GSK Addresses Clinical Outcomes Through Patient-Centric Studies

The need for the biopharmaceutical industry to demonstrate the value of medical products to both regulatory bodies and payers is changing clinical trial design in order to generate data from real world settings. In this interview, Dr. Catherine Bonuccelli, Vice President and Therapeutic Area Head of Respiratory at GSK, discusses the Salford Lung Study, its patient-centric design, and how it is different from the traditional randomized clinical trial.   Moe Alsumidaie: Can you describe the differences between a regular randomized clinical trial (RCT) and Salford’s real-world study design? What were the study’s endpoints and objectives?  

Catherine Bonuccelli

Catherine Bonuccelli: Traditional RCTs are fundamental to our review and approval process, and the key purpose is to establish critical safety and efficacy data in a highly selective patient population. RCTs are very controlled; we ask very narrow questions by focusing on uncovering drug efficacy and safety. However, confounding factors, such as patient preferences, and the experiences of everyday clinical practice are removed; it doesn't get to some of the fundamentals of what people really want to know, such as how patient comorbidities, behaviors, and preferences affect how the drug performs.    In order to address that gap, GSK spearheaded the Salford Lung Study, which we believe is the world's first pragmatically designed study to get this kind of complementary data to the traditional RCT data. The Salford Study allowed us to look at a question of the effectiveness of our new COPD medicine (Breo Ellipta) in that real-world environment (i.e., everyday clinical practice). Planning for the study started while the traditional pivotal RCTs were in progress. It was initiated after they had completed, but prior to the product receiving a marketing license. The primary endpoint was the annual rate of moderate to severe exacerbation of COPD. There is a parallel study in asthma. The COPD study enrolled 2,800 patients. The one in asthma has enrolled 4,000 patients.   MA: What drove GSK to design and initiate the Salford study? What will this data be used for?   CB: In healthcare right now there is a patient centric revolution. Patients know more about healthcare, and patients are doing their own work on making those healthcare decisions; they want different kinds of information to weigh and consider. The other factor is that the healthcare community as a whole, including providers, want a different kind of information set than what the traditional RCTs give. They want to know what the value and impact of these medicines are in a less controlled environment. So the idea behind the Salford Lung Study is ‘let’s go to those patients where they live, go to their world and conduct our study in their normal practice setting to see how effective and safe the medicine is in that broad population and setting.’ It’s an evolution in trial design. The Salford Study is a first step in the use of new research to better understand the medicine's full potential in everyday clinical practice, but is still scientifically rigorous.   MA: Is the Salford Study design going to be the future of clinical trials, as it relates to both regulatory and payer approval?   CB: I believe that both regulators and payers want to understand if new medicines provide significant benefits compared to what already exists. In order to answer that question, we need to constantly change how we conduct our clinical trials. We are always looking for new ways to get the kind of evidence that both payers and regulators want and this is one way. It was a big undertaking and can only be done in an environment where all parties are in collaboration--from the EMR systems, physicians and hospitals, so in that way it may or may not become the way of the future. I believe that this type of study is complementary to the efficacy and safety data from traditional RCTs, but not a replacement. I think we will see more of this kind of novel research design trying to get to the fundamental question of how we can better capture the impact of these new medicines in settings where you can get any other existing therapy.   MA: Please describe how the Salford study is patient centric.   CB: This is my favorite aspect of the study because we are really trying to think more about how to put the patient in the center of not only in how we market medicines, but also in how we do research. The way that I see this as being patient centric, is if you think of a traditional clinical trial we pluck the patient from where they normally get their care, we put them in a controlled setting where research staff do investigational studies for a living. We put the patient in a bit of an artificial place. So recruiting patients into those studies has gotten harder and harder over time even though we have more and more questions to answer. Studies like Salford put the patient at the center by going where the patient normally gets their clinical care and then collecting the information we need to extract through the use of things like the technology platform that was available in Salford: the integrated electronic health record. So for us, it was a way that we could include patients in a trial that closely mimics their experience outside the parameters of an RCT.   Catherine Bonuccelli will expand on the research mentioned in this article on October 10-11 in Philadelphia, at the eyeforpharma Patient Centered Clinical Trials Summit.