlighted in this ASCPT session, digital biomarkers can be powerful tools for accelerating drug development and tailoring clinical studies to small populations. This can be done in several ways. Remote data collection by means of digital health technologies, such as wearable sensors, can enable frequent, dense data collection, which can reduce measurement error and increase study power. Our paper on mobile spirometry in patients with asthma shows that this approach is feasible. Additionally, remote data collection by means of body-worn sensors and mobile apps can enable patient participation by bringing clinical studies to patients’ homes, as described in our joint publication with Merck.

Our research in partnership with pharma companies and academic institutions continues to foster out-of-the-box ideas and novel designs to move beyond conventional methods and meet this unmet need.

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