Historically, regulatory approval was the primary focus of drug development, and everything drove toward achieving that milestone. In today’s world, the focus has extended well beyond product approval and launch. Payers, regulatory authorities, physicians, patients and sponsors want to know how a product performs in the real world. They’re looking for a clear picture of real drug performance in a diverse population of patients. The new focus is on the evaluation of what actually happens regarding safety and effectiveness when patients are using a newly launched product.
What is real world research and why is it important?
Let’s start with the differences between evidence and outcomes research. In developing a new product, we have a great deal of existing evidence regarding primary safety and efficacy in a controlled population over a relatively short duration. For example, we know the results of clinical trials that were carefully designed, monitored, and reported. What don’t we know? Exactly what will happen once the product is out on the market and used over time.
This is where real world research comes in. Whereas clinical trials give us information about a relatively small number of patients, millions of patients can end up using the product in the real world. So from real world research, we can learn all kinds of things—
How is a drug being prescribed? Which types of physicians are prescribing and treating patients? How are patients using it? What does the safety profile look like in more diverse patient groups over a longer period of time?
Who uses real world outcomes and why?
Real world research is important for different stakeholders. Payers want this information because they don’t want a “me too” product on formulary or reimburse a premium price point. They want real world proof that a product will satisfy an unmet need in the marketplace and provide health economic value. They want to support a product that can demonstrate that it performs better than the rest—not just in clinical trials, but in clinical practice.
Regulatory agencies often want to see outcomes as well. They may mandate further real world research before or after approving a new product. In many countries, any new product that comes to market must be followed in the real world.
For companies developing new treatment, evaluation of clinical outcomes early in the product lifecycle can help prepare them answer questions regulatory agencies may have. This information can also provide valuable insights to help support physician understanding, strengthen competitive strategies, and get a clearer picture of product performance, challenges, and opportunities.
Why haven’t all companies sponsored outcomes studies?
Traditionally, companies completed Phase II and Phase III studies, and then pass the baton to the commercial team for launch activities. There wasn’t always connection or strategic vision across the development cycle. As a result, companies sometimes waited untill later stages to ask themselves, “What health outcomes do we need?” Or they were surprised by a mandate from a regulatory agency, concerns from physicians to change their treatment patterns, or questions from payers causing delays in reimbursement. Today, more and more companies understand the critical need for this evidence early in the drug development process.
How do you collect real world data?
At PRA, we have a two-fold approach to collection and analysis of real world data. We first look at our extensive secondary data assets to determine if we can answer some of the clinical questions from data that we already have. For additional evidence needs, we implement studies to collect specific primary data. In this case, our teams take advantage of new technology to make it easy for patients to participate in these studies. For example, we use an interactive platform on the patient’s mobile device for eConsent, ongoing engagement, wearable data collection and to report outcome data on their phone, laptop or iPad. They participate in the research on their own time, in the privacy of their homes.
What’s it like to work on real world research?
Real world research is completely different from randomized clinical trials. Real physician practices and patients’ lives are not predictable and dictated by a protocol as you would have in a controlled trial. That’s what makes it fun! Participants in real world research are people like you and me. And every product, every disease, every patient group is different, requiring a different protocol and analysis to get exactly the information that we need. It’s no surprise that the specialists that work within PRA’s Real World Solution team are adventurous, inquisitive, innovative, and have great passion for the work that they do. I’ve been involved in a variety of clinical research for 30 years—and I find real world to be the most interesting and rewarding work yet.