The healthcare industry is undergoing change at an unprecedented rate. Because of new technologies, financial pressures, and the increasing demand for evidence, the way in which both individuals and organizations make decisions in this sector is being altered. For leaders in the field of health economics and outcomes research (HEOR), the evolution of healthcare means that there will be a huge amount of data available, and comprehensive analysis of that data will result in a longer time span between innovation and its real-world impact.
Ten key themes are influencing how evidence is generated and how value is measured. These include Artificial Intelligence (AI), real-world evidence, value-based care, and patient centricity, among others. Yet all of these trends have one thing in common: they are concerned with how the growing volume of data can be made use of in order to achieve clinical, operational, and financial outcomes. The article examines where each of these areas might be going and what this means for evidence teams.
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Artificial Intelligence: How Do We Use It Responsibly?
AI is already integrated in nearly all areas of healthcare, from drug discovery & clinical trials to HEOR. With large language models (LLMs) able to process large databases and machine learning able to create predictive analytics, these tools will make systematic literature reviews and dataset organisation faster. Agentic AI, where AI is used to automate tasks as well as decision-making processes without direct human intervention, is revolutionizing the operational processes across the healthcare sector. As these technologies develop, the risks grow too; data privacy and biases are a concern, not to mention generating incorrect outputs.
What it means: Faster outputs are useful only when validated. Also, human oversight and governance should be part of AI workflows from the beginning.
How can IeB help?
We help teams leverage AI without compromising accuracy, offering AI-assisted literature reviews and evidence synthesis validated by our experts. We also provide guidance around the workflows and quality checks needed to maintain credibility with payers and regulators.
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Real-World Evidence: Can We Trust the Data Behind It?
Regulatory authorities are showing an inclination towards real-world evidence (RWE). The European Medicines Agency (EMA) announced a 47.5 percent increase in RWE studies over the last reporting period, and the FDA has utilized real-world data in risk assessment for post-market drug safety. However, missing data integration still remains a key obstacle, with transparency efforts, for example, the HARPER protocol template cited in the International Council for Harmonisation (ICH) M14 guideline, working to improve studies’ reproducibility.
What it means: Decision-makers trust RWE when they can see how it was built, so protocol transparency is becoming a baseline expectation.
How can IeB help?
IeB’s role in RWE begins with data mapping and protocol design. We then plan the study around regulator and HTA expectations, so the evidence can be used for regulatory, reimbursement, and commercial purposes.
Read More: Building a Global Evidence Strategy for Overseas Reimbursement Success
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Value-based Healthcare: How Do We Prove Value Beyond Efficacy?
Value-based healthcare links payment to patient outcomes, rather than volume of services. For life sciences companies, patient cost savings, manpower shortages, and an upward trend in case volume make value-based care more relevant. The scope has expanded beyond clinical outcomes to include information regarding healthcare resource utilization, variation in care, nursing time, storage, wastage, and treatment capacity. Health technology assessment (HTA) agencies take account of how these factors affect resource allocation and the opportunity costs that may be incurred while adopting new therapies.
What it means: Value needs to be considered at approval and adoption, not after launch, and it has to answer operational questions as well as clinical ones.
How can IeB help?
For value-based models, we build evidence plans and economic models that are grounded in real-world care practices. This allows manufacturers to answer provider and payer questions about utilization, implementation, and effectiveness.
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Drug Pricing: How Do We Protect Access and Affordability?
Pricing pressure has reached a global level. The most-favored-nation (MFN) status, international reference pricing, and Medicare negotiations are transforming choices around new launches. A move towards reference pricing across the US is currently exerting upward pressure on prices at comparator companies. In response, value-based pricing models are emerging, such as outcomes-based contracts, instalment-based payments, subscription access, and indication-specific pricing. A recent review revealed some 1,000 risk-sharing arrangements during a decade in 28 nations, over 50% focused on oncology drugs. However, limited price transparency around confidential rebates means actual transaction prices remain hidden.
What it means: Pricing decisions in one market can affect other markets. Therefore, launch plans should account for these impacts.
How can IeB help?
We support reimbursement policy, launch sequencing, and payer studies. Our experts help structure managed entry and risk-sharing options that link price to value.
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Innovative Therapies: How Do We Get Breakthroughs to Patients?
The innovation incentive is under pressure. The pipeline consists mainly of treatments for rare diseases. Of 50 new medicines approved by the FDA’s Center for Drug Evaluation and Research (CDER) in 2024, 26 had orphan designations. Most of these medicines struggle to reach patients since payers and HTA agencies require more evidence than regulators. The US generated 64-78% of global pharmaceutical profits, so pricing reforms to US prices also affect the incentives to fund innovation worldwide. R&D activities are shifting to eastern countries. In China, trial initiations surged from just over 600 in 2017 to nearly 2,000 in 2023.
What it means: Accessibility planning can begin before approval, and competitive intelligence now has to look beyond one region.
How can IeB help?
We combine market access planning, global evidence strategy development, and patent & competitive intelligence to help bring high-cost and orphan medicines to the market early in their lifecycle.
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Patient Centricity: How Do We Bring in the Patient Voice?
The EMA (European Medicines Agency), the U.S. FDA, and Canada’s Drug Agency have issued guidance on how patient experience data should be collected and used. Wearables and other digital devices gather real-time health data to permit volunteers from all patient groups to take part in trials. Regulators and reviewers are placing more value on patient-generated data. The focus is shifting from a passive research phase where patient experiences can be studied to an active co-creation phase. Significant progress has been made in rare disease journeys; still, six of ten Americans who have chronic diseases have experienced much less-relatively little to date-progress.
What it means: Patient input carries weight only when it is structured alongside clinical and economic data.
How can IeB help?
We design patient-reported outcome & preferences research strategies and incorporate the findings into new HTA submissions and payer dossiers, providing patient experience that can inform decisions about coverage and value.
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Relevance of HEOR: How Do We Show Its Role in Decisions?
With data piling up exponentially, decisions related to healthcare are getting more difficult, while systems are dealing with cost escalation, ageing population, and workforce strain. Each region has its own challenge, like fragmented incentives in the US, ageing demographics in Europe and Asia, and funding gaps in China. HEOR helps cut through the noise for everyone. Manufacturers use HEOR to demonstrate value, regulators use it to help evaluate real-world evidence for rare disease management, payers use it for coverage determination, and clinicians & patients use it to make clear choices.
What it means: HEOR is moving from a supporting analytical activity to the core part of decision-making, so findings need to be translated into actionable insights.
How can IeB help?
We turn analysis into messages that each stakeholder can act on, through evidence communication, publication planning, and KOL engagement.
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Health Technology Assessment: How Do We Prepare for Cross-Country Collaboration?
HTA agencies are assessing new technologies with limited evidence and uncertain long-term results, which makes collaboration attractive in markets with fewer resources. The European Union HTA Regulation has launched a series of Joint Clinical Assessments for new cancer drugs and regenerative medicines, with a pilot of joint scientific consultations allowing developers to discuss evidence planning at an early stage. Managed access models, like those in Ireland, cover more than 30 medicines and conditions. While some analyses have questioned modest savings from managed entry agreements, they allow payers and manufacturers to know how the product performs in practice.
What it means: A single evidence package that works across markets saves time and cost, so it pays to design for several HTA bodies from the start.
How can IeB help?
We support JCA readiness, joint scientific consultation preparation, and multi-country evidence planning, designing studies with several HTA bodies in mind from day one.
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Value Measurement: How Do We Define Whole-Health Value?
Healthcare systems are calling for a broader definition of value beyond physical and mental health. A comprehensive view of health value takes into account financial security, housing, and food security. The majority of these lie outside the traditional healthcare system. Countries like Australia, the UK, the Netherlands, and Denmark have started embedding measures around equity, productivity, and environmental sustainability into their HTA approaches. Methods need to be flexible enough to catch up unmet needs, multisector perspectives, and consider time horizons long enough for patients to feel the impact.
What it means: A broader concept of value is useful only if it is quantifiable and can be justified, which requires the application of methodologies beyond those currently in use.
How can IeB help?
We help extend value frameworks to cover a broader range of endpoints through thorough economic and budget impact modeling. IeB supports HTA dossiers following an assessment based on evolving criteria.
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Digital Health: How Do We Turn New Data Streams into Evidence?
Wearables, digital therapeutics, and digital twins are producing real-world data. Digital twins could support more appropriate use of synthetic control arms. The FDA had approved ten mental health-related apps; including the first for depression by the end of 2024. The significant benefits will be realised in low- and middle-income countries, where mobile applications can help address critical gaps in health-system infrastructure. One WHO estimate predicts health-related cost savings of up to $11 billion in the African Region by 2030.
What it means: With more adoption, it will become crucial to define the quality of data and evidence, because not every device signal converts into a relevant outcome.
How can IeB help?
We provide evidence strategies for digital therapeutics and digital biomarkers, backed by patent landscape analysis and RWE models that treat device data in the same way as pharmaceutical data.
Also Read: Oncology Market Access Beyond Approval: Evidence, Payer, and Delivery Barriers to Address
What Does This Mean for HEOR Leaders?
Together, these trends show how HEOR’s role is shifting from supporting decisions to shaping how value is defined and demonstrated. Credible evidence alone is no longer enough. It has to be linked across data sources and stakeholders, and used in clinical, reimbursement, and policy decisions.
The common thread across these trends is connection: between data and methods, between stakeholders, and between insight and action. Teams that plan evidence early, pair AI automation with human oversight, and prepare for broader HTA and payer questions will be better placed to show value. At IeB, we help build evidence strategies that span your entire portfolio.
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