The greatest impact of real world research is the industry-wide realization that it's is not a “nice to have”—it’s a “must have.” If companies want premium reimbursement, they must show payers that their product’s benefit-risk profile demonstrates clinical, economic, and humanistic value. Being incrementally better than existing therapies may not be enough. And outcomes research—following the product’s impact on the lives of patients over time—is an important step toward providing the evidence to help pharma companies achieve this.
Our Maria Harrison explores:
How has outcomes-based reimbursement impacted the pharma industry?
In the past, companies were primarily focused on the research required for submission. Conducting clinical trials, gaining product approval, making product available to prescribers—that is what spelled success. Today, product launch is still the big event, but it is no longer the finish line. It is simply an important milestone in the lifecycle of the product. What happens after launch is also key: How will the product behave when it goes to market? How will it affect possibly thousands of patients over months and years of use? How does the treatment affect the different types of patients that were excluded from the clinical trials? These are questions that clinical trials alone can’t answer.
This focus on outcomes-based research has been transformative for the pharma industry. And reimbursement is not the only factor driving real world studies. Increasingly, regulatory agencies are mandating further outcomes research. We’re seeing this globally as well: in many countries, any product that comes to market must be followed in the real world to obtain additional safety and/or effectiveness data.
Innovative developers are starting early to prepare for what they’ll need to know long-term. Clearly, companies that have planned real world studies early in product development are well-positioned to capture important information, proactively satisfy regulatory agency requirements, and leverage data that inform strategic decisions in a competitive marketplace. To stay ahead of the game, companies need to have answers today to the questions they are going to be asked tomorrow.
How has this landscape changed how pharma companies develop their products?
Ten years ago, pharma companies were only conducting post-marketing studies to meet regularly commitments or to support Medical Affairs activities. In the current reimbursement and regulatory landscape, that is no longer the model. Today, companies are realizing the value of well-designed real-world studies, and they are planning them as early as Phase 1. They are excited about the possibilities of what they can learn from real-world research, beyond providing mandated supplemental data and creating brand awareness.
As regulatory and advocacy groups demand to see outcomes much sooner, so has real world research migrated earlier in the product development planning process. One example is a natural history study of patients with Crohn’s disease, which was initiated early in Phase II and has been underway for nine years. Research like this provides an unprecedented opportunity to understand how a disease progresses before, during, and after treatment. We can look at years of information about the patient’s experience with their disease and its treatment in a naturalistic setting. We can see if treatment slows progression and how it impacts quality of life. We can get to know the disease space in a new way. This research also provides a baseline dataset of untreated patients to compare against treated patients. In short, we can understand the true benefit of a treatment in the lives of the patients who need it.
Pharma companies are now realizing that real world studies can be powerful and revealing. They may uncover strengths and weaknesses of treatment or point product developers in new directions. When collected and analyzed correctly, real world outcomes have the potential to reveal truths that aren’t seen within the structured and limited confines of clinical trials. When a company gets a mandate to do outcomes research, there is an opportunity to ‘make lemonade out of lemons’. It can be a great opportunity to gather more information to support a product. As a result of the demand for real world data, more and more companies are prioritizing the long view—investing in this research, optimizing what they can learn from it, and using it to inform their products for physicians and patients.
What do payers want from pharma companies?
For payers, regulatory approval isn’t enough. They aren’t interested in supporting a product just because it’s new to the market. Payers want pharma companies to provide them with convincing evidence of a product’s benefit over existing therapies. They are looking for robust clinical outcomes, comparative data, and peer-reviewed published articles that demonstrate the brand’s value in a broader context. They are asking where the product fits in the treatment paradigm. Payers want to know that a new product isn’t just a “me too,” but that it will truly satisfy an unmet need. And they expect pharma companies to come to the table with proof.
When pharma companies take the initiative to sponsor real-world research, everyone wins. Payers get real world product data. Companies get invaluable intelligence about their product’s impact on the lives of the patients who use it. And patients get access to effective new therapies. New technologies are making it easier to reach patients in efficient, meaningful new ways. For example, at PRA, we reach patients with an interactive platform on the patient’s mobile device. This seamlessly integrates a study into a patient’s daily life, allowing them to report outcome data with ease, at their convenience, and in the privacy of their homes. This becomes an ecosystem of support and connection that literally puts study participation in the palms of patients’ hands.
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