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New technologies are revolutionizing clinical trials and transforming drug and biologic development in ways that are unprecedented. In the process, digital data capture technologies allow for increasing data types and volume, improved accuracy and precision, and reductions in variance. They are also opening a window for capturing “real-world” data, minimizing patient inconvenience, increasing patient compliance, and decreasing site-monitoring costs. These new technologies are helping to transform the concept of what should constitute a medicine, so that new types of tech-enabled therapeutics will advance the ways in which both medical treatments are delivered and how clinical trials are performed.
There are many goals associated with using the new digital technologies in clinical trials. Among these are using wearable biosensors (or “unawearables”) and “smart delivery systems” to collect large amounts of new types of data, as well as to analyze them in sophisticated ways in “near real-time.” These approaches are helping to transform the mechanics of clinical research so that site-less trials are now becoming possibilities in some therapeutic areas. The pace of ongoing evolution has been rapid, and according to the editors of Applied Clinical Trials, “the ability to amalgamate, organize and analyze real-world data sets with structured data sets… can provide new, provable indications of hidden relationships that can precipitate better support, new hypotheses and provoke new, potentially life-saving questions that we didn’t think to ask before.”
Pharmaceutical companies are attempting to leverage the newer digital technologies to help develop targeted therapeutics faster, more efficiently, and more cheaply than ever before. In many cases, the technologies to do so have already been developed. Yet, to date, they are not being used at scale. So, why isn’t the world of clinical trials changing for the better more rapidly? The answer is the need for clinical validation (and qualification) of what amounts to a new world of digital biomarkers. As with wet biomarkers, these must be rigorously validated both technically and clinically to ensure that they are proper indicators of meaningful clinical changes – ones that are both useful and robust.
One of the current challenges faced by pharmaceutical companies is that there is an intrinsic mismatch between the development times for high assurance “smart medical devices” and overall drug development processes due to intrinsic cycle time differences. As an example, consumer devices/ software can typically be developed in 6-18 months (or less). For devices with medical-grade functions, development times can range up to 3-5 years, with iterative device upgrades occurring every 1-2 years. In contrast, drug/ biologic development programs generally follow a more conservative and risk-averse strategy. The resulting timelines can be lengthy due to the lifecycle/ biology of the disease under consideration, and they often require up to 6-8 years (i.e., patients must be recruited and retained in clinical trials to assess differences in hard clinical outcomes).
In general, medical device development is iterative and guided by Design Control and Quality System principles that foster rapid engineering processes and quick development times. To accelerate these processes further, medical device manufacturers have attempted to adopt agile development paradigms that retain the necessary compliance characteristics from a regulatory standpoint. This faster pace introduces a complication: Numerous versions of a medical device may be developed over the course of a single drug development program, resulting in the need to re-qualify and cross-validate the outputs of the devices to ensure that their data are consistent. These must then be mated to analytical paradigms that are fit-for-purpose and qualified to assess clinically meaningful differences and treatment effects. Lastly, the regulatory landscape for “digital medical devices” is evolving in different and non-equivalent ways in the United States, EU, and Japan.
In summary, currently available digital technologies represent a likely tipping point for the way that clinical trials of the future will be conducted. The challenge is to determine how to deploy currently available technology sensibly in order to advance the clinical trial process and existing drug pipelines faster.
The speaker, Gerard G. Nahum, MD (Vice President of Clinical Development at Bayer Pharmaceuticals, Head of Medical Devices & eHealth), will address these various issues from a pharmaceutical industry perspective and outline the challenges that explain why we haven't yet seen adoption of these new digital technologies in clinical trials at scale.
Gerard G. Nahum is Vice President of Global Research & Development, Medical Devices & eHealth at Bayer Pharmaceuticals. Previously, he was Vice President of Global Clinical Development for General Medicine at Bayer HealthCare, and before that Senior Director of US Medical Affairs at Berlex/ Bayer HealthCare. Dr. Nahum has also been FDA Medical Officer at the Center for Drug Evaluation and Research in the Office of New Drugs, Associate Professor at the Duke University School of Medicine, and Adjunct Professor at the Uniformed Services University of the Health Sciences. He was in private practice for 10 years, followed by six years at the Duke University Medical Center.
Dr. Nahum is the first physician in the world to successfully deliver a set of surviving twins from a woman with a rudimentary horn uterus, initially by Cesarean section and then vaginally (births separated by eight days). He is the BIO and FDA-approved Industry Representative to the FDA’s BRUDAC Advisory Committee, in addition to the Industry Representative for the FDA Risk Communication Advisory Committee. He is a graduate of Yale University (BS, Engineering and Chemistry, 1978) and of the Stanford University School of Medicine (MD, 1984). He has authored 65 peer-reviewed articles and chapters, in addition to three books.
Dr. Nahum currently leads a team of experts providing internal advice to the Bayer Pharmaceutical R&D Division regarding Medical Devices and relevant aspects of eHealth. The group supports the success of Medical Device utilization and the advancement of eHealth platforms in a highly competitive and rapidly changing environment.
His group’s responsibilities include:
- the support of global clinical strategies for Medical Devices and relevant aspects of eHealth
- assisting in clinical trial design with the aim of streamlining study execution and optimizing product value (e.g., including new indications, new formulations, combination products, etc.)
- assisting in the evaluation of Medical Devices for mobile electronic data capture and R&D aspects of eHealth, including advanced analytics and relevant aspects of artificial intelligence
- supporting the search, evaluation, and execution of in-licensing opportunities in the Medical Device and eHealth spaces.
