ISPOR, the Professional Society for Health Economics and Outcomes Research (HEOR), has released its fifth biennial report, ‘ISPOR 2024-2025 Top 10 HEOR Trends.’ The report highlights key trends shaping global healthcare decision-making:
- Real-world evidence (RWE) remains a top trend.
- Drug pricing, especially in the U.S., is a significant concern.
- Artificial intelligence (AI) re-emerges as a major trend.
- Fostering innovation with affordable pricing is a new focus.
- Health equity is a continued priority.
- Accelerated drug approvals, value measurement, patient centricity, precision medicine, and public health are also on the radar.
HEOR’s role in informing healthcare decisions is crucial, and ISPOR remains committed to improving patient outcomes globally.
ISPOR 2024-2025 Top 10 HEOR Trends
REAL-WORLD EVIDENCE (RWE)
Real-world evidence (RWE) retains its top position among the top 10 trends in healthcare decision-making. RWE studies offer insights into real-world patient experiences, proving faster and more inclusive than traditional clinical trials.
Regulatory agencies like the FDA, EMA, NICE, and CADTH have issued guidance on RWE. Key areas of focus include data quality, suitability, registries, and transparent reporting. RWE is now used as primary evidence of efficacy in regulatory decisions.
ISPOR prioritizes RWE, focusing on transparency and data quality. Collaborating with organizations like ISPE, the Duke-Margolis Center, and the National Pharmaceutical Council, ISPOR promotes transparency through initiatives like the Real-World Evidence Registry.
In 2023, ISPOR hosted an RWE Summit with ISPE and the Duke-Margolis Center, summarizing the outcomes in Value & Outcomes Spotlight.
DRUG PRICING
High drug prices are a growing concern in healthcare, moving from #6 to #2 in the 2024-2025 report. The U.S. Inflation Reduction Act (IRA) and the EU pharmaceutical strategy are key factors. The IRA marks a major change by introducing drug price negotiations for Medicare. The EU-JCA aims to improve access.
The consequences remain uncertain, with potential impacts on global drug innovation and intellectual property. ISPOR and the HEOR community closely follow these developments.
ARTIFICIAL INTELLIGENCE
Ranked #3 in the 2024-2025 report (up from #10), artificial intelligence (AI) is gaining significance. OpenAI’s ChatGPT exemplifies AI’s integration into daily life.
Large language models (LLMs) like ChatGPT assist clinicians in note-writing, data analysis, and diagnostics. In health economics and outcomes research (HEOR), machine learning (ML) enhances data analysis, literature reviews, and modeling.
ISPOR emphasizes AI’s assistive role in healthcare decision-making, particularly in electronic health records and patient care.
FOSTERING INNOVATION
In the 2024-2025 report, fostering innovation is a new topic. It’s crucial for introducing innovative health technologies to the market. However, balancing affordable pricing with incentivizing innovation remains a critical challenge.
Innovations are essential to address unmet needs in healthcare, but they often come with high costs. For example, the gene therapy Hemgenix for hemophilia B is priced at $3.5 million. Similarly, treatments like Leqembi for Alzheimer’s disease can strain budgets at $82,500 per patient per year.
Areas like antibiotic development struggle due to limited financial incentives. Initiatives like the Inflation Reduction Act and government efforts aim to foster innovation in drug development.
ISPOR explores fostering innovation through conferences and events, focusing on outcomes-based pricing and value assessment.
HEALTH EQUITY
Health equity returns to the trends report, emphasizing the importance of addressing disparities. Achieving health equity means eliminating unfair differences among populations, ensuring everyone can attain their full health potential.
Health disparities contribute to lower life expectancies and higher rates of preventable conditions, particularly in disadvantaged communities.
Efforts to integrate health equity in research and clinical practice are rising. Measuring equity effects and applying social determinants of health (SDoH) to health economics and outcomes research (HEOR) are key developments.
ISPOR supports this work through various initiatives, including the Health Equity Research Special Interest Group and webinars on distributional cost-effectiveness analysis and health disparities.
ACCELERATED DRUG APPROVALS
Accelerated drug approvals make a comeback as a prominent trend in healthcare decision-making. The urgency to bring innovative therapies to market swiftly, particularly for rare diseases, drives this trend.
In 2022, many novel drug approvals in the U.S. and Europe utilized expedited programs. These approvals often rely on limited clinical evidence, such as short-term trials or surrogate markers, to indicate treatment efficacy.
Health economics and outcomes research (HEOR) plays a crucial role in filling evidence gaps for accelerated-approval therapies. It can validate surrogate markers with clinical outcomes data, reducing uncertainty. However, concerns arise when drugmakers delay confirmatory studies.
VALUE MEASUREMENT
Measuring and assessing healthcare value is vital as health systems shift towards value-driven care. The quality-adjusted life-year (QALY) is a commonly used measure but has faced criticism for undervaluing the quality of life in certain populations.
In the U.S., the Inflation Reduction Act prohibits the use of QALYs in drug price negotiations, aiming for equal consideration of all patients’ lives.
Efforts to address QALY-related issues include the ISPOR Value Flower, the Generalized Risk-Adjusted Cost-Effectiveness (GRACE) approach, the Online Personal Utility Function (OPUF) for individual-specific value measurement, and the evLYG measure.
ISPOR actively engages in this discussion through conferences, articles, and education, focusing on innovative approaches to value assessment.
PATIENT CENTRICITY
Patient centricity remains a key focus in the 2024-2025 report. Despite recognizing its significance, clinical research often lacks patient engagement.
In health economics and outcomes research (HEOR), centering on patients is crucial. Patient-centered research yields insights into the patient experience, preferences, and their definition of treatment success.
Government guidance, like the U.S. FDA’s initiatives, emphasizes involving patients in regulatory processes—various global organizations, such as EUPATI, PCORI, EURORDIS, and others, champion patient engagement.
However, challenges persist, including late or absent patient inclusion and addressing health equity. Diverse data sources like patient-generated health data, electronic health records, and real-world evidence are vital for informed patient engagement.
PRECISION MEDICINE
Precision medicine, the tailoring of treatments to individual characteristics, continues to advance. Oncology leads in this field, but challenges remain in terms of costs, clinical trials, regulation, benefits, and equity.
Precision medicine goes beyond biomedical aspects, considering psychological and social dimensions. Regional disparities in adoption, like in India, exist due to limited accessibility and affordability.
ISPOR actively addresses precision medicine through conferences and publications. The society’s Precision Medicine and Advanced Therapies Special Interest Group emphasizes consensus-building, transparent expectations, and value generation.
PUBLIC HEALTH
Public health returns as a top trend amid global challenges like climate change, social unrest, war, and economic recession. Climate change threatens health with increased deaths, infectious diseases, and vulnerable communities’ risks.
Social unrest disrupts healthcare access and safety. Economic recessions, especially in emerging markets, correlate with higher mortality, notably child mortality.
Dealing with these issues requires multidisciplinary collaboration between public health and HEOR professionals to effectively tackle unpredictable and chronic health threats.
Resource: ISPOR, January 16, 2024
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