Janssen Pharmaceuticals and PRA Health Sciences recently designed the first-ever completely decentralized, mobile, indication-seeking clinical trial, which will assess the effectiveness of INVOKANA (canagliflozin) in adults with heart failure.
 
This represents the first fully virtual trial to support approval of a new drug indication, in which patients can leverage their smartphones and wearable devices.   
 
The study, which started recruiting in early 2020, will rely completely on the decentralized platform – patients will enroll from home, the drug will be shipped directly to their homes, and patients will be required to provide guided assessments of their symptoms.  
 
Until now, virtual trials have been observational using wearable device data. With PRA’s model, actual drug trials can be designed with patient burden, trial costs and timelines for completion reduced.
 
Contract Pharma spoke with PRA’s Kent Thoelke, Chief Scientific Officer & Executive Vice President and Greg Licholai, Senior Vice President & Chief Medical Information Officer about the alliance with Janssen, advances that have made virtual clinical trials possible, and the obstacles that remain. –KB
 
Contract Pharma: Please briefly describe PRA’s partnership with Janssen and the goal of the alliance
 
PRA and Janssen have partnered to utilize PRA’s patient facing mobile platform to launch the first-ever completely decentralized virtual clinical trial to assess the effectiveness of INVOKANA (canagliflozin) in adults with heart failure, with or without type 2 diabetes. Instead of relying on traditional in-person medical visits, the study will rely on PRA’s mobile platform to remotely track patient healthcare data via smartphones and wearable devices, and patient-reported outcomes will also be tracked and analyzed. The goal of this partnership is to develop a clinical study model that will lower the patient burden to participate in clinical trials and give patients more access to clinical research as a treatment option. For PRA and Janssen, it’s all about changing the way clinical research is conducted while lowering patient burden and improving access for patients.   
 
CP: How does PRA’s virtual platform work and what services are provided?
 
In lieu of in-person clinical visits, participants will utilize PRA’s mobile platform, smartphones, and wearable devices to complete the study. Participants will enroll in the trial from their homes, have their drugs delivered to them, and provide guided assessments for their symptoms all via interactions on the PRA mobile platform. Participants will also have their daily physical activity data recorded using wearable devices that deliver data via smartphone.
 
By using PRA’s mobile platform that allows patient data to be collected continuously in real time, better results should be collected because of the higher level of interaction with patients and reliance on directly collected metrics. This study design methodology will allow us to engage with patients more often than a traditional clinical trial design, including increasing compliance by pushing reminders via the platform and asking them to complete specific study tasks. Data from the study will be used to support a clinical program to evaluate drug efficacy.
 
CP: What advances have been made to allow for the implementation of virtual clinical trials? 
 
There are multiple advances that have made the implementation of virtual clinical trials possible. These include, but are not limited to, the technology applications and mobile healthcare that allow patients to interact directly with their healthcare. Additionally, connected healthcare devices that integrate into mobile platforms have allowed us the ability to collect patient level healthcare data in real time in ways we have not been able to do in the past.

However, regulatory support is critical for these novel trial designs to work. Pharmaceutical companies will need to feel adequately confident the trials will produce the sufficient data and evidence needed for regulatory approval by the U.S. Food and Drug Administration. To that end, the FDA has been willing to work with Janssen and PRA to approve the current trial design and model. Much of the willingness of the FDA to work on novel trial approaches such as this one is the 21^st Century Cure Act which gave the FDA a framework to promote innovative clinical trial designs to provide broader access to clinical research as a care option. The FDA has recognized that it can help encourage digital health and decentralized trials by making its policies and processes more efficient and modernizing its regulatory tools. 

CP: What are some of the obstacles that remain for the success of virtual trials?
 
The primary obstacle that remains today is the willingness of pharma and biotech companies to deploy these novel designs. Fear of departing from a long history and experience of deploying traditional trial design paradigm is a considerable barrier. While the Janssen/JNJ/PRA study is fully virtual/decentralized, for most sponsor companies it is more likely that hybrid approaches will be used initially – deploying some digital components or replacing some in-person visits with virtual visits utilizing mobile/connected and telehealth platforms as well as local providers and home healthcare nurses.
 
Data continues to show that patients are increasingly preferring to receive their healthcare in a digital, interactive model. As healthcare continues to expand into telehealth and decentralized models, the attractiveness of participating in the traditional clinical trial model continues to decrease. The barriers to participating in traditional trial models continue to be high – resulting in a current participation rate of <5%. Given this paradigm shift in how healthcare is delivered, it is essential that the pharma and biotech industry adopt trial designs that more closely resemble how healthcare is being delivered while lowering the burden for patient participation. 
 
The current drug development paradigm is simply unsustainable from a cost and time perspective. The ability to deploy decentralized/virtual trials allows more patients to participate in clinical trials by lowering the barriers to entry and participation. This will translate into far higher study recruitment rates, thereby significantly decreasing overall trial timelines and total development costs.
 
As the industry moves to the decentralized model, we will see more patients get involved in clinical research – that’s good news for our industry and for overall patient health and well-being. This transformation and new approach could significantly change the $60 billion drug development industry.