Why biopharma and biotech companies must understand the availability, strengths, and limitations of RWD methods
Barbara Arone, VP of real-world solutions for IQVIA, focuses her efforts on the development and operations of real-world evidence (RWE) research methods for regulators, clinicians, patients, and payers.
She recently joined Pharma Commerce to discuss not only the importance of understanding and demonstrating the value of RWE, but facilitating its application as well.
Pharma Commerce: In terms of the strengths and limitations of RWE, what do biopharma and biotech companies need to understand to determine the most appropriate applications?
Barbara Arone: The most important aspect of utilizing real-world data (RWD) is understanding the correct application for the specific goal and audience. Different RWD types will be appropriate in different places. One source may be appropriate and compelling for a payer but not for a regulator, and what may be suitable for a regulator may not be a fit for a physician.
For example, while a regulator may be impressed that a product targeting progressive vision loss can allow a patient to stay on the same row of an eye chart, the physician and patient would be more concerned about whether the patient will be able to continue driving their car. It’s about what the sponsor wants to prove and to whom they are trying to prove it. Therefore, biopharma and biotech companies should evaluate how RWD will help them achieve their goal, whether it’s securing FDA approval, or marketing a new treatment to physicians and their patients.
The expectation is that the industry’s view of RWE will shift from “nice to have” to an integral part of new product development and a required element to demonstrate value to stakeholders. How do you see this shift evolving in the next few years? What kind of timeframe are we looking at?
The increased adoption of RWE is a testament to the growing industry focus on rare diseases and precision medicine. For more personalized treatments to be identified, researchers need to know what disease classification each patient has, including the type of mutation, the imaging at certain stages, and more. In order to get someone to invest in the product, even in the earliest stages, key metrics such as market size and patient burden must be demonstrated to prove the need is there. RWD is an excellent tool for estimating the market size and the patient burden, which is key to demonstrating value to stakeholders and can act as a foundation for external comparators.
The growing pressure on regulators from industry and patients regarding approvals for treatments supported by RWD is a great start. Still, the true shift in regulatory operating procedures will be very slow moving.
What do you think has been the effect of the pandemic on the attitude and approach to RWE, both on the part of industry and patients?
The pandemic pushed industry leaders and regulators to reevaluate their views of what was possible in terms of moving away from traditional, on-site clinical trial processes. Before the pandemic, it was widely believed that shipping drugs directly to the patients and allowing them to record their own symptom progress without on-site clinical support would be infeasible, if not impossible. Fast-forward to 2022, and patients are getting treatments shipped to them, recording their own symptom progress, and meeting with their care teams without leaving their homes.
From the patient perspective, there has been a desire for more convenient and efficient processes for managing their health and participating in clinical trials. The reality is that many patients have wanted to be involved in the research process but haven’t been able to do so for various reasons (e.g., lack of transportation, limited access to childcare or time off work, etc.). So, in many ways, the clinical research and care changes that allow them to be more actively involved in their healthcare have been long-awaited upgrades.
However, it is too early to tell what industry attitudes will be toward the role of RWE in the new clinical trial process. There will always be situations where the use of RWE isn't appropriate, but the pandemic has pushed the industry to open its eyes more to the situations that would benefit from using RWE.
What are the regulators doing to promote the use of RWE? Is enough being done to urge companies to embrace it further?
With the RWE program and recent draft guidances, the FDA has sent a strong signal that it's willing to evaluate treatments that have been developed using RWD. The FDA has shown support for RWE adoption, so the best path forward is for industry and patient communities to partner on ways to drive momentum. That could include the compilation of case studies highlighting the effectiveness of RWE in treatment outcomes or creating industry standards that are complementary to the FDA standards but offer more concrete pathways. The door is open for patients and organizations to push the needle forward without waiting for a neon sign from regulators.
Similarly, how is the technology around RWE evolving and helping to facilitate its application more smoothly?
The biggest hindrance to widespread adoption and implementation of RWE is access to high-quality, structured data that’s organized in an accessible way. It’s difficult for people to conceptualize the benefits of using something if they can’t see it and evaluate its relevance to their primary objectives. To show how rich of a resource RWD is in the research process, technology is needed to pool the data, structure it, and make it accessible. With the implementation of the 21st Century Cures Act, US Core Data for Interoperability, and information blocking regulations, in addition to accessibility of artificial intelligence, meaningful steps may be made to address the data utilization hurdle.