With the drug pricing pressures mounting on a global scale, there is a clear and imminent need for industry to demonstrate the value of therapies to payers, health technology assessment (HTA) agencies, and regulators. Real-world evidence (RWE) is continuing to be positioned as a go-to tool for biopharma as they define the value and effectiveness of drugs.

On December 3, 2019, Aetion gathered leaders dedicated to driving the value  conversation and use of RWE forward for a webinar with eyeforpharma, “Let’s talk about value: Use RWE to determine drug price and performance.” The lively discussion featured Carolyn Magill, CEO of Aetion; Eddie Lee, Pharm.D., Health of U.S. HEOR Strategy at UCB; Dr. Pamela Bradt, M.D., M.P.H., Chief Science Officer at the Institute for Clinical and Economic Review (ICER); and Enkeleida Nikai-Fitzgerald, Head of RWE and Senior Director at Janssen Europe, Middle East, and Africa.

Over one-third of the webinar audience, which was primarily comprised of biopharma, stated that RWE is becoming a universal and necessary standard in their work. The panelists discussed a number of opportunities for biopharma to maximize the utility of RWE in value discussions, including: 

  • The importance of investing in data and analytics; 
  • The growing need for collaboration among stakeholders, including alignment on a common platform for transparent analyses; and
  • The need for clear and concise guidance from regulatory, HTA, and payer bodies on how RWE should be generated, and how it will be used in decision-making.  

Importance of investing in data and analytics 

In a poll, the audience highlighted one of the greatest areas of opportunity for biopharma to advance RWE adoption—increasing investment in data sources and analytic capabilities for RWE generation. It’s still early days for RWE adoption, but, as the panelists discussed, RWE's potential to complement RCT data in an evidence package makes it a valuable area for increased investment by biopharma.

Nikai-Fitzgerald described her experience seeking early advice from payers and HTA agencies on value assessment, and how the conversations typically prioritize the RCT clinical development plan over RWE generation. Increased focus on RWE can expand the types of evidence available to payers and HTAs, especially when demonstrating cost-effectiveness.

Dr. Bradt explained that long-term effectiveness is key to payer and HTA decisions: “Unless a company can demonstrate long-term effectiveness with data, including RWD, they are going to have a difficult time getting the price they want.” 

This focus on effectiveness and RWE generation starts with determining what data is fit-for-purpose. Multiple panelists discussed that biopharma needs to demonstrate where real-world data (RWD) is fit-for-purpose and how analytical tools can be applied to generate evidence needed for payer and HTA value conversations. This investment in RWD sources can inform many aspects of drug development, not just the value conversation.

“There is a way to leverage RWE to optimize the endpoints or outcomes that we choose for our clinical studies to allow for better translation into the effectiveness that we would see in the real world,” said Dr. Lee. 

Panelists called on biopharma to invest in and explore fit-for-purpose data sets and analytic capabilities to increase RWE generation and to recognize RWE as an essential component of drug development and approval from both regulatory, payer, and HTA perspectives.  

The growing need for collaboration among stakeholders, including alignment on a common platform for transparent analyses

A major theme that drove the webinar conversation was the growing need for collaboration among all stakeholders and a common analytics platform that can facilitate conversations that focus on the impact of RWE. 

Around 60 percent of the webinar audience was interested in collaborating with HTAs and regulators on RWE initiatives. 

“In the same way that we design RCTs, we need to bring everyone around the table to design RWE studies,” proposed Nikai-Fitzgerald, noting that the collaboration should include patients as well as industry, regulators, HTAs, and payers. 

Magill detailed some of Aetion’s work to drive multi-stakeholder collaboration:  “One of the things that Aetion is working on with our clients is thinking through the types of data that are most reflective of patient goals, and how we can capture these outcomes, which are typically not captured in claims.” 

Panelists noted that the idea of collaboration should not be done on an individual manufacturer-regulator basis. Instead, the discussions around how to generate the highest-quality RWE should be done on a collective basis and on a common platform. This would allow us all to align on things like transparency, replicability, scalability, and the credibility of evidence and it will facilitate a common way to evaluate RWE and to use it more consistently.

The opportunity for clear and concise guidance from regulatory, HTA, and payer bodies on how RWE should be generated, and how it will be used in decision-making

Underlying the need for increased investment in RWD and RWE, is the need for standards on how RWE should be generated—and how evaluators will use it in decision-making. Industry panelists stated that biopharma welcomes a framework for how and when RWE will be incorporated into HTAs’ value assessments. This would help industry refine their HEOR strategy and improve their ability to align the right research questions and study execution with the agreed-upon standards. 

Dr. Bradt and Magill agreed that as an industry, we are making progress down the path of framework development. They identified areas where RWE can be especially useful—including when evaluating adherence, utilization, and other inputs into cost-effectiveness modeling. These elements will take us a long way forward as stakeholders continue to advance the science of RWE to facilitate decision-making.