The consumer mobile health technology revolution has dramatically altered the way we view health care. Personal health tracking apps and wearables provide real-time and continuous data to the user, allowing them to understand, track, and trend everything from heart rate to blood pressure, from cognitive function to memory, and everything in between. Caregivers can even be notified in the event troubling data is received. The revolution doesn’t just stop there, though. Sensors don’t just provide data on users; they are also embedded in everything from cars to consumer packaging, giving real-time data on surroundings, informing on viability of product, or any of a host of other data points. Finally, tools like telemedicine can provide on-demand knowledge of a medical professional with the convenience of at-home care.
Given that clinical drug trials are testing innovative pharmaceutical and biopharmaceutical products, one might expect these trials to leverage modern mobile health tools in the same way they’re leveraged in the consumer space. Unfortunately, even a cursory look at a typical late-phase clinical trial provides clear evidence that mobile health isn’t leveraged as effectively as it could be. For example, in order to understand if patients took their prescribed medications on time, pharmaceutical companies rely on patients to self-report their compliance (often using a paper diary). Medication is then reconciled by counting pills after the patient returns their supply. Patients have to make multiple trips to the clinic to get medication, have blood drawn, or any of a host of reasons. This is inconvenient for most patients, and can be completely prohibitive if the patient doesn’t live nearby the clinical site.
Finally, data on a patient’s disease state (and ultimately the efficacy of therapy) is only gathered during site visits, and occasionally via patient diary. For a typical six month clinical trial, this could mean only a handful of snapshots of the patient’s health over the course of more than 4,000 hours of treatment. What’s worse – given the limited amount of this data, it’s much more likely to be influenced by a host of external factors, from “white coat syndrome” to whether the patient is having a bad day or is distracted, and may not be fully representative of the patient’s response to therapy over the full course of treatment.
Adopting Mobile Health Should be a Win-Win – But it’s Not That Simple
With the wealth of possibilities, and considering the pace at which digital medicine is evolving in the consumer arena, it begs the question – why isn’t the Pharmaceutical Industry leveraging the full power of mobile health technologies in clinical trials to create a more patient-centric clinical trial experience? What if the patient could be engaged via wearable technology that collects relevant continuous real-time data, at their convenience? Wouldn’t that be a win-win for patients and the pharmaceutical industry? To be fair, there are quite a few challenges to jumping into the mobile health technology pool.
- Clinical trials are planned and executed at a very rapid pace
- Lack of internal expertise (people and process), coupled with negative influencing components of company culture (aversion to change, lack of leadership support, etc.)
- Return on investment can be difficult to justify, as there is often substantial up-front investment without clear quantitative value
- Data collected must meet all applicable standards for reliability, quality, compliance, traceability, and security. It must also take into account the evolving regulations impacting access to patient data (such as the European General Data Protection Regulation, GDPR)
- Lack of clear global regulatory standards. This is especially true for digital endpoints, where equivalency between a newly collectable digital data stream (for example, data collected from a blood pressure sensor) must be validated against the gold standard in-clinic measurement for it to be used in proving medication efficacy
- Impact on patient experience – ideally, the technology should improve patient experience. However, if a technology is too burdensome, invasive, or difficult to use, it may cause a patient to use it incorrectly, or dissuade them from using it entirely
All told, this seems quite daunting. However, there are efforts both within and outside of MSD, to overcome these challenges.
MSD’s Smart Trials team is a cross-functional team, with the goal of creating a more patient-centric and effective clinical trial through use of technologies. Smart Trials investigates and pilots technologies in rapid and clinically-relevant learn-and-confirm cycles. These pilots can be as simple as an internal test by MSD employee volunteers, or they can be conducted as a small trial using a clinical site. Multiple technologies can be piloted in a single trial. These pilots provide a venue where viability of a technology for standard clinical use can be confirmed – kinks worked out, deployment modifications made, business case evaluated, training materials developed, user feedback sought, etc. Pilots include technologies in the following categories:
- Smart Dosing: Technologies that provide insight into medication intake
- Smart Sampling: Solutions to provide flexibility of at-home sampling for pharmacokinetic, pharmacodynamics, or biomarker analysis
- Smart Analytics: Digital platforms to facilitate patient engagement, capture patient feedback, and capture/integrate data from a myriad of sources
- Digital Evidence Capture: Digitally-captured clinical endpoints, or disease-state information. This data can be novel (couldn’t be captured previously), or the digital equivalent to an existing clinical measure
MSD has successfully run several pilots with technologies from these categories, with more planned in 2018. These pilots have provided critical information to help enable adoption of technologies from these categories within standard clinical development programs.
Industry Collaboration through TransCelerate
In addition to MSD internal efforts, there was a clear need for an industry-wide solution to help encourage and facilitate adoption of mobile health technology. TransCelerate, a non-profit organization that works across the biopharmaceutical research and development community to improve the health of people around the world, has organized a Patient Technology initiative. This initiative is focused on supporting the adoption of patient-centric technology in clinical trials. Over 30 participants from 15 biopharmaceutical companies (sponsors) are working together to level-set awareness of the current state of patient-centric technology use in clinical trials, and provide tools to aid in the implementation of these technologies in future trials. The TransCelerate Patient Technology initiative is led by Jeff Ramsey (Eli Lilly and Company; Indianapolis, IN) and Matt Moyer (Merck & Co, Inc, Kenilworth, NJ, USA), and is planning to publish a number of items in 2018 to further this goal:
- A point-of-view paper, describing current landscape of technology use, and forward-looking collaboration opportunities
- A framework for sponsors to share knowledge, as appropriate, about patient-centric technologies used in clinical trials
- A toolkit that sponsors can use, describing the spectrum of considerations when looking to utilize technologies
In addition to TransCelerate, there are also consortia comprised of industry, regulatory agencies, and the private sector aimed at addressing the challenges of using mobile health technologies in clinical trials. Consortia such as the Clinical Trials Transformation Initiative (CTTI) and the European Innovative Medicines Initiative (IMI ) act as collaborative conduits, and provide clear evidence that regulators are willing to work together to advance the science and convenience of clinical trials, in the context of improving patients’ lives. Further evidence of regulatory agency interest can be found in a recent pre-certification pilot that is being facilitated by the FDA. This pilot is, in part, helping better define requirements for mobile health solutions in clinical trials. However, there is still much to be done.
The Blue Sky Opportunity of Mobile Health Technologies
Mobile health technologies offer almost limitless opportunities for patient engagement and richer data collection. Patients, Investigators, Agencies, and the Pharmaceutical Industry are all interested in targeted use of mobile health technologies for clinical drug development research. These types of technologies can provide a digital avenue for endpoint data collection, enable real-time interpretability of clinical trial results, and engage more readily with patients, ultimately reducing their burden and enhancing their experience. Efforts within MSD, coupled with broader collaborations across industry and with regulatory agencies, will make the modernization of clinical trials via mobile health technologies a reality.