February 1, 2019 | It’s an exciting time for real-world data and evidence, says Martin Marciniak, US Medical Affairs Vice President leading the Customer Engagement Value Evidence and Outcomes Team at GlaxoSmithKline. With more data available than ever—and an FDA commissioner who views the use of real-world data to improve regulatory decisions as a key strategic priority for the FDA—our options for including new data sources cover the spectrum of drug discovery and clinical trials.

In his role at GSK, Marciniak has the opportunity to find synergies between evidence generation and the end user, whether it be patients, healthcare practitioners, population-based decisions makers or policy makers. His organization’s unique makeup and positioning, evidence generation and communication gives him perspective on what’s next for real-world data and evidence generation.

On behalf of Clinical Research News, Marina Filshtinsky spoke with Marciniak about the types of real-world data, how these data types will change trials, and what this means for patients.

Editor’s note: Marina Filshtinsky, Executive Director of Conferences at Cambridge Healthtech Institute, is planning a track dedicated to Late Stage Research Strategy and Operations at the upcoming Summit for Clinical Ops Executives, SCOPE, in Orlando, February 18-21. Marciniak be speaking on the program. Their conversation has been edited for length and clarity.

Clinical Research News: On December 6, there was a statement from FDA Commissioner Scott Gottlieb on the Agency’s new strategic framework to advance the use of real-world evidence to support development of drugs and biologics. Would you mind commenting on that?

Martin Marciniak: It's a very exciting time. Healthcare data is expanding rapidly in its many different and unique forms. Dr. Gottlieb has been a champion of different approaches to data and evidence generation and their use in drug development for a number of years. Seeing him come forward proactively in the discussion around what that might look like moving forward is very exciting. When you think about real-world data in healthcare, you also think about real-world evidence: the transformation of data in its many forms to be used in medical decision-making. It is important to note that data has context based on the end user of the evidence generated. This contextualization includes, but is not limited to regulators, clinicians, population-based decision makers, and increasingly patients.

Real-world data in healthcare is now collected from a variety of sources and offers unique opportunities for its utilization. Traditionally, real-world data would focus on administrative claims. This data is the result of having prescriptions filled at your local pharmacy, or through medical claims for physician or hospital visits or services being adjudicated by an insurance plan. In recent years, this type of data augmented through electronic medical records – bridging the gap between the administrative claim and clinical outcomes. Today, we have data from what might be considered non-traditional sources: genetic observational data, and social media. This new data represents the “undiscovered country” of opportunities in drug target development, post-marketing surveillance studies, and health technology assessment.

Regardless of the data source or its ultimate transformation into evidence, the goal is to leverage the available information to make informed health care decisions. The goal depends on where you sit in healthcare. If you are a patient, you have more information about the therapies you are taking and how they make work in you. If you are a clinician, you have real world evidence to help make a more informed decision about your patient’s care. If you are a population-based decision maker, it is about the appropriate placement of therapies and interventions on a health plan’s formulary. If you are a pharmaceutical company, it is combination of goals, therapy area targets for development, safety and surveillance, and health technology assessments. In the end, it is about providing answers that matter to make better and more informed decisions for patients.

The applications of real-world data go across all the stages of drug development and beyond. Does GSK have a system in place that allows it to take advantage of real-world evidence throughout a life cycle?

Like many companies, we do. It utilizes several different approaches ranging from drug discovery to safety and surveillance work. It includes but is not limited to organizations such as statistics, clinical operations and epidemiology, as well as health outcomes and market research. Many companies have observational data warehouses where they can do analyses to help us better understand a therapy as it moves through development process. In some cases, the research may seek to identify an unmet therapeutic need. In other cases, it may focus on patient identification for a clinical development program. With unique real-world data, it may be more focused on identification of a therapeutic target for further exploration, or the detection of an early safety signal. In the end, these are pieces of the puzzle. The data represents an opportunity for evidence generation and leverage its use in the many forms of medical decision making.

A key element for real-world data is that it has context such as regulatory, clinical, and population-based decision makers. New data entrants add an important voice—the patient. This is most directly observed in social media where patients are directly sharing their personal stories and experiences. In other cases, it may be sharing their genetic information. In all cases, there are considerations to be made: is the data fit for the question the patient, practitioner, or scientist is asking. It gets to whether the data is both relevant and of sufficient quality and takes place prior to methodological consideration. There are a number of different initiatives both within and external to pharmaceutical companies which are very important in formulating an overall strategy for how data gets used to bring answers that matter to different patient populations. These initiatives often focus on providing greater accessibility to end users. Innovation in this area is rapid, with many tools being created to leverage data use. Pharmaceutical companies play a role in this innovation but are only one piece of a complex puzzle operating in this space. They may also not be the most important player in this area.

How can your data change conditional clinical trials?

I think that there's a great deal of excitement about this topic. The FDA has used this type of data recently in label extensions. With that in mind, it’s not just a question of how it is going to change the clinical trials. It's about how is going to change the nature of research moving forward, which also includes observational studies, prospective observational studies, pragmatic clinical trials and clinical trials. With respect to prospective clinical trials, I believe everybody recognizes that they are very expensive research programs. The opportunity to use real-world data in its many different forms allows us the opportunity to consider different in clinical development and post marketing programs options. . First, from a development perspective, real-world data affords the clinical development teams the opportunity to optimize their studies (e.g. patient identification, study enrichment et al.) That affords the opportunity to change the cost structure of studies, and bend the overall cost curve associated with drug development programs. Second, from a post-marketing perspective, real-world data affords us the opportunity to interweave and tell a layered evidence driven story from a development, regulatory, clinical and population based decision making perspective concerning therapies. The FDA’s recent incorporation of this type of evidence into their decision-making process, in combination with 21^st Century Cures, makes this a very exciting time for individuals innovating and working in this space.

Who has the ownership of the real-world data and how are real-world evidence and real-world data changing the role of patient in clinical and observational research?

At this time, there is more data available to inform medical decisions—and it is only going to grow faster in the coming years. Real world data, and ultimately the evidence generated by it, put the patient at the center of health care decision making. This affords the patient the opportunity to play a more proximal role in their health care and own the decisions in a different way than they have had the opportunity to do so previously.