Unveiling The Key Trends and Topics: Axtria's Insights from ISPOR US 2024

This year's ISPOR annual conference theme was "HEOR: A Transformative Force for Whole Health." Axtria conducted two podium presentations and exhibited nine posters. The conference started with the plenary session on "Advancing Whole Health: How do We Know When We're Succeeding?" The aim of the first plenary was to delve into the concept of "whole health," emphasizing the interconnectedness of physical, behavioral, and socioeconomic factors in shaping individual health outcomes. It covered three core topics: 1) Patient to Person, 2) Healthcare the Health, and 3) Invisible to Visible. Overall, the session highlighted the importance of data and measurement in advancing healthcare, encouraging the audience to innovate within this framework.

Axtria's takeaways from the conference include:

• The growing acceptance of causal inference methodologies in real-world evidence (RWE) – Several presentations discussed guidelines and important considerations for generating RWE with causal inference methods.

• The transformative impact of Generative AI – Several workshops, a plenary session, and poster presentations focused on how GenAI is revolutionizing RWE/HEOR research.

• The use of novel, real-world data (RWD) – There is a growing emphasis on patient-centric approaches involving the collection of patient-reported outcomes (PROs) and patient-generated health data (PGHD) to capture the patient's experience and preferences.

• Digital Twins – Although not prevalent at this year's ISPOR, several sessions touched on the importance and practicality of applying digital twins, mostly for clinical trial simulations.

A Deep Dive Into Key Topics

• The growing acceptance of causal inference methodologies in real-world evidence (RWE)

  Keeping with the top trend from recent years, the 2024 ISPOR Conference featured many advanced analytics methods often used to tackle challenges inherent to RWD and to generate reliable RWE. In particular, causal inference methodologies, including G-computation and targeted maximum likelihood estimation (TMLE), are becoming more widely understood and used by RWE/HEOR analytics teams and pharmaceutical companies alike. When combined with machine learning (ML) algorithms like Super Learning (SL), these methods can be used to generate valid estimates of the effectiveness of treatments from RWD while accounting for confounders, or factors that influence the exposure and outcome. However, the question remains whether regulatory bodies are ready to accept these kinds of analyses based on RWD and rigorous statistical techniques in place of the gold standard: randomized control trials. At the 2024 ISPOR conference, pioneers of TMLE offered their guidance on implementing these complex methods in a regulatory context as well as the payer setting, suggesting growing confidence in the widespread adoption of the methods and ultimate approval by regulatory authorities. To ensure approval, those implementing such solutions must have appropriate evidence supporting their findings and should be able to answer regulators' questions on the complex methodology. Axtria's RWE, HEOR, and Evidence Synthesis team is proud to have the capability to generate regulatory-grade evidence in collaboration with causal inference and TMLE subject matter experts.

• The transformative impact of generative AI

  At the ISPOR 2024 conference, artificial intelligence (AI) was spotlighted for its transformative role in health economics and outcomes research, particularly through its application in literature reviews and economic model design. Axtria's presentation on "The Use of NLP in Literature Reviews" exemplified how natural language processing (NLP) technologies can streamline the selection process and extraction of relevant information, enhancing the efficiency and comprehensiveness of literature surveys. This accelerates the initial phases of research and improves the accuracy and depth of the gathered data, which is crucial for building robust economic models and making informed healthcare decisions.
  
  Another significant highlight from Axtria was "An Updated Landscape of FDA-approved AI/ML-enabled Medical Devices from 1995-2023," which showcased the evolution and impact of AI technologies in medical devices. This presentation underlined the growing integration of AI tools in healthcare technologies and their approval by regulatory bodies such as the US Food and Drug Administration (FDA). It also emphasized the importance of a human/AI team. Combining human expertise with artificial intelligence is indispensable in developing more effective, personalized medical interventions and enhancing patient care. The emphasis on this partnership shows how AI can supplement human capabilities, ensuring that the technology is a powerful addition to traditional analytical methods in healthcare. To learn more about Axtria's point of view on NLP, we invite you to read our white paper "The Use of Natural Language Processing in Literature Reviews.”

• The use of novel real-world data (RWD)

  As the evidence-generation process evolves, incorporating the patient’s voice has become crucial. The patient benefits by having their perspectives, the impact of their illness, and their unmet needs recognized. Biopharmaceutical companies benefit by having more complete communications with clinicians and payers. Incorporating patient-reported data into research investigations can be expanded by integrating information from claims and electronic medical record systems, leading to a more comprehensive understanding. While social media listening represents one method for capturing the patient's perspective and validating PROs, it is essential to explore additional avenues. The integration of different data sources, such as electronic health records (EHRs), wearable devices, and social media, enables a comprehensive and holistic view of patient health and treatment outcomes.
  
  On this theme, Axtria’s team presented a podium on “Patients’ Perspectives on Muscular Dystrophy: Insights from Social Media Listening” and a poster on “Anhedonia-Related Wording in Social Media: An Application of Natural Language Processing.” Both presentations were well received and sparked engaging discussions with the audience. Rare diseases remain in focus at ISPOR with several presentations on critical challenges in evaluating rare disease technologies. To learn more about Axtria’s point of view on rare diseases, we invite you to read our blog “Modeling the ‘Difficult to Model’ Disease: Considerations for Evaluating the Cost-Effectiveness in Rare Diseases.”

• Digital Twins

  Finally, we believe the next big discussion at ISPOR will be on study methodologies using digital twins (DT). A digital twin refers to a virtual model that accurately represents a physical object or system. It is continuously updated using real-time data and incorporates simulation, machine learning, and reasoning to aid decision-making. DTs are the future of personalized medicine and innovation as they can accelerate evidence generation, improve clinical trials, and reduce cost. Within our RWE, HEOR, and Evidence Synthesis practice, we have developed a strong capability in simulation modeling, where RWD can be used to create counterfactual copies of real-world patients that follow a variety of flexibly defined treatment strategies. The next step is to use a multi-model framework, predicting future inputs to the endpoint model, and establishing the Digital Thread for physical and digital twin feedback. To learn more about Axtria’s current simulation modeling capabilities, we invite you to read our white paper “RWD-based Simulations: Generating Medical Evidence from Clinical Trial and Real-World Data.”

We hope you find this summary of conference highlights valuable. If you were unable to attend the conference and would like more information on Axtria’s offerings in RWE, HEOR & Evidence Synthesis, please feel free to contact us at connect@axtria.com.

We look forward to attending the ISPOR Europe conference in Barcelona in November and hope to see you there.

Until next time,

Won Chan Lee, Ph.D.
Principal, RWE & HEOR Practice Lead – Scientific and Strategic Oversight

Jennifer Ken-Opurum, Ph.D.
Associate Director, RWE & HEOR – Client Engagement Lead

Alex Almond
Senior Director, RWE & HEOR Practice - Head of Client Strategies

David Hood
Senior Manager, RWE & HEOR – Client Engagement Lead

Coby Martin
Project Leader, RWE & HEOR – Client Engagement Lead

Alexandra Koumas
Project Lead, RWE & HEOR – Client Engagement Lead