patient reported outcomes

The drug development environment is in crisis. The time and cost to develop a new drug has skyrocketed to $2.6 billion—an increase of 145% since 2003. With development costs reaching unsustainable levels, we must modernize our most fundamental activity—how we conduct a clinical trial.

One approach that holds great promise is telemedicine, which allows healthcare professionals to evaluate, diagnose and treat patients using telecommunications technology. This enables them to meet the needs of more patients in less time while eliminating the need for travel on both ends.

This technology can have a tremendous impact on clinical research as it allows researchers to access new patient populations, decrease trial burden on participants, and lower the overhead cost of running multiple under-utilized facilities. It also creates an environment where a few leading experts can oversee patient care at many facilities from a central locale.

And it is important that healthcare leaders act today in driving this technology adoption. Technology giants like Apple and Google are already building platforms that attract and aggregate millions of end consumers, and with recent developments, including Apple's ResearchKit and Google's partnership with Novartis, it is apparent that they have plans for the health sector.  These companies have the ability to become powerful players in any health sector that reaches large populations, which is why it is urgent for companies like QuintilesIMS and its partners to acquire the knowledge needed to compete in a world of virtual trials.  

In an effort to achieve this goal and to demonstrate the effectiveness of telemedicine as a clinical research tool, QuintilesIMS and Biogen recently evaluated a telemedicine platform to study remote detection of Alzheimer’s disease and categorization of severity. The project utilizes a telehealth platform to conduct an endpoint validation study in prodromal Alzheimer’s disease comparing the quality of endpoint data collected remotely to a standard in-person visit encounter. This validation study is a necessary first step to receive FDA clearance to conduct a clinical trial in prodromal Alzheimer’s disease relying upon remote assessment of endpoint data through a telehealth platform.

I will be presenting the results of this study at the DIA annual meeting June 18-22 in Chicago.

A hub and spoke model for AD research

Innovative applications of telemedicine could be particularly useful for AD research to reduce the time and cost of trials while minimizing disruptions in the lives of patients and their families. In the study, we implemented telemedicine technology in a hub-and-spoke referral structure where primary care physicians who are not AD experts served as the principal investigators, with links to two neurology experts at a cognitive neuroscience (CNS) specialty site.

Using the hub and spoke telemedicine model, we worked with a site network with active clinical practices that have 450,000 patients receiving clinical care, 25% were 65 and older, which would roughly represent a population of patients with AD (based on medical literature) of 15,000. We identified 12 PCP investigators within this network who served as PI’s and connected them via the telemedicine platform to a CNS expert site (also in the site network). This linking of PCP to specialist site allowed us to recruit briskly patients for this study while confirming the diagnosis and administering cognitive ratings via the platform by CNS experts.

Moreover, we  conducted endpoint validation of the cognitive assessments, that is, we wanted to ensure that the cognitive data collected remotely from patients interviews was comparable to the quality of data collected remotely, using the telemedicine platform. Using a 2-period crossover design for this assessment, we found that cognitive assessments administered remotely had good-to-excellent intra-rater reliability depending on the instrument.  

Finally, patient and caregivers participating in the trial reported high levels of satisfaction with participating virtually in the trial.

In my presentation at DIA, which will be part of the Are We Ready for Telemedicine-Powered Clinical Trials? session, I will share the details of the trial and how it was conducted, and what it means for the future of clinical research.

The scientific, financial, and social benefits of using telemedicine in clinical research is clear. This technology creates an environment where more patients can participate, potentially affecting speed of recruitment (and thus impacting clinical development costs) and lowering patient and family burden related to participation remotely in studies.

This AD study demonstrates the benefits of the hub and spoke telemedicine model, and should be considered as a tool to innovate clinical trials across therapeutic areas.  In the near term, this technology will drive the era of the virtual trial. As the industry seeks more efficient ways to conduct clinical research, telemedicine or virtual trials likely will be a viable approach.