Setting Real-World Standards at ISPOR 2023
Ashley Jaksa, MPH
Market Access Scientific Strategy Lead
Nirosha Lederer, PHD
Head of US Government Partnerships
Aetion joins other industry experts at ISPOR 2023 to discuss standard-setting and innovation
As real-world evidence (RWE) continues to affect positive change in the healthcare industry’s approach to evidence generation, efforts to standardize its use for regulatory and health technology assessment (HTA) decision-making in the form of guidance have accelerated. Recent examples of this include the RWE-focused amendments in the Food and Drug Omnibus Reform Act of 2022 (FDORA), the RWE provisions contained within the 21st Century Cures Act, as well as in the FDA’s RWE framework and associated guidance. Similarly, NICE issued its RWE Framework in 2022. These standard-setting efforts will only continue as RWE utilization increases globally and its successful use cases proliferate. Currently, experts are preparing to answer new questions and address methodological challenges that will inevitably arise.
This year, several thought leaders and experts from Aetion, along with other researchers and academicians, regulators, HTA agencies, payers, and policymakers, will gather in Boston, Massachusetts, to discuss these issues (and more!) at the annual meeting of the International Society for Pharmacoeconomics and Outcomes Research, also known as ISPOR. Over the course of ISPOR 2023 (May 7-10, 2023), Aetion will be represented across six presentations where our scientists will endeavor to identify, discuss, and debate the most effective, scientifically-valid, and scalable approaches for the uses of real-world data (RWD) and RWE.
Six conversations about RWE
“Navigating the RWE Landscape – Successes, Struggles, and the Path Forward”
On Sunday, May 7, ISPOR will host its inaugural RWE Summit, a one-day meeting focused specifically on the key issues surrounding RWE in regulatory decisions and HTAs. In session four of the Summit entitled, “Navigating the RWE Landscape – Successes, Struggles, and the Path Forward” (4:30-5:20 p.m. ET), panelist Ashley Jaksa, Market Access Scientific Strategy Lead at Aetion, will take part in a discussion regarding challenges and lessons learned in specific instances when RWE was used for regulatory or HTA decision-making. This session will be moderated by Nancy Dreyer (IQVIA) and also features panelists Dr. Stephan Duffield (NICE), Dr. Wim Goettsch (National Care Institute), and Dr. Tae Hyun Jung (CDER/FDA).
“Can US Healthcare Data Inform HTAs in Other Parts of the World?”
During the first full day of ISPOR programming on Monday, May 7, Ashley Jaksa will continue exploring the topic of HTAs during the workshop, “Can US Healthcare Data Inform HTAs in Other Parts of the World?” (1:45-2:45 p.m. ET), alongside moderator Dr. Karen Facey (RWE4Decisions) and co-panelists Dr. Laurie Lamber (CADTH) and Joshua Ray (Roche). RWD derived from US sources can serve as a useful alternative for RWE generation in other countries where access to local, high-quality data isn’t accessible or available. Despite this, questions remain as it relates to data quality which is often compromised by healthcare data that is disconnected across jurisdictions. In this workshop, attendees will hear about many of the challenges associated with using US RWD in other countries and jurisdictions, as well as a number of case studies that illustrate how US RWD has been used by industry experts thus far to inform decisions in the EU.
“External Control Arms (ECA) in Phase 3 trials: What is Needed Now and in the Future to Enable Acceptance with Regulatory and HTA Bodies?”
Aetion’s Chief Science Officer, Dr. Nicolle Gatto, will take the stage on Monday, May 7 (4:45-5:45 p.m. ET), where she will speak on the panel, “External Control Arms (ECA) in Phase 3 trials: What is Needed Now and in the Future to Enable Acceptance with Regulatory and HTA Bodies?” Dr. Gatto, in company with moderator Dr. Hélène Karcher (Novartis), and panelists Emma Hawe (RTI Health Solutions) and Dr. Nicole Mittmann (CADTH), will discuss key questions and challenges associated with considering an external control arm (ECA). ECAs are control arms comprised of data collected outside of a clinical trial to a support single-arm trial, particularly in rare disease or oncology trials where a traditional comparator arm is unfeasible or unethical. As the conditions for the acceptance of ECAs differ between regulatory and HTA bodies, debates continue over the proper methods and data that should be used for these trials. This workshop will provide payers, regulators, and leaders with a fuller understanding of the function of ECAs in the context of Phase 3 trials, as well as the role ECAs can continue to play in the future of evidence-generation frameworks for new pharmaceutical products.
“Medicare Coverage with Evidence Development for Drugs Approved for Alzheimer’s Disease: Which Study Designs and Data Sources Would Best Answer Medicare’s Questions?”
Also on day one of ISPOR (May 7, 4:45-5:45 p.m. ET), Dr. Nirosha Lederer, Head of US Partnerships at Aetion, joins the discussion, “Medicare Coverage with Evidence Development for Drugs Approved for Alzheimer’s Disease: Which Study Designs and Data Sources Would Best Answer Medicare’s Questions?” In this session, Dr. Lederer will accompany Dr. Sean Tunnis (Rubix Health), Dr. Maria Glymore (UCSF), and Dr. Emily Zeitler (Dartmouth-Hitchcock Medical Center) to discuss evidence generation approaches for new Alzheimer’s Disease (AD) therapies. Following the FDA approval of the first disease-modifying therapy for AD in 2021, the Center for Medicare & Medicaid Services (CMS) issued the decision that patients receiving these therapies must participate in “prospective comparative studies.” Dr. Lederer and her co-panelists will each propose their own approach to evidence generation for coverage with evidence development (CED) studies of drugs for AD, debating the potential pros and cons of each.
“Standardized Assessment Tool Designed to Assist in Evaluation of RWE on Drug Effectiveness and Safety”
On Tuesday, May 8 (10:15-11:15 a.m. ET), Ashley Jaksa returns to the ISPOR stage to moderate the session, “Standardized Assessment Tool Designed to Assist in Evaluation of RWE on Drug Effectiveness and Safety.” There, she will be joined by panelists Dr. Katsiaryna Bykov (BWH/HMS), Dr. Jessica Franklin (Optum), and Dr. Cynthia Girman (CERobs Consulting) to debate current efforts to improve the quality of RWE evaluation, including the implementation of a new assessment tool developed to support HTA and payer decision-makers when evaluating RWE studies of medication safety and effectiveness. This session marks the introduction of this tool to the wider ISPOR community and will give participants – particularly those HTA representatives, payers, industry analysts preparing RWE submissions to HTA agencies – the opportunity to provide feedback on the effectiveness of this tool to support their needs.
Closing Plenary: “Causal Inference with US Electronic Health Record (EHR) Data: Practical Solutions to Recurrent Methodologic Challenges”
ISPOR 2023 will culminate in the closing plenary, Causal Inference with US EHR Data: Practical Solutions to Recurrent Methodologic Challenges, on Wednesday, May 10 (11:30 a.m. – 12:45 p.m. ET). There is great potential for the use of EHR data to supplement claims-based RWE, particularly in those areas – like oncology, post-transplant care, and hemophilia – where the associated care is structured and intense, thus yielding an abundance of detailed clinical information. In this plenary presentation, Aetion Co-Founders, Drs. Jeremy Rassen and Sebastian Schenesswiess (BWH/HMS) will join Dr. Rebecca Hubbard (UPenn), Dr. Jannick Weberpals (BWH/HMS), and Dr. Richard Wyss (BWH, Sentinel) to share their expert approaches for harnessing the potential of EHR data, as well as how to overcome many of the methodological challenges associated with using this data to estimate causal treatment effects.
We hope you will join Aetion at #ISPORAnnual. In addition to the presentations outlined above, you can meet our team on the Exhibition floor at Booth 723. If you’re interested in setting up a meeting with our experts at ISPOR, please click here. We look forward to seeing you soon.