Janssen says the design will “reimagine” drug development and is the “next evolution” of clinical trials

The study promises to improve clinical research with an innovative and patient-friendly approach. It will use PRA’s mobile platform and wearable devices to more efficiently gather real-world evidence directly from patients. The clinical trial is called CHIEF [an acronym for Canagliflozin: Impact on Health Status, Quality of Life, and Functional Status in Heart Failure (CHIEF-HF)] and will assess the effectiveness and safety of Janssen’s canagliflozin (INVOKANA) in adults with HF.

The CHIEF trial breaks new ground in several ways. This will be the first fully “virtual” trial to support approval of a new drug indication, meaning that patients can participate from home using their phones and computers. Previous mobile trials, such as Apple Heart, were “observational” and had no drug intervention. Other studies combine traditional site visits and mobile data collection, but CHIEF is the first to rely completely on the new decentralized platform in order to seek regulatory approval for a medication.

Another novelty is the use of patients’ own assessments called Patient Reported Outcomes (PROs) as the primary endpoint. Traditionally complex diseases like HF require objective endpoints such as mortality to approve medications. Earlier this year the Food and Drug Administration (FDA) updated its policy on HF to permit approvals based on symptom improvement even without survival benefit in order to help address the desperate need for better drugs in this difficult condition. Since patient responses will be collected using smart devices there will be no required hospital or doctor’s office visits.

This partnership is a major step forward to make clinical trials faster, less expensive, and more patient-centric. This type of new approach could significantly disrupt the $60 billion drug development industry.

Over the past several years there has been much attention from pharmaceutical companies on these types of decentralized, mobile Virtual Clinical Trials (VCTs). A number of small companies generated early excitement, but most encountered difficulties in sustaining recruitment and moving from observational trials to interventional drug approval trials. Various social media outreach efforts got participant attention, but eventually recruitment rates fell to or below industry averages of only about 3% of eligible patients.

Heart failure is a leading cause of hospitalization and causes up to one in nine deaths in the U.S. Unfortunately, treatment options have been limited to supportive care or diuretics and some patients eventually need implantable devices or heart transplants. Canagliflozin is a new class of drugs called sodium-glucose transport protein 2 (SGLT2) inhibitors that were initially approved in diabetes and found to be also effective in heart failure.

PRA is excited about this partnership and its impact on HF patients moving into the future of healthcare.

November 19, 2019 

https://prahs.com/