ISPOR 2024 daily round-ups: Day 1

Written by The Evidence Base
Events coverage ISPOR

During May 6–8, 2024, The Evidence Base team are in Atlanta, GA, USA attending ISPOR 2024, the annual international conference for ISPOR—The Professional Society for Health Economics and Outcomes Research (#ISPORAnnual). Bringing you insights from the plenary sessions as well as daily round-ups, here we cover ISPOR 2024 Day 1.

Click below to read the coverage of Days 2 and 3.

Day 2 Day 3

Our partners

The Evidence Base is delighted to be working with several ISPOR 2024 event partners to bring coverage of their sessions and posters: Avalere, Cardinal Health, Cencora, Flatiron Health, Genesis Research Group, Lane Clark & Peacock LLP and Medlior Health Outcomes Research. Register on The Evidence Base to receive regular updates and be notified when this coverage is published on the site.

Register here

And, many thanks also to Maxis Clinical Sciences for their support and welcome Carrot Recruitment, our Talent Hub partner.

Welcome address

Kicking off the first day, Brian O’Rourke welcomed over 4000 delegates to ISPOR 2024 and officially launched the conference. O’Rourke thanked the Program Committee comprised of 30 HEOR experts from the USA, Europe, Asia, the Middle East, and Latin America and led by three Program Co-Chairs:

Plenary 1 – Advancing Whole Health: How do We Know When We’re Succeeding?

Day 1 of ISPOR 2024 began with the highly-anticipated plenary session, “Advancing Whole Health: How do We Know When We’re Succeeding?” The discussion was led by Charlene Wong (Centers for Disease Control and Prevention, USA) and included Denise Webb (The Partnership For Southern Equity, USA), Seth Berkowitz (University of North Carolina at Chapel Hill, USA) and Eric Schneider (National Committee for Quality Assurance [NCQA], USA). With whole person health requiring a holistic approach that considers multiple factors that promote health or disease, the session focused on the challenges of designing and evaluating integrated interventions aimed at promoting whole person health.

For more insights into Plenary 1, check out our full coverage here.

Spotlight session – Medicare Drug Price Negotiation: Lessons from the First 10 Drugs Selected 

Moderator Sean D Sullivan (The CHOICE Institute School of Pharmacy, University of Washington, USA) introduced the spotlight panelists Kristi Martin (Center for Medicare and Medicaid Services, USA) and Inmaculada Hernandez (Skaggs School of Pharmacy and Pharmaceutical Sciences, University of California San Diego, USA).

Sean D Sullivan began by setting the scene, introducing the audience to the current ‘state of play’ in the drug Medicare drug price negotiation process and offering insights into the experience from the selection of the first 10 drugs. Sullivan predicted what might be learnt after the maximum fair prices (MFPs) are published in the summer after first round of negotiation, such as how the lowest MFP is reached and what evidence is most valued by CMS.

Fellow panelist Kristi Martin then provided the CMS viewpoint, explaining how the agency is balancing the process of negotiations for the first cycle of 10 selected drugs whilst also preparing for the second cycle of negotiation where up to 15 drugs will be selected for negotiation. Having released draft guidance for this second cycle on May 3, 2024, the revised guidance draws on the learnings from the previous selection process, with numerous adjustments including improved dialogue with manufacturers, new ways to collect relevant data and improved patient-listening focused events to ensure there is better engagement and potentially two-way discussions amongst the speakers. Martin noted CMS have an ‘open door’ commenting process and are welcoming the opportunity for stakeholders to comment on the new draft guidance, with feedback to be submitted by July 2, 2024 to ensure it is considered during the development of final guidance.

Inmaculada Hernandez provided an overview of the Skaggs School of Pharmacy and Pharmaceutical Sciences research project funded by The Commonwealth Fund, sharing ‘scenarios’ looking at price benchmarking for some of the 10 drugs selected for negotiation and the therapeutic alternatives. Hernandez signposted the audience to the forthcoming peer-reviewed publication of the project for more in-depth analysis of the data.

Concurrent sessions

Following the plenary session, ISPOR 2024 included a broad range of concurrent sessions, including Issue Panels, Workshops, Forums and other Breakout sessions. Topics covered on Day 1 reflected the current trends in the field, including real-world data (RWD) and real-world evidence (RWE) methodology, health equity, health technology assessment (HTA) and health policy. Laura Dormer and Joanne Walker of The Evidence Base attended several sessions on Day 1 summarized below.

Issue Panel – Target Trial Emulation in HTA RWD Submissions: A Best Practice Not Embraced – Methodological Challenge or Manufacturers’ Reluctance? 

Moderator Sreeram Ramagopalan (Health Analytics, Lane Clark & Peacock, UK), a regular contributor to The Evidence Base, was joined by his colleague Alejandra Castanon (Health Analytics, Lane Clark and Peacock, UK) and panelists Stephen Duffield (National Institute for Health and Care Excellence, UK) and Rob Reynolds (GSK, USA), to discuss the use of target trial emulation (TTE) in observational studies. 

Castanon began by providing background on trial emulation frameworks and the relevance of TTE to HTA agencies. The importance of providing high-quality data for decision-making was emphasized. Reynolds expressed concerns about the limited adoption of TTEs in practice, and the speakers discussed the difficulties in applying RWD analysis in HTA, including the need for explicit frameworks and adjusting for confounding. The importance of transparency and characterization of the target trial approach in evidence development was also highlighted, with Duffield emphasizing the benefits of visualizing study design as a diagram and pre-specifying sensitivity analyses.  Reynolds stressed the need to marry target population, cohort study practices, and observational studies to ensure effective emulation, and highlighted the importance of exchangeability in emulating trial population selection variables and outcomes.

Exhibit Hall Theater – No Longer Business As Usual: Establishing Value in an Uncertain and Dynamic Landscape

During the afternoon session sponsored by Avalere, Laura Housman and Michael Ciarametaro (Avalere, USA) was joined Jan McKendrick (Avalere, UK) to discuss the major shifts in the global landscape of value assessment and the evidence generation required to support this. Providing the US perspective, Ciarametaro discussed the lack of centralization in value assessment in the US, touching on the various requirements from manufacturers across the country, such as through drug price negotiation and part D reform mandated by the Inflation Reduction Act (IRA). Jan McKendrick drew on the European perspective, echoing the increased demand in evidence generation by manufacturers as part of the forthcoming joint clinical assessment under the EU’s new HTA regulation. The topic of patient engagement was tackled, with all speakers highlighting the importance of patient centricity and the iterative approach that would be needed in both the IRA and JCA. The Evidence Base will be providing a ‘deep dive’ into this session to be published on the site over the next few weeks – don’t forget to register to be notified when this is published.

Exhibit Hall Theater – Payer Perceptions of Health Inequalities in Reimbursement Decision Making: Implications for the Pharmaceutical Industry

Sponsored by Genesis Research Group, in this session Kuldeep Singh and Tijana Ignjatovic (Genesis Research Group, UK) explored how health inequality is presently factored into HTA and payer coverage decisions, sharing insights from the Institute for Clinical and Economic Review (ICER) and from National Institute for Health and Care Excellence (NICE) frameworks. Ignjatovic presented interesting findings from a recent a qualitative survey of payers in six countries on the perceptions of health equity and its current and future role in decision-making. The results indicated a number of key drivers of health inequalities and national-level variations on what is important. Be sure to check out a fuller in depth write up of this session on The Evidence Base to be published soon.

Workshop – Targeted Learning for Causal Inference Using Real-World Data

The workshop was led by discussion leader Suzanne McMullen (Medlior Health Outcomes Research, Canada) and included her colleague John Paul Ekwaru (Medlior Health Outcomes Research, Canada) as well as Mark van der Laan (University of California Berkeley, USA) and Stephen Duffield (NICE, UK). RWE can be used in assessing treatment effectiveness, while reflecting diverse patient populations and actual clinical practice. However, navigating confounding factors, intercurrent events and informative drop-outs poses significant challenges. To navigate this terrain, doubly robust causal inference methods, like the targeted maximum likelihood estimator (TMLE), are emerging as important tools, and the speakers delved into the nuances of these advanced methods throughout the workshop. Readers can learn more about the advanced methodologies discussed in this session in our extended coverage coming soon.

Educational Symposia – Staying Ahead of the Curve: Navigating Policy Changes and Ensuring Patient Access in the Era of IRA and EU HTA

During this Cencora-supported symposium, Corey Ford (Cencora, USA) welcomed attendees to the session that promised to delve deep into the complexities of two pivotal areas in healthcare policy – the IRA in the US and JCA in Europe. Kimberly Westrich (National Pharmaceutical Council, USA) provided an overview of the IRA’s objectives, timelines, and ‘unintended consequences’, offering strategies and suggestions to CMS towards mitigating these consequences. Michael Drummond (University of York, UK) discussed JCA as an opportunity to harmonize processes and accelerate patient access in Europe. Casper Paardekooper (Vintura, part of Cencora) discussed how the key objectives of EU JCA align with the revised EU pharmaceutical legislation and broader health care policy landscape in Europe. This was followed by a lively Q&A discussion, which The Evidence Base will be providing insights from in our forthcoming deep dive into this session.

On demand ISPOR 2024 sessions

ISPOR are continuing the popular ‘Digital Conference Pass‘ option providing on-demand access to recordings of nearly all the educational sessions, plenaries, spotlights, and other content. Some of the sessions we were unable to attend but will be catching up on demand from Day 1 of ISPOR include:

  • “Computable Phenotypes”— Understanding Their Importance in Regulatory Submissions of RWE – Discussion leader: David Thompson, Rubidoux Research, US
  • Applications of Real-World Data and Real-World Evidence in Decision-Making in Asia Pacific – Moderator: Larry Liu, Merck & Co., Inc., US
  • Innovations in RWE: Natural Language Processing, Synthetic Data, and Drug Repurposing – Moderator: Sandipan Bhattacharjee, MS, PhD, Bayer US LLC, US
  • Harnessing the Power of AI: Revolutionizing HEOR and Market Access – Panelists: James Creeden, CGC Consulting, Switzerland and Francesco De Solda, Janssen Global Services LLC, US
  • Progress or Regress…Does CMS’ Maximum Fair Price Negotiation Align With Patient-Centered Evidence Assessment in Healthcare Decision-Making? – Moderator: Kirsten Axelsen, Charles River Associates, US
  • VBAs and Warranties in an Uncertain CMS World: Opportunities or Challenges? – Moderator: Ambarish J Ambegaonkar, APPERTURE LLC, US
  • Making Sense of Sensors: Challenges in Developing Patient-Centered Sensor-Based Outcomes (eg Wearables) for Use in Medical Product Decision-Making and Challenges to Adoption in the Real World – Discussion Leader: Katelyn Keyloun, AbbVie, US

ISPOR’s Strategic Plan 2030

Touched on briefly in the opening remarks ISPOR President, Brian O’Rourke, facilitated a panel session including Eberechukwu Onukwugha (President-Elect), Rob Abbott (CEO), Laura Pizzi (CSO) and Mitch Higashi (ACSO). The speakers outlined the ISPOR vision and strategic goals for 2030, with the ‘Big Themes’ of affordability, digital transformation of health care, and wholistic health. To manifest these themes, ISPOR has set out two main goals:

  • Goal 1: Lead the definition, measurement, and use of value for health decision-making
  • Goal 2: Be the trusted HEOR authority that leads change in health policy worldwide

These goals cement ISPOR’s essential position in the industry as a trusted ‘voice’, leading the way to enable the application and implementation of the great science taking place and, ultimately, leading to change.

Welcome Reception

Day 1 came to a close with the well-attended Welcome Reception, providing attendees with a chance to socialize with exhibitors while enjoying wine and reflect on the insights gained from the conference’s first day.