Introduction to

Health Economics
and Outcomes
Research (HEOR)
Health Economics and Outcomes Research (HEOR) is a critical
field that bridges the gap between clinical research and real-
world healthcare decision-making. By evaluating the economic
and clinical impacts of healthcare interventions, HEOR provides
the evidence base needed to ensure that limited healthcare
resources are used efficiently to maximize patient outcomes and
public health.
Defining HEOR
1 Definition 2 Scope
HEOR is a field of study that The scope of HEOR encompasses a wide range
evaluates the economic and clinical of analyses and methodologies, such as cost-
outcomes associated with healthcare effectiveness analysis, budget impact analysis,
interventions, including treatments, comparative effectiveness research, and
devices, and healthcare systems. It patient-reported outcomes research. HEOR
aims to provide evidence about the studies can evaluate various aspects of
value of healthcare services, focusing healthcare, including the efficiency of
on the balance between costs and treatment protocols, the effectiveness of new
benefits, including quality of life and technologies, and the impact of healthcare
clinical outcomes. policies on patient outcomes and system
sustainability.
The Importance of HEOR in
Healthcare Decision-Making
Evidence-Based Supporting Clinical Market Access and
Policy Making Decisions Reimbursement

HEOR provides the Clinicians rely on For pharmaceutical

evidence base needed for outcomes research to companies and medical
policymakers to make make informed treatment device manufacturers,
informed decisions about choices that balance HEOR is crucial in
healthcare funding, efficacy, safety, and demonstrating the value
reimbursement, and patient preferences. of new products to payers
resource allocation. By HEOR data can guide and regulators. Well-
analyzing the cost- clinical practice by conducted HEOR studies
effectiveness of highlighting which can support market
interventions, HEOR helps treatments offer the best access and
ensure that limited outcomes for specific reimbursement
healthcare resources are patient populations. negotiations by providing
used efficiently to evidence of a product's
maximize patient economic and clinical
outcomes and public benefits compared to
HEOR and Patient-Centered Care
1 Patient-Reported Outcomes 2 Advancing Public Health
By including patient-reported HEOR contributes to the broader
outcomes and quality of life goals of public health by
measures, HEOR emphasizes identifying effective healthcare
the importance of patient- interventions and strategies
centered care. This focus that can prevent disease,
ensures that healthcare improve health outcomes, and
interventions not only achieve reduce health disparities across
clinical objectives but also populations. By providing
address the needs and comprehensive insights into the
preferences of patients, economic and clinical impacts of
improving overall satisfaction healthcare interventions, HEOR
and health outcomes. supports evidence-based
decisions that can lead to
improved health outcomes and
more sustainable healthcare
Key Methodologies in HEOR
Comparative Effectiveness
Research
Cost-Effectiveness Analysis
Comparative effectiveness research
HEOR professionals use cost- compares the real-world performance of
effectiveness analysis to evaluate the different healthcare interventions,
balance between the costs and benefits enabling clinicians and policymakers to
of healthcare interventions, helping to make informed decisions about the most
identify the most efficient use of limited appropriate treatments for specific patient
resources. populations.

1 2 3

Budget Impact Analysis

Budget impact analysis examines the financial
implications of adopting new healthcare
technologies or interventions, providing crucial
information for policymakers and payers.
The HEOR Ecosystem

Policymakers Clinicians Patients Pharmaceutical

Companies
HEOR provides the Clinicians rely on HEOR emphasizes
evidence base HEOR data to make the importance of For pharmaceutical
needed for informed treatment patient-centered companies, HEOR
policymakers to choices that care by is crucial in
make informed balance efficacy, incorporating demonstrating the
decisions about safety, and patient patient-reported value of new
healthcare funding, preferences. outcomes and products to payers
reimbursement, quality of life and regulators.
and resource measures.
allocation.
The Future of HEOR

Advancing Integrated Decision- Global Collaboration

Technologies Making
HEOR experts from
Emerging technologies, As healthcare systems around the world will
such as real-world data, strive for greater efficiency continue to collaborate,
artificial intelligence, and and value-based care, sharing best practices
digital health solutions, HEOR will become and developing
are expected to expand increasingly integrated into harmonized
the capabilities and the decision-making methodologies to ensure
applications of HEOR, process, guiding the the consistent and
leading to more selection and reliable evaluation of
comprehensive and implementation of healthcare interventions
personalized insights. healthcare interventions across diverse
that deliver the best healthcare systems and
outcomes for patients and populations.
Conclusion: The
Transformative Power of
HEOR
Health Economics and Outcomes Research is a transformative
field that is reshaping the way healthcare decisions are made. By
providing comprehensive insights into the economic and clinical
impacts of healthcare interventions, HEOR supports evidence-
based decisions that can lead to improved health outcomes,
greater patient satisfaction, and more sustainable healthcare
systems. As the healthcare landscape continues to evolve, the
critical role of HEOR in guiding effective, efficient, and equitable
healthcare policies and practices will only become more vital.
Core Concepts in
Health Economics
Health economics is a vital field that applies economic theories
and principles to analyze healthcare decisions, the allocation of
resources, and the optimization of health outcomes. By
evaluating the costs and benefits of healthcare interventions,
health economics provides a framework for making informed
decisions that maximize health benefits while minimizing costs.
This section introduces the core concepts in health economics,
including cost-effectiveness analysis (CEA), cost-utility analysis
(CUA), and cost-benefit analysis (CBA).
Cost-Effectiveness Analysis (CEA)
1 Definition 2 Application
Cost-effectiveness analysis is a CEA is widely used in healthcare
method used to compare the relative decision-making to guide the
costs and outcomes (effects) of two allocation of resources towards
or more courses of action. CEA helps interventions that offer the most
determine which intervention significant health benefit per unit of
provides the best outcome for the cost. It is particularly useful when
cost incurred, typically expressed as budget constraints exist and helps
a ratio of cost to health outcome prioritize interventions that provide
(e.g., cost per life-year gained). the greatest value for money.
Cost-Utility Analysis (CUA)
Definition Application

Cost-utility analysis is a specific type of CUA is instrumental in assessing the value

CEA that compares the cost of an of medical interventions that affect both
intervention to the benefit it provides in the length and quality of life, such as
terms of quality-adjusted life years (QALYs) chronic disease treatments or preventative
or disability-adjusted life years (DALYs). measures. It enables policymakers and
This allows for the comparison of healthcare providers to make decisions
interventions with diverse outcomes by that consider both the economic and
converting them into a common metric humanistic aspects of healthcare
that reflects both the quantity and quality interventions.
of life.
Cost-Benefit Analysis (CBA)
Definition Application
Cost-benefit analysis evaluates the CBA is used in healthcare to assess the
financial costs and benefits of an overall economic impact of interventions,
intervention, with both costs and policies, or programs. It supports
benefits quantified in monetary terms. decisions that promote economic
This analysis provides a direct efficiency by ensuring that the benefits
comparison of the economic value of of healthcare interventions exceed their
different interventions, allowing costs. CBA can be particularly useful in
decision-makers to assess whether the public health and policy contexts, where
benefits of an intervention justify its it is essential to demonstrate the
costs. economic rationale for investments in
healthcare.
Types of Outcomes
Patient Reported Outcomes (PROs)
PROs encompass any report of the
Clinical Outcomes
status of a patient's health condition
These refer to the medical or health that comes directly from the patient,
status outcomes directly related to without interpretation by a clinician or
healthcare interventions, such as anyone else. This includes measures of
survival rates, reduction in disease quality of life, satisfaction with care,
symptoms, or incidence of adverse and perceived benefits or downsides of
effects. treatment.

1 2 3

Economic Outcomes
Economic outcomes assess the financial implications of
healthcare interventions, including direct costs (e.g.,
treatment and hospitalization costs) and indirect costs
(e.g., lost productivity due to illness).
Outcomes Research

Clinical Outcomes Economic Outcomes Patient Reported

Outcomes (PROs)
Outcomes research Outcomes research
investigates the results of examines the financial Outcomes research
healthcare interventions implications of healthcare encompasses patient-
and strategies on patient interventions, including reported measures of
health outcomes. Unlike direct costs (e.g., treatment quality of life, satisfaction
clinical trials that may and hospitalization costs) with care, and perceived
focus on immediate or and indirect costs (e.g., lost benefits or downsides of
short-term effects, productivity due to illness). treatment, providing
outcomes research seeks valuable insights into the
to understand the long- humanistic aspects of
term impact of care on healthcare interventions.
patients' lives.
Applying Outcomes Research

Inform Decision- Improve Quality of Enhance Patient

Making Care Engagement
Outcomes research By understanding the Incorporating patient-
provides valuable data to long-term impacts of reported outcomes into
inform healthcare healthcare interventions healthcare decision-
decision-making, guiding on patient outcomes, making promotes
the allocation of providers can make more patient-centered care
resources and the informed decisions and and fosters greater
selection of interventions implement strategies to patient engagement,
that offer the best value improve the quality of empowering individuals
for patients and the care and optimize health to be active participants
healthcare system. outcomes. in their own health
management.
The Future of Health Economics
Personalized Medicine Advancements in genomics and
precision diagnostics are enabling more
targeted and cost-effective healthcare
interventions, leading to improved
patient outcomes and reduced overall
costs.
Digital Health Technologies The integration of digital health tools,
such as wearables and mobile apps, is
transforming the way healthcare data is
collected and analyzed, providing new
insights to inform health economics
decision-making.
Value-Based Care The shift towards value-based care
models, which emphasize quality of care
and patient outcomes over volume of
services, is driving healthcare providers
to optimize resource allocation and
deliver the most effective interventions.

Global Collaborations Increased international cooperation and

data sharing in health economics
research are facilitating the
development of more robust and
comprehensive analyses, leading to
better-informed healthcare policies and
decisions.
Data Sources and
Methodologies in
HEOR
Health Economics and Outcomes Research (HEOR) relies on a
diverse array of data sources and analytical methods to
generate insights into the efficiency, effectiveness, and impact
of healthcare interventions. The careful selection of data sources
and analytical approaches is crucial for the validity and
applicability of HEOR findings. This presentation will explore the
pivotal role of real-world data (RWD), real-world evidence (RWE),
primary data collection, and secondary data analysis in the
context of HEOR.
Real-World Data (RWD) and Real-
World Evidence (RWE)
Real-World Data (RWD) Real-World Evidence (RWE)

RWD encompasses data related to patient RWE refers to the clinical evidence
health status and healthcare delivery regarding the usage, benefits, and risks of
collected from various sources outside medical products derived from the
traditional clinical trials. This includes analysis of RWD. Regulatory bodies such
electronic health records (EHRs), claims as the FDA and EMA increasingly recognize
and billing activities, product and disease the value of RWE in understanding the
registries, patient-generated data, and effectiveness and safety of healthcare
information from mobile devices. interventions in broader patient
populations and diverse real-world
settings.
Applications of RWD and RWE in HEOR

1 Post-Market 2 Comparative 3 Health Policy

Surveillance Effectiveness Decision-Making
Research
RWD and RWE are HEOR studies
instrumental in RWD and RWE leveraging RWD and
monitoring the long- provide insights into RWE can inform
term performance, how healthcare healthcare policies,
safety, and potential interventions perform clinical guidelines,
adverse effects of in routine clinical and patient care
healthcare practice, strategies by offering
interventions after complementing the a comprehensive
they have been findings from understanding of the
approved and randomized real-world value and
introduced to the controlled trials impact of healthcare
market. (RCTs) and interventions.
contributing to
Primary Data Collection Methods in
HEOR
Surveys Clinical Trials

Surveys are a primary data collection While clinical trials are primarily
method used to gather specific associated with the pre-market
information directly from patients, assessment of new medical interventions,
healthcare providers, or the general they also serve as a valuable source of
population. In HEOR, surveys can collect data for HEOR. Well-designed trials can
data on patient-reported outcomes, incorporate economic and quality-of-life
treatment adherence, healthcare evaluations alongside clinical efficacy and
utilization, and quality of life. safety assessments.
Secondary Data Analysis in HEOR

Administrative Registries Advantages of

Databases Secondary Data
Administrative databases Registries systematically Secondary data analysis in
contain large volumes of data collect data on patients with a HEOR allows for the
on healthcare utilization, particular disease, condition, examination of healthcare
costs, and outcomes, or exposure to healthcare interventions in diverse and
collected by healthcare interventions. They are large populations, offering a
providers, insurance valuable for HEOR studies as broader understanding of
companies, and government they provide detailed their impact across different
agencies. HEOR researchers information on clinical healthcare settings and
use these databases to practices, patient outcomes, subgroups. This method can
conduct retrospective studies and long-term effects of complement findings from
on the effectiveness and treatments in specific patient RWD and primary data
economic impact of populations. collection, enriching the
healthcare interventions. evidence base for healthcare
Conclusion: The Evolving Role of
Data in HEOR

Comprehensive Informing Evolving Importance

Insights Healthcare
As healthcare continues
Decisions
By leveraging RWD, RWE, The data sources and to evolve, the importance
and both primary and methodologies used in of diverse and robust
secondary data collection HEOR play a critical role data sources in HEOR will
methods, HEOR studies in generating evidence only increase, driving
provide comprehensive that informs healthcare improvements in
insights into the real- policies, clinical healthcare outcomes and
world effectiveness, guidelines, and patient efficiency.
safety, and value of care strategies.
healthcare interventions.
The Future of HEOR: Harnessing the
Power of Data
1 Artificial Intelligence and Machine Learning
Advancements in AI and ML are revolutionizing HEOR by enabling the analysis
of large, complex datasets to uncover hidden insights and patterns, further
enhancing the value of RWD and RWE.

2 Integrated Data Platforms

The development of integrated data platforms that seamlessly combine
various data sources, including EHRs, claims, and patient-reported data, is
crucial for providing a comprehensive view of healthcare outcomes and costs.

3 Patient-Centric Approach
HEOR is increasingly embracing a patient-centric approach, leveraging
patient-generated data and incorporating patient preferences and experiences
into the evaluation of healthcare interventions.
The Evolving Landscape of HEOR
Regulatory Guidance Collaborative Initiatives
Regulatory bodies, such as the FDA and Partnerships and collaborative initiatives
EMA, are providing more guidance on among researchers, healthcare
the use of RWD and RWE in the providers, payers, and patient advocacy
evaluation of healthcare interventions, groups are crucial for advancing the
further solidifying their role in HEOR. field of HEOR and ensuring the optimal
use of data and methodologies.

Ethical Considerations Interdisciplinary Approach

As the use of data in HEOR continues to HEOR requires an interdisciplinary
evolve, it is essential to address ethical approach, bringing together experts
concerns, such as patient privacy, data from various fields, including
ownership, and the responsible use of healthcare, economics, data science,
sensitive information. and epidemiology, to generate
comprehensive and actionable insights.
Modelling
Techniques in
HEOR
Health Economics and Outcomes Research (HEOR) employs a
diverse range of modelling techniques to predict the impacts of
healthcare interventions on costs, outcomes, and overall
healthcare system efficiency. These models are critical tools for
decision-makers, allowing them to assess the value of medical
interventions and make informed choices about healthcare
policies and practices. This presentation will delve into the key
modelling techniques used in HEOR, including decision-analytic
modelling, budget impact analysis, Markov models, and discrete
event simulation.
Decision-Analytic Modelling
Overview Applications Key Features

Decision-analytic Decision-analytic models Decision-analytic models

modelling is a systematic are used to assess the are highly versatile,
approach used in HEOR to cost-effectiveness of allowing researchers to
evaluate the medical interventions, simulate the long-term
consequences of different guide clinical decision- impacts of healthcare
healthcare decisions. making, and inform policy interventions on patient
These models integrate and reimbursement outcomes, resource
data from various sources decisions. They can utilization, and overall
to compare the outcomes incorporate a wide range healthcare costs. They
and costs associated with of data, including clinical enable policymakers to
different treatment trial results, real-world make informed decisions
options. evidence, and patient that balance clinical
preferences, to provide a efficacy, cost-
comprehensive view of effectiveness, and budget
treatment options. impact.
Budget Impact Analysis

1 Definition 2 Purpose
Budget impact analysis (BIA) BIAs are crucial for payers and
estimates the financial consequences healthcare decision-makers,
of adopting a new healthcare providing insights into how a new
intervention within a specific intervention will affect healthcare
healthcare budget context, over a spending. They help ensure that the
defined time period. adoption of new treatments is
financially sustainable and can be
integrated into healthcare systems
without compromising other services.

3 Key Considerations 4 Practical Applications

BIAs take into account factors such as BIAs inform decision-making around
the target patient population, the the reimbursement and adoption of
expected uptake of the new new healthcare technologies, helping
intervention, the costs of the to balance clinical benefits with
intervention itself, and any offset budgetary constraints and ensure the
savings or additional costs that may long-term sustainability of healthcare
arise from its implementation. systems.
Markov Models
1 Conceptual Framework
Markov models are a type of decision-analytic model used to simulate the
progression of diseases over time. They describe health states related to a
disease and the probabilities of transitioning between these states, allowing
for the estimation of long-term outcomes and costs.

2 Model Structure
Markov models divide the disease process into distinct health states, such as
"Healthy," "Mild Illness," "Severe Illness," and "Death." The model then
simulates the patient's movement between these states over a series of time
intervals, based on the transition probabilities associated with each state.

3 Applications in HEOR
Markov models are particularly useful for chronic diseases with well-defined
stages and transitions, such as cancer or cardiovascular disease. They enable
researchers to project the lifetime costs and outcomes of interventions,
accounting for the changing risks and benefits as a patient's condition
evolves.
Discrete Event Simulation
Concept Advantages
Discrete event simulation (DES) is a DES allows for the detailed analysis of
modelling technique that simulates the how different interventions influence
pathways of individual patients through patient outcomes, resource use, and
healthcare systems, based on the healthcare costs over time. It is often
occurrence of discrete events (e.g., applied in situations where patient
receiving treatment, experiencing side pathways are complex and
effects). DES models are highly flexible individualized, such as in emergency
and can capture complex patient departments or for treatments with
interactions and variations in care varied patient responses.
processes.

Modeling Approach Applications

DES models simulate the flow of DES is a powerful tool for evaluating
individual patients through a healthcare the impact of healthcare interventions,
system, tracking the occurrence of redesigning care pathways, and
relevant events and the resulting optimizing resource allocation in
impacts on costs, resource utilization, complex healthcare systems. It
and patient outcomes. This level of provides valuable insights that can
granularity enables researchers to inform decision-making and lead to
explore the complex dynamics of more efficient and effective healthcare
healthcare delivery and identify delivery.
Conclusion

Versatile Tools Informed Optimizing Driving

Decision-Making Healthcare Innovation
Modeling
techniques in By leveraging These models are Continued
HEOR provide decision-analytic instrumental in advancements in
powerful tools for modelling, budget guiding the HEOR modelling
understanding the impact analysis, efficient allocation will play a crucial
implications of Markov models, of healthcare role in shaping the
healthcare and discrete event resources, future of
decisions in terms simulation, optimizing patient healthcare,
of costs, outcomes, healthcare care, and ensuring fostering
and system-wide decision-makers the sustainability of innovation and
impacts. can evaluate the healthcare improving
value of medical systems. outcomes for
interventions patients and
across different healthcare systems
Patient-Reported
Outcomes and
Quality of Life
Measures
In the realm of Health Economics and Outcomes Research (HEOR),
Patient-Reported Outcomes (PROs) and measures of quality of life
stand as critical components in evaluating the impact of healthcare
interventions from the patient's perspective. These outcomes and
measures provide insights into the effectiveness of treatments
beyond traditional clinical endpoints, emphasizing the significance
of patient experience and satisfaction in healthcare decision-
making.
Importance of PROs in HEOR

1 Definition 2 Evaluating Treatment Impact

PROs are direct reports from patients PROs are essential for evaluating the
about how they feel or function in broader impact of treatments,
relation to a health condition or its offering a comprehensive view that
treatment, without interpretation by encompasses the physical,
healthcare professionals or anyone emotional, and social dimensions of
else. They include symptoms, health. They help to ensure that
functional status, and overall quality healthcare interventions address the
of life. needs and concerns most relevant to
patients.
3 Informing Clinical and Policy 4 Enhancing Drug Development
Decisions and Market Access
By incorporating the patient's voice, Pharmaceutical companies use PRO
PROs inform clinical guidelines, data to support drug development
regulatory decisions, and healthcare and market access strategies.
policies, promoting patient-centered Demonstrating positive effects on
care. They are particularly valuable in patient-reported outcomes can be
shared decision-making processes, pivotal for regulatory approval,
where patients and providers jointly reimbursement, and differentiation in
determine the course of treatment. competitive markets.
Instruments for Measuring PROs and
Quality of Life
Generic Instruments Disease-Specific Symptom-Specific
Instruments Instruments
These tools, such as the
Short Form Health Survey Disease-specific PRO These tools focus on specific
(SF-36) and the EuroQol five- measures are tailored to the symptoms or side effects,
dimension questionnaire symptoms and issues such as pain or fatigue, that
(EQ-5D), measure general relevant to particular can be relevant across
health status and quality of conditions, such as the multiple conditions. The Brief
life across different diseases Asthma Control Test (ACT) or Pain Inventory (BPI) and the
and populations. They are the Health Assessment Fatigue Severity Scale (FSS)
useful for comparing health Questionnaire (HAQ) for are examples of symptom-
outcomes across studies and rheumatoid arthritis. These specific measures.
interventions. instruments can detect
changes in disease
symptoms and impact that
might not be captured by
Utility Measures
Assessing Health-Related Quality of Life
Utility measures, like the EQ-5D, assess health-related quality of life in terms
of utility values, which represent the preference for specific health states.
These values are crucial for cost-utility analyses, allowing for the calculation
of quality-adjusted life years (QALYs).
Integrating PROs into HEOR
1 Comprehensive Perspective
By integrating PROs into HEOR, the evaluation of healthcare
interventions takes a more holistic approach, considering the
multifaceted aspects of patient well-being beyond just clinical outcomes.

2 Patient-Centered Care
Incorporating PROs supports a patient-centered healthcare system
where treatment success is gauged not only by physiological outcomes
but also by improvements in quality of life and patient satisfaction.

3 Informing Decisions
The utilization of PROs in HEOR ensures that the patient's voice is heard,
informing clinical guidelines, regulatory decisions, and healthcare
policies to better address the needs and preferences of those receiving
care.
The Evolving Role of PROs

Expanding Influence Patient-Centered Informed Decisions

Approach
As healthcare continues to The utilization of PROs in
evolve, the role of PROs in Incorporating PROs into HEOR ensures that the
shaping treatment HEOR supports a patient- patient's voice is heard,
guidelines, policy decisions, centered healthcare informing clinical
and healthcare practices system where treatment guidelines, regulatory
will undoubtedly expand, success is gauged not only decisions, and healthcare
underscoring the by physiological outcomes policies to better address
importance of listening to but also by improvements the needs and preferences
and prioritizing patient in quality of life and patient of those receiving care.
voices. satisfaction.
Conclusion
Patient-Centered Approach
Incorporating PROs into HEOR supports a patient-centered healthcare
system where treatment success is gauged not only by physiological
outcomes but also by improvements in quality of life and patient
satisfaction.
Comprehensive Evaluation
By integrating PROs, HEOR emphasizes the significance of treatment effects
on patients' daily lives, beyond what is typically captured by clinical
measures alone, providing a more holistic approach to healthcare evaluation
and decision-making.
Prioritizing Patient Voices
As healthcare continues to evolve, the role of PROs in shaping treatment
guidelines, policy decisions, and healthcare practices will undoubtedly
expand, underscoring the importance of listening to and prioritizing patient
voices in the quest for improved health outcomes.
Key Takeaways

1 Comprehensive Perspective 2 Informing Decisions

PROs provide a comprehensive view By incorporating the patient's voice,
of the impact of healthcare PROs inform clinical guidelines,
interventions, encompassing the regulatory decisions, and healthcare
physical, emotional, and social policies, promoting patient-centered
dimensions of patient well-being. care.

3 Enhancing Drug Development 4 Evolving Role of PROs

Pharmaceutical companies use PRO As healthcare continues to evolve,
data to support drug development the role of PROs in shaping treatment
and market access strategies, guidelines, policy decisions, and
demonstrating positive effects on healthcare practices will undoubtedly
patient-reported outcomes. expand.
Role of HEOR in
Drug Development
Health Economics and Outcomes Research (HEOR) plays a
crucial role in the drug development process, from the early
phases to regulatory approval and reimbursement negotiations.
HEOR provides valuable insights that inform clinical trial design,
target areas of unmet medical need, and support value-based
pricing strategies.
Informing Clinical Trial Design
Relevant Endpoints
In the early stages of drug development, HEOR can
guide the design of clinical trials to ensure they capture
data relevant to economic and outcomes-based
evaluations. Incorporating endpoints that measure
quality of life, patient satisfaction, and direct and
indirect costs can provide early evidence of a drug's
potential value proposition.

1 2

Unmet Medical Needs

HEOR analyses help identify areas of significant unmet
medical need where new therapies could have the most
substantial impact. By focusing on these areas,
pharmaceutical companies can prioritize drug
development efforts where there is a clear opportunity
to improve patient outcomes and demonstrate
economic value.
HEOR's Impact on Pricing and
Reimbursement
Value-based Pricing Reimbursement Negotiations

HEOR provides the evidence base needed Comprehensive HEOR studies are crucial
for value-based pricing strategies, where for reimbursement submissions, offering
the price of a drug is aligned with the payers a detailed understanding of a
benefits it delivers to patients and the drug's economic and clinical benefits
healthcare system. Economic models and compared to standard care. This evidence
outcomes research data support can be decisive in securing favorable
negotiations with payers, demonstrating reimbursement terms, ensuring that
the drug's value in terms of improved patients gain access to new therapies.
outcomes and potential cost savings.
Regulatory Agencies and HEOR
1 Incorporation into Evaluations 2 Evidence Submission Guidance
Regulatory agencies, including the Agencies provide guidance to
U.S. Food and Drug Administration pharmaceutical companies on
(FDA) and the European Medicines submitting HEOR data as part of their
Agency (EMA), have begun to market authorization applications.
incorporate HEOR data into their This may include information on cost-
evaluation processes for new medical effectiveness, the impact on quality
products. While the primary focus of life, and comparative effectiveness
remains on safety and efficacy, there against existing therapies.
is an increasing acknowledgment of
the importance of economic and
patient-reported outcomes.
3 Post-Marketing Requirements
In some cases, regulatory approvals are granted with stipulations for post-marketing
studies that include HEOR components. These studies can help to fill evidence gaps
regarding a drug's long-term effectiveness, real-world outcomes, and impact on
healthcare resource utilization.
Payers and HEOR Decision-Making

Reimbursement Drug Price Negotiations Formulary Decisions

Assessments
HEOR data provides a basis Payers use HEOR data to
Payers, including insurance for payers to negotiate make informed formulary
companies and drug prices with decisions, deciding which
government healthcare pharmaceutical companies. drugs to include on their
programs, rely heavily on By demonstrating the list of covered medications.
HEOR data to assess the economic value of a drug, Drugs that show clear
value of new drugs and including potential savings benefits in terms of
treatments. Cost- from reduced improved patient outcomes
effectiveness analyses and hospitalizations or and cost savings are more
budget impact models are improved disease likely to be prioritized for
key tools used by payers to management, payers can inclusion.
determine whether an advocate for pricing that
intervention should be reflects the drug's real-
covered and at what level world impact.
Coverage Policy Development
HEOR Data
HEOR data informs the development of broader coverage policies,
guiding how resources are allocated across different therapies and
services.

Evidence-Based Practices
This ensures that payer policies are aligned with evidence-based
practices that optimize patient outcomes and resource use.

Optimal Resource Allocation

By leveraging HEOR data, payers can make informed decisions on
coverage policies that lead to optimal resource allocation and
improved patient outcomes.
HEOR's Comprehensive Impact
Drug Development HEOR guides clinical trial design and
identifies unmet medical needs.

Regulatory Approval Agencies incorporate HEOR data into

evaluation processes and require post-
marketing studies.
Pricing and Reimbursement HEOR supports value-based pricing and
reimbursement negotiations with payers.

Payer Decision-Making Payers use HEOR to assess value,

negotiate prices, make formulary
decisions, and develop coverage policies.
The Future of HEOR
As healthcare systems continue to evolve, the role of HEOR will
become increasingly pivotal in driving informed decision-making
and ensuring the sustainable delivery of high-quality, cost-
effective care. With its multidisciplinary approach and focus on
real-world evidence, HEOR will remain a cornerstone of drug
development, regulatory approval, and reimbursement
processes, ultimately benefiting patients, healthcare providers,
and society as a whole.