Written by Paloma Charlesworth
Introduction
From the 17th–20th May 2026, the Professional Society for Health Economics and Outcomes Research (ISPOR) annual conference took place in Philadelphia, convening stakeholders from across the health economics and outcomes research (HEOR), pharmaceutical, biotechnology, payer, and health technology assessment (HTA) communities.
This month’s HTA insights summarises key themes emerging from ISPOR.
European Joint Clinical Assessment
The implementation of the European Union (EU) HTA Regulation continued to be a key discussion at ISPOR. Since January 2025, all new oncology medicines and advanced therapy medicinal products (ATMP) have been subject to Joint Clinical Assessment (JCA), requiring a single EU-level clinical assessment to support national HTA processes.
May 2026 also saw the completion of the first JCA for tovorafenib (Ojemda™) in paediatric low-grade glioma, with publication expected imminently.
Several ISPOR presentations examined emerging experiences with JCA submissions, with a particular focus on aligning JCA requirements with national HTA expectations, including differing national population, intervention, comparator(s), and outcome (PICO) requirements, comparator preferences, and evidence needs. Discussions also highlighted the increasing use of indirect treatment comparisons (ITC), external control arms, and real-world evidence (RWE) to address gaps in comparative evidence.
Surrogate endpoints
Surrogate endpoints remained a prominent topic, particularly in oncology and rare diseases, where mature overall survival (OS) data are often unavailable at launch.
Several ISPOR presentations examined the use of endpoints such as progression-free survival (PFS), objective response rate (ORR), and minimal residual disease (MRD) in HTA submissions. A recurring finding was the continued variation in how HTA bodies assess surrogate outcomes and the level of evidence required to demonstrate a relationship with patient-relevant outcomes such as OS or quality of life (QoL).
Presentations also highlighted the increasing use of RWE and post-launch data collection to address uncertainty where decisions are based on surrogate endpoints.
Artificial Intelligence in HEOR
Several ISPOR presentations assessed the performance of large language models (LLM) and artificial intelligence (AI)-enabled workflows within HEOR activities, often comparing outputs against traditional approaches. Alongside efficiency gains, recurring discussion points included transparency, reproducibility, auditability, and documentation requirements for AI-generated outputs.
All of these presentations highlighted the need for governance frameworks to support the use of AI in evidence generation and HTA submissions, particularly as HTA agencies and regulators continue to evaluate acceptable standards for validation and oversight.
US policy spillover into global HTA
Several ISPOR presentations examined the implications of US pricing reforms, particularly the proposed Most Favoured Nation (MFN) approach, for global market access strategies. Discussions focused on how linkage between US and international prices could affect launch sequencing, pricing decisions, and market prioritisation.
Presenters highlighted increased interaction between US pricing policy and global HTA processes, with manufacturers needing to consider how pricing and reimbursement decisions in one market could influence outcomes elsewhere.
Where to go for more information
If you would like to learn more about any of these topics, please see the additional insights below written by members of the Source team:
- The European Union Joint Clinical Assessment (EU JCA) – an overview
- What are the implications of joint clinical assessment (JCA) on systematic reviews?
- RAISE guidance – maintaining SLR standards in an AI assisted future
- Advances in artificial intelligence: ChatGPT and the implications for medical writing
- Source’s HTA quarterly updates (for insights on surrogate endpoints)
If you would like to find out more about our HTA submissions (including systematic reviews, health economic modelling, and medical writing), please contact us at Source Health Economics, a consultancy specialising in evidence generation, health economics, and communication.
