Harnessing the Potential of Emerging Digital Health and Biological Sampling Technologies for Clinical Drug Development: Promise to Reality

Abstract

Advances in emerging innovative technologies have led to optimistic outlooks on their transformative potential for healthcare and clinical trials. Given the increased attention, this white paper by the International Consortium for Innovation and Quality in Pharmaceutical Development (IQ) presents perspectives on pharmaceutical and biotechnology industry trends for innovative digital health, adherence, and outpatient sampling technologies. As stimulus for cross-company scientific dialog points to consider for adoption, implementation, and recommendations to broaden uptake are proposed.

Can Digital Health Enable Higher Quality Real‐World Evidence‐Based Decisions?

“…The use of digitally enabled RWD is being increasingly recognized in a number of recent regulatory frameworks or enabling actions . Yet, across a sampling of the pharmaceutical industry, digital health currently is largely in nascent stages of clinical exploration and broad‐scale adoption across the portfolio is lacking . The intent of this commentary is to spur further action in two key areas of interest to clinical and quantitative pharmacology scientists.…”

“…There is growing interest across drug development in leveraging digital health for decision making. Previously, the authors examined the current state, opportunities, and challenges for digital health and patient centric sampling tools in drug development . This commentary expands the dialog to ask: “How can digital health intersect with real‐world data (RWD) to expand the evidence‐base for drug development and regulatory decision making?”…”

“…Importantly, randomized clinical trials (RCTs) and RWD should not be considered mutually exclusive . Digital health is a broad term that describes the convergence of digital technologies intended to collect, share, and manipulate healthcare data with other elements of medical practice, including precision medicine tools to improve and individualize the management, diagnosis, and tracking of disease or health and wellness . It is recognized that as the fields of RWD/RWE and digital health progress, working definitions of these terms will continue to evolve.…”

Digitally Enabled, Patient‐Centric Clinical Trials: Shifting the Drug Development Paradigm

“…All these benefits will ultimately improve the patient experience during the trial and enable improved drug development decisions and understanding of drug and disease effects. 8 Despite all these potential improvements, the relative "explosion" in both the number of digital health technologies as well as their capabilities, and an increased adoption of consumer-grade health-tracking devices in the marketplace, adoption of use of such technologies in pharmaceutical trials has been lagging by comparison. [9][10][11] Some of the challenges to pharmaceutical trial adoption include questions around patient privacy, lack of sufficient validation for digital endpoints, lack of transparency for calculation of endpoints ("black box" algorithms), challenges related to patient adherence and burden of wearing and using devices, operational and data transfer challenges, and regulatory unknowns.…”

Beyond the Randomized Clinical Trial: Innovative Data Science to Close the Pediatric Evidence Gap

“…The use of wearables creates the possibility of continuous data collection in an at-home setting, which supports characterizing the intra-individual and inter-individual variability in disease and drug response, as well as quantifying exposureresponse relationships for drugs in the pediatric population. 28 The latter is especially true if the clinical outcome or a surrogate end point can be quantified at home. Similar to mobile applications, the wearable itself might not only be used to collect data but also to motivate desirable behavior.…”