November 21, 2019 Advisory Board's take: 3 ways this approach could disrupt traditional clinical trials

Johnson & Johnson's Janssen pharmaceutical subsidiary will launch the company's first-ever solely digital trial to test its diabetes drug Invokana, the company announced at the American Heart Association conference last weekend.

Digital trial details

For the trial, called CHIEF-HR, Janssen will collaborate with PRA Health Sciences to gather evidence to support a new cardiovascular indication for Invokana. However, instead of relying on in-person medical visits to gather data, Janssen will rely on remote tracking via smartphones and wearables.

The nine-month trial will recruit about 1,900 U.S. adults who were previously diagnosed with preserved or reduced ejection fraction heart failure, regardless of their Type 2 diabetes status. The researchers will use participants' personal mobile devices to screen and onboard participants. 

For the first phase of the trial, patients will be randomly assigned to take either Invokana or a placebo for three months.

After three months, the researchers will inform participants of which treatment they were receiving, and patients will be allowed to decide whether they want to continue with their current therapy or switch therapies.

Throughout the trial, participants will use their personal mobile devices to fill out app-based questionnaires that researchers will use to gauge participants' quality of life. Participants will also log any daily physical activity data, including number of steps and stairs climbed, using wearables that deliver data via smartphone.

Data from the study will be used to support a clinical program for the drug, according to Janssen.

The future of digital trials

According to Paul Stoffels, vice chair of the executive committee and chief scientific officer at J&J, the trial marks "the first-ever completely decentralized, mobile, indication-seeking clinical study."

Stoffels said he believes digital devices will become more popular in clinical trials because "the cost of clinical trials is so high." He added, "[I]t very quickly will change the entire field. Virtual recruitment and inclusion, people being able to be treated at home, digital consultations—that will all help to do faster and simpler trials," he said.

That said, Stoffels acknowledged that "[t]raditional trials are undeniably essential in medical research but are often long and costly."

Separately, C. Michael Gibson, founder of the PERFUSE academic research organization at Beth Israel Deaconess, added that digital trials could be useful in the earlier phases of drug development to determine whether it's necessary to continue the trials. "[It] will help you identify very quickly the most viable candidates" in the earlier stages of the clinical trials process, Gibson said.

The digital trials will also allow researchers and clinicians to return health data to patients in a way "that allows them to understand how treatment and lifestyle choices affect their health," according to Paul Burton, Janssen's VP of medical affairs for internal medicine (Hale, FierceBiotech, 11/16; Muoio, MobiHealthNews, 11/18; PharmaTimes, 11/19).

Advisory Board's take

Janssen's virtual clinical trial is an exciting development given the endless buzz around wearable devices, real-world evidence, and "patient centricity" in health care. We're excited to see that Janssen is disrupting the traditional model of clinical trials and hope the change will lead to more diversity and representation in clinical evidence, as well as more accurate data about patients' true experiences.

We're also interested to see how this model of data ownership plays out. By giving patients direct ownership of their data, Janssen may be able to overcome the data privacy and ownership barriers that would otherwise prevent cross-industry evidence generation. Overall, it will be indicative of the increasing opportunity for pharmaceutical manufacturers to leverage digital capabilities to overcome barriers in clinical trial evidence generation and better prove value to payers, providers, and cross-industry stakeholders.

More specifically, we see three key ways this innovative trial (and future digital approaches) could disrupt traditional methods:

  1. Digital recruitment will increase diversity of patients enrolling in clinical trials—making clinical evidence more representative of real patient populations. Clinical trials today are often scrutinized for their homogenous patient populations and criticized for lacking insight into how treatments work for different subgroups of patients (e.g. those with multiple co-morbidities, age differences, or impacted by the social determinants of health). However, payers and providers want clinical evidence that allows them to predict treatment efficacy in the individual patients they serve (which impacts their approach to formulary/purchasing decisions, guideline development, or prescribing choices). If the digital trial recruitment expands patient representation, it could help advance appropriate treatment use.
  2. Virtual trials can track patients' quality of life—whereas evidence today typically focuses on safety and efficacy. Pharmaceutical companies rarely capture patient-reported outcomes (e.g. quality of life measures, pain scores) in their clinical evidence, but payers and providers often want this information to guide appropriate use. In fact, when we recently held a Cross-Industry Value Summit aimed at understanding how stakeholders across the industry assess treatment value, many participants named the "elephant in the room" that we often assume we know what patients value in their treatment without ever asking them directly. Generating patient-reported outcomes through virtual trials could help provide needed insight.  
  3. Digital data generation can eliminate data ownership issues that currently prevent cross-industry collaboration. Many stakeholders today struggle to build lasting partnerships for evidence generation because of contentious issues of data ownership. However, Janssen's digital trial obviates the need for other parties who may complicate data ownership and compromise privacy rights (such as payers, providers, technology companies). It could also allay patient privacy concerns over data ownership and use—empowering those to participate who may not have otherwise.

However, it's worth approaching this trial with a bit of caution. While this study is exciting, it's not the company's first trial using digital tools in some capacity. Indeed, Janssen's parent company, Johnson & Johnson, helped launch the Apple Watch trial to detect atrial fibrillation (AFib)—a study that, while novel, is currently receiving some pushback for its high drop-off rate and lack of significant physician-patient follow up. While this new study is different from the Apple Watch trial in that all enrolled patients must have diabetes (whereas anyone, regardless of health status could enroll in the AFib study), we can hope that Janssen will identify better ways to keep patients engaged in using the digital platform.

Are you interested in innovative ways to generate evidence and measure value? In light of new, complex therapeutics (e.g. gene therapy, biosimilars) and improvements in technology analytics (e.g. AI, real world evidence), Advisory Board is researching how cross-industry stakeholders define and measure treatment value. If you're interested in sharing your thoughts on value for our upcoming research, let us know by taking this survey or email Pam at DivackP@advisory.com.

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