ISPOR Goes Real-World
Ashley Jaksa, MPH
Scientific Partnerships Lead, Aetion
Nirosha Lederer, PhD
Head of US Government Partnerships, Aetion
Regulators, HTAs, and pharma leaders discuss RWE trends with Aetion experts.
Real-world evidence (RWE) continues to gain traction as part of clinical research. Regulators are more and more accustomed to reviewing RWE in submissions, payers are considering RWE to inform reimbursement, and physicians and patients want to see proof of real-world results as part of their treatment decision-making.
That said, product sponsors are still uncertain on how exactly to incorporate real-world data (RWD) into their clinical development and commercialization programs, and how to best use it to explain the safety, efficacy, and pricing for their products.
At Aetion, these are topics we talk about every day, and we are excited to share our insights at this year’s International Society for Pharmacoeconomics and Outcomes Research (ISPOR) annual conference (May 15-18, 2022, in the Washington DC area). After a two-year hiatus due to the pandemic, ISPOR will be the first big event gathering industry leaders together in-person to talk about how we can use data and technology to drive greater insights that benefit patients, public health, and revenue results.
At this year’s conference, Aetion is hosting four panels on how sponsors and decision makers can use RWE to support decision-making and assess the effectiveness and value of new treatments. We have partnered with several industry leaders, including experts from Duke-Margolis Center for Health Policy, the United States’ Food and Drug Administration (FDA), the Institute for Clinical and Economic Review (ICER), the National Health Council, the National Institute of Health and Care Excellence (NICE), PhRMA, as well as several innovative life sciences companies to share insights and answer questions about these important topics.
Let’s get real: Four conversations about RWE
On Monday, May 16 (10:15-11:15 a.m. ET), Aetion’s Dr. Nirosha Lederer will be talking about pricing reforms in a panel entitled What is the Preferred Approach to US Drug Pricing Reforms – International Reference Pricing (IRP) or Value Based Pricing (VBP)? Panelists include Randy Burkholder (PhRMA), Dr. Marianne Hamilton Lopez (Duke-Margolis Center for Health Policy), and Dr. Margaret Labban (Global Pricing Innovation), all of whom will discuss the benefits and challenges of IRP versus VBP, and what impact both approaches will have on the future of drug development. The panelists will debate their preferred approach for pricing reforms in the US.
This event will be a great opportunity for attendees to understand how these pricing models will impact innovation, access, and development, as well as how they can participate in this important industry conversation.
Tuesday morning’s (10:15-11:15 a.m. ET) workshop, Lessons Learned From Health Technology Assessment (HTA) Pilot Projects to Assess RWE’s Usefulness in Reassessments of Effectiveness and Value, will explore the use of RWE in HTA reassessments. Aetion’s Ashley Jaksa will be joined onstage by Dr. Jon Campbell (ICER), Dr. Gregory Daniel (Eli Lilly), and Dr. Seamus Kent (NICE).
Most HTA agencies complete only one assessment at time of launch, but many are considering a shift toward assessing drugs at multiple timepoints throughout a product’s lifecycle. To better understand the role of RWE in serial HTA assessments, ICER and NICE conducted pilot projects to explore the use of RWE in reassessments.
Panelists will unveil lessons learned from these projects and how they may inform future RWE guidance. The speakers will solicit public comments, and participants will have a chance to provide their perspective regarding their own RWE best practices.
Tuesday afternoon (4:30-5:30 p.m. ET), Dr. Nirosha Lederer will join a conversation on the topic of Getting Real with RWE for Regulatory Decision Making: Where Are We Going?
In this session, she will be joined by Dr. Elisabeth Oehrlein (National Health Council), Dr. Donna Rivera (FDA), and Dr. Mehmet Burcu (Merck), and who will discuss current and future applications for RWD and RWE in regulatory decision-making. We will look at the current global regulatory landscape, explore techniques for designing effective observational research models, and consider the value of including patient perspective(s) in efforts to address public health needs.
In our final panel on Wednesday (10-11 a.m. ET), Aetion will examine the Relevance of Using International RWD in Regulatory and HTA Decision Making. In this session, Ashley Jaksa, Dr. Rami Ben-Joseph (Jazz Pharmaceuticals), Dr. Kelvin K.W. Chan (Sunnybrook Odette Cancer Centre), and Dr. Pall Jonsson (NICE) will discuss when and where HTAs will consider international RWD for local assessments.
Ideally, sponsors will submit country-specific RWD to demonstrate effectiveness, safety, and value, but that data doesn’t always exist. In these cases, HTA and regulatory bodies have signaled receptivity to RWD gathered outside of their country if the data sets are similar, but there is a lack of guidance on what constitutes similarity in healthcare systems, patient populations, and clinical practice.
This panel will discuss the ideal settings to use international RWD, what gaps exist in current guidance, and what a working definition of “similarity” might look like.
Aetion’s sessions at ISPOR 2022 will offer participants a chance to hear the latest insights from leading experts on using RWE in clinical research and provide opportunities to ask questions and help shape the ongoing dialog about how regulators and HTAs should use this data to inform decision making.
We hope you will join us at #ISPORAnnual. You can also meet our team on the Exhibition floor at booth #714. If you’re interested in setting up a meeting with our experts onsite, please click here to find a time that works. We look forward to seeing you soon.