How one pharmaceutical company is reinventing the clinical trial

Millions of Americans— most of us, in fact — rely on medications, from prescription drugs for high cholesterol, diabetes, asthma, cancer, and the like to over-the-counter medicines for allergy, acne, and other conditions.

While the myriad medications we take differ from one another, they have something important in common: Before they were sold to a single person, they were tested by people who volunteered to take part in clinical trials. It was only after these trials demonstrated the drugs had favorable benefit-risk profiles that they were able to make their way to pharmacy shelves.

Clinical trials are as essential today as drug discovery for bringing new drugs to market. But while new tools and technologies have helped advance techniques for new drug discovery, we’ve seen far less innovation in clinical trials. The process continues to be weighed down with regulatory paperwork, medical jargon, and participants’ time-intensive trips to trial sites.

At the same time, increasingly complex clinical study designs and the growing demand for larger and more diverse pools have made it harder to find eligible clinical trial participants. Two-thirds of study sites fail to meet their enrollment goal for a given trial, which significantly slows the pace of bringing new medicines to patients.

The struggle continues after patients enroll, as dropouts are common. The average dropout rate across all clinical trials is as high as 30 percent. It’s clear that patient engagement is just as important to the success of a trial as patient recruitment.

Dropouts and failing to meet enrollment goals are troubling and expensive problems, felt deeply across the pharmaceutical industry. Creating solutions for them is the goal of my team at Janssen Clinical Innovation, part of Janssen Research & Development, LLC. We’re exploring digital health, non-conventional trial models, enhanced patient communications, and industry-wide collaborations to make trials more engaging, rewarding, and streamlined for patients. We believe doing that will not only bring down the cost of drug development but will also help new drugs reach the marketplace sooner. Here’s a look at several key projects we have underway:

Bring the trial to the patient

Digital technologies open new possibilities for clinical research. They can, for example, allow patients to participate in trials from their homes. Direct-to-patient trial models, or “siteless” clinical trials, use tools such as telemedicine along with wearable devices and sensors for remote data collection.

Janssen recently collaborated with Scripps Translational Science Institute, Aetna, and iRhythm Technologies to understand how digital technology can improve large-scale observation and treatment programs. A home-based study of 2,659 volunteers, called mSToPS (short for mHealth Screening To Prevent Strokes), evaluated a wearable electrocardiogram patch as a new way to remotely detect atrial fibrillation.

The mSToPS results, published this summer in the Journal of the American Medical Association, showed that patients with asymptomatic atrial fibrillation were diagnosed earlier with the patch than those who received only routine medical care. This is a big deal, as untreated atrial fibrillation is associated with a fivefold increased risk for stroke.

We’ve since expanded this direct-to-patient approach to study individuals at risk for rheumatoid arthritis. We are using real-world data, including insurance databases from Aetna’s Healthagen business, to identify participants with the condition who are eligible to be invited to join study. More pilots, looking at different aspects of this direct-to-patient approach, will be needed to fully understand where it can add value for patients and sponsors and where a traditional, site-based approach is the best choice.

Empower patients by giving them what they want: their health data

People are hungry for personal health data. This isn’t breaking news. But until now, sharing data with clinical trial participants hasn’t been standard operating procedure. Recent industrywide research shows that clinical trial participants often have to go through cumbersome processes to obtain data about them that is collected in the trial, either because they want to share it with a doctor or just because they are interested.

In the wake of that research, Janssen created a way to give patients some of their data during trials without compromising the integrity of the research.

Through the program, participants have access to lab results and other health data collected during trial visits. If, for some reason, a patient drops out of a trial or doesn’t qualify after an initial evaluation, he or she can still use the data to find other clinical trial options, share the information with caregivers, or donate it for the common good. In one Phase 3 study, participants who signed up for the “data return” platform received a complete set of the health data collected during their initial health examination. In a Phase 3 extension study, participants accessed their lab results via the platform.

This initiative is part of an umbrella project called Global Trial Community, which aims to keep patients engaged before, during, and after they participate in clinical trials — for example, to learn about the outcome of the trial (think lay summaries), give feedback on their trial experiences, or stay in touch with a community of like-minded patients. We aim to expand this platform to 26 countries by the end of 2018 and believe it represents a new standard for responsible clinical trial data sharing. It is part of our goal to transform passive trial participants into engaged research partners.

Using technology to enable more efficient clinical trials

In conventional clinical trials, participation isn’t burdensome just for patients. Study sites also struggle with labor-intensive data collection, as well as with less than state-of-the-art administrative and supply tracking processes. Fortunately, new technologies are replacing paperwork while at the same time resulting in richer, more accurate data. Electronic tools can help improve logistics, patient communications, and data reporting, all of which are critical factors in keeping trials running smoothly and efficiently.

We recently incorporated a digital technology platform into a Phase 2 registrational clinical trial that addresses many of the paperwork and logistical challenges common in clinical studies. The platform, called iSTEP (short for Integrated Smart Trial & Engagement Platform), uses scanners to track when medication kits arrive and are returned, and sends patients customized information like dosing instructions and tutorial videos that used to come in booklet form. It also includes electronic drug labels in a patient’s preferred language that can be updated immediately to inform patients of protocol changes after a trial has begun. An “eAdherence” tool uses tech-enabled blister packs to track each pill as it is removed, providing a signal for real-time intervention if patients don’t remove a dose at the scheduled time.

Janssen isn’t alone in its efforts to improve the clinical trial landscape: Major pharmaceutical companies around the world are seeking to modernize clinical trials in one way or another through independent efforts and in concert with one another.

Collaboration and knowledge sharing happens through organizations such as the nonprofit TransCelerate BioPharma, through which our company works with other pharma companies to create tools available for industrywide adoption. An example is the Investigator Databank, which seeks to reduce the administrative burden for investigators and helps drug sponsors find the right investigators for specific study protocols.

I believe we are at the start of transformational shift in which clinical trials will one day offer the same level of customer focus that people have come to expect in other areas of their lives. The industry has much work to do before our collective efforts are scaled up to the point that they’re making a visible difference in global patient recruitment, retention, and satisfaction. And it goes without saying that at every step of the way we must diligently adhere to the stringent rules for clinical trial conduct and reporting set by regulatory authorities.

In other words, challenges abound. But we are up for it, knowing that our work has the potential to make clinical trials more rewarding for patients and investigators, and speed the delivery of new therapies for improving health.

Andreas Koester, M.D., is vice president and global head of Janssen Clinical Innovation.